Autosomal recessive disease
MONDO:0006025Autosomal recessive form of disease.
Also known as: autosomal recessive disease or disorder, autosomal recessive hereditary disease, autosomal recessive hereditary disorder, autosomal recessive inherited disease, autosomal recessive inherited disorder, disease or disorder, autosomal recessive, disease, autosomal recessive, recessive hereditary disorder (autosomal)
873 clinical trials for this condition and its sub-types.
Follow this condition — get notified about new trialsSub-types
Broader categories
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Experimental drug aims to halt rare neurological decline
Disease control Not yet recruitingThis Phase 3 trial tests whether vatiquinone can slow the progression of Friedreich's ataxia, a rare genetic disorder that damages the nervous system and impairs movement. About 120 adults with moderate symptoms will take the drug for 24 months. Researchers will measure changes i…
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jul 04, 2026 00:00 UTC
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New hope for rare immune disorders: upadacitinib trial launches
Disease control Not yet recruitingThis study tests the drug upadacitinib (Rinvoq) in 30 people with rare genetic disorders that overactivate the JAK-STAT immune pathway. The trial has four phases over 12 months, including an open-label phase to find the best dose and a randomized withdrawal phase to compare the d…
Phase: PHASE1, PHASE2 • Sponsor: Lisa Satter • Aim: Disease control
Last updated Jul 04, 2026 00:00 UTC
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New drug aims to slow kidney damage in rare alport syndrome
Disease control Not yet recruitingThis study tests a drug called exaluren in 24 people aged 12 and older with Alport syndrome caused by specific genetic errors. The drug is given as a daily shot for 32 weeks. Half the group starts exaluren right away, while the other half gets a placebo first and then switches to…
Phase: PHASE2 • Sponsor: Eloxx Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jul 03, 2026 00:00 UTC
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New drug aims to preserve sight in rare genetic blindness
Disease control Not yet recruitingThis phase 3 trial tests whether NPI-001, taken twice daily for 24 months, can slow the loss of light-sensing cells in the eyes of adults with Usher syndrome, a genetic condition that causes both hearing and vision loss. About 80 participants will receive either the drug or a pla…
Phase: PHASE3 • Sponsor: Nacuity Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jul 03, 2026 00:00 UTC
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Tiny tablets could make PKU management easier for patients
Disease control Not yet recruitingThis study tests a new microtablet protein substitute for people with phenylketonuria (PKU), a condition requiring a strict low-protein diet. The microtablets combine protein with vitamins and minerals, aiming to be easier to take than current liquid or powder options. About 20 a…
Phase: NA • Sponsor: Nutricia UK Ltd • Aim: Disease control
Last updated Jun 28, 2026 00:00 UTC
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Could a pill cut transfusions for sickle cell patients?
Disease control Not yet recruitingThis Phase 3 trial tests whether the drug mitapivat can reduce the need for blood transfusions in people with sickle cell disease. About 159 participants aged 12 and older will receive either mitapivat or a placebo daily for a year. The main goal is to see if more people can avoi…
Phase: PHASE3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
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Could a simple gel boost gum healing in diabetes?
Disease control Not yet recruitingThis study tests whether adding hyaluronic acid gel to standard deep cleaning (scaling and root planing) improves gum healing in people with both diabetes and advanced gum disease. Twenty-three participants will receive the standard cleaning on all teeth, and then two gum pockets…
Phase: NA • Sponsor: Marmara University • Aim: Disease control
Last updated Jun 27, 2026 13:05 UTC
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Could a special supplement boost health in cystic fibrosis?
