Disorder of development or morphogenesis

This study tests a weekly injection called TransCon CNP in children aged 2 to 10 with achondroplasia, the most common form of dwarfism. The goal is to see if it can safely improve growth over one year. Half the children receive the drug and half receive a placebo, and all childre…

This phase 2 pilot study tests whether adding the investigational drug vidutolimod to the immunotherapy nivolumab can destroy more melanoma tumors in lymph nodes or nearby skin areas before surgery. Participants have stage IIIB/C/D melanoma that has spread but not to distant orga…

This trial investigates how the Fontan surgery, which reroutes blood flow in children born with a single heart ventricle, affects the liver and heart. Researchers use MRI scans and blood tests to measure fibrosis (scarring) and lymphatic congestion. They also test whether spirono…

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 the drug larotrectinib in 215 adults with various solid cancers that have a specific change in the NTRK gene. The drug is taken orally and works by blocking the action of the altered gene. The main goal was to see how many patients had their tumors shrink or dis…

This early-phase study tested the safety of combining two drugs—pembrolizumab (an immunotherapy) and AMG386 (which targets blood vessel growth)—in 62 people with advanced solid tumors like melanoma, ovarian, renal, or colorectal cancer. The main goals were to find the safest dose…

This trial investigates whether doxercalciferol, a form of vitamin D, can prevent bone loss in the spine and thigh bone in postmenopausal women with low bone density. Participants take either doxercalciferol or a placebo daily for two years, with bone density measured at the star…

This study tests whether a wide-spectrum nutritional supplement can improve mitochondrial function in children with autism spectrum disorder (ASD). Mitochondria are the energy factories in cells, and some children with ASD have abnormal mitochondrial activity. The trial enrolls c…

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 5-week community-based program that teaches protective behavioral strategies to reduce alcohol use in men aged 35–44 with hazardous drinking habits in rural Vietnam. Participants learn to set safe drinking limits, choose nonalcoholic drinks, and plan ahead, wit…

This phase II trial tests whether giving two targeted drugs (dabrafenib and trametinib) before and after surgery can improve outcomes for people with stage IIIB-C melanoma that has a BRAF V600 mutation. 35 participants will take the drugs for 12 weeks before surgery, then continu…

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 two drugs, mirabegron and oxybutynin, in 52 children over age 3 who had surgery for posterior urethral valves. The goal was to see which drug better improves bladder and kidney function and reduces urinary tract infections. Researchers also checked safety by …

This study tested a new approach for people with advanced melanoma that can be surgically removed. Participants received a combination of two immunotherapies: one injected directly into the tumor (Tavo) and one given through a vein (nivolumab). After 12 weeks, they had surgery to…

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 study tested the safety of combining two drugs, alpelisib and fulvestrant, in 40 men and post-menopausal women with a certain type of advanced breast cancer (HR+, HER2-, with a PIK3CA mutation) that had worsened after hormone therapy. The goal was to track side effects. The …

This completed Phase 3 study tested TAK-771, a combination of immune globulin and hyaluronidase, in 26 Japanese adults with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) or multifocal motor neuropathy (MMN). The main goals were to check for side effects and see…

This phase 3 trial tested whether a yeast-derived immune stimulant (GM-CSF) and a peptide vaccine, alone or together, could prevent melanoma from returning after surgery. 815 patients with no visible cancer after complete surgical removal of advanced melanoma were randomly assign…

This phase 3 trial tested the safety of giving a monoclonal antibody (dinutuximab) along with immune-boosting drugs and a vitamin-like drug to children with high-risk neuroblastoma after a stem cell transplant. The goal was to see if this combination could be given safely and hel…

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 large study tested whether evolocumab (Repatha), a cholesterol-lowering injection, could reduce major cardiovascular events like heart attacks, strokes, or death in over 12,000 adults at high risk but without a prior heart attack or stroke. Participants received either evolo…

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 early-phase trial tested an experimental drug called KVA12123, given alone or with the immunotherapy Keytruda, in 40 adults with advanced solid tumors (like lung, breast, or colon cancer) that had stopped responding to standard treatments. The main goals were to check safety…

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 the drug nivolumab in 26 people with stage IIB or IIC melanoma that had been completely removed by surgery. The goal was to see if nivolumab, an immunotherapy that helps the immune system attack cancer cells, could prevent the melanoma from coming back. Particip…

This study tests whether a therapeutic education program helps teenagers and young adults with congenital heart disease transition from pediatric to adult medical care. Participants aged 13 to 25 will either receive the education program or standard follow-up. Researchers will me…

This study offers ongoing treatment with alectinib or crizotinib to about 200 adults with ALK- or RET-positive cancer who were already benefiting from these drugs in a previous Roche trial. The main goal is to monitor safety and side effects as patients continue therapy after the…

This early-phase study tested an experimental drug called APX005M added to two standard immunotherapies (nivolumab and ipilimumab) in 26 people with advanced melanoma or kidney cancer who had not received prior immune therapy. The main goals were to check safety and find the best…

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 implanting a new aortic valve (TAVI) without first using a balloon to open the narrowed valve is safer and as effective as the standard procedure with balloon predilatation. 250 adults with severe aortic stenosis who were at high risk for open-heart surg…

This study tested whether a two-year course of teriparatide (a bone-building drug) followed by a single infusion of zoledronic acid (a bone-strengthening drug) could reduce fractures in adults with osteogenesis imperfecta, also known as brittle bone disease. 350 participants were…

This study tested a device called ShortCut that splits the leaflets of a failing artificial heart valve before placing a new valve inside it. The goal was to prevent the old leaflets from blocking the coronary arteries, which can cause a heart attack. Eight people at risk for thi…

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 early-phase study tested a personalized cancer vaccine (RO7198457) in 273 patients with advanced solid tumors like melanoma, lung, and breast cancer. The vaccine was given alone or with an immunotherapy drug (atezolizumab) to see if it was safe and how the body responded. Th…

This study tested whether the drug abaloparatide (Tymlos) can reduce bone loss after total knee replacement in people with osteoporosis or osteopenia. 58 participants took the drug for 18 months. The main goal was to see if bone density around the new knee improved compared to be…

This small pilot study tested the safety and effectiveness of the oral drug sirolimus for reducing nosebleeds in people with hereditary hemorrhagic telangiectasia (HHT). Ten adults with moderate to severe nosebleeds took 2 mg of sirolimus daily for 3 months. Researchers tracked b…

This study tested a new approach for stage IIIB/C melanoma, the deadliest form of skin cancer. Nineteen participants received a single infusion of the immunotherapy drug nivolumab, followed by cryotherapy (freezing) of a cancerous lymph node, and then an injection of another immu…

