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
- NCT00801489RecruitingPhase 2
New treatment for AML or high-risk MDS using combination chemo
This trial tests a combination treatment (chemo drugs plus a growth-factor medicine) for people newly diagnosed with acute myeloid leukemia (AML) or certain high-risk myelodysplastic syndromes (MDS). It may help by targeting cancer cells in the blood and bone marrow and improving remission chances.
Houston, TexasAges 18 years+ - NCT01624805RecruitingPhase 2
Immunosuppressant treatment to help low blood counts in some bone marrow disorders
This trial tests a group of immune-suppressing medicines (with steroids and growth-factor injections) for people with aplastic anemia or certain low-risk myelodysplastic syndromes. The goal is to improve bone marrow function and reduce problems caused by low blood counts, such as fatigue, infections, or bleeding.
Houston, TexasAges 18 years+ - NCT02115295RecruitingPhase 2
Venetoclax plus chemotherapy for AML, high-risk MDS, or blast CML
This trial tests a combination treatment (venetoclax plus several chemotherapy medicines) for people with AML, high-risk MDS, or CML that has progressed into a blast phase. It may help by killing leukemia-like blood cells and improving disease control.
Houston, TexasAges 18–65 - NCT05432349Recruiting
Rett syndrome genetic registry for MECP2 changes
This trial is a registry that collects health and genetic information from people with Rett syndrome. It may help researchers better understand how MECP2 loss-of-function changes lead to symptoms and how to support patients.
Birmingham, AlabamaAges birth–99 years - NCT07450612Recruiting
Blood test and AI for early colorectal cancer detection
This study uses a simple blood test (liquid biopsy) and computer analysis to find early signs of colorectal cancer, precancerous growths, and Lynch syndrome. It aims to improve early detection and monitor for any remaining cancer after treatment.
Milan, LombardyAges 18 years+ - NCT07674290RecruitingPhase 4
Study of MC4R Agonist for Bardet-Biedl Syndrome and Genetic Obesity
This trial tests a drug called setmelanotide for people with Bardet-Biedl Syndrome (a rare genetic condition) or other severe genetic obesity. It looks at how well the drug works in real-world settings, not just in a lab.
EssenAges Any age - NCT07671482Recruiting
Kinesio vs McConnell taping for knee pain relief
This study compares two types of taping (Kinesio and McConnell) to see which works better for people with pain behind the kneecap (patellofemoral pain syndrome). You may help researchers learn how to best relieve knee pain during daily activities.
Beirut, BeirutAges 18–50 - NCT07381985Enrolling by invitation
Managing hereditary cancer risk in rural areas
This study tests a way to help people with inherited cancer gene mutations manage their health, especially if they live in rural areas. You join if you found out about the mutation more than a year ago.
Burlington, VermontAges 18 years+ - NCT06915077Enrolling by invitation
Personalized treatment for sleep apnea
This study tests alternative treatments for people with moderate to severe sleep apnea who cannot use CPAP. It aims to find more tolerable options.
Wilrijk, AntwerpenAges 18 years+ - NCT06914726Enrolling by invitation
Cancer risk follow-up care for hereditary syndromes
This study tests a tool to help primary care doctors provide better cancer prevention care for people with hereditary breast and ovarian cancer (HBOC) or Lynch syndrome (LS). It aims to ensure you're up-to-date on recommended screenings and preventive steps.
Minneapolis, MinnesotaAges 18 years+ - NCT06306157Enrolling by invitationPhase 4
Low dose naltrexone for complex regional pain syndrome
This trial tests whether a low dose of naltrexone, a medication that may help calm overactive immune and pain signals, can reduce pain and improve daily life in people with complex regional pain syndrome (CRPS), a chronic condition that often causes severe pain and swelling.
New York, New YorkAges 18–85 - NCT05521958Enrolling by invitation
Gene activity study in severe leg muscle swelling after fracture
This study looks at gene activity (what genes are “turned on or off”) in the lower leg in people with acute compartment syndrome after a tibia (shin bone) injury. The goal is to better understand what is happening in the body and potentially guide future treatment decisions.
