Clinical trials
Inherited Disease clinical trials
Below are recruiting inherited disease clinical trials, each written for real people, not researchers. We’re tracking 44 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06749639RecruitingEarly Phase 1
Gene therapy trial for RDH12-related eye disease
This trial tests an injection called PUMCH-E101 for people with vision loss caused by changes in the RDH12 gene. The goal is to see if it is safe and if it can help improve vision.
Beijing, Beijing MunicipalityAges 8–45 - NCT05906953RecruitingPhase 1/Phase 2
HG004 gene therapy for RPE65 vision loss
This Phase 1/2 trial tests the safety and early effectiveness of HG004 (a gene therapy) in people with Leber congenital amaurosis caused by RPE65 gene changes. It aims to see whether treating the eye can improve or stabilize vision-related measures while monitoring side effects.
Sacramento, CaliforniaAges 6–50 - NCT07085533Recruiting
A Study of Inherited Retinal Diseases
This study is looking at how inherited retinal diseases affect color vision over time. It may help researchers understand these conditions better and is a good fit for people with a known diagnosis who can describe what they see.
WuhanAges Any age - NCT06330324Enrolling by invitation
Reproductive choices for genetic skin conditions
This study looks at the choices families have when they carry a genetic skin condition. It aims to learn more about how prenatal testing and IVF with genetic testing can help.
Maastricht, LimburgAges Any age - NCT05587439Recruiting
Study of inherited genetic risk in lung and related cancers
This study looks for inherited (family-passed) genetic changes that may raise the risk of lung cancer and related thoracic cancers. It may help people and families understand their genetic risk by collecting health information and sometimes specimens from patients and relatives.
Boston, MassachusettsAges 18 years+ - NCT00004568Recruiting
Study of inherited neurological disorders and family genetics
This research study evaluates people with inherited neurological conditions (nervous system disorders that run in families) and their relatives. Researchers aim to better understand these conditions and improve diagnosis through clinical exams and genetic testing.
Bethesda, MarylandAges 2–120 - NCT05589714Recruiting
Study your rare eye gene and track retinal disease over time
This study builds a registry and tracks the natural history of rare retinal dystrophies while also testing people for disease-causing gene variants. It may help researchers better understand what causes retinal degeneration and how it progresses.
Little Rock, ArkansasAges 4 years+ - NCT07266584Recruiting
Retinal implant for central vision loss in inherited eye disease
This trial tests a new implant (PRIMA) placed under the retina to help restore central vision in people with inherited retinal degeneration. If you have very poor central vision and a large enough area of retinal damage, this device may help you see better.
Sydney, New South WalesAges 18 years+ - NCT05721911Recruiting
Study using brain-cell tests to better understand Parkinson’s risk
This study collects blood and related samples to create a “biobank” and uses lab tests to study genetic risk in Parkinson’s disease and idiopathic REM sleep behavior disorder (RBD). It may help researchers understand why symptoms develop and improve future prevention or treatment approaches.
Milan, ItalyAges 18 years+ - NCT01793168Recruiting
Rare disease registry and history study
This study keeps a registry (a structured list) of people with rare diseases and may also track how the condition changes over time. It can help researchers better understand rare illnesses and find gaps in care, which may improve future treatments.
Sioux Falls, South DakotaAges Any age - NCT00869817Recruiting
Study for adults at risk of inherited Alzheimer’s
This study follows people who are at risk for a specific inherited form of Alzheimer’s disease (because a parent has it). It helps researchers learn how the disease starts and changes over time, especially in people who still feel cognitively normal.
Jacksonville, FloridaAges 18 years+ - NCT02077894Recruiting
Genetic testing to help identify inherited eye conditions
This study uses whole exome and whole genome genetic tests to look for DNA changes linked to inherited or congenital eye problems. It may help families and doctors better understand the cause of the eye condition and guide future care.
Bethesda, MarylandAges 1 day–120 years - NCT02414178Enrolling by invitation
Tau PET brain scan for inherited Alzheimer’s disease
This study uses a special brain scan to measure “tau” buildup in people with an inherited form of Alzheimer’s. It may help researchers better understand disease changes and evaluate whether the scan can track them over time.
St Louis, MissouriAges 18 years+ - NCT04232085RecruitingPhase 2
Stem cell transplant to rebuild immunity in genetic immune disorders
This Phase 2 trial tests a stem cell (bone marrow) transplant approach to restore bone marrow function and immune function in people with certain inherited immune problems. If you have one of these conditions and a suitable donor, the study may offer a path to rebuild blood and immune systems.
Baltimore, MarylandAges 4 months–50 years - NCT04414046RecruitingPhase 2
Stem cell transplant using T-cell cleanup for immune and metabolism disorders
This Phase 2 study tests a specialized stem cell transplant that removes certain “T-cells” (a type of immune cell) to treat children with serious immune system or inherited metabolic problems. The goal is to improve or stabilize disease by resetting the immune system and supporting the body’s function.
St. Petersburg, FloridaAges Up to 21 years - NCT03880708Recruiting
Registry for inherited dangerous heart rhythm conditions
This study is a heart rhythm registry for people with inherited conditions that can cause abnormal heart beats. By collecting health and testing information, researchers hope to better understand these conditions and improve care.
Beijing, Beijing MunicipalityAges 1 hour–90 years - NCT01351545Recruiting
Cryopreserved cord blood access program for transplant patients
This trial studies an organized way to obtain and distribute unlicensed (not FDA-approved for general use) stored cord blood units that have been frozen and kept. It may help people who need a cord blood transplant but don’t fit the “licensed cord blood only” options.
Birmingham, AlabamaAges Any age - NCT06958640Enrolling by invitation
Da Qing offspring health study
This study looks at the health of children, grandchildren, and great-grandchildren of people who took part in a diabetes prevention study in Daqing, China. It aims to understand how their family history affects their health over time.
Beijing, Beijing MunicipalityAges Any age - NCT05916287Recruiting
STANISLAS cohort follow-up visit for heart and kidney health
This study is a follow-up visit for people who joined the STANISLAS family health cohort. Researchers look at how genes and environment may affect heart and kidney (kidneys) health over time.
Vandœuvre-lès-Nancy, LorraineAges Any age - NCT06065852Recruiting
National registry for rare kidney diseases
This study creates a registry to collect health information from people with rare kidney diseases. It aims to improve understanding and future treatments by tracking patient experiences.
Bristol, South WestAges Any age
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Common questions
- Are there clinical trials for inherited disease?
- Yes. Clin2 currently lists 44 recruiting inherited disease 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 inherited disease 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 inherited disease 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.