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 looked at how different recreational exercises—swimming, Pilates, and walking—affect muscle strength and balance in women at risk for osteoporosis. 60 women who had not exercised regularly took part. The goal was to see which activity might help prevent falls and fract…

This study tested a device designed to catch and remove debris that can break loose during transcatheter aortic valve replacement (TAVR), a procedure to replace a narrowed heart valve. The device uses two filters placed in arteries leading to the brain. The trial involved 260 peo…

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 looked at how well the Ellipsys Vascular Access System works for creating arteriovenous (AV) fistulas in people with end-stage kidney disease who need dialysis. A total of 142 patients were followed for 12 months to see how long the access stayed open and how safe the …

This study tested giving chemotherapy before surgery to children with Wilms tumor, a rare kidney cancer. The goal was to shrink tumors so surgeons could remove less kidney tissue, helping preserve kidney function. 249 children took part, and the approach aimed to reduce the need …

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 Phase 2 trial tested whether adding bosentan, a drug used for blood vessel conditions, to standard chemotherapy (dacarbazine) could slow tumor growth in people with stage IV melanoma. The study enrolled 80 adults with advanced melanoma and compared the combination to chemoth…

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 adding two immunotherapy drugs (dinutuximab and GM-CSF) to standard chemotherapy is safe and doable for children newly diagnosed with high-risk neuroblastoma. Forty-two children took part. The goal was to see if the extra drugs cause too many side effect…

This study looked at 216 people with advanced melanoma that had stopped responding to standard treatments. Researchers tested each person's tumor for specific genetic changes and then gave them a drug designed to target that change. The goal was to see if this personalized approa…

This study tested a drug called indoximod, taken as a pill, together with standard immune checkpoint inhibitors (ipilimumab or pembrolizumab) in 131 adults with advanced or metastatic melanoma that could not be removed by surgery. The goal was to see if the combination was safe a…

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 tested whether adding other treatments to the drug pembrolizumab can help people with advanced melanoma that hasn't been treated before. Researchers checked safety and how many people's tumors shrank or disappeared. The trial involved 335 participants and was part of a…

This study tested a new drug called XTX202 in 95 people with advanced solid tumors that had not responded to standard treatments. The goal was to check safety, find the right dose, and see if the drug could shrink tumors. The trial is now complete.

This phase 2 trial tested two drugs, selumetinib and sirolimus, in 21 people with advanced malignant peripheral nerve sheath tumors (MPNST), a rare cancer often linked to neurofibromatosis type 1. The goal was to see if the combination could shrink or stabilize tumors. The study …

This early-stage trial tested a new drug called AZD6244 combined with standard chemotherapies in 140 people with advanced solid tumors (breast, colon, lung, melanoma, kidney). The main goal was to see if the combination is safe and tolerable, and to find the best dose. Researcher…

This study tested a device called VasQ that wraps around a surgically created blood vessel connection (fistula) used for dialysis in people with kidney failure. The device is designed to improve blood flow and reduce complications. 144 patients received the device and were follow…

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 early-phase study tested a single dose of an experimental antibody (MEDI0562) given before surgery to 13 patients with advanced head and neck cancer or melanoma. The goal was to see if the drug safely activates the immune system to fight the cancer. Researchers compared givi…

This study tested whether empagliflozin (Jardiance), a diabetes drug, can help the heart recover after aortic valve replacement. 206 patients took either the drug or a placebo for six months. The goal was to see if the drug reduces heart muscle thickness and improves symptoms.

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 study looked at the long-term safety and effectiveness of a drug called pegunigalsidase alfa for adults with Fabry disease, a rare genetic condition. Participants received an infusion every four weeks for up to several years. The goal was to see if the drug is safe over time…

This study looked at whether giving a drug called protamine to all patients after a minimally invasive heart valve replacement (TAVI) reduces bleeding complications compared to giving it only when needed. The trial involved 1000 adults with aortic valve stenosis. The goal was to …

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 study followed 134 adults in Italy with active secondary progressive multiple sclerosis who were prescribed siponimod (Mayzent) as part of their routine care. Over 36 months, researchers measured how many patients experienced a confirmed worsening of disability or cognitive …

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 looked at 80 children with a type of hypospadias (a birth defect where the urine opening is not at the tip of the penis) who had a narrow urethral plate. The researchers compared two different surgical repair methods to see which had fewer complications and better cosm…

This study tested a special dental appliance to correct underbite (Class III malocclusion) in 36 children aged 7 to 12. The device combines a fixed inclined plane on the lower front teeth with rotated brackets on the upper front teeth to encourage proper jaw growth. After 6 month…

This early-stage study tested a new drug called LBL-007 combined with two other medicines (toripalimab and axitinib) in 79 people with advanced melanoma that could not be removed by surgery or had spread. The main goals were to check safety and find the best dose. Researchers als…

This phase II trial tested whether adding stereotactic body radiation therapy (SBRT) to the immunotherapy drug pembrolizumab (Keytruda) works better than pembrolizumab alone for advanced Merkel cell carcinoma, a rare and aggressive skin cancer. Nine patients with metastatic disea…

This study looked at the side effects of using ethanol-amine oleate injections to treat venous malformations (abnormal veins) on the face and jaw in 15 children. The goal was to see how well the treatment worked and what complications might occur. The study is already completed.

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 looked at over 2,600 people with multiple sclerosis to compare two drugs: Kesimpta and Ocrevus. Researchers used insurance claims data to see which drug led to fewer relapses and lower healthcare costs. The goal is to help patients and doctors choose the best treatment…

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 early-stage study tested a custom-made yeast vaccine designed to train the immune system to recognize and attack leftover cancer cells. Five adults with various cancers (like colorectal, breast, or lung) who had completed initial treatment received the vaccine. The goal was …

This study tested whether a new way of processing tissue for Edwards heart valves is safe and works well. 777 adults who needed a new aortic or mitral valve took part. Researchers checked for valve problems like wear or calcification over about a year, and will follow patients fo…

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 a new way to replace a narrowed heart valve without open-heart surgery. It involved 782 people with severe aortic stenosis who were considered too high-risk for traditional surgery. The procedure, called TAVI, uses a thin tube to place a new valve inside the hea…

This study tested whether membranes made from a patient's own blood (L-PRF) can seal the skull base after sinus surgery as well as standard fibrin glues. 220 adults with sellar lesions were randomly assigned to receive either L-PRF or commercial sealants. The main goal was to com…

This Phase 2 trial tested a combination of targeted drugs (vemurafenib and cobimetinib) and immunotherapy (atezolizumab and tiragolumab) given before surgery to 64 patients with high-risk stage III melanoma. The goal was to see if the treatment could eliminate or shrink the cance…

