Disorder of development or morphogenesis

This study tested a new RNA cancer vaccine in 119 people with advanced melanoma. The vaccine was designed to train the immune system to attack four proteins found on melanoma cells. The main goal was to check the vaccine's safety and find the best dose, while also looking for sig…

This study tests whether a two-step drug treatment can improve bone density in people with long-term spinal cord injury who have bone loss. Participants receive either romosozumab (a bone-building drug) or a placebo for 12 months, followed by denosumab (a bone-strengthening drug)…

This early-stage trial tests a drug called PH-762, injected directly into skin tumors, for people with squamous cell carcinoma, melanoma, or Merkel cell carcinoma. Participants receive four weekly injections into one tumor, then have it surgically removed about two weeks later. T…

This Phase 3 study tests whether a gel containing patidegib can lower the number of new basal cell carcinomas (a type of skin cancer) on the face of adults with Gorlin syndrome. Participants apply the gel or a placebo to their face twice daily for a year. The study will compare h…

Researchers are testing an experimental immunotherapy called GNR-051 in adults with advanced lung cancer, kidney cancer, or melanoma that has returned or not responded to other treatments. The Phase 1 trial aims to find the highest dose people can tolerate and to track side effec…

This early-phase study tests a new drug called ERAS-601, alone or with other cancer treatments, in adults with advanced solid tumors that have not responded to standard therapies. The main goals are to find safe doses and check for side effects. About 90 participants will take pa…

This trial tests whether a new intraoral device, the Ertty Gap III, can correct skeletal Class III malocclusion (underbite) in growing children aged 9–12 as effectively as the standard facemask therapy. The study measures changes in jaw alignment using X-rays before and after tre…

Researchers are testing whether intensive blood pressure lowering slows heart and valve damage in people with asymptomatic moderate to severe aortic valve stenosis. The trial enrolls 60 adults with high blood pressure and a narrowed aortic valve but no symptoms. Half receive stan…

Lentigo maligna is an early form of melanoma that appears on sun-exposed skin, often on the face. Doctors usually remove it with surgery, but it can be hard to decide how much tissue to take. This trial tests whether applying imiquimod cream for four weeks before surgery can shri…

Researchers are testing an experimental drug called TAK-935 in children aged 2 to 17 with Dravet syndrome or Lennox-Gastaut syndrome, two severe forms of epilepsy that often resist treatment. The trial compares TAK-935 added to standard anti-seizure medications against a placebo …

Researchers are testing a two-step radioimmunotherapy for people with medullary thyroid cancer that has come back. Participants receive a bispecific antibody followed by a radioactive peptide. The goal is to see if this targeted radiation can shrink tumors and how safe it is.

This early-phase trial tests a new drug called AI-061, which combines two immune-boosting antibodies (anti-PD-1 and anti-CTLA-4) into one treatment. The study will enroll 18 adults with advanced solid tumors that have spread or cannot be removed. The main goals are to find a safe…

This trial tests a treatment called Melphalan/HDS for people whose eye melanoma has spread to the liver. The approach delivers high-dose melphalan, a chemotherapy drug, straight into the liver through a device system while limiting exposure to the rest of the body. Researchers wa…

This phase 2 trial tested a combination of an injected drug (LTX-315) and an intravenous immunotherapy (pembrolizumab) in 23 people with advanced melanoma that no longer responded to standard PD-1/PD-L1 inhibitors. The goal was to see if the combination could shrink tumors or slo…

This study tests a drug called apatinib combined with two chemotherapy drugs (irinotecan and temozolomide) in children aged 5 to 18 with neuroblastoma that has returned or not improved after standard treatment. The goal is to see if the combination can shrink tumors and help chil…

This phase 2 study tests a combination of two nucleoside drugs, doxecitine and doxribtimine, in 47 people with thymidine kinase 2 (TK2) deficiency, a rare genetic disorder that weakens muscles. Participants already receiving nucleoside therapy continue treatment to see if the dru…

This phase 2 trial tests whether adding an experimental drug called pixatimod to the immunotherapy nivolumab can shrink tumors in people with advanced melanoma, non-small cell lung cancer, or colorectal cancer that has stopped responding to prior treatment. One group with colorec…

This phase 2 trial tests an oral drug called ARD-101 in adults with Prader-Willi syndrome, a genetic condition that causes an unrelenting feeling of hunger. Researchers give the drug twice daily for 28 days to see if it safely reduces hyperphagia, the intense drive to eat, using …

This study tracks 245 adults aged 18 to 60 who receive a Vivacit-E liner during hip replacement surgery. The liner is a plastic cup that fits inside the hip socket to allow smooth movement. Researchers measure how long the liner stays in place without needing removal, and they ch…

This trial tests whether combining two bone-building drugs, teriparatide and cinacalcet, works better than teriparatide alone for men with low bone mass. Researchers will give one group the combination and another group teriparatide plus a placebo for 11 months. They will measure…

This study tested a drug called RTA 408 (omaveloxolone) in 172 people with Friedreich's ataxia, a rare genetic disease that affects movement and coordination. The goal was to see if the drug is safe and can improve exercise ability and daily function. Participants took the drug o…

This trial tests whether repairing an isolated cleft palate at 6 months of age leads to better speech than waiting until 12 months. About 650 infants across the UK, Scandinavia, and Brazil will be randomly assigned to one of the two surgery times. Doctors will measure how well th…

This trial tests whether regular walking with a robotic exoskeleton can improve bone health and quality of life in people with spinal cord injury. Participants will train three times a week for 26 weeks, aiming for an hour of continuous walking each session. Researchers will meas…

This trial tests an experimental drug called SNS-101, which targets a protein called VISTA that may help tumors evade the immune system. Researchers give SNS-101 alone or with another immunotherapy, cemiplimab, to adults with advanced solid tumors that have not responded to stand…

This study follows adults who had cataract surgery with Clareon Vivity or PanOptix intraocular lenses, which are designed to restore vision at multiple distances. Researchers will track visual acuity and watch for complications like lens dislocation or retinal detachment over sev…

This study tests whether eating wild blueberry powder every day can lower blood pressure and improve blood vessel health in middle-aged and older men and postmenopausal women with slightly high blood pressure. Researchers will measure artery function and stiffness over time. The …

This study looked at whether it is safe for people with multiple sclerosis to receive their natalizumab (Tysabri) infusions at home instead of at a hospital. Researchers compared serious side effects, quality of life, and costs over one year in 295 patients. The goal was to see i…

This early-phase trial tested whether giving activated natural killer (NK) cells, with or without a protein called rhIL15, is safe and feasible for children and young adults with solid tumors that didn't respond to other treatments. Sixteen participants received chemotherapy to l…

This trial is testing whether combining the immunotherapy drug pembrolizumab with other experimental agents can help people with advanced melanoma whose cancer has stopped responding to standard anti-PD-1 treatment. The study includes several treatment arms, each pairing pembroli…

This study tests whether giving probiotics to pregnant women with diabetes can improve their babies' brain development. Researchers will give 60 mothers a probiotic supplement and then measure their infants' brain responses to sounds and sights at 1 and 6 months old. The goal is …

This early-phase trial is testing whether combining two drugs—pembrolizumab (Keytruda) and ibrutinib (Imbruvica)—can safely treat advanced melanoma that cannot be removed by surgery. Pembrolizumab helps the immune system fight cancer, while ibrutinib blocks enzymes that help canc…

This trial tests two devices, a face mask and the Ertty Gap appliance, in 30 growing patients with a deficient upper jaw (skeletal Class III malocclusion). Each patient receives one of the two devices to pull the upper jaw forward. Researchers use cone beam CT scans and 3D cephal…

This trial tests whether performing surgery within 72 hours of birth improves outcomes for female newborns with bladder exstrophy, a rare birth defect where the bladder develops outside the body. Half the participants receive surgery within 72 hours, while the other half wait unt…

This phase II trial tests whether combining two immunotherapy drugs, nivolumab and relatlimab, can shrink tumors in people with metastatic melanoma who have not yet received immunotherapy. Nivolumab helps immune cells attack cancer, while relatlimab blocks a different brake on th…

This trial tests whether adding a synbiotic supplement to a Mediterranean diet can improve quality of life and reduce disability in people with relapsing-remitting multiple sclerosis. Participants follow the diet for 12 weeks and take either a synbiotic or a placebo pill daily. R…

This trial tests whether a cutting balloon, a catheter with tiny blades that score the vessel wall before expanding, can safely widen branch pulmonary arteries that resist standard low-pressure balloon dilation. Researchers will compare the cutting balloon to high-pressure balloo…

This trial is testing whether a minimally invasive version of isolated limb perfusion is feasible. Isolated limb perfusion is a procedure that delivers high-dose chemotherapy directly to a limb affected by cancer, but the standard approach requires a large incision. The study wil…

This phase 2 trial is testing whether combining two chemotherapy drugs—temozolomide and topotecan—can help children with neuroblastoma, brain tumors, or other solid tumors that have come back or not responded to standard treatment. Participants receive temozolomide by mouth and t…

This trial compares giving zoledronate, a yearly intravenous bone-strengthening drug, within 5 days of hip fracture surgery versus waiting 3 months. The goal is to see if early treatment is as safe and effective at reducing bone turnover and preventing future fractures. Participa…

This study is testing whether a subcutaneous (under the skin) injection of ocrelizumab works as well as the standard intravenous (IV) infusion for people with relapsing or primary progressive multiple sclerosis. The trial will compare how the drug is absorbed, its safety, and its…

This trial compares two ways of making the skin incision during upper eyelid surgery (blepharoplasty) to correct droopy or puffy eyelids. Each participant will have one eyelid cut with an electrocautery device and the other with a traditional scalpel, so the results can be compar…

This study tested a drug called leuprorelin to control central precocious puberty, a condition where children start puberty too early. About 80 children received an injection every 12 weeks for 6 months. The goal was to suppress hormones that drive early puberty, helping children…

This trial tests whether a nasal spray containing bevacizumab, a drug typically used for cancer, can reduce frequent nosebleeds in people with hereditary hemorrhagic telangiectasia (HHT). The study enrolls 20 adults who have at least 10 nosebleeds per month. The goal is to see if…

This trial tests whether cladribine tablets change key immune cells and nerve damage markers in the spinal fluid of people with relapsing forms of multiple sclerosis. Participants take cladribine as prescribed and undergo up to two lumbar punctures to measure these biomarkers ove…

This study tests whether doing Pilates exercises three times a week for 12 weeks can improve bone density in breast cancer survivors aged 40-60 who are taking hormone therapy (aromatase inhibitors). These women are at higher risk for bone loss and osteoporosis. The trial will mea…

This trial investigates whether two common osteoporosis medications, denosumab and alendronate, can reduce liver fat and scarring in postmenopausal women who also have nonalcoholic fatty liver disease (NAFLD). The study will compare changes in liver fat and stiffness over 12 mont…

This trial investigates whether people with knee osteoarthritis who also have a bowleg deformity (tibia vara) experience different pain relief after a partial knee replacement compared to those without the deformity. The study will measure medial knee pain using a standard pain s…

This study looks at whether adding a statin (atorvastatin or rosuvastatin) to standard osteoporosis treatment can improve bone density in postmenopausal women. Participants are women aged 55 and older with osteoporosis who also have high cholesterol. The trial compares standard t…

