Clinical trials
Down Syndrome clinical trials
Below are recruiting down syndrome clinical trials, each written for real people, not researchers. We’re tracking 3,187 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT07771855RecruitingPhase 2
Antibody treatment for chronic lung disease after stem cell transplant
This study tests a new antibody medicine called tezelumab for patients who develop a breathing problem called bronchiolitis obliterans syndrome (BOS) after a stem cell transplant. The trial aims to see if this medicine can reduce flare-ups and improve lung function.
BesançonAges 18 years+ - NCT07727538RecruitingPhase 3
Olezarsen for children with familial chylomicronemia syndrome
This trial tests a drug called olezarsen in children with a genetic condition that causes extremely high triglycerides. It aims to lower triglyceride levels and prevent pancreatitis.
Birmingham, AlabamaAges 2–17 - NCT04206215Recruiting
Noninvasive brain stimulation for carpal tunnel pain relief
This study tests whether a noninvasive brain stimulation treatment can reduce chronic carpal tunnel (CTS) pain, especially when pain spreads beyond the usual nerve area. If you have long-lasting CTS pain that hasn’t improved with standard treatments, you may be able to try this therapy while being closely monitored for safety.
Charlestown, MassachusettsAges 18–80 - NCT07722247Recruiting
Eye stiffness and healing in cataract surgery with PEX
This study looks at how your eye's outer layer (cornea) heals and changes shape after cataract surgery, specifically comparing people who have pseudoexfoliation syndrome (PEX) with those who do not. The goal is to see if PEX affects the final vision result after cataract removal.
Split, Splitsko-dalmatinska CountyAges 50–85 - NCT07503444RecruitingPhase 3
Fenfluramine for Rett syndrome symptoms
This study tests whether fenfluramine, a medication already used for seizures, can help people with Rett syndrome. It aims to see if it improves daily life and reduces symptoms.
Little Rock, ArkansasAges 5–35 - NCT06577441RecruitingPhase 2
Adding enasidenib to usual care for higher-risk MDS with IDH2 mutation
This trial tests whether adding enasidenib (a targeted pill) to the standard chemotherapy (cedazuridine-decitabine) works better than the chemotherapy alone for people with higher-risk myelodysplastic syndrome (MDS) whose cells have a specific change in the IDH2 gene.
Berkeley, CaliforniaAges 18 years+ - NCT07758842Recruiting
Lifestyle changes for erectile dysfunction in men with metabolic syndrome
This study tests whether a six-month lifestyle modification program (including diet and exercise) can improve erectile function in men who have both erectile dysfunction and metabolic syndrome. If you're eligible, you may get support to make healthier lifestyle changes, which could help improve your symptoms.
CairoAges 30–65 - NCT07450664Enrolling by invitation
Observational study of VYKAT XR for Prader-Willi syndrome
This study follows people with Prader-Willi syndrome who are already taking or starting VYKAT XR, including those switching from an earlier version of the drug (DCCR). It aims to understand how the treatment works in everyday life.
Phoenix, ArizonaAges 4 years+ - NCT04462770RecruitingPhase 3
Clemizole trial for children and adults with Dravet syndrome
This Phase 3 study tests EPX-100 (clemizole) to help reduce seizures in people with Dravet syndrome whose seizures are not fully controlled by current anti-seizure medicines. You may consider this study if your Dravet diagnosis is confirmed and your medical situation fits the specific rules about medicines and seizure causes.
Birmingham, AlabamaAges 2 years+ - NCT07211685RecruitingPhase 2
Testing a new drug for adults with Alport syndrome
This study tests a new medicine called BAY 3401016 to see if it can help adults with Alport syndrome, a genetic condition that affects the kidneys. The drug is given as an infusion or injection and the study will check how well it works and if it is safe.
Birmingham, AlabamaAges 18–45 - NCT07754149Recruiting
Progressive resistance training for PMS in teen girls
This study tests whether a 6-week progressive strength training program can reduce symptoms of premenstrual syndrome (PMS) in teenage girls. It compares different exercise intensities to see what works best and may offer a drug-free way to manage PMS.
Islamabad, FederalAges 11–19 - NCT07102628RecruitingPhase 3
Early treatment with inclisiran for heart attack patients
This study tests if starting inclisiran soon after a heart attack helps lower cholesterol further in patients already on statins. The goal is to see if early treatment can reduce future heart risks.