Disease control Not yet recruitingThis study will test whether a nutrition supplement with different forms of certain nutrients works better than standard supplements for adults with cystic fibrosis. Sixty participants will take the supplement for six weeks, and researchers will measure nutrient levels in their b…
Phase: NA • Sponsor: Ohio State University • Aim: Disease control
Last updated Jun 27, 2026 13:05 UTC
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New hope for sickle cell: Half-Matched transplants aim to cut complications
Disease control Not yet recruitingThis study tests a stem cell transplant from a half-matched family donor for children and young adults with sickle cell disease. The goal is to control the disease while reducing serious side effects like graft-versus-host disease. Up to 45 participants will receive a special dru…
Phase: PHASE2 • Sponsor: St. Jude Children's Research Hospital • Aim: Disease control
Last updated Jun 27, 2026 13:01 UTC
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Gene editing breakthrough aims to stop sickle cell pain crises
Disease control Not yet recruitingThis early-stage study tests a one-time gene therapy (CS-206) in 5 teenagers with severe sickle cell disease. The treatment uses the patient's own blood stem cells, modified with a gene-editing tool, to help produce healthy red blood cells. The goal is to reduce painful blockages…
Phase: EARLY_PHASE1 • Sponsor: Children's Hospital of Fudan University • Aim: Disease control
Last updated Jun 27, 2026 12:32 UTC
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New enzyme could help digest food for those with pancreatic problems
Disease control Not yet recruitingThis study tests a new lab-made enzyme called NHS7108 in people with exocrine pancreatic insufficiency (EPI), a condition where the pancreas doesn't make enough enzymes to digest food. About 66 adults will take either NHS7108 or the standard enzyme Zenpep for 14 days each, while …
Phase: PHASE2 • Sponsor: Aimmune Nestlé Health Science US R&D, LLC • Aim: Disease control
Last updated Jun 27, 2026 12:26 UTC
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New combo therapy aims to spare organs in High-Risk rectal cancer
Disease control Not yet recruitingThis phase 3 trial tests whether short-course radiation followed by chemotherapy and an immunotherapy drug (serplulimab) works better than standard long-course chemoradiation for high-risk locally advanced rectal cancer. 612 patients will be randomly assigned to one of the two tr…
Phase: PHASE3 • Sponsor: Fudan University • Aim: Disease control
Last updated Jun 27, 2026 12:23 UTC
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Virus cocktail targets tough lung bugs in cystic fibrosis trial
Disease control Not yet recruitingThis early-stage trial tests a new treatment called AchromoPhage, a mix of four viruses that specifically kill Achromobacter bacteria, in 12 adults with cystic fibrosis and chronic lung infections. Participants will receive the treatment by inhalation, IV, or both over three week…
Phase: PHASE1 • Sponsor: Ghady Haidar • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC
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Gene therapy injection aims to save sight in rare genetic disease
Disease control Not yet recruitingThis early-stage trial tests a single injection of AXV-101 gene therapy into one eye of 12 children and teens (ages 4-17) with Bardet-Biedl syndrome type 1, a rare genetic condition that causes progressive vision loss. The main goal is to check safety and find the right dose, whi…
Phase: EARLY_PHASE1 • Sponsor: Axovia Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:02 UTC
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Early screening and care for sickle cell in newborns could save lives
Disease control Not yet recruitingThis study will test 120 babies in Côte d'Ivoire for sickle cell disease using a simple point-of-care test. Babies who test positive will start comprehensive care early, including medicines and vaccines. Researchers want to see if this approach reduces serious health problems and…