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 two osteoporosis medications—romosozumab and denosumab—in 54 people who take long-term steroids and have weak bones. Participants first received either romosozumab for 12 months followed by denosumab, or denosumab alone for 24 months. Then everyone stayed on …

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 phase 2 trial tested whether a reduced dose of chemotherapy drugs (fludarabine and cyclophosphamide) given before infusing a patient's own tumor-fighting immune cells (lifileucel) could work for advanced melanoma that has spread or can't be removed. Eleven adults with stage …

This study compared two surgical techniques for treating Hirschsprung's disease in children: the traditional Soave method and a newer heart-shaped anastomosis. The trial involved 177 children under 14 years old across multiple hospitals. The goal was to see which approach leads t…

This phase 2 trial tested a combination of a radioactive drug (MIBG), intensive chemotherapy, and a stem cell transplant in 50 children with relapsed or hard-to-treat neuroblastoma. The goal was to see if this approach could shrink tumors and improve survival. The treatment aims …

This study tested a new surgical procedure for people with Chiari malformation and type II skull base depression, a condition where brain tissue extends into the spinal canal. The surgery combined decompression, joint release, and fusion to relieve pressure and stabilize the spin…

This early-stage trial tested a radioactive drug called 177Lu-DOTATATE in one child with neuroblastoma that had come back or not responded to standard treatments. The drug targets tumor cells that have a specific marker (somatostatin receptors). The goal was to find a safe dose. …

This study tested the drug trametinib in 25 people aged 12 to 60 with complicated arteriovenous malformations (AVMs), which are abnormal blood vessel tangles that can cause pain, bleeding, and heart problems. The goal was to see if the drug could shrink the malformations and impr…

This study tested a special skin graft called X A-DERM™ mADM in 16 people who had Mohs surgery to remove skin cancers like basal cell carcinoma or squamous cell carcinoma. The graft has a micro-textured surface designed to help the wound heal faster and leave a better scar. Resea…

This study followed 77 people who had a procedure called Subchondroplasty for hip bone defects. Doctors injected a calcium phosphate bone filler into the damaged area. The goal was to see if it improved pain, movement, and daily activities over two years. Results were measured us…

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 looked at 45 people with osteoporosis caused by long-term steroid use. Participants had stopped taking denosumab and were switched to either zoledronic acid or alendronate at different times. The goal was to see which timing and drug best controlled bone loss markers. …

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 early-phase trial tested a combination of three drugs—vemurafenib, cetuximab, and irinotecan—in 47 people with advanced solid tumors that have a specific BRAF V600 mutation and could not be removed by surgery or had spread. The main goal was to find the safest dose and under…

This phase 2 trial tested the drug samotolisib in 18 children and young adults with advanced solid tumors, non-Hodgkin lymphoma, or histiocytic disorders that had spread, returned, or stopped responding to treatment. Only patients whose tumors had specific mutations in the TSC or…

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 early-stage trial tested an experimental drug called CTX-471, given alone or with the approved drug Keytruda, in 100 adults with advanced cancers like lung cancer, melanoma, and head and neck cancer. The goal was to see if the combination is safe and tolerable for patients w…

This early-phase study tested a combination of three oral drugs (irinotecan, temozolomide, and cefixime) in 30 children whose high-risk neuroblastoma had come back or not responded to standard treatment. The goal was to find the safest dose of irinotecan when given with the other…

This study followed 107 adults in Switzerland with relapsing multiple sclerosis who were already taking Kesimpta (ofatumumab) as part of their routine care. Researchers tracked how many patients had no signs of disease activity, including relapses, new brain lesions, or worsening…

This trial tested a new gene therapy called ST-920 for Fabry disease, a rare genetic condition. The therapy uses a harmless virus to deliver a working gene that helps the body produce an enzyme it's missing. 36 adults with Fabry disease received a single intravenous dose and were…

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 study tested two new heart valves (Portico and Navitor) in 30 Indian patients with severe aortic stenosis, a condition where the heart's main valve narrows. The goal was to see if the valves are safe and work well. Researchers tracked complications and heart function for up …

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 early-phase study tested a new approach for children with neuroblastoma that did not respond to standard treatments. It used two rapid infusions of a radioactive drug (I-MIBG) to target cancer cells, followed by a stem cell transplant to restore healthy blood cells. The goal…

This study tested a new antibody (hu14.18K322A) added to standard chemotherapy for 153 children with high-risk neuroblastoma, a serious nerve tissue cancer. The goal was to see if the combination improves tumor shrinkage and delays the cancer from coming back. While the treatment…

This early-phase study tested a combination of two chemotherapy drugs (irinotecan and vincristine) plus a radioactive drug (131I-MIBG) in 26 children with high-risk neuroblastoma that had come back or not responded to standard treatment. The goal was to find the safest dose of th…

This early-stage trial tested a new oral form of the chemotherapy drug fenretinide in 80 children whose neuroblastoma had come back or resisted standard treatment. The goal was to find the safest dose and understand side effects. Researchers also looked at how the body processes …

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 a drug called tazemetostat in 20 children whose cancers had come back or weren't responding to treatment. The children had specific gene changes (EZH2, SMARCB1, or SMARCA4) in their tumors. The goal was to see if the drug could shrink or slow the growth of these…

This study looked at how well ofatumumab works in 562 people with relapsing multiple sclerosis who switched from other drugs (fumarates or fingolimod) because their disease was still active. The main goal was to see how many relapses they had per year. Researchers also tracked si…

This early-phase trial tested the safety of combining two drugs—L-NMMA and pembrolizumab (Keytruda)—in 12 adults with various advanced cancers. Pembrolizumab helps the immune system recognize cancer cells, while L-NMMA may remove a chemical barrier that blocks immune attack. The …

This study looked at whether surgery helps people over 65 with mild primary hyperparathyroidism (high calcium levels) compared to just monitoring them. The goal was to see if surgery improves bone density and lowers fracture risk. A total of 102 participants were randomly assigne…

This early study tested whether giving a single dose of nivolumab with either ipilimumab or relatlimab before surgery is safe and possible for people with melanoma that has spread to the brain. Only one person took part, so results are very limited. The goal was to see how the dr…

This phase 2 study tested a combination of oral azacitidine (a chemotherapy-like drug) and pembrolizumab (an immunotherapy) in 24 people with advanced melanoma that had spread. The goal was to see if the combo could shrink tumors or slow the disease, both in patients who had neve…

This study tested whether adults with CIDP, a nerve disorder causing weakness and numbness, could safely switch from regular intravenous immunoglobulin (IVIg) infusions to a new injectable drug called efgartigimod PH20 SC. Twenty-three participants who were stable on IVIg were gi…

