Clinical trials
Down Syndrome clinical trials
Below are recruiting down syndrome clinical trials, each written for real people, not researchers. We’re tracking 3,177 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT07243327Recruiting
Shockwave therapy vs standard rehab for hip pain
This trial compares two treatments—shockwave therapy and conventional physical therapy—for long-lasting pain on the outside of the hip, often called trochanteric pain syndrome. It aims to find which option works better for people whose hip pain has persisted for months.
IstanbulAges 18–65 - NCT07135050RecruitingPhase 1/Phase 2
Gene therapy study for Pitt Hopkins syndrome
This trial tests a new gene therapy called MZ-1866 for people with Pitt Hopkins syndrome. The therapy is given as a single injection into the fluid around the brain to help replace the missing or faulty TCF4 gene.
Oakland, CaliforniaAges 2–25 - NCT07045259Recruiting
Exergaming vs. exercise for memory and movement in seniors
This study compares fun video games that get you moving (exergaming) with standard muscle-strengthening exercises. It aims to see which one better helps older adults who have both slower walking speed and memory concerns.
BalıkesirAges 65 years+ - NCT07747909Recruiting
Orlistat for metabolic and hormonal health in PCOS
This study tests whether the weight-loss drug orlistat can improve blood sugar, hormones, and other metabolic measures in women with polycystic ovary syndrome (PCOS). If you have PCOS and are overweight, this treatment might help with symptoms like irregular periods and excess hair growth.
Abbottābād, Khyber PakhtunkhwaAges 18–45 - NCT07531745RecruitingPhase 1/Phase 2
ION337 gene therapy for children with Dravet syndrome
This study tests a new gene therapy called ION337 designed to treat Dravet syndrome, a severe childhood epilepsy caused by a SCN1A gene mutation. The therapy is given as a spinal fluid injection and aims to reduce seizures in children ages 2–12.
Ann Arbor, MichiganAges 2–12 - NCT07434037Recruiting
Trust and the brain in Down syndrome and Fragile X
This study explores how people with Down syndrome or Fragile X syndrome learn to trust others. It uses simple tasks and a brain scan (MRI) to compare their responses with those of typically developing children and young adults.
BronAges 3–29 - NCT06872125RecruitingPhase 3
Testing zorevunersen for Dravet syndrome
This study tests an experimental drug called zorevunersen for people with Dravet syndrome. It aims to see if the drug can reduce seizures and improve safety.
Phoenix, ArizonaAges 2–17 - NCT07742735RecruitingPhase 3
Study of apraglutide for short bowel syndrome with intestinal failure
This study tests a new medicine called apraglutide, given as a shot, to see if it can help adults with short bowel syndrome and intestinal failure reduce their need for intravenous nutrition (parenteral support). The study compares apraglutide to a placebo (a shot with no medicine) to check if it is safe and effective.
Los Angeles, CaliforniaAges 18 years+ - 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+ - NCT05677100Recruiting
Aortix device for acute heart failure
This trial is testing a small pump placed in the aorta to help the kidneys remove extra fluid when standard IV diuretics aren't working well enough for severe heart failure. It may help you if you're hospitalized and still very congested (swollen, fluid buildup) despite strong water pills.
Phoenix, ArizonaAges 21 years+ - NCT07743242Recruiting
Study of hemolytic anemia in antiphospholipid syndrome
This study looks at how autoimmune hemolytic anemia (when the body attacks its own red blood cells) relates to antiphospholipid syndrome (a condition that increases blood clot risk). It aims to better understand this connection, which may help improve care for people with both conditions.
Al Khārjah, Kharga OasisAges 18 years+ - NCT07585097Recruiting
Long-term Safety Study of Odevixibat for Alagille Syndrome
This study follows patients with Alagille syndrome who are taking odevixibat to see how safe the medicine is over time. Alagille syndrome is a rare liver disease that affects bile flow, and odevixibat is a treatment that may help reduce itching and improve liver function.
BronAges 6 months+ - NCT06850038Recruiting
Long-term study of odevixibat in Alagille syndrome
This study follows people with Alagille syndrome who are already taking or starting odevixibat (Bylvay). It looks at how well the medicine works and how safe it is over a long period of time.