Salt Lake City, UtahAges 18–90 - NCT07262983RecruitingPhase 1
Baricitinib for Job syndrome with lupus or eczema
This study tests if baricitinib, a pill that changes how your immune system works, is safe and tolerable for people with Job syndrome who also have lupus-like disease or severe eczema. It might offer a new treatment option for these skin and immune problems.
Bethesda, MarylandAges 12–120 - NCT06789913RecruitingPhase 2
Testing RLY-2608 for growth disorders linked to PIK3CA mutations
This trial tests a new medicine, RLY-2608, that targets the specific genetic change (PIK3CA mutation) causing overgrowth conditions. It aims to see if the drug can slow or stop the growth of affected tissues.
Phoenix, ArizonaAges 2 years+ - NCT05535361Recruiting
Testing a bowel device for short bowel syndrome patients
This trial studies whether the Eclipse XL1 system is safe and may help people with short bowel syndrome, in both children and adults. It will check how well the approach works and watch for side effects over the study period.
Palo Alto, CaliforniaAges 3 months–65 years - NCT07412028Recruiting
Gene study in women with severe insulin resistance and PCOS
This study is looking for women with a type of severe insulin resistance linked to genetics, especially those who have PCOS or a condition called lipodystrophy. The goal is to better understand the genes involved so doctors can offer more targeted treatments.
ParisAges 18–45 - NCT00027274Recruiting
Cancer risk in inherited bone marrow failure syndromes
This study looks at why people with inherited bone marrow failure syndromes (rare genetic conditions affecting blood cell production) have higher cancer risk. Researchers want to understand the genetic and medical factors that increase cancer likelihood, which could help doctors monitor and protect affected patients and their families.
Bethesda, MarylandAges 1 day–100 years - NCT05047354Recruiting
Study cholesterol metabolism in rare genetic conditions
This study looks at body chemistry (cholesterol-related lab findings) and physical features in people with Smith-Lemli-Opitz syndrome and related rare genetic cholesterol disorders. It may help researchers better understand these conditions and how they affect the body.
Bethesda, MarylandAges 1 day–100 years - NCT06033144Recruiting
Tapia's syndrome study in breathing recovery unit
This study looks at how common Tapia's syndrome (a rare nerve problem after breathing tube use) is in patients recovering from long-term breathing support. If you've had a breathing tube for more than 48 hours, you may be able to help researchers learn more about this condition.
Férolles-Attilly, Seine-et-MarneAges 18 years+ - NCT05938439Recruiting
Testing a magnetic device for Candy Cane Syndrome
This trial studies how well a small magnetic device works to relieve problems caused by Candy Cane Syndrome, such as pain, nausea, vomiting, or reflux-like symptoms. It will also check whether the device is safe for your anatomy and overall health.
BrusselsAges 18–85 - NCT05306080RecruitingEarly Phase 1
Rescue treatment for CAR T side effects in adults
This trial tests a rescue medicine given to treat severe immune over-reactions that can happen after CAR T cell therapy, such as cytokine release syndrome and an HLH-like syndrome. It may help calm the immune response if you are already enrolled in a specific CAR T study.
Philadelphia, PennsylvaniaAges 18 years+ - NCT03303716Recruiting
Study of the health history of people with ASXL disorders
This study looks at your medical history and health changes over time in people diagnosed with an ASXL-related disorder. It may help researchers better understand how these conditions progress and what needs to be measured in future treatments.
Los Angeles, CaliforniaAges Any age - NCT07075445Recruiting
Living with long QT syndrome types 2 and 3
This study looks at how long QT syndrome types 2 and 3 affect your daily life and health. It asks you to wear a smartwatch and answer questions to help researchers understand the challenges you face.
Greenville, North CarolinaAges 18 years+ - NCT06538181RecruitingPhase 1
Testing pacritinib for VEXAS syndrome
This trial tests a new drug called pacritinib for people with VEXAS syndrome, a rare autoinflammatory condition. The goal is to see if pacritinib can help control symptoms like rashes, joint pain, and low blood counts.
St Louis, MissouriAges 18 years+
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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.