Phase: PHASE4 • Sponsor: Swiss Tropical & Public Health Institute • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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New inhaled mRNA therapy aims to fix cystic fibrosis at its source
Disease control Not yet recruitingThis early-phase study tests an inhaled mRNA drug (BMD003) in 24 people aged 12 and older with cystic fibrosis. The goal is to see if it is safe and can improve lung function by delivering instructions to make a working CFTR protein. Participants will receive multiple doses and b…
Phase: EARLY_PHASE1 • Sponsor: Peking Union Medical College Hospital • Aim: Disease control
Last updated Jun 27, 2026 11:03 UTC
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Gene therapy hope for sickle cell: tiny trial tests One-Time fix
Disease control Not yet recruitingThis early study tests a gene therapy called KL003 in just 3 people with severe sickle cell disease. The therapy uses the patient's own stem cells, modified to produce healthy hemoglobin, to reduce painful crises and the need for transfusions. The main goals are to see if the tre…
Phase: NA • Sponsor: Institute of Hematology & Blood Diseases Hospital, China • Aim: Disease control
Last updated Jun 27, 2026 09:09 UTC
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New combo therapy aims to shrink Hard-to-Treat colorectal tumors before surgery
Disease control Not yet recruitingThis study tests whether giving immunotherapy drugs (nivolumab with or without ipilimumab) or immunotherapy plus radiotherapy before surgery can improve outcomes for people with a specific genetic type of colorectal cancer called MSI-H/dMMR. About 114 participants will be randoml…
Phase: PHASE2 • Sponsor: Fudan University • Aim: Disease control
Last updated Jun 27, 2026 09:04 UTC
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Sound waves and immunotherapy join forces against deadly brain tumor
Disease control Not yet recruitingThis early-phase study tests whether combining focused ultrasound with the immunotherapy drug pembrolizumab is safe and feasible for people with recurrent glioblastoma, a severe brain cancer. Only 8 participants with a specific genetic feature (mismatch repair deficiency) will be…
Phase: PHASE1 • Sponsor: Jennifer Leddon • Aim: Disease control
Last updated Jun 27, 2026 09:01 UTC
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Could a drug combo replace the knife for some endometrial cancers?
Disease control Not yet recruitingThis study tests two immunotherapy drugs (iparomlimab and tuvonralimab) in 30 women with a specific type of endometrial cancer that is mismatch repair-deficient. The goal is to see if the treatment can make the cancer disappear or shrink enough to avoid surgery. Participants must…
Phase: PHASE2 • Sponsor: Women's Hospital School Of Medicine Zhejiang University • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC
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New combo therapy aims to wipe out stomach cancer before surgery
Disease control Not yet recruitingThis phase 2 trial tests two different drug combinations given before surgery for people with a specific type of stomach cancer (dMMR/MSI-H). Participants will receive the immunotherapy drug adebrelimab plus either chemotherapy or another immunotherapy drug (SHR-8068). The goal i…
Phase: PHASE2 • Sponsor: Shanghai Zhongshan Hospital • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC
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New drug hopes to stop bone loss in rare aging disease
Disease control Not yet recruitingThis phase 2 trial tests a drug called Progerinin in up to 5 adults with Werner syndrome, a rare condition that causes premature aging and bone loss. Participants take the drug twice daily for about a year. The main goal is to check safety and tolerability, not yet to prove it wo…
Phase: PHASE2 • Sponsor: PRG Science & Technology Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 08:13 UTC
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Could a higher drug dose help sickle cell patients get stem cell transplants?