This study tested an experimental drug called BMS-986016 (anti-LAG-3) alone or combined with nivolumab in people with advanced solid tumors that had spread or couldn't be removed by surgery. It included several cancer types like lung, stomach, liver, kidney, bladder, head and nec…

This study tested a drug called palbociclib in 23 children and young adults (ages 1–21) whose cancers had come back or didn't respond to treatment. The drug works by blocking proteins that help cancer cells grow. The goal was to see if it could shrink tumors or slow the disease i…

This early-phase trial tested a new personalized therapy called TBio-4101 for people with advanced melanoma that had stopped responding to standard treatments. The therapy uses a patient's own tumor-fighting immune cells, which are collected, multiplied in the lab, and then infus…

This study followed 21 boys with Hunter syndrome who started taking Elaprase before age 6. Researchers tracked their height and weight for at least 5 years to see if the drug helps them grow more like other children. The study also monitored safety and compared growth data to unt…

This study tests a special SPECT imaging agent that seeks out a protein called LRRC15, found on many aggressive cancers like pancreatic, breast, lung, and brain tumors. The goal is to see if this scan can help doctors diagnose and stage these cancers more accurately. Healthy volu…

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 looked at whether functional MRI can accurately measure kidney function in children with pyelo-ureteral junction syndrome, a common urinary blockage. Researchers compared MRI results to the standard scintigraphy scan in 270 children aged 0-17. The goal was to see if MR…

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 completed study looked at 313 children to see if blood tests and advanced heart scans could help detect congenital heart disease earlier and predict which children might need surgery. Researchers compared children with heart defects to healthy controls using blood biomarkers…

This study tested whether a simple breath test can predict if cancer patients will respond to immunotherapy drugs. Researchers used an electronic nose device to analyze breath samples from 190 patients with lung, melanoma, kidney, bladder, or head and neck cancers. The goal is to…

This study tested whether a computerized stethoscope that uses artificial intelligence (AI) can accurately detect two common heart valve conditions: aortic stenosis and mitral regurgitation. Researchers at Mayo Clinic recorded heart sounds from 400 adults and compared the AI's an…

This study tested a new ultra-low-dose chest CT technique in 3 children (ages 1-17) with hereditary hemorrhagic telangiectasia (HHT) to see if it could detect abnormal blood vessels in the lungs (PAVMs) using much less radiation than standard CT. The goal was to find the lowest p…

This large study in China compared how well primary care doctors detect congenital heart disease in children using a regular stethoscope versus an AI-assisted electronic stethoscope. Nearly 20,000 children aged 0-18 from schools and kindergartens were screened. The goal was to se…

This completed trial compared how well primary care doctors detect congenital heart disease in children using either their own listening skills or an AI-assisted stethoscope. The AI analyzes heart sounds in real time and gives an immediate result. The study enrolled 212 children …

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 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 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 looked at whether taking resveratrol (a natural compound found in grapes and red wine) every day could help prevent bone loss in postmenopausal women with low bone mass. 52 women who were 1 to 10 years past menopause took part. Researchers measured bone health through …

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 procedure called Periodontally Accelerated Osteogenic Orthodontics (PAOO) in 20 adults getting braces. The goal was to see if it could prevent bone loss and gum recession on the front of the lower teeth. The procedure combines a small cut in the gum with a bon…

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 study tested whether quercetin, a natural substance found in some foods, could help prevent a type of mouth cancer in people with Fanconi anemia, a rare genetic condition that raises cancer risk. 48 participants took quercetin to see if it reduced certain cell changes linked…

This study tested whether giving paracetamol (a common painkiller) early can prevent a heart opening called PDA from staying open in extremely premature infants (born before 30 weeks or under 1250 grams). 40 babies were randomly given either paracetamol or the standard drug indom…

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…

This trial investigates whether a non-invasive technique called transcranial direct current stimulation (tDCS) can help with fatigue, depression, anxiety, and cognitive problems in people with multiple sclerosis (MS). tDCS uses a weak electrical current applied to the scalp to ge…

This study tested whether bright light therapy could help people with multiple sclerosis who have trouble sleeping. 27 participants used a light box at home. The goal was to see if it was safe and if it improved sleep and daily function.

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 tests whether adding task-oriented training—like walking on different surfaces, climbing stairs, and reaching for objects—to standard aerobic and strength exercises can improve functional mobility, balance, and fatigue in people with relapsing-remitting multiple sclero…

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…

This study explores whether a 9-month online mind-body exercise program is practical and acceptable for people with multiple sclerosis (MS) living in Portugal and Spain. The program combines physical fitness, Pilates, Tai-Chi, mindfulness, and relaxation in small group sessions v…

This study tested whether ear acupressure (small pellets on specific ear points) plus basic breast care can reduce pain and blocked milk ducts in women after cesarean delivery. 72 new mothers with blocked ducts received either real ear acupressure or a sham version for 3 days, al…

This study compares two gentle exercise programs—tai chi and square stepping—in women with multiple sclerosis. Researchers want to see if these activities improve knee joint awareness, leg strength, and fatigue. About 39 women aged 20-45 with mild MS will participate, helping to …

This study tested whether a special nerve block given during cleft palate repair can lower pain and reduce the need for opioids in children. Forty children having their first cleft palate surgery received the nerve block. Researchers measured pain scores and opioid use to see if …

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 a live-video-supervised exercise program could improve fitness in children and teens (ages 8-19) born with a single ventricle, a condition often treated with the Fontan procedure. Participants exercised three times a week for three months, then a mainten…

This study investigates whether an online Pilates program designed for people with hypermobile Ehlers-Danlos syndrome or hypermobility spectrum disorder can reduce pain and improve function. Participants follow 25-minute guided videos three times a week for eight weeks. Researche…

This study tested a new type of finger splint made with shape memory alloy—a smart metal that gently and continuously corrects stiff finger joints. Twenty adults with finger stiffness were split into two groups: one used the new splint at home, the other did standard stretching e…

This study looked at 120 children aged 1 to 10 with webbed fingers (congenital syndactyly) who had surgery to separate them. After separation, a skin graft was needed to cover the gap. The study compared taking the graft from the foot (plantar area) versus the groin, to see which…

This study tested two 8-week stress management programs delivered via secure video calls for 196 adolescents aged 12–17 with neurofibromatosis (NF1 or NF2). The goal was to see if these programs could improve emotional, social, and physical quality of life. Both programs taught c…

This study tested whether a new prosthetic hand, the SoftHand Pro, helps people with below-elbow limb loss perform daily activities better than a commonly used hand, the i-Limb. Thirty-six adults who already use a myoelectric hand took part over 8 weeks. Researchers measured how …

This study tested an eight-week visually-guided gait training program in 40 people with multiple sclerosis. The training combined eye movement exercises with precision walking and obstacle avoidance to improve balance and mobility. Researchers measured balance, walking ability, a…

This study tested whether giving the pain medicine sufentanil as a continuous drip during heart surgery helps patients wake up and leave the ICU sooner compared to giving it as single shots. 65 adults having planned heart surgery took part. The goal was to see if the drip method …

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 a nasal spray containing vasopressin, a natural hormone, can improve social functioning in children with autism. 157 children aged 6 to 17 took part. Researchers measured changes in social responsiveness and overall symptoms over 8 weeks.