This study compares two ways of creating scleral tunnels during flanged intrascleral intraocular lens (IOL) fixation surgery, a procedure used when the eye's natural lens support is missing or damaged. Researchers are testing whether making the tunnel horizontally (parallel to th…

This study tested a once-weekly injection called TransCon CNP in 84 children aged 2 to 11 with achondroplasia, the most common form of dwarfism. The goal was to see if it safely increases growth rate over a year compared to a placebo. After the first year, all children could rece…

People with inflammatory bowel disease (IBD) have a higher risk of colorectal cancer and need regular colonoscopies to catch early warning signs. This study compares two techniques—narrow-band imaging (a special light) and an artificial intelligence (AI) system—to see which finds…

This study tests a new drug called pegilodecakin, a lab-made version of a natural immune protein (interleukin-10), in people with advanced solid tumors such as melanoma, lung, breast, prostate, ovarian, kidney, colorectal, and pancreatic cancers. The drug is given as a daily shot…

This trial tests whether repeatedly adding sterile fluid into the womb can help fetuses with severe kidney failure grow enough lungs to survive after birth. Pregnant women with early pregnancy kidney failure can choose to receive the infusions or just be monitored. Babies who sur…

This study tests whether using continuous positive airway pressure (CPAP) or positive end-expiratory pressure (PEEP) during resuscitation helps extremely low birth weight infants (born before 28 weeks) breathe better and avoid lung damage. Half of the babies receive standard care…

This study tested an ointment called tirbanibulin (Klisyri®) on adults with actinic keratosis, a common precancerous skin condition caused by sun exposure. The trial included 280 participants and applied the ointment to larger skin areas (up to 100 cm²) on the face or balding sca…

This completed phase 2 trial tested whether adding alpha interferon to standard surgery helps prevent skin melanoma from returning. 278 participants with stage I-IV melanoma were assigned to one of six groups, including surgery alone, low or high dose interferon, chemotherapy, or…

This trial investigates whether cardiac resynchronization therapy (CRT) — a special type of pacing — can improve how far people can walk in six minutes. It includes adults with congenital heart disease where the right ventricle pumps blood to the body (systemic right ventricle). …

This study tests a new, minimally invasive surgical technique to repair open neural tube defects (spina bifida) in unborn babies. Instead of opening the uterus, surgeons use a tiny camera and tools inserted through small incisions. The goal is to close the spinal defect effective…

This study looked at whether using a soft hip brace (abduction splint) for two months in one-month-old babies with mild hip dysplasia (stable hips but abnormal ultrasound) improves hip development. 90 infants were enrolled. The goal was to see if the brace makes hip ultrasound me…

This trial tests whether a personalized physical activity plan can help children aged 5–18 with congenital heart disease increase their exercise capacity and daily activity levels. The plan is designed with input from parents, teachers, and a pediatric cardiologist to address saf…

This study tests whether a graft made from a patient's own or donor stem cells can repair the cornea's surface in people with limbal stem cell deficiency, a condition that causes pain, scarring, and vision loss. A small biopsy of limbal tissue is taken from a healthy eye or donor…

This study looked at 144 people with aggressive relapsing multiple sclerosis to compare two treatment plans over three years: monthly natalizumab infusions versus a six-month course of mitoxantrone followed by another immune-modulating drug. Researchers measured cost-effectivenes…

This study tested a special stent graft (WRAPSODY) against the usual balloon treatment for vein blockages in people on dialysis. The goal was to see if the stent graft could keep the vein open longer and reduce the need for repeat procedures. A total of 357 patients took part acr…

This study tested whether adding natural ingredients like garlic oil and nattokinase to standard hormone replacement therapy (HRT) could reduce the risk of blood clots in menopausal women. The trial involved 60 women divided into premenopausal and postmenopausal groups. Researche…

This early-phase trial tested a new treatment for relapsing-remitting multiple sclerosis (RRMS) that uses a patient's own red blood cells. The cells are coated with specific peptides and given back to the patient to teach the immune system to stop attacking the body. The study in…

This study tested whether blood thinners (DOACs) work better than aspirin to prevent valve damage and serious heart problems after a less invasive aortic valve replacement (TAVI). 360 patients aged 65-80 were randomly assigned to take either a DOAC or aspirin daily. The main goal…

This study tested whether N-acetylcysteine (NAC), an antioxidant drug, can improve motor skills and learning in children with neurofibromatosis type 1 (NF1). Twenty-five children aged 8 to 16 took NAC or a placebo for 8 weeks. The goal was to see if NAC is safe and helps with mov…

This study followed 500 people who received the Amplatzer PFO Occluder, a device that closes a small hole in the heart called PFO. Researchers tracked serious complications within 30 days and longer-term issues like stroke or blood clots over 3 years. The goal was to confirm the …

This study tested a new, less invasive surgery for children aged 4 to 10 with a stubborn clubfoot deformity called 'bean-shaped foot.' Instead of cutting bones, surgeons placed a small metal plate across a joint in the foot to gently guide growth and straighten the foot over time…

This study tested a combination of two drugs, sitravatinib (a pill) and tislelizumab (an IV infusion), in 16 people with a rare eye cancer (uveal melanoma) that had spread to the liver. The goal was to see if the combo could shrink liver tumors better than current treatments. Par…

This study tested the safety of a drug called maralixibat in 27 infants under 12 months old with Alagille syndrome or progressive familial intrahepatic cholestasis (PFIC), two rare liver diseases that cause bile buildup. The drug is given as an oral solution and aims to reduce bi…

This study tested whether combining a Mediterranean diet with progressive resistance training could improve bone density in adults aged 65 and older. Over 12 months, 22 participants followed the diet and exercise program, while 19 received no structured intervention. Researchers …

This study followed 650 low-risk patients with severe aortic valve narrowing who received a less invasive valve replacement called TAVI. Researchers tracked deaths, strokes, and return hospital visits for heart problems. The goal was to see how well TAVI works in everyday practic…

This study looked at whether using half the usual dose of heparin during heart surgery (bypass or valve replacement) could reduce bleeding afterward. Heparin is a blood thinner used to prevent clots during surgery, but it can increase bleeding. The study involved 238 adults and c…

This study tested whether a dietary supplement called propionic acid is safe and helpful for people with multiple sclerosis (MS). Twenty-two adults with stable MS took propionic acid capsules alongside their usual MS medications. Researchers measured nerve damage markers and thin…

This completed Phase 3 trial tested the oral drug dimethyl fumarate (Tecfidera) against an injected interferon in 156 children aged 10 to 17 with relapsing-remitting multiple sclerosis. The main goal was to see if the drug could prevent new brain lesions and control the disease s…

This study tested the drug sunitinib (Sutent) in 71 people with advanced thyroid cancer that had spread or could not be removed by surgery. The goal was to see if the drug could shrink tumors when standard treatments like radioactive iodine no longer work. Participants took sunit…

This phase 2 trial tested sodium valproate, a drug used for seizures, in 41 children with Rubinstein-Taybi syndrome (RTS), a rare genetic condition causing intellectual disability. Participants aged 6 to 21 received either the drug or a placebo for one year. Researchers measured …

This early-phase study tested a new drug called CCT3833 in 31 adults with advanced solid tumours, including melanoma. The main goal was to find a safe dose and check for side effects. Researchers measured how the drug moves through the body and looked for the best dose to use in …

This study tested whether adding the drug bevacizumab (Avastin) to standard chemotherapy helps children with neuroblastoma that has returned or not responded to treatment. The trial enrolled 225 children aged 1 to 21. Researchers looked at tumor shrinkage and how long the disease…

This study tested whether giving very low birth weight preterm infants (born at or before 32 weeks, weighing under 1250 grams) a weekly high-dose oral vitamin A supplement could reduce their risk of developing bronchopulmonary dysplasia (BPD), a chronic lung disease. The trial ra…

This study tested a new, simple device to reduce the size of wide gaps in the jawbone (alveolar clefts) in 15 people aged 12-25 with repaired cleft lip and/or palate. The device slowly pulls the bone together before a bone graft, aiming to make the graft more successful. The stud…

This study tested a treatment called extracorporeal photopheresis (ECP) in 15 people with multiple sclerosis. ECP is a procedure that filters a person's blood, treats it with light, and returns it to the body. The goal was to see if it is safe and if it can improve walking, hand …

This study tested a new calcium phosphate putty to repair bone gaps in the gums of 84 children with unilateral cleft lip and palate. The putty was compared to the standard approach of using the child's own bone. Researchers measured how much bone remained after one year, as well …

This study tested the drug spironolactone in 15 adults with chronic right-sided heart failure. The goal was to see if it is safe and can improve heart function and reduce stress on the heart. Participants took either the drug or a placebo to compare effects.

This pilot study tested whether colchicine, an anti-inflammatory drug, can slow the buildup of calcium in the aortic valve. 24 adults with mild-to-moderate aortic stenosis were randomly assigned to take colchicine or a placebo for 6 months. The study measured changes in valve cal…

This study looked at whether a high-fat diet changes how children with mitochondrial disease burn energy. 36 children aged 5 to 21 were randomly assigned to eat either a normal or high-fat diet for a month, then switched. Researchers measured their resting energy use and body com…

This study tested whether taking two targeted drugs (dabrafenib and trametinib) for 12 months after melanoma surgery could keep the cancer from returning. It included 870 adults with high-risk stage III melanoma that had a specific BRAF V600 mutation. Results showed the drug comb…

This study compared two different artificial heart valves (Sapien and Myval) used in a procedure called TAVI to replace a narrowed aortic valve. Over 1,000 patients were randomly assigned to receive one of the two valves. The goal was to see which valve led to fewer major heart p…

This phase 2 trial tested the drug darovasertib in 15 people with high-risk uveal melanoma (a rare eye cancer) who were scheduled for eye removal surgery. Participants took the drug for up to 4 weeks before surgery, and some continued for 6 months afterward. The main goals were t…

This study followed 26 people with Anderson-Fabry disease, some taking the enzyme replacement drug Replagal® and some not, to see how their hearts changed over two years. Researchers used special MRI scans to measure heart tissue relaxation time, which may indicate early damage. …

This Phase 3 trial tested a gene therapy called GS010 in 98 people with Leber hereditary optic neuropathy (LHON), a genetic condition that leads to rapid vision loss. Participants received injections of the therapy or a placebo into both eyes. The study measured changes in vision…

This early-phase study tested an intravenous form of the drug fenretinide in 17 children whose neuroblastoma had come back or didn't respond to standard treatments. The main goals were to find the safest dose and understand side effects. Researchers also looked at how the drug mo…

This study looked at the safety and how the body processes two doses of nirsevimab, a medicine to prevent RSV, given 5 to 6 months apart. It included 33 Japanese infants up to 12 months old with heart or lung disease, weak immune systems, Down syndrome, or who were born early. Re…

This study compared two surgical methods for repairing distal hypospadias, a birth defect where the urinary opening is on the underside of the penis. 120 boys aged 2-12 received either a standard double dartos flap or that plus a Byar's flap. The goal was to see which technique l…

This study tested whether denosumab, a drug given as a shot every 6 months, can improve bone density in people with osteoporosis caused by systemic mastocytosis. 24 adults with the condition took part. The goal was to see if the drug strengthens bones and prevents fractures bette…

This early-phase study tested whether adding a TNF-blocking drug (infliximab or certolizumab) to standard immunotherapy (nivolumab and ipilimumab) is safe for people with advanced melanoma that has spread. The main goal was to find the best dose and check for serious side effects…

This study tested a mix of treatments—biofeedback, osteopathic care, behavioral training, diet changes, and electrical stimulation—to help children with spina bifida control their bladder. 66 kids aged 10-17 took part, receiving either the combo therapy or standard care for 10 we…

This study tested whether sildenafil, a drug used for high blood pressure in the lungs, is safe for premature infants at risk of bronchopulmonary dysplasia (BPD), a serious chronic lung disease. 109 babies born before 29 weeks and needing breathing support were given either silde…

This study tested a gel called Picato (ingenol mebutate) on 20 people with a very early form of melanoma on the face, called lentigo maligna. Participants applied the gel once a day for three days. The goal was to see if the gel could make the cancer disappear completely, offerin…

This study tested a special device (GORE® CARDIOFORM ASD Occluder) to close a common type of hole in the heart called an atrial septal defect (ASD). The goal was to see if the device could safely and effectively seal the hole using a tube inserted through a blood vessel, avoiding…

This study tested a new delivery system for implanting devices that close holes in the heart, such as PFO and ASD. It involved 254 patients and measured how well the system worked and how safe it was. The goal was to confirm that the system performs well in real-world use.