Disease control Not yet recruitingThis study tests whether a high dose of plerixafor can safely collect enough stem cells from people with sickle cell disease for a future transplant. Twelve patients aged 18-25 will receive the drug and have their stem cells collected through a process called apheresis. The goal …
Phase: PHASE1, PHASE2 • Sponsor: Hospital Israelita Albert Einstein • Aim: Disease control
Last updated Jun 27, 2026 08:11 UTC
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New hope for rare genetic disorder: tailored chemo before stem cell transplant
Disease control Not yet recruitingThis study tests whether a lower dose of the chemotherapy drug treosulfan, given before a stem cell transplant, can help people with Nijmegen breakage syndrome. The condition causes immune problems and a high risk of cancer. The trial will enroll 24 patients, giving a lower dose …
Phase: PHASE2 • Sponsor: Federal Research Institute of Pediatric Hematology, Oncology and Immunology • Aim: Disease control
Last updated Jun 27, 2026 08:08 UTC
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New blood processing method could cut transfusion needs for sickle cell patients
Disease control Not yet recruitingThis study tests whether red blood cells processed to be low in oxygen (hypoxic) last longer in the body than standard cells for people with sickle cell anemia who need regular transfusions. About 48 participants aged 7 and older will receive both types of blood in a crossover de…
Phase: NA • Sponsor: Hemanext • Aim: Disease control
Last updated Jun 27, 2026 08:08 UTC
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One-Shot gene therapy could free PKU patients from strict diet
Disease control Not yet recruitingThis early-phase trial tests a single injection of a gene therapy called GS1168 in 9 adults with PKU whose condition is not well controlled by diet or other treatments. The therapy uses a harmless virus to deliver a working copy of the PAH gene, which is faulty in PKU, to help th…
Phase: EARLY_PHASE1 • Sponsor: Gritgen Therapeutics Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 08:08 UTC
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New hope for stomach cancer: immunotherapy combo targets resistant tumors
Disease control Not yet recruitingThis phase 2 trial will test a combination of immunotherapy drugs and chemotherapy given before surgery for people with a specific genetic subtype of stomach cancer (dMMR/MSI-H). The study aims to see how well this approach clears the tumor and to understand why some patients do …
Phase: PHASE2 • Sponsor: Shanghai Zhongshan Hospital • Aim: Disease control
Last updated Jun 27, 2026 08:08 UTC
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New pill aims to ease sickle cell anemia without pain crises
Disease control Not yet recruitingThis study tests a drug called SIL-8301 (senicapoc) in 105 adults with sickle cell disease who have low hemoglobin but few pain crises. Participants take the drug or a placebo daily for 24 weeks, along with their usual hydroxyurea. The main goal is to see if the drug raises hemog…
Phase: PHASE2 • Sponsor: Biossil Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:04 UTC
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Breast cancer drug tamoxifen tested as new hope for cystic fibrosis
Disease control Not yet recruitingThis study tests whether tamoxifen citrate, a drug commonly used for breast cancer, is safe and tolerable for adults with cystic fibrosis who cannot take current CFTR modulator drugs. Thirty-five participants will take one tablet daily for six months and visit the clinic every 28…
Phase: PHASE2 • Sponsor: Azienda Ospedaliera Universitaria Integrata Verona • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC
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Virus cocktail takes on superbugs in cystic fibrosis trial
Disease control Not yet recruitingThis early-stage study tests whether adding specially-made viruses (phages) to standard antibiotics can safely treat stubborn lung infections in adults with cystic fibrosis. About 30 participants will receive a 7-day course of both treatments. The main goal is to check safety, bu…
Phase: PHASE1 • Sponsor: University of California, San Diego • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC
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New drug YOLT-204 aims to reduce transfusions in blood disorders
Disease control Not yet recruitingThis early-phase trial will test a single dose of a new drug called YOLT-204 in 18 children and teens with transfusion-dependent β-thalassemia or sickle cell disease. The goal is to see if it is safe and can raise fetal hemoglobin levels enough to reduce the need for blood transf…