This early-stage trial tested a new oral drug called MTR-601 in 89 healthy adults to see if it is safe and how the body processes it. The drug aims to reduce muscle stiffness and spasms, which could help people with conditions like cerebral palsy, multiple sclerosis, or stroke. S…

This study tested a 4-month remote exercise program designed for African-Americans with multiple sclerosis living in rural areas. 121 participants were randomly assigned to either the exercise program or a stretching control group. The goal was to see if the program could improve…

This study tested whether a small, at-home device that stimulates a nerve in the ear can improve symptoms like pain, fatigue, sleep, and mood in people with hypermobile Ehlers-Danlos syndrome (hEDS). Twenty-five adults with hEDS used the device daily for two weeks, with some rece…

This study tested a newer trifocal lens implant (Clareon PanOptix Pro) against an older one (Clareon PanOptix) in 71 adults having cataract surgery in both eyes. Each person got one of each lens, so their eyes could be compared directly. The goal was to see if the newer lens prov…

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 study tested whether the drug benralizumab can safely reduce skin rashes caused by cancer treatments like checkpoint inhibitors. The goal was to lower the number of high-grade rashes to milder ones, which could improve quality of life and help patients stay on their cancer t…

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 compared two types of ultrasound-guided nerve blocks (caudal block and dorsal penile block) to control pain after hypospadias repair in 62 boys aged 1-6 years. The main goal was to see how long it took before children needed extra pain medicine. The results could help …

This study tested whether a home-based program combining motor imagery (mentally practicing movements) and action observation (watching videos of movements) could help people with multiple sclerosis improve balance, walking, and fatigue. Twenty-nine adults with MS participated in…

This study tested whether using the ABLE Exoskeleton—a robotic walking aid—once a week for a year could help people with multiple sclerosis maintain their physical and mental health. Nineteen adults with MS took part in weekly 60-minute gait training sessions and were evaluated e…

This study tested a wearable device that delivers mild electrical stimulation to the ear in 30 adults with hypermobile Ehlers-Danlos syndrome (hEDS). The goal was to see if it could improve quality of life by reducing symptoms like pain, fatigue, sleep problems, and anxiety. Part…

This study tested whether hip surgery (PAO) followed by exercise is better than exercise alone for reducing pain in adults with hip dysplasia. 69 people aged 18-45 took part. The main goal was to see which approach improved hip pain and function more after 12 months.

This study tested a 6-week program that combines home exercises with fun, game-like balance activities (like stepping on colored circles and Twister-inspired moves) for people with multiple sclerosis. Thirty participants were split into two groups: both did home exercises, but on…

This study compared two therapies—myofeedback and neuromuscular electrical stimulation—to see which better improves arm and hand function in children aged 6 to 14 who have one-sided weakness (hemiplegia) after brain tumor surgery. Forty-five children participated, and researchers…

This study tested whether using virtual reality glasses during physical therapy can reduce pain, improve blood oxygen levels, and help children walk farther after open heart surgery. Forty children aged 6 to 12 took part. One group used VR glasses during their cardiac rehab exerc…

This completed study tested hyaluronic acid fillers injected into the penis to increase width and girth in 20 healthy men aged 21 to 65. The main goal was to see if size increased after one year using 3D imaging. The treatment is a temporary cosmetic procedure, not a cure for any…

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 completed clinical trial tested a high-intensity focused ultrasound (HIFU) device called ULTRAFORMER MPT for lifting eyebrows without surgery. 180 adults aged 20-65 with mild to moderate forehead skin laxity received either a single HIFU treatment or no treatment. The study …

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 types of nerve blocks to see which provides better pain relief for children aged 1 to 6 after hypospadias surgery. Sixty kids received either a pudendal nerve block or a dorsal penile nerve block, both guided by ultrasound. Researchers measured pain levels a…

This phase 2 study tested whether pitolisant, a drug that promotes wakefulness, can safely reduce excessive daytime sleepiness in people with Prader-Willi syndrome. The trial enrolled 65 participants aged 6 to 65 and compared pitolisant to a placebo over 11 weeks, followed by an …

This study looked at whether aerobic exercise and virtual reality games could improve functional capacity, activity, and participation in children aged 6-10 living in care facilities. Sixty-three children were split into groups: one did aerobic exercise, one played VR games, and …

This study tested whether a single dose of dexmedetomidine, given during surgery, can prevent severe agitation in children aged 1-7 after cleft palate repair. Sixty children were randomly assigned to receive either the drug or a placebo (saline). The main goal was to measure emer…

This study looked at whether high-intensity walking exercise can help reduce fatigue and improve walking in people with multiple sclerosis (MS). Twelve adults with MS who had walking problems and significant fatigue took part. Researchers measured changes in fatigue, walking spee…

This study tested whether an ultrasound-guided belly muscle nerve block (TAP block) can reduce pain and movement during a minimally invasive heart valve replacement (TAVI). Fifty adults having elective TAVI received either the block plus sedation or sedation alone. The goal was t…

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 adding Dynamic Neuromuscular Stabilization (DNS) exercises to standard pelvic floor muscle training can improve bladder control in women with multiple sclerosis. Twenty-four women aged 28-54 completed an 8-week program, with half doing pelvic floor exerc…

This study tested a home-based telerehabilitation system for veterans with multiple sclerosis. The system provided exercise instructions and tracked daily logs to help improve balance and mobility. Fifty-one participants used the system to see if it was feasible and acceptable, w…

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 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 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 Emotional Freedom Techniques (EFT), a therapy that combines talking with gentle tapping on acupressure points, in 56 people who had been treated for melanoma and were in remission. The goal was to see if EFT could reduce fear of the cancer coming back and improv…

This study tested whether MitoQ, a dietary supplement, can reduce fatigue in people with multiple sclerosis. 45 participants took either 20 mg, 40 mg, or a placebo daily for 12 weeks. Researchers measured changes in fatigue using a standard survey. The goal is to find a safe, sim…