This study tested whether giving acetaminophen (a common pain and fever medicine) to extremely preterm babies within the first 12 hours after birth can help close a blood vessel in the heart that normally closes on its own. The trial involved 804 babies born between 23 and 28 wee…

This phase 3 trial tested a single injection of GS010 gene therapy in 39 people with Leber Hereditary Optic Neuropathy (LHON) who had vision loss for 6 months or less. The treatment aims to improve vision by delivering a working copy of the ND4 gene to the eye. Results measured c…

This study tested a one-time gene therapy injection, GS010, in 37 adults with Leber Hereditary Optic Neuropathy (LHON), a genetic condition that causes rapid vision loss. Participants had vision loss for 6 to 12 months before treatment. The goal was to see if the therapy could im…

This phase 3 trial tested a combination of two targeted drugs, LGX818 and MEK162, against standard treatments in 921 people with advanced melanoma that has a BRAF V600 mutation. The study compared the combo to vemurafenib alone or LGX818 alone to see which worked better at delayi…

This study tested a monthly injection of ofatumumab in 180 people with very early relapsing-remitting multiple sclerosis (MS). The goal was to see if the drug could stop all signs of disease activity—like relapses, disability worsening, and new brain lesions—over 18 months. Resea…

This study tested an experimental drug called BIIB091, alone or combined with an approved MS drug (diroximel fumarate), in 127 people with relapsing multiple sclerosis. The goal was to check safety and see if it reduces brain inflammation on MRI scans. Participants took pills twi…

This study tested the drug sorafenib (Nexavar) in 32 adults with uveal melanoma that had spread to other parts of the body. The goal was to see if the drug could stop tumors from growing for at least 24 weeks. Sorafenib is already used for other cancers, and researchers wanted to…

This phase 3 trial tested a new version of the drug agalsidase beta (AGA BETA BS) in 20 people with Fabry disease who were already stable on the standard drug Fabrazyme. Participants switched to the new drug for 54 weeks to see if it worked just as well. The main goal was to keep…

This phase 2 trial tested whether giving the chemotherapy drug dacarbazine (DTIC) before re-trying immunotherapy could help patients with advanced melanoma whose cancer had stopped responding to immunotherapy. The study enrolled 38 adults with metastatic melanoma that had progres…

This study tested whether the anesthetic sevoflurane protects the heart better than propofol during aortic valve replacement surgery. 82 patients were randomly assigned to receive either sevoflurane or propofol during and after surgery. The researchers measured troponin levels, a…

This study tested whether adding a personalized cancer vaccine (RO7198457) to the immunotherapy drug pembrolizumab works better than pembrolizumab alone for people with advanced melanoma that hasn't been treated before. The trial involved 131 participants and measured how long th…

This early-stage trial tested an experimental drug called CyPep-1, injected directly into tumors, either alone or with the immunotherapy pembrolizumab. The study involved 60 adults with advanced solid cancers that had stopped responding to standard treatments. The main goals were…

This study looked at how well a special telescopic nail (JuniOrtho) works and how safe it is in children with osteogenesis imperfecta (brittle bone disease). Fourteen children aged 18 months to 18 years who needed surgery for broken bones or bone straightening took part. The main…

This study looked at whether the drug sildenafil is safe for premature infants with a severe lung condition called bronchopulmonary dysplasia (BPD). The study included 125 babies in hospital intensive care units. Researchers monitored for dangerously low blood pressure as the mai…

This study tested whether injecting a child's own heart stem cells during a planned surgery could help their heart work better. It involved 25 children with hypoplastic left heart syndrome, a condition where the left side of the heart is underdeveloped. The main goals were to see…

This study tested a new drug called OMT-28 in 28 people with primary mitochondrial disease, a condition that affects how cells produce energy. Participants took the drug once daily for 6 months, and researchers measured safety, blood markers of inflammation, and symptoms like fat…

This study looked at whether people with relapsing multiple sclerosis can safely switch from an intravenous (IV) anti-CD20 antibody treatment to a self-injected drug called ofatumumab. 111 adults took part, and researchers tracked their brain scans and side effects for a year. Th…

This study tested whether giving the steroid methylprednisolone before and during heart surgery could reduce dangerous inflammation in infants under one year old. Over 1,200 babies were randomly assigned to receive either the steroid or a placebo (salt water). Researchers tracked…

This study tested whether a special material made from a child's own blood, called platelet-rich fibrin (PRF), can reduce complications after surgery for a rare bladder condition called bladder exstrophy. Twenty children having their first bladder repair surgery were split into t…

This completed study in Spain looked at 310 people with relapsing multiple sclerosis who were taking ofatumumab (Kesimpta®). Researchers wanted to see how well the drug reduced relapses and how safe it was in everyday medical practice. The study collected information from medical…

Women with pathological nipple discharge often need surgery to find the cause, but standard mammograms and ultrasounds can miss cancer. Researchers are testing a non-invasive breast scan called high-resolution magnetic resonance ductography (HR-MRD) that uses a powerful magnet an…

This study is testing a portable ultrasound device called POROUS R4C that measures bone in the lower leg without radiation. Researchers want to see if it can estimate bone mineral density as accurately as the standard X-ray method (DXA). The study will include 350 healthy men and…

This Phase 3 trial tests a radioactive imaging agent called 18F-mFBG to see if it can accurately detect neuroblastoma, a type of childhood cancer. The study involves 43 participants who either have or are suspected of having neuroblastoma. Researchers will compare the new PET sca…

When a general practitioner spots a skin lesion that might be melanoma, the patient is referred to a dermatologist, but some never go and others wait too long. This trial tests whether the GP calling the dermatologist directly and emailing a smartphone photo of the lesion shorten…

Researchers are studying whether a blood test can detect the BRAF V600E mutation in people with melanoma. The trial enrolls patients with advanced melanoma and those with earlier-stage disease after surgery. By comparing blood samples to standard tumor tissue tests, they aim to s…

Researchers are testing whether 3D photographs can accurately measure facial asymmetry in adults aged 18 to 40 who have no craniofacial deformities. The study uses stereophotogrammetry to create a 3D image and then calculates an asymmetry index to classify faces as symmetric, sli…

This study looked at whether a special PET scan (18FDG dual-point) can tell the difference between real tumor growth and harmless inflammation caused by immunotherapy. Researchers studied 100 people with advanced lung cancer or melanoma who were being treated with drugs like nivo…

This study tests a new MRI technique called ENRAGED FLAIR that aims to capture two types of brain images (T2-FLAIR and T1-weighted) in a single sequence, instead of the usual two separate scans. Researchers will compare the image quality and time savings against standard MRI in 1…

Researchers are testing whether a handheld device that shines a special light on the inside of the mouth can help clinicians spot suspicious areas that might be precancerous or cancerous. The study will have three dentists with different levels of experience examine 200 adults, i…

This trial tests whether indocyanine green, a dye injected before surgery, can find sentinel lymph nodes as reliably as the standard technetium-99 radioactive tracer. About 744 adults with various cancers will receive both agents, and surgeons will compare detection rates using a…

This trial evaluates a new blood test (ELISPOT) that looks for immune cells in a newborn's cord blood that produce antibodies against the Toxoplasma parasite. The goal is to see if this test can diagnose congenital toxoplasmosis—an infection passed from mother to baby during preg…

This trial explores whether a standardized breakfast and a continuous glucose monitor can replace the traditional glucose drink test for diagnosing diabetes and prediabetes in people with Dunnigan's lipodystrophy, a rare genetic condition that causes severe insulin resistance. Th…

This study tests whether a new device that records chest vibrations from a distance can detect heart and lung diseases. Researchers will compare the vibration patterns of people with known cardiac or pulmonary conditions to those of healthy volunteers. If the technique works, it …

This trial tests whether a new hybrid tracer, ICG-99mTc-nanoscan, can replace the standard tracer for sentinel node biopsy in people with penile cancer, melanoma, or oral cancer. The hybrid tracer combines a fluorescent dye with a radioactive marker, potentially allowing surgeons…

This trial evaluates an artificial intelligence algorithm, AutoAS, that analyzes ultrasound images of the heart to detect aortic stenosis — a narrowing of the heart's main valve. Researchers will collect images from adults scheduled for a standard echocardiogram and test how well…

This study compares an artificial intelligence (AI) system called WebCeph with the traditional way orthodontists diagnose and plan treatment for misaligned teeth (malocclusion). Researchers will use routine dental records from 120 new patients aged 12 and older. Each case will be…

This trial tests whether a magnetic sensor placed on the skin can accurately measure blood pressure in the jugular vein and radial artery, compared to standard invasive methods using catheters. The study involves 10 adults with complex congenital heart disease or chronic right he…

This study tested whether a special MRI scan called T1 mapping can detect kidney damage in people with Fabry disease, a rare genetic disorder. Researchers compared MRI results from 70 adults with and without Fabry disease. The goal was to see if this non-invasive scan could help …

This study looked at 15 patients with a rare heart condition called wild-type transthyretin amyloid cardiomyopathy (ATTRwt) who also had signs of aortic stenosis. Researchers used dobutamine stress echocardiography—a test that uses a drug to make the heart pump harder while imagi…

This study tested 776 people with chronic pain of unknown cause to see if they had Fabry disease, a rare inherited disorder that can cause pain, kidney, heart, and nerve problems. Researchers used enzyme tests in men and genetic tests in women to find cases. The goal was to see h…

This study tested whether a special PET scan (called 18F-DOPA PET-CT) can find brain metastases as well as or better than standard MRI in people with lung cancer, breast cancer, or melanoma. 32 adults with untreated brain metastases took part. The goal was to see if this PET meth…

This study tested whether a tablet-based eye-tracking device (ETNA-MS) can accurately estimate disability levels in people with Multiple Sclerosis. Sixty adults with MS took eye-tracking tests, and the device's estimates were compared to standard neurologist assessments. The goal…

This study looked at whether newborns who fail their hearing screening can be diagnosed with congenital CMV infection and have a full hearing test before they turn one month old. Researchers tested 235 babies to see how quickly they could get results. The goal is to improve early…