Phase: EARLY_PHASE1 • Sponsor: Guangzhou Women and Children's Medical Center • Aim: Disease control
Last updated Jun 27, 2026 07:51 UTC
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New At-Home PKU test could give results in under an hour
Diagnosis Not yet recruitingThis study will test a new device called PheCheck™ that measures phenylalanine levels from a single fingerstick drop of blood. About 60 people with PKU or hyperphenylalaninemia, aged 1 year and older, will use the test at home and compare results to standard lab testing. The goal…
Sponsor: Aptatek BioSciences, Inc • Aim: Diagnosis
Last updated Jun 27, 2026 12:24 UTC
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New sweat patch could make cystic fibrosis testing easier
Diagnosis Not yet recruitingThis study will test a new device called the micro Sweat Test Patch (µSTP) that measures chloride levels in sweat to help diagnose and monitor cystic fibrosis (CF). Researchers will compare the new patch to the standard sweat test in 300 newborns, children, and adults with CF. Th…
Phase: NA • Sponsor: Emory University • Aim: Diagnosis
Last updated Jun 27, 2026 09:05 UTC
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Mini organs could solve cystic fibrosis diagnosis puzzle
Diagnosis Not yet recruitingThis study aims to develop a more accurate test for cystic fibrosis (CF) using lab-grown mini organs called organoids, made from a small piece of gut tissue. The researchers will compare this new test with current methods like sweat tests and genetic testing in 80 people who are …
Sponsor: Royal Brompton & Harefield NHS Foundation Trust • Aim: Diagnosis
Last updated Jun 27, 2026 09:05 UTC
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Liquid biopsies may let lynch syndrome patients skip half their colonoscopies
Diagnosis Not yet recruitingThis study tests whether annual liquid biopsies (from blood, urine, stool, and vaginal swabs) can safely replace colonoscopies every 18 months for people with Lynch syndrome. 2,000 participants will be split into two groups: one gets standard care (colonoscopy every 18 months), t…
Phase: NA • Sponsor: UNICANCER • Aim: Diagnosis
Last updated Jun 27, 2026 08:13 UTC
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Tuning in to relief: music therapy app tested for sickle cell pain
Symptom relief Not yet recruitingThis study will test whether a 4-week daily music therapy app can reduce chronic pain and improve quality of life in 25 youth with sickle cell disease. Participants will either use the app or receive standard educational materials. Researchers will also look at changes in immune …
Phase: NA • Sponsor: Emory University • Aim: Symptom relief
Last updated Jul 03, 2026 00:00 UTC
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Could a VR headset ease sickle cell pain?
Symptom relief Not yet recruitingThis study explores whether using a virtual reality headset can help reduce pain for adults with sickle cell disease during a painful crisis. Participants will try different types of headset experiences—like watching a 2D video or exploring a 3D world—during their infusion center…
Phase: NA • Sponsor: University of Maryland, Baltimore • Aim: Symptom relief
Last updated Jul 01, 2026 00:00 UTC
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Can a VR app ease the pain of sickle cell disease? new study aims to find out
Symptom relief Not yet recruitingThis study tests a virtual reality app called Amani designed to help adults with sickle cell disease manage pain, emotional distress, and social isolation. Researchers will enroll 40 adults to see if the app is easy to use and helpful for improving coping skills and peer support.…
Phase: EARLY_PHASE1 • Sponsor: Massachusetts General Hospital • Aim: Symptom relief
Last updated Jun 27, 2026 14:00 UTC
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Could a simple saltwater mist make breathing easier for lung patients?
Symptom relief Not yet recruitingThis study looks at whether adding a continuous saltwater mist to warm, humidified air can make thick lung mucus easier to clear. 35 adults hospitalized with lung conditions like COPD or cystic fibrosis will receive both treatments on separate days. Researchers will measure mucus…
Phase: NA • Sponsor: Cliniques universitaires Saint-Luc- Université Catholique de Louvain • Aim: Symptom relief
Last updated Jun 27, 2026 13:00 UTC
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Could an old antibiotic shield the brain in sickle cell disease?