This study tested whether a virtual rehabilitation program called VIVA is feasible for people with spine fractures caused by osteoporosis. The program includes weekly online sessions to help reduce pain and improve movement and quality of life. The study involved 34 participants …

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 whether a robotic walking system called Atalante could help people with multiple sclerosis walk better. Sixty participants were randomly assigned to either use the robot or receive standard physical therapy for 8 weeks. The main goal was to see if the robot impr…

This study looked at whether music can help children aged 6-9 feel less anxious and experience less pain during a routine dental procedure (applying sealants). Each child had three treatments: one with no music, one with classical music chosen by the dentist, and one with their o…

This early study tested a tiny dexamethasone insert placed in the tear duct of 15 children after retinal surgery or laser treatment. The goal was to see if it could safely reduce pain and eye inflammation. Researchers measured pain using a standard scale and tracked any side effe…

This study looked at whether a special nerve block (erector spinae plane block) can reduce pain and improve sleep in infants having surgery for congenital lung cysts. Sixty-eight babies were enrolled, and their pain and sleep quality were measured after surgery. The goal is to fi…

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 tested whether a 12-week exercise program using coordinated arm movements could improve brain function and movement in people with progressive multiple sclerosis (MS). Five adults with progressive MS did the exercises three times a week. Researchers measured brain acti…

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 a home exercise program, supported by a smartwatch and weekly coaching, is practical and acceptable for people with mitochondrial disease. Ten participants followed an 8-week program with remote monitoring. The goal was to see if people would stick with …

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 completed phase 3 trial tested whether a patient outreach program—regular phone calls every two weeks—could help people with advanced medullary thyroid cancer spend less time experiencing moderate-to-severe side effects from the drug vandetanib. The study enrolled 205 adults…

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 whether combining two types of dermal fillers—Sculptra and Restylane—can improve cheek volume, wrinkles, and skin quality in 41 adults with moderate-to-severe cheek aging. Participants had a history of weight loss or were taking GLP-1 medications. They received …

This study tested whether the drug Northera (droxidopa) is safe and helpful for adults with Menkes disease or Occipital Horn Syndrome who have trouble with low blood pressure and other symptoms of dysautonomia. Three participants took either the drug or a placebo in a crossover d…

This study tested a micro-coring device that removes small pieces of skin to reduce moderate to severe cheek wrinkles. Thirty adults aged 40 to 70 participated. Researchers measured wrinkle improvement at 90 days and tracked any side effects.

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 pilot study tested two different computerized brain training programs that also involve light physical activity, like reaching or stepping, in people with multiple sclerosis who have memory or attention problems. The goal was to see if these programs could improve thinking s…

This study tested whether applying shock wave therapy to the soles of the feet could improve balance and walking in people with multiple sclerosis (MS). Fifty-four participants received the therapy three times a week for six weeks. Researchers measured changes in foot sensation, …

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 an 8-week online program to help people with multiple sclerosis (MS) improve their problem-solving and coping skills. The goal was to see if the program was easy to use and if it helped reduce anxiety and depression. Thirty adults with MS in Lebanon took part. T…

This study looks at how good or poor sleep quality influences quality of life, depression, and fatigue in people with multiple sclerosis. Researchers will survey about 121 adults with MS to measure their sleep patterns and link them to these health outcomes. The goal is to better…

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…

This study looks at rare genetic disorders where the body can't make cholesterol properly, which can cause birth defects and learning problems. Researchers collect blood, urine, and tissue samples from affected people and their families to learn more about these conditions. The g…

This study looked at people with suspected bone-related connective tissue disorders (like brittle bone disease) and their healthy family members. The goal was to learn more about these conditions, train doctors, and collect samples for future research. Participants received stand…

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 nearly 1,400 people with rare conditions like PPNAD, Carney Complex, and Peutz-Jeghers syndrome. The goal was to find the genetic causes and link them to specific symptoms. Researchers used clinical exams and genetic testing to better understand how these dis…

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 followed 88 children and young adults with types III and IV osteogenesis imperfecta (brittle bone disease) to track how the condition changes over time. Researchers measured bone density, lung and heart function, hearing, and movement abilities. The goal was to better …

This study tested a new risk score that combines genetic and environmental factors to predict multiple sclerosis (MS). Researchers studied 181 people, including MS patients and their close relatives. The goal was to see if this score could identify who might develop MS in the fut…

This study looked at people with neurofibromatosis type 1 (NF1) to better understand how harmless tumors (plexiform neurofibromas) turn into a rare cancer called MPNST. Ten participants had MRI, PET scans, and tumor biopsies. Researchers tested whether a special PET scan (FLT PET…

This study tests whether wearable sensors and digital home tasks can track changes in movement, speech, and falls in people with ALS. About 20 participants will wear neck and wrist sensors and complete speech and handwriting tests over 48 weeks. The goal is to see if these digita…

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 looked at 200 men and women who had surgery to repair a small ascending aortic aneurysm (5.0-5.5 cm). Researchers wanted to see if gender affects early and long-term outcomes like death, heart attack, or bleeding. The goal is to understand why women might have worse re…

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 looked at how well surgery works for people with an enlarged aorta (aneurysm) in the chest. Researchers compared outcomes in 50 patients who had surgery versus those who did not, tracking deaths and how fast the aorta grew over time. The goal was to better understand w…

This study brought together four expert skin pathologists to see how well they agree when diagnosing moles that might be melanoma. The goal was to create a reliable standard for future research. The experts reviewed images and discussed tricky cases to reach a consensus.

This study looked at 60 children aged 1 to 4 years who had surgery for developmental dysplasia of the hip. Doctors compared two ways of cutting the hip capsule: a U-shaped cut and a T-shaped cut. The goal was to see which method was faster and needed fewer stitches. The results m…

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…

This study tested two different methods of closing the main leg artery after a minimally invasive heart valve replacement (TAVR). One method used two small stitch-like devices, the other used one stitch device plus a plug. The goal was to see which approach caused fewer bleeding …

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 tests whether special low-energy MRI settings can make it safe to use guidewires during heart catheterization. Guidewires help position catheters but can heat up in standard MRI. Researchers will enroll 22 adults who need a right heart catheterization to see if this ap…

This study tested a new way to measure nose and lip shape in 14 infants with cleft lip and palate using 3D facial scans. Researchers compared these scans to standard photos and manual measurements to see if the digital method is accurate. The goal is to find a better, less invasi…

This study tested a mobile health program called Heart2Heart for teens aged 12-18 with congenital heart disease. The goal was to see if teens liked the program and found it easy to use. Seventeen teens completed surveys, used the app, and gave feedback in interviews.