This study tested whether an artificial intelligence (AI) system can help detect melanoma, a dangerous form of skin cancer. Researchers took photos of suspicious skin spots from 105 adults using a smartphone camera and let the AI analyze them. They compared the AI's results to di…

This study tested whether advanced ultrasound techniques can predict if an arteriovenous fistula (AVF) will work for dialysis in people with end-stage kidney disease. Researchers measured artery stiffness and blood flow in 76 patients before and after AVF surgery. The goal was to…

This completed study tested whether an AI device could help 16 doctors (10 general practitioners and 6 dermatologists) more accurately diagnose 13 different skin conditions, including melanoma, psoriasis, and acne. The doctors reviewed 29 medical images, first giving a diagnosis …

This study tested whether an artificial intelligence (AI) tool could help primary care doctors diagnose skin conditions more accurately. Nine doctors reviewed 30 images of various skin problems, first giving their own diagnosis and then a final diagnosis after seeing the AI's sug…

This study looked at whether a 6-minute walk test done over a short 6-meter distance is a valid and reliable way to measure walking ability in people with multiple sclerosis. Twenty-one adults with MS participated. The goal is to see if this shorter version works as well as the s…

This study tested a portable ultrasound device to measure bone thickness and porosity in 50 adults over 50 years old. The goal was to see if this radiation-free method could help identify people at risk for osteoporotic fractures as accurately as standard X-ray scans. The approac…

This study tested two different MRI sequences to see which one better detects active inflammatory lesions in the brains of people with multiple sclerosis (MS). 180 adults with known central nervous system inflammatory disease who needed an MRI after a recent clinical event took p…

Researchers are testing whether a six-month supervised exercise program can improve forearm muscle strength and support radial bone health in older adults living in nursing homes. Thirty-four participants are assigned to either an exercise group or a control group. The exercise g…

Very premature infants born before 28 weeks often have a blood vessel near the heart, called the patent ductus arteriosus, that stays open when it should close. A large opening can strain the heart and may affect brain development. In this trial, researchers use an early echocard…

This trial tests whether educating primary care health professionals can improve prevention of repeat fractures in older adults. It involves 40 primary health care centers in Stockholm, where some clinics receive special training and referral support for patients who have had a f…

This trial tests whether giving azithromycin—a common antibiotic—twice a year to young children in Niger can reduce deaths. It compares giving the drug to all children aged 1-59 months versus only to infants 1-11 months, who are at highest risk. The study also checks how these ap…

This study tests a program called FLARE that teaches families how to protect children from the sun. It includes children ages 8 to 17 who have a parent who survived melanoma and who have had at least one sunburn in the past year. Families are randomly assigned to either the FLARE…

This study tested whether a single infusion of zoledronic acid (Reclast) can prevent fractures in men and women aged 60 and older with Parkinson's disease or related conditions. Over 2,700 participants were randomly assigned to receive either the drug or a placebo, and were follo…

This study tested whether colchicine, an anti-inflammatory drug used for gout, can prevent new-onset atrial fibrillation and the need for a permanent pacemaker in people undergoing transcatheter aortic valve replacement (TAVI). About 120 older adults with severe aortic stenosis w…

This study tested whether a single infusion of zoledronic acid can prevent bone and muscle loss in adults aged 35 and older who are undergoing bariatric surgery. Fifty-nine participants were randomly assigned to receive either the drug or a placebo. The main goal was to measure c…

This study tested three methods of keeping teeth dry while applying protective sealants to prevent cavities in children's permanent molars. Thirty children at high risk for cavities took part, and each method was compared for how long it took, how well the sealant stayed on after…

This study tested whether a special sitting position that centers the hip joint can prevent hip dislocation in children with severe cerebral palsy. Seventeen children aged 1-6 years participated, using either the experimental or usual sitting method for at least 5 hours daily ove…

This study tested whether atenolol, a widely used beta blocker for heart conditions, can prevent bone loss in postmenopausal women. 420 healthy women aged 50-75 took either atenolol or a placebo daily for a period. The main goal was to see if the drug slowed bone density loss in …

This study tested a drug called conestat alfa in 141 people undergoing a minimally invasive heart valve replacement (TAVI) for severe aortic stenosis. The goal was to see if the drug could reduce the risk of small strokes and kidney damage that can happen after the procedure. Par…

This pilot study tested a virtual program designed to help older men at high risk for fractures reduce their risk of falls and broken bones. The program included personalized exercise plans delivered through a smartphone app and virtual nutrition counseling. Researchers enrolled …

This early-stage trial tested a new drug called rfhSP-D in 18 premature babies born before 30 weeks. The goal was to find the safest dose that might prevent chronic lung disease, a common complication in preemies. The drug was given along with standard care to babies on breathing…

Researchers are testing whether a combined exercise program can improve muscle thickness, balance, and fall risk in people with multiple sclerosis who also have sarcopenia, a condition involving loss of muscle mass and strength. Participants in the exercise group attend 60-minute…

Researchers are comparing two diets to see whether either one reduces fatigue in people with relapsing-remitting multiple sclerosis. Participants follow their usual diet for 12 weeks, then are randomly assigned to the Swank low-saturated-fat diet or the Wahls Elimination modified…

This study tested a new type of leg brace designed to help children with cerebral palsy and other movement disorders walk more easily. The brace was custom-made for each child and could be adjusted to provide different kinds of support. Researchers measured how well children walk…

Researchers are comparing two ways of delivering exercise therapy to people with multiple sclerosis: supervised sessions with a physiotherapist and a program followed over the internet. The study enrolls 34 adults with moderate to severe disability, measured by an EDSS score of 5…

Researchers are testing whether propionic acid, a short-chain fatty acid, can slow nerve damage and ease fatigue in people with multiple sclerosis. The trial enrolls 101 adults aged 18 to 60 who have stable MS and signs of immune activity in their spinal fluid. Participants recei…

This study tests whether a custom wrist brace made with 3D scanning and printing can improve quality of life for people with Ehlers-Danlos Syndrome (EDS) compared to standard braces. The brace aims to reduce pain, prevent joint dislocations, and help with daily activities like co…

This study tested whether providing overnight infant care and parenting support helps new mothers with substance use disorder living in residential treatment programs. 41 mothers participated, and researchers measured how many enrolled, completed the program, and gave feedback. T…

Newborns with complete bilateral cleft lip and palate often wear a custom molding appliance for a few months before surgery to bring the upper jaw segments closer together. This trial compares two versions: a vomer molding appliance and a conventional molding appliance. Researche…

Boys with hemophilia often miss out on games and activities because of concerns about bleeding and joint damage. Researchers developed a 12-week muscle-strengthening exercise programme with input from children, parents, and physiotherapists. The DOLPHIN-II trial compares this pro…

Fatigue is one of the most common problems in multiple sclerosis, and it can make walking, balance, and thinking harder. Researchers want to measure how fatigue changes these functions during a long walking test using wearable sensors. They are also testing whether a high-intensi…

Researchers at Ataturk University are testing whether watching nature videos through virtual reality glasses can improve sleep quality and mental well-being in people with multiple sclerosis. The trial enrolls 42 adults aged 18 to 80 who have MS and are not in an attack. One grou…

This pilot study tested whether a discreet, hands-free wearable breast pump could help Black mothers of critically ill infants in the NICU pump more often and produce more milk. Forty mothers were randomly assigned to use either the wearable pump with a tracking app or a standard…

Researchers are testing whether a hybrid cardiac rehabilitation programme can help adults recover after heart surgery. Participants start with two weeks of supervised exercise at the hospital, then continue training at home for six weeks using heart-rate monitors, a cloud care pl…

This trial tests whether an 8-week Clinical Pilates program can improve balance and physical function in adults with multiple sclerosis. Participants are randomly assigned to Clinical Pilates, standard physiotherapy, or a control group with no structured exercise. Researchers mea…

This study tests whether custom-made 3D-printed masks fit children better than standard commercial masks for non-invasive ventilation (NIV). Fifteen children aged 8-12 who already use NIV but have poor adherence will try a custom mask made from a 3D facial scan. Researchers will …

This study is testing a robotic lower-limb exoskeleton combined with functional electrical stimulation (FES) to help people with conditions like spinal cord injury, brain injury, or multiple sclerosis improve their walking. The device provides robotic support and electrical stimu…

This study tests whether a web-based training program helps people with COPD who use a noninvasive ventilator (NIV) at home stick to their therapy and sleep better. Participants receive online lessons on using the device, and researchers measure their compliance and insomnia seve…

This trial tests whether adding nalbuphine to a standard numbing medicine improves pain control and reduces agitation in children aged 1 to 7 after cleft palate repair. Half of the 60 participants receive the standard nerve block with bupivacaine alone, while the other half recei…

This pilot study tests whether a mindfulness program delivered through a web app can help hospitalized neurological patients and their caregivers cope with stress and anxiety. Participants learn mindfulness techniques in an initial face-to-face session, then practice via weekly o…

This trial tests HEARTPrep, a virtual counseling program designed to support mothers who are expecting a baby with congenital heart disease. The program aims to reduce depression, anxiety, and social isolation during pregnancy. Researchers will enroll 30 mothers and their partner…

This trial tests whether a structured patient education program can lower the risk of severe immune-related side effects in people receiving immunotherapy for certain cancers, including melanoma, lung, kidney, and head and neck cancers. Participants either attend five educational…

This early-phase trial is testing whether an experimental form of radiation therapy, called FLASH, can safely treat skin lesions from metastatic melanoma. FLASH delivers radiation in a fraction of a second, which may spare healthy tissue while still targeting tumors. The study wi…

This study tests whether a new high-intensity exercise program is feasible and can improve dynamic balance and physical activity in adolescents with cerebral palsy or spina bifida. Participants will perform a series of exercises twice a week, with researchers measuring walking sp…

This study tested whether a low-histamine version of the Mediterranean diet could help people with relapsing-remitting multiple sclerosis (MS) feel better. 51 adults were split into three groups: one followed the low-histamine Mediterranean diet, another a standard Mediterranean …

This trial compares two simple educational tools—an animated video and a brochure—against routine care to see which best lowers anxiety and fear in children aged 7 to 10 who need an echocardiogram. All participants have a congenital heart condition and are scheduled for a follow-…

This study tested whether a high-intensity virtual reality boxing program is safe and feasible for people with multiple sclerosis. Five participants used a VR headset to exercise for 10 minutes twice a week over 8 weeks, alongside their usual therapy. Researchers measured side ef…

This trial compares two exercise programs—chin tuck against resistance (CTAR) and proprioceptive neuromuscular facilitation (PNF) neck stretches—for people with multiple sclerosis who have trouble swallowing. Over 8 weeks, participants perform supervised sessions three times a we…

This trial tests whether a structured memory training program, called Strategy-Based Training to Enhance Memory (STEM), can improve learning and recall in people with multiple sclerosis who report memory difficulties. Participants attend eight training sessions over four weeks an…

This study tested a robotic exoskeleton called Atalante to see if it is easy and safe for people with multiple sclerosis (MS) to use. 15 adults with MS who had trouble walking tried the device. The goal was to see if the suit could help them walk better and if they liked using it…

This study tests whether a wheelchair-mounted robotic arm called Jaco helps people with tetraplegia perform daily tasks like grasping and reaching at home. Participants use the device for two months after training. Researchers measure changes in performance and satisfaction with …