Symptom relief Not yet recruitingThis study tests whether minocycline, an antibiotic that reduces brain inflammation, can safely prevent or reverse memory and thinking problems in adults with sickle cell disease. About 30 participants will take either a low dose, high dose, or placebo for one year. Researchers w…
Phase: PHASE1 • Sponsor: University of Cincinnati • Aim: Symptom relief
Last updated Jun 27, 2026 12:34 UTC
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Could a simple seed ease sickle cell pain? new study hopes to find out
Symptom relief Not yet recruitingThis study will test whether eating flaxseed, a source of omega-3 fatty acids, can help reduce pain and improve daily life for children with sickle cell disease. Twenty children aged 5 to 18 will take part. The main goal is to see if it's possible for them to eat flaxseed regular…
Phase: NA • Sponsor: University of Alabama at Birmingham • Aim: Symptom relief
Last updated Jun 27, 2026 12:07 UTC
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New combo therapy aims to clear lungs and boost stamina in cystic fibrosis
Symptom relief Not yet recruitingThis study tests whether adding exercise training to a standard chest-vibration vest (HFCWO) helps people with cystic fibrosis clear mucus, cough less, and walk farther. Twenty-four adults aged 16–35 will either get the vest plus exercise or the vest alone for 8 weeks. The goal i…
Phase: NA • Sponsor: Riphah International University • Aim: Symptom relief
Last updated Jun 27, 2026 12:05 UTC
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Remote rehab: a new hope for cystic fibrosis patients battling frailty
Symptom relief Not yet recruitingThis study tests whether an 8-week tele-rehabilitation program can improve muscle mass, reduce frailty, and enhance quality of life in adults with cystic fibrosis. Participants will do supervised exercise sessions at home via video calls three times a week. The goal is to see if …
Phase: NA • Sponsor: King Saud University • Aim: Symptom relief
Last updated Jun 27, 2026 12:02 UTC
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New hope for sickle cell pain: could iloprost cut opioid use?
Symptom relief Not yet recruitingThis study tests if iloprost, a drug that widens blood vessels and reduces inflammation, can help adults with sickle cell disease during painful vaso-occlusive crises. About 144 participants hospitalized for a crisis will receive either iloprost or a placebo, plus standard care. …
Phase: PHASE3 • Sponsor: University Hospital, Rouen • Aim: Symptom relief
Last updated Jun 27, 2026 12:00 UTC
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Could a single dose of ketamine keep sickle cell patients out of the hospital?
Symptom relief Not yet recruitingThis study tests whether giving ketamine within an hour of pain onset can prevent hospital admission for children and young adults with sickle cell disease. 90 participants aged 6-24 will receive either ketamine or a placebo by mouth. If admitted, they can still receive standard …
Phase: PHASE3 • Sponsor: Boston Children's Hospital • Aim: Symptom relief
Last updated Jun 27, 2026 11:00 UTC
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Exercise and drug combo may fight fatigue in rare disease
Symptom relief Not yet recruitingThis study looks at whether doing aerobic exercise at home, with or without the drug omaveloxolone, can safely improve fatigue and heart-lung fitness in people with Friedreich's ataxia. About 30 adults with the condition will take part. The goal is to see if the combination helps…
Phase: NA • Sponsor: Scott Barbuto • Aim: Symptom relief
Last updated Jun 27, 2026 09:09 UTC
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Could a common numbing drug cut opioid needs in sickle cell emergencies?
Symptom relief Not yet recruitingThis study tests whether adding lidocaine, a numbing medicine, to standard pain treatment can lower the amount of strong opioids needed during severe sickle cell pain crises. About 104 adults with sickle cell disease who are in the intensive care unit for a pain crisis will take …
Phase: PHASE3 • Sponsor: Nantes University Hospital • Aim: Symptom relief
Last updated Jun 27, 2026 08:06 UTC
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Could lying on your stomach ease a deadly sickle cell complication?
Symptom relief Not yet recruitingThis study tests whether awake prone positioning (lying on the stomach while awake) can help sickle cell patients with severe acute chest syndrome in the ICU. The approach aims to improve oxygen levels and breathing by evening out air flow in the lungs. Fifteen adults will be enr…
Phase: NA • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Symptom relief
Last updated Jun 26, 2026 13:15 UTC
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Virtual reality fights pharmacy stigma for sickle cell patients
Knowledge-focused Not yet recruitingThis study tests whether a computer simulation can help pharmacists and pharmacy students better understand the pain struggles of sickle cell disease patients. About 200 healthcare professionals will use a point-of-view simulation and then complete surveys to see if their misconc…
Phase: NA • Sponsor: Ohio State University • Aim: Knowledge-focused
Last updated Jul 03, 2026 00:00 UTC
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Air pollution may worsen cystic fibrosis in kids, study hopes to find out
Knowledge-focused Not yet recruitingThis study will enroll 200 children with cystic fibrosis (100 Hispanic, 100 non-Hispanic white) to measure indoor and outdoor air pollution and see how it relates to lung function, inflammation, and airway bacteria. Researchers hope to understand why Hispanic children often have …
Sponsor: Seattle Children's Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:02 UTC
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Scientists track hidden threat of antibiotic resistance in lung patients
Knowledge-focused Not yet recruitingThis study follows people with chronic lung diseases like bronchiectasis, cystic fibrosis, and COPD to understand how bacteria become resistant to antibiotics. Researchers will collect samples and health data over time to map resistance patterns and genetic causes. The goal is to…
Sponsor: Imperial College London • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:01 UTC
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Can a light cap boost brain power in kids with sickle cell?