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 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 examines the patient profiles and treatment patterns of adults with melanoma in Japan who were prescribed either dabrafenib with trametinib or encorafenib with binimetinib. Researchers are looking at data from 2012 to 2021 to understand patient characteristics and how …

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 completed pilot study aimed to estimate how common osteoporosis is in France, starting with the Alpes-Maritimes region. Researchers enrolled 800 participants who each received a bone densitometry scan. The study was observational and did not test any treatment.

This study aims to understand how satisfied both doctors and patients are with current multiple sclerosis (MS) treatments in everyday medical practice. Researchers will collect information from 261 patients and their physicians across several countries, including the UK, France, …

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 tested a new prosthetic foot for children with leg amputations or birth defects. Thirteen kids walked with the device and gave feedback on stiffness and performance. The goal was to gather ideas to improve future foot designs.

This study checked whether women over 50 who had severe osteoporotic fractures (like hip or spine breaks) were scheduled for osteoporosis treatment at Nîmes University Hospital. Researchers reviewed hospital records of 500 patients to see if treatment was planned. The goal was to…

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 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 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 looked at 112 people with known differences in their sex chromosomes (X and Y) to better understand how these variations affect health, especially infertility. Participants underwent physical exams, blood and urine tests, imaging, and sensory checks over about 5 days. …

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 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 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 completed study reviewed the medical records of 210 people with aortitis (inflammation of the aorta) or retroperitoneal fibrosis (scar tissue in the abdomen). The goal was to find patterns in symptoms, lab tests, and imaging that could help doctors tell different types of th…

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 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 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 used a quick, painless eye scan called OCTA to compare blood flow in the optic nerve between 54 people with multiple sclerosis and healthy volunteers. The goal was to see if MS changes optic nerve blood vessels and if measuring this could help doctors track the disease…

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 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 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 study used advanced brain imaging (MRI and MEG) to look for differences in brain structure, chemistry, and activity between people with schizophrenia and healthy volunteers. Over 3,200 adults took part. The goal was to better understand the brain changes linked to schizophre…

This study watched 152 melanoma patients who had their tumors surgically removed and then started nivolumab as part of their normal care. Researchers tracked how long patients stayed cancer-free, whether the cancer spread, and overall survival. The goal was to see how well the dr…

This study looked at 40 children with congenital heart disease who had heart surgery. During surgery, doctors used a special filtering technique called SBUF-SMUF to remove inflammatory substances from the blood. The goal was to see how this affected inflammation and recovery. The…

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 tested a special MRI technique called magnetic resonance elastography (MRE) to see if it can detect scarring in transplanted kidneys. Researchers compared MRE results with traditional biopsy samples from 73 kidney transplant patients. The goal was to find a non-invasiv…

This study collected DNA, tissue, and medical history from 305 people with Williams syndrome or supravalvar aortic stenosis (SVAS), as well as their family members and healthy volunteers. The goal was to create a resource for future research to understand why symptoms vary so muc…

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 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 looked at new ways to test blood clotting in 200 children with congenital heart disease undergoing surgery. The goal was to see if newer technologies like TEG, ROTEM, and T2MR could help doctors manage bleeding more precisely and reduce the need for blood transfusions.…

This study tested whether smartphone communication and a special questionnaire could help confirm MS relapses without requiring an in-person visit. Researchers worked with 150 people with relapsing-remitting MS to see if this approach improves patient satisfaction and care. The g…

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 collected blood, saliva, and eye fluid samples from 343 people with uveitis, an inflammatory eye disease that can cause vision loss. Researchers aimed to learn which genes and other factors might lead to the disease and affect treatment response. Participants were foll…

This completed study tracked 42 women with multiple sclerosis who became pregnant around the time they received LEMTRADA infusions. Researchers aimed to measure rates of miscarriage and birth defects to better understand the drug's safety during pregnancy. The study did not test …

This small pilot study tested whether a non-invasive eye scan called OCTA can detect changes in the brain related to stroke and other neurological conditions. Researchers compared eye scans from 7 people with brain diseases and healthy volunteers. The goal was to gather early dat…

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 looked at how people with autoimmune diseases like multiple sclerosis or blistering skin conditions respond to COVID-19 vaccines while on B-cell depleting therapies. Researchers measured antibody levels and T-cell activity over time to understand vaccine effectiveness …

This study looked at whether a special honey (Trigona sp.) helps heal wounds after cleft palate surgery better than a standard antibiotic dressing. Twenty-four children aged 18 months to 6.5 years participated. Researchers measured a healing marker in saliva before and after surg…

This study looked at 173 infants with myelomeningocele, a severe form of spina bifida, to see if the timing of their surgery (before or after birth) affects their risk for sleep-disordered breathing. Researchers used sleep studies to measure breathing problems and later checked t…

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 completed study tested a smart wearable device called gaitQ that monitors and cues walking in people with long-term movement conditions like Parkinson's, stroke, multiple sclerosis, and arthritis. Researchers collected movement data from 98 participants in a lab to see if th…

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 completed study looked at 65 people with multiple sclerosis to find specific scores on walking and balance tests that could tell who is at risk of falling. Participants walked on a special mat and stood on a balance platform under different conditions. The goal is to help do…

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 tested a new device called EchoMark LP, which is implanted near a surgically created blood vessel connection (fistula) used for dialysis. The device works with a special ultrasound system (EchoSure) to measure blood flow, depth, and diameter of the fistula. The trial i…

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 at whether a small device called an implantable loop recorder can be safely placed in newborns who have only one working heart chamber. Ten babies received the monitor during their first heart surgery. The goal was to see if the device could continuously track h…

This study collected information from over 15,000 people hospitalized with cardiomyopathy, a heart muscle disease. Researchers looked at how much these hospital stays cost and what the money was spent on. The goal was to understand the real-world financial impact and help manage …

This study looked at a treatment called "Surgery-First" for people with a severe underbite (skeletal Class III malocclusion). Instead of wearing braces before surgery, patients had jaw surgery first to fix the bone problem, then braces afterward. Researchers studied 60 patients u…

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 completed study looked at 29 people with hypophosphatasia, a rare bone disease, who had no known genetic cause from standard tests. Researchers used whole genome sequencing to find hidden genetic changes in the ALPL gene. The goal was to better understand the disease and imp…

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 pilot study tested whether a single dose of an oral ketone supplement could increase blood flow and improve brain connectivity in 15 older adults with subjective or mild cognitive decline. Participants had one 2-hour session including MRI scans and cognitive tests. The goal …

This pilot study looked at how weight loss affects bone density in overweight or obese men. 44 men either followed a reduced-calorie diet to lose weight or maintained their weight for 6 months. Researchers measured changes in bone density and quality to see if weight loss has any…

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 tested whether implanting tiny gold markers near spinal tumors helps deliver radiation more accurately. Researchers also checked if giving slightly more radiation to the nearby esophagus is safe, which might improve tumor control. The study involved 41 adults with vari…

This study looked at 136 premature babies who needed breathing help. Researchers tracked how long they needed support and checked for lung problems. The goal was to learn more about different non-invasive breathing methods, not to test a new treatment.