This study tested whether listening to music and applying acupressure before needle insertion can reduce pain in dialysis patients. 72 participants were split into four groups: music plus acupressure, acupressure alone, music alone, or standard care. Pain was measured during thre…

This study tested whether adding probiotics (found in yogurt and yeast tablets) to standard therapy could improve fatigue, quality of life, mood, and inflammation in 60 people with relapsing-remitting multiple sclerosis. Participants were randomly assigned to take probiotics or n…

This study looked at three different ways to manage pain after abdominal wall repair surgery: an epidural, a nerve block, and local injections. 160 adults who had this surgery were randomly assigned to one of the three methods. The goal was to see which approach provides the best…

This study looked at whether 8 weeks of low-load leg exercises with a blood flow restriction cuff is safe and doable for people with multiple sclerosis who have moderate to severe walking problems. Sixteen adults with MS took part, doing exercises like knee extensions and hip lif…

This study tested whether wearing virtual reality goggles during the needle stick for dialysis can lower pain and anxiety. Fifty adults on hemodialysis were randomly assigned to either watch VR content during the procedure or receive usual care. The results will show if a simple,…

This completed pilot study looked at whether a placebo treatment — sham TENS combined with positive verbal suggestions — could reduce fatigue in people with relapsing-remitting multiple sclerosis. 36 participants were enrolled. The main goal was to measure motor performance (numb…

This study explores whether a powered exoskeleton—a wearable robot—can help people with moderate to severe walking problems due to multiple sclerosis (MS) exercise and walk better. Twenty-four participants will either use the exoskeleton or do standard exercises with a fitness in…

This study looked at 40 infants under 1 year old having kidney surgery. They received a pain block with either bupivacaine alone or bupivacaine plus ketamine. The goal was to see if adding ketamine reduces pain and the need for extra pain medicine after surgery.

This early-phase study tested a freezing technique called cryoablation on 14 adults with painful venous malformations that had come back after standard treatments like surgery or sclerotherapy. The main goal was to check safety and see if it could ease pain and discomfort. Result…

This study tested whether plasma exchange (a blood-filtering treatment) helps people with multiple sclerosis who have severe flare-ups that don't get better with steroids. Thirty-eight adults took part, and researchers compared real plasma exchange to a sham procedure. The main g…

This study tested whether L-carnitine, a natural supplement, can reduce fatigue in people with multiple sclerosis (MS). About 60 adults with MS who had significant fatigue took either L-carnitine or a placebo, then switched. The goal was to see if L-carnitine improved fatigue sco…

This study tested two numbing methods for a common clubfoot procedure in infants: a needle-free jet injection that works in 1-2 minutes versus a topical cream that takes 30 minutes. Researchers measured pain using a standard scale, heart rate, and oxygen levels in 107 babies. The…

This study tested whether acupressure, a technique where pressure is applied to specific points on the body, can help people with multiple sclerosis (MS) manage pain, fatigue, and sleep problems. Researchers enrolled 90 adults with relapsing-remitting MS who had moderate to sever…

This study looked at whether different types of exercise training can help adults with congenital heart disease exercise more easily. 47 participants were split into three groups: interval training, moderate continuous training, or usual care (no training). The main goal was to s…

This study tested whether doing handgrip exercises before and after surgery can improve the size and function of dialysis fistulas in people with advanced kidney disease. 40 patients participated, and the main goal was to see if the fistula diameter increased. The approach is sim…

This study tested a new device called VIVHEART EasyCross™ in 20 people undergoing transcatheter aortic valve replacement (TAVR). The device uses a basket-like tip to center itself in the artery, making it easier and quicker to guide a wire through the heart valve. The goal was to…

This study tested whether N-acetylcysteine (NAC), an over-the-counter antioxidant, could improve learning and motor skills in children with neurofibromatosis type 1 (NF1). Five children aged 8 to 16 took NAC or a placebo to see if it helped with movement and attention problems. T…

This study tested two types of nerve stimulation—one using tiny needles (PENS) and one using skin pads (TENS)—to see if they could improve hand function in people with multiple sclerosis. 28 participants received a single session of stimulation, and their hand dexterity, strength…

This completed Phase 2 trial tested whether blood thinners (rivaroxaban) or antiplatelet drugs (aspirin, clopidogrel) work better than standard migraine medication (metoprolol) for people with both migraine and a small heart hole called PFO. 1,000 participants took one of the fou…

This study tested whether partial-body cryotherapy (standing in a very cold chamber for 2-3 minutes) could help reduce fatigue in people with multiple sclerosis during a rehabilitation stay. 43 participants received either real cryotherapy at -120°C or a sham version at -30°C ove…

This study tested a powered leg exoskeleton (Keeogo) in 50 people with various muscle disorders to see if it is safe and helps them walk better. Participants performed walking tests with and without the device. The goal was to see if the device could improve walking distance and …

This study tested a wearable robotic suit called MyoSuit that assists knee and hip movement in people with various muscle disorders. 32 participants used the device to perform walking tests, and researchers checked for safety and any immediate improvements in walking ability. The…

This study looked at whether a single intravenous iron dose could improve exercise capacity in people with severe aortic stenosis and iron deficiency who were scheduled for a valve replacement. About 100 participants received either iron or a placebo. The main goal was to see if …

This study tested a digital group program called 'Fewer Falls in MS' for people with multiple sclerosis who had fallen in the past year. 240 participants either joined the 8-session online program or received a brochure. Researchers tracked falls and other outcomes over 12 months…

This study tested whether wearing compression garments and doing specific physiotherapy exercises could improve balance and quality of life in people with hypermobile Ehlers-Danlos syndrome (hEDS). Forty patients with hEDS were followed for a year, first receiving standard care, …

This study tested whether listening to positive affirmations along with classical music during a cesarean section could lower anxiety and improve breastfeeding. 99 women were split into three groups: one listened to affirmations with music, one listened to music only, and one had…

This study tested two online programs designed to help people with multiple sclerosis manage spasticity (muscle tightness) in their legs. Participants attended two video classes that included education and stretching exercises. The goal was to see if these programs could reduce t…

This study tested a gel called ZYN002, applied to the skin, in 20 children and teens (ages 4-17) with 22q11.2 deletion syndrome. The main goal was to see if the gel is safe and tolerable over 38 weeks. Researchers also looked at whether it could improve behavior and other symptom…

This study looked at whether adding cognitive behavior therapy (a type of talk therapy) to supervised Pilates exercises could improve balance, walking, and fatigue in people with multiple sclerosis. 90 adults with mild to moderate MS took part. The goal was to see if combining me…

This study tested whether adapted judo training plus nutritional counseling could improve physical fitness, body composition, and stability in children and adolescents with autism spectrum disorder (ASD). One hundred participants aged 7–14, with and without ASD, took part in 10 m…

This study tested whether breathing in lavender essential oil for 15 minutes before skin cancer surgery could reduce anxiety. Sixty-six adults with early-stage melanoma or other skin cancers took part. Researchers measured anxiety using questionnaires and vital signs like heart r…

This study tested whether a nerve block called erector spinae plane block (ESPB) can reduce pain and the amount of anesthesia needed during and after tethered cord surgery in children. The trial involved 73 children aged 18 and under. The goal was to see if the block lowers pain …

This study tested a mobile app called SurgeryPal that teaches teens coping skills before and after major spine surgery. 433 teens aged 12-18 were randomly assigned to use the app or receive standard education. The goal was to see if the app could reduce pain and prevent pain from…

This study tested whether a safe, non-invasive brain stimulation technique called transcranial direct current stimulation (tDCS) could reduce fatigue in people with multiple sclerosis (MS). Ten adults with MS-related fatigue used a home-based tDCS device. The goal was to see if i…

This study tested whether a low dose (4.5 mg) of naltrexone, a drug already used for alcoholism, can improve physical health and quality of life in people with vasculitis. Sixty participants took either the drug or a placebo daily for six weeks. The goal was to see if the drug le…

This study tested a cream made from andaliman fruit extract on 40 adults with seborrheic keratosis, harmless skin growths on the face. Participants applied the cream daily for 12 weeks, and researchers checked for changes in the growths using a special camera. The goal was to see…

Researchers are examining skin cancer among people whose jobs involve long hours in the sun. The study uses national administrative and medical records to describe who develops these cancers, including age, sex, region, cancer type, and disability outcomes. The goal is to underst…

Children with growth hormone deficiency often need daily injections, which can be hard to keep up with. Researchers in Belgium and Luxembourg are asking families in the BELGROW registry to fill out a questionnaire about whether they would consider switching to long-acting growth …

Children with congenital heart disease often need a breathing machine after surgery, and the pressure it creates can affect blood flow to the brain. This study compares conventional ventilation with NAVA, a mode that works with the child's own breathing signals, in 30 children re…

Researchers are studying whether certain immune substances in the blood are linked to physical and mental abilities in people with relapsing-remitting multiple sclerosis. The study includes 20 adults aged 19 to 45 with mild disability. Participants give blood samples, and researc…

This early-phase study tested whether ferumoxytol, an iron-based contrast agent, can help MRI scans better detect inflammation in the brains of people with multiple sclerosis (MS). Fourteen participants (some with MS, some healthy) received a single IV dose of ferumoxytol and had…

Researchers are studying whether adults with Dunnigan lipodystrophy, a rare genetic condition that changes how the body stores fat, have higher levels of inflammation than people without it. The trial enrolls 64 non-diabetic adults on Reunion Island, including those with the cond…

This study tested whether two light-based technologies, fNIRS and DCS, can safely measure brain activity in children with rare neurocognitive disorders like Niemann-Pick disease and Smith-Lemli-Opitz syndrome. 73 participants, including healthy volunteers, wore a cap with lights …

This study looked for genetic causes of congenital heart disease (heart problems present at birth) in families from Uganda and Nigeria. Over 1,200 people took part, including affected children and their parents. Researchers used advanced DNA testing to find gene changes linked to…

This study looked at how blood vessel stiffness affects organs like the heart, brain, kidneys, and gut in people with Williams Syndrome (WS) or supravalvular aortic stenosis (SVAS). Researchers compared 159 participants (ages 3–85) with these conditions to healthy volunteers over…

This study followed 17 adults with neurofibromatosis type 1 (NF1) over two years to learn how their skin tumors (dermal neurofibromas) grow and change. Researchers used special cameras and skin biopsies to measure tumor growth and look for genes that might influence it. The goal …

This study looked at Smith-Lemli-Opitz syndrome (SLOS), a rare genetic condition that affects cholesterol production and causes birth defects and intellectual disabilities. Researchers enrolled 130 patients and their mothers to learn more about the disease's progression, genetic …

This study looked at people with three rare genetic conditions—xeroderma pigmentosum (XP), Cockayne syndrome (CS), and trichothiodystrophy (TTD)—that affect the body's ability to repair DNA. Researchers examined 709 participants to understand how these defects relate to cancer ri…

This study checks whether an artificial intelligence (AI) system trained on Turkish patients can accurately diagnose jaw and bite problems in people from other ethnic backgrounds. Researchers will compare the AI's performance on 5,000 patients from Turkey and Belgium using photos…

Researchers want to learn which molecules on cancer cells the immune system attacks when checkpoint inhibitor drugs work well. They collect tumor and blood samples from adults with solid tumors who respond to these drugs, either during treatment or after surgery. The team then se…