Los Angeles, CaliforniaAges Any age - NCT07469566Recruiting
Studying the natural course of 7q11.23 microduplication syndrome
This study follows people with a rare genetic condition called 7q11.23 microduplication over time to understand how it affects development, learning, and health. Participants will have interviews, thinking and memory tests, and an MRI brain scan.
BronAges 5–50 - NCT06287944RecruitingPhase 1
Targeted radiation and chemo before donor stem cell transplant
This trial tests a new approach: giving a targeted radioactive antibody (attached to a drug that finds CD38 on cancer cells) along with low-dose chemotherapy and targeted radiation to the bone marrow before a donor stem cell transplant. The goal is to prepare the body for the transplant while killing remaining cancer cells more precisely.
Duarte, CaliforniaAges 18–70 - NCT07736144Recruiting
Herbal formula study for fibromyalgia pain relief
This study tests a Chinese herbal formula called Yangyin Roujin (which nourishes yin and softens the muscles) to see if it helps reduce pain and other symptoms of fibromyalgia. If you have fibromyalgia and a pain level of 4 or more, you may be able to join.
Beijing, Beijing MunicipalityAges 18–75 - NCT06529731RecruitingPhase 2
Interferon-Gamma and Donor Cells for Relapsed Leukemia After Transplant
This trial tests combining a protein called interferon-gamma with an infusion of donor white blood cells to treat AML or MDS that returned after a stem cell transplant. The goal is to see if this combination helps the donor cells attack the cancer more effectively.
St Louis, MissouriAges 18 years+ - NCT07142239Recruiting
Blood markers in primary vs. secondary antiphospholipid syndrome
This study looks at three blood markers (MPV, PLR, and NLR) in people with antiphospholipid syndrome (APS) to see if they differ between primary APS (no other autoimmune disease) and secondary APS (with lupus or another autoimmune condition). It may help doctors better understand and manage APS.
Al Khārjah, Kharga OasisAges 18 years+ - NCT05334368RecruitingPhase 3
Depemokimab for hypereosinophilic syndrome with frequent flares
This trial studies depemokimab, a medicine meant to reduce flares in people with hypereosinophilic syndrome (HES). It also checks how safe the treatment is and how well it works over time.
San Diego, CaliforniaAges 18 years+ - NCT07734051Enrolling by invitation
Mobilization for kneecap pain
This trial tests if adding a groin-area mobilization to foot mobilization helps with kneecap (patellofemoral) pain. It may be for you if you have one-sided kneecap pain for at least 3 months, especially when climbing stairs, squatting, or sitting for long periods.
Fāqūs, SharkeyaAges 18–40 - NCT06616636RecruitingPhase 1
Rezatapopt and azacitidine for AML or MDS with a specific TP53 mutation
This trial tests a new drug, rezatapopt, combined with azacitidine for people with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) that has come back or not responded to treatment. It is only for people whose cancer cells have a specific change in a gene called TP53 (the Y220C mutation). The goal is to see if this combination can help control the disease.
Tampa, FloridaAges 18 years+ - NCT07605429RecruitingPhase 3
Rugonersen treatment for Angelman syndrome
This study tests a new medicine called rugonersen for people with Angelman syndrome. The medicine is given as an injection into the fluid around the spinal cord and may help improve symptoms by targeting the genetic cause of the condition.
San Diego, CaliforniaAges 1–50 - NCT07725926Recruiting
Combining therapy techniques for tic-like OCD
This study tests a therapy that combines two approaches—one for tics and one for OCD—to help people whose OCD feels like it's linked to tics. The goal is to see if this combined treatment reduces distressing urges and repetitive behaviors.
London, LondonAges 18–65 - NCT07411222Recruiting
Emotional Eating, Sleep, and Metabolic Health Study
This study examines how emotional eating, sleep quality, and mental state relate to metabolic syndrome (a group of conditions like high blood pressure, high blood sugar, and extra belly fat) in people with stable schizophrenia or bipolar disorder. It aims to help understand and improve overall health.
Bolu, BoluAges 18–65
Hear when a new Down Syndrome trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.
Common questions
- Are there clinical trials for down syndrome?
- Yes. Clin2 currently lists 3,177 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.