Knowledge-focused Not yet recruitingThis study tests whether shining a special light on the head (transcranial photobiomodulation) can improve oxygen use and thinking skills in children with sickle cell disease. The brain needs steady oxygen, but sickle cell can reduce blood flow. Researchers will randomly assign 6…
Phase: NA • Sponsor: St. Jude Children's Research Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC
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Stomach cancer study may spare some patients unnecessary surgery
Knowledge-focused Not yet recruitingThis study looks at whether a tumor's DNA repair status (MMR) changes how well the presence of cancer cells in lymph vessels predicts spread to lymph nodes in stomach cancer. Researchers will review medical records of 3,000 patients who had surgery. The goal is to improve risk as…
Sponsor: Shanghai Zhongshan Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC
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Can mindfulness ease trauma for Justice-Involved black teens?
Knowledge-focused Not yet recruitingThis study looks at whether mindfulness-based stress reduction can help Black teenage girls who have been involved with the justice system and their parents or caregivers. About 90 participants will report on stress, PTSD symptoms, and other outcomes before and after the program.…
Phase: NA • Sponsor: University of Michigan • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC
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New study aims to track dangerous infections in kids with sickle cell disease
Knowledge-focused Not yet recruitingThis study will look at medical records of 350 children with sickle cell disease in France who had serious bacterial infections between 2020 and 2025. The goal is to find out which bacteria are causing these infections today, especially with new vaccines available. The results co…
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:08 UTC
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How strong should you be? new study aims to find out
Knowledge-focused Not yet recruitingThis study will measure leg and handgrip strength in about 2,800 healthy adults and people with chronic lung diseases across Spain, Portugal, and Italy. Researchers want to create normal muscle strength values for these populations and see if the same standards can apply to peopl…
Sponsor: University of Alcala • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:05 UTC
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Tailoring sickle cell drug doses: a new hope for nigerian patients
Knowledge-focused Not yet recruitingThis study looks at how genetic differences affect the way Nigerian sickle cell patients respond to hydroxyurea, a common treatment. Researchers will monitor 100 patients to build a computer model that predicts the best dose for each person. The goal is to reduce side effects and…
Sponsor: Ochuko Orherhe • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:26 UTC
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Can a 90-Minute training improve care for kids with sickle cell?