This study collects tissue and blood samples from 300 people with autoimmune diseases like Crohn's, multiple sclerosis, and celiac disease. Researchers aim to identify the specific targets of immune cells (T cells) that cause inflammation. The goal is to discover new targets for …

This study observes how doctors decide when to remove breathing tubes (extubation) in extremely preterm infants born before 28 weeks. Researchers will track 100 babies in the NICU, asking doctors daily why they choose to extubate or not. The goal is to understand current practice…

Researchers at Nantes University Hospital are studying whether nerves feel stiffer in people with three rare peripheral neuropathies: CIDP, CMT1a, and anti-MAG neuropathy. They use ultrasound shear wave elastography to measure nerve stiffness in 36 participants aged 18 to 65 and …

This study is testing whether a green dye called ICG can help surgeons see tumors more clearly during surgery in children and teens with solid tumors. The dye is injected before surgery and makes tumor tissue glow under a special camera. The goal is to see if this technique helps…

Animal-derived heart valves replace damaged human valves, but many fail within about a decade. Researchers suspect the recipient's immune response may speed up that breakdown. This international study follows people who receive pig, cow, or horse tissue valves, using echocardiogr…

Researchers want to know whether the number of other health conditions a person has affects their quality of life after transcatheter aortic valve implantation. The study follows 150 patients aged 75 and older who receive a new aortic valve through a catheter. Before the procedur…

Researchers are tracking birth outcomes for infants born in Eswatini to mothers who took dolutegravir or other HIV drugs around the time of conception. The study compares rates of neural tube defects and other birth defects among babies born to HIV-positive women on dolutegravir,…

This study investigates whether balance problems explain why fatigue leads to a greater fear of falling in people with multiple sclerosis (MS). Researchers will assess fatigue, balance, and fear of falling in 40 adults with MS who can walk independently or with a device. The goal…

Researchers are following 110 people aged 1 to 65 who have homocystinuria caused by a missing or faulty CBS enzyme. The study observes how the condition changes over time under standard care, without testing any new treatment. Participants give blood samples, complete quality-of-…

This study follows nearly 600 people with Smith-Magenis syndrome (SMS), a rare genetic condition, to track how their health, behavior, and development change over time. Researchers will perform detailed medical exams, genetic tests, and surveys to better understand the syndrome's…

People with multiple sclerosis often do not report side effects from their oral disease-modifying drugs, which leaves safety monitoring incomplete. This trial compares two ways of encouraging reports: a nurse who trains the patient and follows up by phone over six months, versus …

This study followed 180 adults with syringomyelia, a condition where a cyst forms in the spinal cord, for up to 5 years. Researchers tracked symptoms, muscle strength, and MRI scans to learn how the disease progresses and how surgery affects outcomes. The goal was to gather knowl…

This study looked at 47 people with bullous pemphigoid, a blistering skin condition, who were treated with high-dose topical corticosteroids (clobetasol propionate) for 6 months. Researchers measured bone mineral density at the start, at 3 months, and at 6 months to see if the tr…

This study looked at the genetics of Chiari I malformation, a condition where part of the brain pushes into the spinal canal. Researchers analyzed DNA and family histories from 152 participants to find genes that may cause the malformation. The goal was to better understand why i…

This trial tests whether a single dose of the antipsychotic drugs olanzapine or ziprasidone changes how the body responds to insulin, compared with a placebo. The study enrolls healthy young men who are overweight or have early signs of insulin resistance, but who have never take…

This study investigates whether people with multiple sclerosis experience kinesiophobia, a fear of movement, and how it relates to fatigue. Researchers will assess 352 adults with MS using scales for disability, spasticity, walking speed, pain, sleep, anxiety, and depression. The…

This study looked at two community-based wellness programs for adults with disabilities like spina bifida, cerebral palsy, spinal cord injury, and traumatic brain injury. Researchers measured changes in health, mood, quality of life, and healthcare costs over up to 24 months. The…

This study followed 46 children with osteogenesis imperfecta (OI), a rare connective tissue disorder that causes frequent fractures and short stature. Researchers collected medical history, physical exams, hearing and dental tests, X-rays, bone scans, and genetic samples over sev…

This study is testing whether two dietary supplements, Goodphyte IB Defense and Goodphyte Immunity, are acceptable and easy for adults with chronic diseases like IBD, hypertension, anemia, or multiple sclerosis to take. Researchers will also check if the supplements improve how w…

This study is creating a large network of hospitals across Italy to improve the diagnosis and understanding of rare cerebrovascular diseases (rCVDs), such as CADASIL, Fabry disease, and Moyamoya. Researchers will collect medical information and blood samples from 500 patients to …

This completed study by the National Human Genome Research Institute looked at 207 people with Moebius syndrome or similar congenital facial weakness disorders, along with their family members. Researchers aimed to better understand the genetic and clinical features of these cond…

This study looks at people with neurofibromatosis type 1 (NF1) and their families to find out why some have more severe symptoms than others. Researchers will collect medical history, blood samples, and images to study genes and physical traits. The goal is to identify genes that…

Researchers are asking whether people with multiple sclerosis who have trouble reading emotions also face more difficulties in family life, friendships, and social integration. The study compares three groups: MS patients with emotional processing problems, MS patients without su…

This trial tests whether a 15-minute educational video and a list of suggested questions can improve understanding of immunotherapy among patients with advanced cancer and their caregivers. Participants are randomly assigned to receive the educational materials or usual care, and…

This study looked at 10 children born with a missing forearm who used a bionic Hero Arm for one year. Researchers measured how well the children could do everyday tasks and how satisfied they were with the device. The goal was to see if the prosthesis helped with activities like …

This study looked at 166 older adults (65+) on hemodialysis to compare three types of vascular access: fistulas, grafts, and catheters. The goal was to see which option leads to fewer complications and better quality of life. Researchers tracked how many procedures were needed to…

This trial is testing whether contrast-enhanced ultrasound (CEUS) can reliably measure tumor blood flow in people with metastatic cancers like GIST, breast, kidney, colon, rectal, or melanoma. Participants will have two ultrasound scans before starting anti-angiogenic treatment, …

This study looks back at the medical records of 244 adults in France with hereditary hemorrhagic telangiectasia (HHT), also called Rendu-Osler disease, who were treated with the drug bevacizumab. The goal is to describe how the drug was used and to measure its impact on bleeding,…

This study investigates why some girls with Turner syndrome find it hard to make friends, read emotions, and control their feelings. Researchers will compare 35 girls with Turner syndrome to 35 girls with isolated growth hormone deficiency, all aged 7 to 16. Through puzzles, memo…

This trial tests whether an online training course, called MelaMEd, can improve primary care doctors' ability to detect and manage melanoma, the most serious form of skin cancer. Participants—general practitioners, trainees, and pediatricians—answer questions and review skin lesi…

This study looks at how leg muscle size and strength affect circulation in people born with a single-chamber heart, a condition where the heart can't pump blood to the lungs on its own. Researchers will measure muscle mass, oxygen use, and quality of life in 40 adults aged 16-50 …

This study follows people with relapsing-remitting multiple sclerosis who are starting immunosuppressant treatment. Researchers will track changes in cognitive abilities, fatigue, emotions, and overall quality of life over an extended period. The goal is to better understand how …

This study follows 66 people with primary progressive multiple sclerosis (PPMS) and healthy volunteers for two years to understand how thinking and memory change over time. Participants take cognitive tests and undergo MRI scans to see how brain tissue damage relates to mental de…

This trial investigates whether a short stay at high altitude (up to 3,545 meters) is safe for people with asymptomatic moderate or severe aortic stenosis. Participants will undergo heart tests—including ECG, echocardiography, and exercise stress tests—both at low altitude and af…

This study looks at how well joints like the shoulder, knee, ankle, elbow, and wrist work after orthopedic surgery in people with Ehlers-Danlos syndrome, a condition that affects connective tissue. Researchers are using standard joint function scores to measure outcomes in 200 ad…

This study tests whether a remote monitoring program can reliably track the health of people who have had surgery for pheochromocytoma or paraganglioma (PPGL), rare adrenal tumors. Participants use an internet platform to report their data, which is compared to information collec…

This study uses French national health data to compare the risk of major heart problems—like heart attack, stroke, and death—in people with Takayasu arteritis versus similar people without the disease. It also looks at how steroid use may affect these risks. The goal is to better…

This study follows thousands of people with multiple sclerosis (MS) in the UK to see how COVID-19 affects them. Researchers track infections, hospital stays, and deaths using questionnaires and national health data. The goal is to understand the risks and outcomes for this group,…

This study looked at whether asking people with multiple sclerosis (MS) to do two things at once — like walking while thinking — can uncover balance problems that standard tests miss. Researchers studied 24 people with mild MS and low disability. The goal was to see if this dual-…

This study enrolled 500 skin cancer patients in China and analyzed over 1.2 million of their social media posts using artificial intelligence. The goal was to see if AI could detect signs of psychological distress and suicidal thoughts. Researchers hope this approach could lead t…

This completed study looked at 40,000 people with metastatic cancer who received the drug nivolumab. Researchers compared the old way of dosing (based on body weight) with a newer, simpler fixed dose. They used national health data to see if the fixed dose was just as good and mo…

This study tested whether a newer type of MRI called 4D flow can more accurately measure leakage in the pulmonary valve and heart chamber size in people born with tetralogy of Fallot who had corrective surgery. Researchers compared 4D flow MRI with standard 2D MRI in 21 patients …

This study tested whether giving personalized nutrition training to mothers of children with congenital heart disease helps their kids grow better after heart surgery. The training covered feeding from birth to age 3, including breastfeeding and starting solid foods. Researchers …

This completed study looked at 44 patients who had lung cancer surgery to understand how the arteries in the lungs can vary from person to person. Using CT scans and 3D models, researchers mapped these variations to help surgeons avoid complications during lobectomy. The goal was…

This study investigates why people develop obstructive sleep apnea (OSA) and whether the underlying cause affects how they respond to different treatments. Researchers will test three approaches—CPAP, a sleep medication (eszopiclone), and supplemental oxygen—in adults with OSA. T…

This study follows 150 people with bicuspid aortic stenosis—a narrowed heart valve present from birth—who receive a transcatheter aortic valve replacement (TAVI) using the Evolut Pro or Evolut R XL device. Researchers will measure how well the valve works over time, including blo…

This study examined whether a marker of cellular aging called p16Ink4a is linked to bronchopulmonary dysplasia (BPD), a serious lung disease in premature babies. Researchers measured p16 levels in blood from 80 newborns and children, comparing those with and without BPD. The goal…

This study followed 115 people with relapsing-remitting multiple sclerosis for up to 5 years to see if early damage to the spinal cord could predict later walking difficulties. Researchers used MRI scans and walking tests to track changes. The goal was to better understand how ea…

This study observed 30 elderly patients (over 65) who had a specific type of neck fracture (odontoid fracture) from a minor fall or low-energy injury. Researchers measured bone density, vitamin D levels, and other bone health markers to understand why these fractures happen. They…

This study explored whether lab-grown skin models containing a patient's own melanoma cells could predict which drug combinations would work best. Researchers took a small sample of a skin metastasis and used it to create 3D cultures and artificial skin. The goal was to test diff…

This study tested a simple flowchart to help doctors monitor and manage heart conduction problems after transcatheter aortic valve replacement (TAVR). The goal was to see if this approach could safely reduce the need for intensive care and permanent pacemakers. Researchers follow…

This study compared two methods for measuring bioavailable testosterone in the blood: the standard lab test (radioimmunoassay) and a mathematical calculation. Researchers included 270 men and women aged 18 to 90. The goal was to see if the calculation matches the lab test closely…

This study looked at how people with multiple sclerosis and their natural caregivers (like family members) feel about the quality of care they receive. Researchers surveyed over 1,000 participants to understand their expectations and experiences. The goal was to create a tool tha…

This study looked at how patients and healthcare professionals experienced a new way of organizing primary care, where different health workers work together as a team. Researchers interviewed 48 patients and staff at one health centre in France. The goal was to understand what w…

This study used advanced MRI scans to look at energy use in the brains of 40 people with multiple sclerosis and 15 healthy volunteers. The goal was to see if energy problems in brain cells can predict nerve damage over two years. Researchers hope this will help understand how MS …

This study tested whether using a pulse oximeter (a small sensor on the foot or hand) to measure oxygen levels in all seemingly healthy newborns can help find hidden, life-threatening heart defects early. Over 44,000 babies took part. The goal was to see if this screening reduces…

This study looked at bone density in 448 men with hemophilia and healthy volunteers to see how common bone loss is. Participants had a bone density scan and some gave blood samples. The goal was to understand if hemophilia itself, not just lack of activity, leads to weaker bones.