Capital Federal, Buenos AiresAges 18 years+ - NCT06914609RecruitingPhase 3
Study of ION582 for people with Angelman syndrome
This trial tests a new medicine called ION582 for people with Angelman syndrome. It aims to see if it can help with symptoms and is given as a lumbar puncture (spinal tap). You may be able to join if you have a genetic diagnosis of Angelman syndrome and are between 2 and 50 years old.
Los Angeles, CaliforniaAges 2–50 - NCT05320198RecruitingPhase 1/Phase 2
Testing DISC-0974 for anemia in myelofibrosis or MDS
This trial tests an investigational drug called DISC-0974 for people with myelofibrosis or certain types of MDS who have low red blood cell counts (anemia). The goal is to see if this treatment can help improve anemia and reduce the need for blood transfusions.
Gilbert, ArizonaAges 18 years+ - NCT07593391RecruitingPhase 3
NNZ-2591 Treatment for Children with Phelan-McDermid Syndrome
This study tests whether a medication called NNZ-2591 can help children with Phelan-McDermid syndrome, a rare genetic condition that affects brain development and learning. Researchers want to see if this drug can improve how children with this condition develop and learn.
Glendale, CaliforniaAges 3–12 - NCT06668922Recruiting
Diet and irritable bowel syndrome study
This study compares how people with and without irritable bowel syndrome (IBS) react to different foods. It may help find which foods trigger symptoms and which are safe to eat.
Los Angeles, CaliforniaAges 18–75 - NCT07281079RecruitingPhase 3
Testing NNZ-2591 for children with Phelan-McDermid syndrome
This trial tests a new medicine, NNZ-2591, to see if it helps children with Phelan-McDermid syndrome. It is for children aged 3 to 12 who have a specific genetic change (SHANK3) and weigh at least 10 kg.
Birmingham, AlabamaAges 3–12 - NCT07746635Recruiting
Fecal transplant for obese women with polyendocrine metabolic ovarian syndrome
This study tests whether a fecal microbiota transplant (a transfer of healthy gut bacteria) can help women with polyendocrine metabolic ovarian syndrome who are also overweight or obese and planning a frozen embryo transfer. It aims to see if this treatment can improve hormone balance and possibly increase the chance of a successful pregnancy.
Shanghai, Shanghai MunicipalityAges 20–40 - NCT07756203Recruiting
Comparing two hand-held treatments for shoulder pain
This study tests two non-surgical treatments for shoulder pain caused by shoulder impingement. Both treatments use hand-held tools to ease pain and improve movement.
IstanbulAges 40–60 - NCT07636811RecruitingPhase 2
Mivelsiran for Early Alzheimer's in Down Syndrome
This trial tests a new drug called mivelsiran designed to slow early-stage Alzheimer's disease in adults with Down syndrome. The drug works by targeting amyloid, a protein that builds up in the brain and causes cognitive decline.
Maitland, FloridaAges 40–55 - NCT07757789Recruiting
Platelet changes in antiphospholipid syndrome
This study looks at how platelet counts change over time in people with antiphospholipid syndrome (APS), a condition that can cause blood clots and pregnancy problems. It may help doctors better understand and manage low platelets in APS patients.
Al Khārjah, Kharga OasisAges 18 years+ - NCT07562425Recruiting
Knee Strengthening Exercises for Patellofemoral Pain
This study compares three types of thigh muscle exercises to help reduce knee pain in people with patellofemoral pain syndrome (pain around the kneecap). Researchers want to see which exercise style—holding steady, moving through motion, or lowering slowly—works best for your pain.
Elâzığ, MErkezAges 18–50 - NCT06465641RecruitingPhase 4
Methylphenidate for ADHD symptoms in KBG syndrome
This study tests if the medication methylphenidate (commonly used for ADHD) can help manage attention and behavior problems in people with KBG syndrome. Each person acts as their own test case to see if the drug works for them.
Nijmegen, GelderlandAges 6–20 - NCT07750392Recruiting
Dry needling and gentle laser therapy for recent neck pain
This trial tests two therapies—dry needling (thin needles into muscle knots) and low-level laser/microcurrent (gentle energy) treatment—alone or combined, to relieve acute neck pain from muscle knots (myofascial pain). It aims to reduce pain and improve function for people with recent onset symptoms.
Denpasar, BaliAges 20–45
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Common questions
- Are there clinical trials for down syndrome?
- Yes. Clin2 currently lists 3,187 recruiting down syndrome studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a down syndrome trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a down syndrome trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.