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 11 people with a repaired heart condition called tetralogy of Fallot who needed a new pulmonary valve. Researchers used MRI scans to measure heart muscle stiffness (fibrosis) and how well the heart pumped during rest and exercise. The goal was to see if these…

This study looked at how well a procedure called catheter ablation works for treating fast heart rhythms in people with congenital heart disease. Researchers treated 40 patients and followed them for one year to see if the abnormal heart rhythm came back. The goal was to understa…

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 measured levels of an inflammatory protein called HMGB1 in 74 children with congenital heart disease and pulmonary hypertension. Researchers compared these levels to those in children with heart disease alone and healthy controls. The goal was to see if inflammation pl…

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 study compared two common sedation drugs, propofol and dexmedetomidine, in 65 older adults undergoing a less invasive heart valve replacement called TAVR. The goal was to see which drug leads to better kidney function, less confusion, and fewer complications after the proced…

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 looked at whether a multiple sclerosis (MS) drug called Ofatumumab (Kesimpta) can make immune cells less toxic to brain cells. Researchers took blood samples from 40 people with relapsing-remitting MS before and after 6 months of treatment. They tested if the chemicals…

This completed study at Brigham and Women's Hospital used special PET scans to look at brain inflammation and chemical activity in 10 people with multiple sclerosis. The goal was to understand what causes the severe fatigue many MS patients experience. Researchers measured brain …

This study tested whether a small, band-aid-like heart monitor (Zio patch) can be worn by children for up to 14 days. It involved 36 kids aged 1 to 17 years. The goal was to see if the patch sticks well and gives clear heart signals. The study did not test any treatment, only the…

This completed study evaluated existing osteoporosis patient education programs in Danish municipalities. Researchers compared over 3,000 people who took part in group education sessions (covering medication, diet, and exercise) with a matched group who did not. They measured eff…

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 tested a school program called SocLaLola that uses a comic book to teach children aged 8-11 about pain. The goal was to see if it improves kids' understanding of pain and reduces their fear of moving. Eighty-two students from two primary schools took part. The program …

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 giving extra oxygen to pregnant mothers carrying babies with congenital heart disease (CHD) could increase oxygen levels in the fetal brain. Researchers used ultrasound and MRI to measure changes in 53 fetuses with different types of CHD. The goal was…

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 tested whether a special CT scan (DECT) can better predict fracture risk in women with osteoporosis than the usual bone density test (DEXA). Seven women aged 60 or older participated. The goal was to develop a computer tool that analyzes bone structure from the scan to…

This study tested whether using a contrast dye during 3D ultrasound can accurately measure the size and function of the right heart chamber in people who had surgery for congenital heart disease. 25 participants received an injection of Lumason (an ultrasound contrast agent) and …

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 giving extra oxygen to pregnant women can help doctors tell how well blood mixes in the hearts of unborn babies with a condition called transposition of the great arteries (TGA). Researchers used ultrasound and MRI to measure blood flow and heart stru…

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 study looked at 114 boys aged 2 to 7 having urogenital surgery to see if being anxious before the operation leads to more pain or confusion after waking up. Researchers measured anxiety before surgery and then tracked pain, delirium, and recovery speed in the recovery room. …

This study looked at 308 people with myelodysplastic syndromes (MDS) who had a stem cell transplant. Researchers identified each patient's unique genetic mutations and then monitored those mutations in blood and bone marrow samples after transplant. The goal was to see if rising …

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 how different levels of ventilator pressure (PEEP) affect the heart's ability to pump blood in children under 5 with congenital heart disease who are on breathing machines. Researchers measured changes in cardiac output and right heart function in 58 children…

This completed study looked at 322 premature babies born before 32 weeks to compare different ways of diagnosing bronchopulmonary dysplasia (BPD), a common lung disease. Researchers proposed a new classification that separates high-flow nasal cannula from other breathing support.…

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…

Fatigue is a major problem for people with multiple sclerosis (MS), but its cause is unknown and there is no good treatment. This completed study looked at 34 adults with MS to see if a type of immune cell (cytotoxic CD4+ T cells) is linked to how tired they feel. Researchers mea…

This study looked at 22 people with severe aortic stenosis who were scheduled for valve replacement. Before surgery, they had an ultrasound of the heart, and during surgery a tiny sample of heart muscle was taken to check for scarring (fibrosis). The goal was to see if ultrasound…

This study checked how the multiple sclerosis drug ocrelizumab passes from mother to baby during pregnancy. Researchers measured B cell levels in 70 infants whose mothers took the drug around the time of pregnancy. The goal was to see if the drug affects the baby's immune system …

This study examined how conditions like diabetes, smoking, heart disease, and osteoporosis affect the health of dental implants. Researchers looked at 93 people with 314 implants that had been in place for at least three years. They found that these systemic factors can influence…

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 completed study brought together men who have had suicidal thoughts to help design suicide prevention tools. Over up to 10 meetings, 13 men gave feedback on tool drafts, focusing on usability, privacy, and support. The goal was to create resources that truly fit their needs.

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 completed study tested whether a new biomarker called 'mass response' can predict how well a cancer therapy will work. Researchers collected tumor cells from 72 patients with various cancers through fluid drainage or biopsies. The goal was to see if this biomarker could guid…

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 70 elderly women, half with osteoporosis and half without, to see if lower body muscle strength is linked to bone density. Researchers measured hip and knee strength using a special machine and compared it to bone scans. The goal was to better understand risk…

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 completed study looked at how appetite-regulating hormones change in infants with Prader-Willi syndrome during the first four years of life. Researchers collected blood samples from 215 infants to understand why they switch from poor feeding to severe overeating and obesity.…

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 study looked at 60 heart surgery patients to see how two different bypass machines affect blood flow in tiny vessels under the tongue. One method was minimally invasive, the other was the standard approach. The goal was to find out which technique keeps small blood vessels w…

This study examined blood and tumor samples from 25 people with advanced melanoma who were starting standard pembrolizumab treatment. Researchers looked at T cells—key immune cells—to find patterns that might predict whether the therapy works. The goal is to better understand why…

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 followed 271 children with achondroplasia, ages 2.5 to 17, to understand their growth, medical issues, and quality of life over time. No medication or treatment was given. The goal was to gather natural history data to help design future clinical trials.