This study looked at how blood vessel function relates to heart blockage in people with a genetic heart condition called hypertrophic cardiomyopathy. Researchers measured blood flow and vessel flexibility in 40 adults. The goal was to understand the connection, not to test a trea…

This study looked at 320 people whose brain scans showed signs that sometimes lead to multiple sclerosis (MS), even though they had no symptoms. Researchers analyzed blood and spinal fluid samples to find biological markers that could predict whether these silent signs would turn…

This study followed 307 people with relapsing multiple sclerosis in Germany who had recently switched to the drug Kesimpta (ofatumumab) from another treatment. Researchers collected information on why patients made the switch and how many doses they missed over one year. The goal…

This study tested 137 people with chronic pain of unknown cause to see if they had Fabry disease, a rare genetic disorder. Researchers used blood tests to check for the condition. The goal was to find out how common Fabry disease is in this group and help diagnose it earlier.

This study examined spinal fluid from 32 children with tethered spinal cord to see if inflammation plays a role in the condition. Researchers collected fluid samples during surgery and looked for markers of inflammation. The goal was to better understand how the spinal cord gets …

This study followed 220 adults with phenylketonuria (PKU) in France to learn how the disease affects them later in life. Researchers looked for signs of cognitive decline, neurological problems, and how the disease impacts quality of life and social integration. No new treatment …

This study looked at how hormones and birth control pills affect knee ligament looseness in healthy women. Researchers measured knee laxity in 45 women at two points in their menstrual cycle, comparing those on hormonal contraceptives to those not. The goal was to better understa…

This study investigates whether changes in DNA methylation—a kind of chemical switch—might cause certain rare limb malformations that appear without a family history. Researchers will compare DNA samples from 40 people with Amelia (missing two or four limbs) or Femur Fibula Ulna …

This study tested a new way to predict dangerous heart rhythm problems after a common heart valve procedure called TAVR. Researchers combined ECG results with CT scan measurements and implant depth to see if they could safely identify low-risk patients. The study looked back at 2…

This completed study enrolled 33 people with osteogenesis imperfecta (OI), a condition that makes bones fragile. Researchers used special sensors and gait analysis to measure how participants move. The goal was to identify which movement patterns are most useful for understanding…

This study looked at how certain heart devices change a blood-clotting protein called von Willebrand factor. Researchers studied 500 adults with heart failure or aortic stenosis who received a mechanical heart pump or a valve replacement. The goal was to understand when these pro…

This study looked at 31 people with a type of jaw misalignment called skeletal Class III deformity. Researchers compared how the face's soft tissues changed after two types of jaw surgery: a minimally invasive approach and a conventional one. Using 3D imaging, they measured diffe…

This study looked at how teriparatide (Forsteo), a daily injection, builds new bone in postmenopausal women with osteoporosis. Nineteen women took the drug for two years. The goal was not to test if it works, but to understand exactly how it changes bone density and turnover over…

This study looked back at 37 patients who had surgery for webbed fingers (congenital syndactyly) between 2005 and 2019. The goal was to see how well the Gilbert dorsal commissural flap worked, focusing on whether the web space tightened up again and how the scars healed. Doctors …

This completed study looked at 75 people with relapsing-remitting multiple sclerosis (RRMS) who were taking the medication Aubagio. The goal was to see if virtual reality tests and real-world tasks could better detect cognitive (thinking) problems in everyday life compared to sta…

This completed study examined 127 melanoma patients to understand how the IL4I1 enzyme influences disease progression and resistance to treatments like immunotherapy and targeted therapy. Researchers analyzed blood and tumor tissue samples before and after treatment. The goal was…

This study looked at how the way words sound (phonotactic probability) and how they are spelled (orthography) affect vocabulary learning in 23 children with low oral language skills (DLD). All children took part in the same 12-week program, which used real words to improve vocabu…

This study tracked 300 patients with severe aortic stenosis who had a minimally invasive heart valve replacement (TAVI) to see how long they stayed in the hospital and why some stayed longer than 3 days. The goal was to find out what factors, like needing a blood transfusion or a…

This study tested a short, computerized cognitive assessment in 421 French-speaking adults with multiple sclerosis and healthy controls. The goal was to see if a brief battery of computer tests can reliably detect early cognitive issues, like slowed thinking and memory problems, …

This study followed 59 nurses during their first year of work to see if night shifts harm bone health. Researchers measured bone density and markers of bone turnover in night shift nurses compared to day shift nurses. The goal was to understand if shift work increases the risk of…

This study looked at how nerve cells in the eye are lost in people with multiple sclerosis (MS) even when there is no active inflammation. Researchers followed 64 adults with relapsing-remitting MS who were already part of a larger study. By using special eye scans over time, the…

This study used brain scans to understand why some people with multiple sclerosis have trouble with social thinking, like reading emotions or intentions. Researchers compared 35 MS patients to healthy volunteers using MRI while they performed social tasks. The goal was to see whi…

This study screened 233 children aged 6 to 66 months with congenital heart disease for neurodevelopmental disorders like delays in communication, movement, and problem-solving. Researchers used a parent questionnaire to identify children at risk. The goal is to better understand …

This study looked at how obesity and related metabolic changes affect bone development in 358 healthy girls aged 9-12. Researchers measured bone mass, density, and strength over two years. The goal is to understand these links to help prevent fractures and osteoporosis later in l…

This study looked at how Hirschsprung disease, a rare birth defect affecting the bowel, impacts brain development and quality of life in children aged 8 to 13. Researchers tested thinking skills and surveyed families to understand long-term effects after surgery. The goal was to …

This study looked at whether a food safety training program helps food service employees improve their knowledge, attitudes, and practices around safe food handling. About 200 adult workers took part, and the training covered personal hygiene and safe food preparation. The goal w…

This study examined a type of immune cell called 4BL B cells in people with multiple sclerosis (MS) and compared them to healthy controls. Researchers wanted to see if these cells are more common in MS and might contribute to inflammation. The study involved 125 participants and …

This completed study collected blood and urine samples from 240 people with ciliopathies—rare genetic disorders that often lead to kidney failure. Researchers analyzed these samples to find biological markers that could predict how the disease will progress. The goal is to develo…

This study tested whether a family history app can help doctors collect and use information about patients' family health risks. Over 600 adults aged 30-69 took part. The goal was to see if the app leads to better screening and genetic referrals for conditions like cancer, heart …

This study examined how indoor air quality impacts breathing symptoms and lung function in 165 school-aged children (6-12 years old) who were born prematurely and have bronchopulmonary dysplasia (BPD), a chronic lung disease. Researchers measured respiratory symptoms and lung fun…

This study tested how well an Apple Watch measures heart and fitness data compared to standard medical equipment. About 50 adults with heart conditions or athletes wore the watch during exercise. The goal was to see if smartwatches can reliably help monitor heart disease.

This study looked at whether a special MRI scan (4D Flow) can accurately measure blood flow to the lungs in people with both congenital heart disease and pulmonary hypertension. The standard test for this is a heart catheterization, which is invasive and uncomfortable. The study …

This completed study involved 116 people with advanced melanoma. Researchers aimed to create a platform that goes beyond standard genetic testing to deeply profile tumors using advanced molecular technologies. The goal was to discover new biomarkers and help doctors make better t…

This study looked at whether adding hands-on simulation training to standard classroom teaching helps nurses cause fewer problems when inserting needles into the arm access (arteriovenous fistula) of dialysis patients. About 9600 adults on chronic hemodialysis took part. The goal…

This study looked back at the records of 1,000 adults who came to the emergency room with very low sodium levels. The goal was to find out how many had brain swelling from the low sodium itself, and how many later developed nerve damage from correcting the sodium too quickly. Res…

This study looked at whether certain gene variations affect how well a transplanted kidney works over time. Researchers checked 80 kidney transplant recipients for specific gene markers and measured their kidney function for up to three years. The goal was to find clues that coul…

This study used a special eye camera (adaptive optics) to look at blood vessel inflammation in the retina of 50 people with multiple sclerosis (MS) and healthy volunteers. The goal was to see if this inflammation differs between MS types and could serve as an early marker of dise…

This study looked at whether the difference between how long it takes someone to actually perform a 'get up and walk' test versus how long they imagine it takes can predict future falls. Researchers followed 67 osteoporosis patients for a year after the tests. The goal was to see…

This completed study looked at 361 people recently diagnosed with multiple sclerosis to see if experiencing 'flow' (being fully absorbed in an activity) and mindfulness are linked to better mental health. Participants filled out online questionnaires about their mental health, an…

This study looked at over 2,200 people with hereditary hemorrhagic telangiectasia (HHT), a rare condition that causes abnormal blood vessels. The goal was to find out what makes some people with HHT more likely to have a brain bleed. Researchers collected health information, bloo…

This study looked at 541 premature babies with a heart condition called patent ductus arteriosus (PDA). Half received ibuprofen to close the vessel, while the other half got only supportive care like fluids or diuretics. Researchers compared closure rates and complications to see…

This study involved 60 people with advanced melanoma who were already scheduled to receive immunotherapy drugs. Researchers collected blood samples and, when possible, tumor samples before and during treatment to look for biological markers that might predict whether the treatmen…

This study looked at whether eating only during a 9-hour window each day (time-restricted eating) affects bone health, weight, and gut bacteria in overweight or obese older women. 49 postmenopausal women were randomly assigned to either eat within a 9-hour window or a 12-hour win…

This study looked at 70 adults having open heart surgery (aortic valve or bypass). It compared a newer, smaller heart-lung machine to the standard one. The goal was to see which method better protects the body's tissues and the brain during surgery. Results will help doctors choo…

This study looked at why girls with Rett syndrome often develop weak bones (osteoporosis) at a very young age. Researchers checked bone density, looked at medical history, and measured certain proteins in the blood. The goal was to find risk factors and understand the biological …

This study looked at how families are told their child has craniosynostosis, a condition where skull bones fuse too early. Researchers interviewed and surveyed 574 parents and children to understand the emotional impact and improve the diagnosis process. The goal is to make the a…