Knowledge-focused Not yet recruitingThis study tests a 90-minute virtual training for healthcare providers that teaches them to see each patient's unique perspective and communicate more clearly. The goal is to improve how doctors talk to children with sickle cell disease. Thirty providers from three hospitals will…
Phase: NA • Sponsor: Connecticut Children's Medical Center • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:25 UTC
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Breathing test may replace overnight sleep studies for CPAP settings
Knowledge-focused Not yet recruitingThis study looks at whether a quick, non-invasive breathing test called impulse oscillometry (IOS) can help doctors find the right pressure settings for sleep apnea machines. Currently, patients often need an overnight sleep study to determine the correct pressure. The study will…
Sponsor: Mustafa Kemal University • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:24 UTC
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Rare skin disease patients develop even rarer lymphoma – scientists investigate why
Knowledge-focused Not yet recruitingThis study looks at the blood immune cells of 10 adults with a rare inherited skin condition called NIPAL4 ichthyosis. Researchers want to understand why three patients with this skin disease also developed a very rare lymphoma (Sezary syndrome). By describing the normal immune c…
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:02 UTC
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Anemia may skew diabetes test results, new study warns
Knowledge-focused Not yet recruitingThis study looks at how different types of anemia—like iron deficiency or thalassemia—can change HbA1c levels, a common test for diabetes, in people who do not have diabetes. Researchers will compare HbA1c results between 100 non-diabetic adults with various anemias and a healthy…
Sponsor: Sohag University • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:00 UTC
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New MRI method could track cystic fibrosis lung damage without X-Rays
Knowledge-focused Not yet recruitingThis study explores whether MRI can produce clear images of the lungs and airways in people with cystic fibrosis (CF) and healthy volunteers. Researchers will use computer assistance to measure airway size and shape, and repeat the MRI two years later to see if changes occur. A s…
Sponsor: The Hospital for Sick Children • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:10 UTC
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New european registry aims to unlock mysteries of rare breast disease
Knowledge-focused Not yet recruitingThis study is creating a European registry for people with idiopathic granulomatous mastitis (IGM), a rare inflammatory breast condition that causes pain, redness, and abscesses. Researchers will collect information from 1,000 participants to better understand symptoms, treatment…
Sponsor: European Breast Cancer Research Association of Surgical Trialists • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:08 UTC
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PKU mystery: why do some patients thrive despite high phe levels?
Knowledge-focused Not yet recruitingThis study looks at how blood phenylalanine (Phe) levels affect thinking, emotions, and daily functioning in adults with PKU. Researchers will ask 30 adults with PKU and their relatives to complete questionnaires. The goal is to understand why some people with high Phe levels hav…
Sponsor: Central Hospital, Nancy, France • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:05 UTC
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New study to track cystic fibrosis drugs in pregnancy and breast milk
Knowledge-focused Not yet recruitingThis study looks at how cystic fibrosis medications (CFTR modulators) change in the body during pregnancy and after childbirth in 30 women. Researchers will measure drug levels in blood, breast milk, and umbilical cord blood. The goal is to understand how these drugs work in preg…
Sponsor: University of Minnesota • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:12 UTC
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Scientists investigate why cystinosis causes early aging
Knowledge-focused Not yet recruitingThis study looks at how energy production inside cells (mitochondria) works in people with cystinosis, a rare disease that causes cystine buildup and early aging. Researchers will measure cell energy activity in 25 patients and compare it to healthy people. The goal is to underst…
Phase: NA • Sponsor: Hospices Civils de Lyon • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:08 UTC
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New study aims to unlock secrets of sickle cell pain crises in kids
Knowledge-focused Not yet recruitingThis study will review medical records of 25 children with sickle cell disease to see how often they experience painful vaso-occlusive crises and what complications arise. Researchers hope to identify risk factors that could lead to better care. No new treatments are being tested…
Sponsor: Assiut University • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:53 UTC
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New study aims to unmask hidden causes of childhood joint stiffness
Knowledge-focused Not yet recruitingThis study will look at 35 children with joint problems that are not caused by inflammation, such as stiffness or deformity. Researchers will use exams, lab tests, and imaging to find the true cause, which could be rare genetic conditions like mucopolysaccharidoses or osteogenesi…
Sponsor: Assiut University • Aim: Knowledge-focused
Last updated Jun 26, 2026 17:17 UTC
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Brain speed test could transform sickle cell care
Knowledge-focused Not yet recruitingThis study examines how regular blood transfusions (exchange transfusions) affect thinking speed in adults with severe sickle cell disease. Researchers will test 85 participants remotely using a simple symbol-matching task and other cognitive assessments at different points in th…
Sponsor: Centre Hospitalier St Anne • Aim: Knowledge-focused
Last updated Jun 26, 2026 16:17 UTC