This completed study tested whether giving families of children with long-term illnesses (like cancer, heart disease, or premature birth) a tablet computer with a special app helps them manage care at home. Researchers measured satisfaction, cost-effectiveness, and how well famil…

This completed study looked at 86 patients who had a fracture possibly due to weak bones (osteoporosis). Researchers wanted to see if the calcium supplements patients were prescribed matched what they actually got from food. Many patients get calcium pills even if their diet alre…

This study reviewed medical records of 901 infants who had surgery for aortic coarctation (a narrowed aorta) before age one. Researchers wanted to find out how many later developed high blood pressure or re-narrowing of the aorta. The goal is to better understand these risks and …

This study looked at 22 people with advanced (Stage IV) melanoma to see if DNA floating in their blood could show genetic changes in their cancer before and after chemotherapy. Researchers compared the DNA found in blood samples with the original tumor's genetics. The goal was to…

This study looked at 36 adults with spina bifida to understand why they often have bowel issues. Researchers took small tissue samples from the colon during a routine scope and measured how the rectum works. The goal was to see if nerve damage affects the gut's lining and nerves,…

This study looked at the DNA changes (mutations) in eye cancers called ocular melanoma. Researchers analyzed tumor samples from 58 patients to understand how these cancers develop and spread. The goal was to identify key mutations that could lead to better treatments in the futur…

This completed study looked at 57 people with CURRARINO syndrome, a rare condition that can include a tumor near the tailbone. Researchers used yearly MRI scans and blood tests to track tumor growth and understand genetic links. The goal was to find out who is most at risk and wh…

This study examined 33 preterm babies born before 30 weeks to mothers with preeclampsia. Researchers measured certain proteins in the mother's blood, umbilical cord blood, and amniotic fluid to see if they were linked to a lung condition called bronchopulmonary dysplasia (BPD). T…

This completed study looked at how women with multiple sclerosis (MS) respond to high-intensity interval training and strength training at different points in their menstrual cycle. Fifteen women with MS and 15 without MS took part in exercise sessions and reported their satisfac…

This study looked at a process called autophagy, which is how cells clean out damaged parts, in 82 women over 65 with osteoporosis. Some also had Alzheimer's disease. Researchers measured autophagy levels in bone cells from hip samples to see if poor cell cleanup links bone loss …

This completed study looked at 60 babies who had heart surgery for a condition called transposition of the great vessels before 6 months of age. Researchers used a special questionnaire to check for sensory integration disorders one week after surgery, and again at one month and …

This study looked at 46 people with progressive multiple sclerosis who have more weakness on one side of their body. Researchers used MRI scans to see how damage in the brain and spinal cord relates to muscle strength in each limb. The goal was to better understand what causes ph…

This completed study looked for genetic causes of Chiari type I malformation, a condition where part of the brain pushes into the spinal canal. Researchers took blood samples from 40 people with the condition and their relatives to analyze their DNA. The goal was to find specific…

This study followed 60 heart transplant patients for one year to see if levels of a harmless virus called TTV in the blood are linked to infections or organ rejection. Researchers took monthly blood samples alongside routine care. The goal is to find a new way to monitor transpla…

This study looked at 86 people with severe aortic valve stenosis who had valve replacement surgery. Researchers used special MRI scans to measure scarring in the heart muscle before and one year after surgery. The goal was to see if the scarring improves and whether MRI could hel…

This study analyzed MRI scans from 732 people with primary-progressive multiple sclerosis who took part in the ORATORIO trial. Researchers wanted to see if ocrelizumab changes a part of the brain called the choroid plexus, which may be involved in inflammation. The study was obse…

This study tested whether automated alerts in electronic health records help doctors identify and treat patients with severe aortic stenosis or mitral regurgitation. Over 2,100 patients were included, and some doctors received alerts while others did not. The goal was to see if a…

This completed study looked at 132 people with advanced melanoma to see if levels of a protein called interferon-gamma (IFN-γ) in their tumors or blood could predict how well they would respond to first-line immunotherapy drugs like pembrolizumab, nivolumab, or a combination. The…

This study looked at 44 people with a rare inherited eye condition called Leber Hereditary Optic Neuropathy (LHON) that causes vision loss. Researchers reviewed medical records and asked participants about their vision, health, and quality of life. The goal was to better understa…

This study looked at children aged 6 to 18 who had heart surgery before they were 3 months old. Researchers asked parents to fill out a questionnaire about their child's emotions, behavior, and social skills. The goal was to see if these children face any developmental challenges…

This completed study created a French national registry for people with rare overgrowth syndromes caused by PIK3CA gene mutations. Researchers collected medical and genetic information from 28 patients to better understand these conditions and estimate how many people are affecte…

This study looked at 105 babies born to mothers who smoked during pregnancy, comparing those whose mothers quit versus those who continued. Researchers checked health indicators like birth weight, Apgar scores, and developmental milestones up to 24 months. The goal is to understa…

This study tested whether an artificial intelligence (AI) tool could help doctors diagnose rare and tricky skin conditions like generalized pustular psoriasis (GPP). Fifteen doctors, including both general practitioners and dermatologists, looked at 100 skin images. They first ga…

This study tested a program to help people with severe aortic stenosis get a TAVI heart valve procedure faster. The program included a website and booklet for patients, plus better coordination among hospital teams. Researchers measured whether more patients could be treated with…

This study tested a new way to control a robotic arm mounted on a power wheelchair. The arm uses a camera to help guide movements, making tasks like grabbing a drink easier. 21 adults who use power wheelchairs and have trouble with everyday tasks took part. They compared the new …

This study looked at 20 adults with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) or multifocal motor neuropathy (MMN) who were starting intravenous immunoglobulin (IVIg) treatment. Researchers measured changes in IgG levels in the blood after the first dose to…

This study looked for two rare genetic diseases, Gaucher disease and acid sphingomyelinase deficiency (ASMD), in 122 adults with an unexplained enlarged spleen or who had their spleen removed for unknown reasons. Researchers used a simple blood spot test to check enzyme activity …

This study looked at how a genetic test that checks 25 genes linked to hereditary cancers is used in clinics. Over 1,500 people with a personal or family history of cancer took part. Researchers collected blood samples and had participants fill out questionnaires over five years …

This study tested a French version of a questionnaire that measures physical activity levels in people with physical disabilities. Fifty participants with conditions like stroke, multiple sclerosis, or Parkinson's disease filled out the questionnaire twice, two weeks apart. The g…

This study looked at how the drug cladribine affects the immune system in people with multiple sclerosis (MS). Researchers analyzed blood samples from 77 MS patients and healthy donors to understand the drug's anti-inflammatory effects. The goal was to learn more about how cladri…

This study compared traditional pencil-and-paper tests with virtual reality tasks (like driving simulations and a virtual supermarket) to assess attention and executive function in 60 people with relapsing-remitting multiple sclerosis. The goal was to see if VR can better detect …

This study looked at whether specially engineered versions of a natural immune protein (IL-2) can selectively boost regulatory T cells, which help calm the immune system. Researchers took blood samples from 67 people with various autoimmune or inflammatory conditions and tested t…

This study followed 10 males who had surgery for hypospadias (a birth defect where the urine opening is on the underside of the penis) to see how they fared long after puberty. Researchers performed physical exams, bladder and testicle ultrasounds, and blood tests to check for co…

This study looked at 78 teens with adolescent idiopathic scoliosis to see if the size of their spinal curve (Cobb angle) is linked to how much pain they feel and how they see their body. Participants were grouped by curve severity (mild, moderate, moderate-to-severe). Researchers…

This study looked at 38 infants under 1 year old with congenital heart disease who needed heart surgery. The goal was to understand why some babies get brain injuries during surgery and to gather data for developing better monitoring tools. Researchers used MRI scans and blood sa…

This study tested whether a powerful DNA test called high-throughput exome sequencing can find genetic causes of multiple birth defects in fetuses when standard exams fail. Researchers studied 100 fetuses with at least two malformations and no prior diagnosis. They compared the n…

This study tested a new imaging system called VERAFEYE in 18 people already scheduled for standard heart procedures like ablation or closure. Doctors used the device during the procedure and gave feedback via a questionnaire. The goal was to gather information to improve the syst…

This study looked at whether the time of day a person has heart surgery changes how their heart muscle tolerates the stress of the procedure. Researchers took small tissue samples from the hearts of 88 patients having planned heart surgery, either in the morning or afternoon. The…

This study looked at how the COVID-19 lockdown changed the health habits and medical care of 1,343 people with chronic conditions like heart disease, multiple sclerosis, and hemophilia. Researchers used phone surveys to track medication use, lifestyle changes, and access to healt…

This study used brain scans to understand how speech therapy helps children with cleft palate who have trouble speaking clearly. Researchers looked at brain connections and structure before and after therapy in 164 children aged 4 to 16. The goal was to see if therapy changes abn…

This study tested whether a fast type of genetic test called rapid genome sequencing can find the cause of birth defects seen on ultrasound more quickly than current methods. Researchers included 184 pregnancies with certain ultrasound findings. The goal was to see if results cou…

This study looked at 81 people with mild memory problems who were getting a heart valve replacement (TAVR). Researchers tested their thinking and memory with a 5-minute quiz before the procedure and again 30 days and 6 months after. The goal was to see if fixing the heart valve c…

This completed study tracked 132 children with obstructive sleep apnea who had their tonsils removed. Researchers recorded how many had breathing problems after surgery, especially those with other health issues. The goal was to create a simple flowchart to help doctors decide wh…

This study looked at whether a simple blood sample can detect genetic mutations in patients with advanced melanoma. Currently, doctors need to take a piece of the tumor (biopsy) to check for key gene changes that guide treatment. The goal was to see if circulating tumor DNA in th…

This study looked at 143 girls to see if two natural chemicals in the blood, spexin and phoenixin-20, can help doctors tell the difference between true central precocious puberty and a harmless condition called premature thelarche. Researchers measured these chemicals in girls wi…

This study looked at whether the smallest meaningful change in bone density measurements is different for people with obesity compared to those without. Researchers measured bone density twice in 120 adults with a BMI over 30. The goal was to improve accuracy when using bone scan…

This study followed 76 women with congenital toxoplasmosis (a parasitic infection present from birth) to see if pregnancy increases the risk of eye inflammation (retinochoroiditis). Researchers checked eye exams done during pregnancy and after childbirth, comparing them to non-pr…

This study looked at 188 children (ages 0-18) with Fetal Alcohol Spectrum Disorders (FASD) in Reunion Island to better describe the condition and find a unique "epigenetic signature" in their DNA. Researchers collected blood and cheek swabs from a small group of children with FAS…

This study looked at how much of the multiple sclerosis drug ocrelizumab gets into breastmilk and whether it affects babies' immune cells. Twenty-six breastfeeding mothers with MS or clinically isolated syndrome took part. Researchers measured drug levels in breastmilk and checke…

This study followed about 400 adults with advanced BRAF-mutant melanoma who were taking targeted therapy (BRAF/MEK inhibitors) and using a smartphone app called TavieSkin. The goal was to describe patient characteristics, how they used the app, their quality of life, and treatmen…

This study looked at whether briefly pressing on the phrenic nerve during lung surgery could help reduce complications like air leaks. Researchers compared 55 patients who had this temporary nerve compression with those who did not. They measured diaphragm activity, lung function…