Clinical trials
Hereditary Diseases clinical trials
Below are recruiting hereditary diseases clinical trials, each written for real people, not researchers. We’re tracking 55 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06526741Recruiting
Alport Syndrome Patient Registry
This registry collects health information from people with Alport syndrome to help researchers better understand the condition and improve future treatments.
Scottsdale, ArizonaAges birth+ - NCT06330441Recruiting
Pancreatic cancer screening for high-risk groups
This study screens people at high risk for pancreatic cancer using imaging tests. It aims to catch cancer early in those with certain genetic conditions or strong family history.
BrnoAges 18 years+ - NCT06374719Recruiting
TNNT1 myopathy natural history study
This study follows people with a rare genetic muscle condition called TNNT1 myopathy over time. Researchers will track how the disease progresses to better understand it and help plan future treatments.
Gordonville, PennsylvaniaAges Any age - NCT07303296RecruitingPhase 2
Gene therapy study for vision loss from LHON
This trial tests a gene therapy called GS010 given as an injection into the eye for people with vision loss from LHON. It aims to see if it can help improve vision in both eyes.
ParisAges 15 years+ - NCT05354622Recruiting
Genetic testing study for inherited leg stiffness
This study looks at genes (DNA) to understand an inherited condition that causes gradually worsening leg stiffness. It may help researchers learn what causes the condition and potentially guide future care.
Boston, MassachusettsAges 1 month–30 years - NCT03011541Recruiting
Stem cell treatment for damaged retina or optic nerve
This study looks at stem-cell treatment to help vision in adults whose retina or optic nerve damage is not expected to improve on its own. You may be a candidate if your eye exam shows documented, stable or slowly changing damage and you can safely complete follow-up eye visits.
Westport, ConnecticutAges 18 years+ - NCT00390104Recruiting
Study uses blood and skin samples to learn muscular disease causes
This study looks at samples from people with muscular dystrophy and from close relatives, to better understand neuromuscular diseases. You may be asked to provide a skin biopsy for research to help with genetic and molecular analysis.
Boston, MassachusettsAges 1 week–100 years - NCT00026884Recruiting
Blood and tissue sample collection for cancer research
This study collects blood and tissue samples from people with cancer or suspected cancer, or from their blood relatives. Researchers use these samples to better understand cancer and develop new treatments.
Bethesda, MarylandAges 2 years+ - NCT05641142Recruiting
Testing blood thinners in adults with inherited bleeding vessel disease
This study looks at people with Rendu-Osler (hereditary hemorrhagic telangiectasia) who have just started blood-thinning medicines. It aims to understand how antiplatelet and/or anticoagulant therapy affects safety and outcomes soon after starting.
AngersAges 18 years+ - NCT06213402Recruiting
European registry for rare anemia disorders
This study is creating a European registry (database) to collect information about people with rare anemia disorders. By joining, you help researchers better understand these conditions and improve care for yourself and others.
Barcelona, CataloniaAges birth–100 years - NCT07136844Recruiting
Walking and arm movement study for nerve and muscle conditions
This study uses motion analysis to understand walking and arm function in adults with certain neurological or metabolic diseases. The goal is to find patterns that help doctors better manage these conditions.
LiègeAges 18 years+ - NCT04903782Recruiting
Family whole-genome testing for children with newly diagnosed cancer
This study offers whole-genome testing (a way to read a person’s DNA) to families after a child is newly diagnosed with cancer. It aims to find inherited changes that may explain why cancer happened and guide future care or monitoring.
Newcastle, New South WalesAges Up to 21 years - NCT00830557Recruiting
Share information and donate tissue samples for pancreatic illnesses
This study collects health information and tissue samples from people with pancreatic cancer or other pancreatic conditions. Your participation may help researchers better understand these diseases and improve future care.
Phoenix, ArizonaAges 18 years+ - NCT04891029Recruiting
Early detection testing for uterine or ovarian cancer risk
This study looks for early signs of uterine (endometrial) or ovarian cancer, especially in people with inherited risk. It may help doctors catch problems earlier, when treatment can be more effective.
Montreal, QuebecAges 45–75 - NCT07601425RecruitingPhase 1/Phase 2
ATV-1601 Treatment for Hereditary Hemorrhagic Telangiectasia
This trial tests a new drug called ATV-1601 in people with HHT, a genetic condition that causes abnormal bleeding and blood vessel problems. The drug aims to reduce bleeding and anemia caused by HHT.
Phoenix, ArizonaAges 18 years+ - NCT05848271Recruiting
Natural history study for people with HPDL gene changes
This study follows people who have an HPDL gene change to better understand how symptoms and health progress over time. It may help clinicians learn more about the condition and plan future treatments.
San Diego, CaliforniaAges Any age - NCT07700992Recruiting
A blood test to find pancreatic cancer early
This study uses stored blood samples from people at higher risk for pancreatic cancer (due to family history or certain pancreatic cysts) to see if a simple blood test can detect the disease early or help monitor it over time.
Milan, MIAges 18–99 - NCT03981276Recruiting
Study aims to understand inherited walking stiffness disorders
This study looks at the causes and biology (“biomarkers”) of hereditary spastic paraplegias and related conditions. You may join as someone with the condition, a family member without symptoms, or a healthy person, to help researchers understand what’s happening in the nervous system.
InnsbruckAges Any age - NCT05904301Recruiting
Registry for autoimmune and autoinflammatory disease patients
This study builds a nationwide list (registry) of people with certain autoimmune or inflammation-related illnesses. It helps researchers understand these diseases better over time, which can guide future care.
YerevanAges Any age - 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 - NCT07478172Recruiting
Electrical muscle stimulation exercise for neuromuscular disease
This study tests whether whole-body electrical muscle stimulation (using small electrodes on the skin) can help adults with certain neuromuscular diseases exercise and improve muscle strength. It may be an option if you can stand for about 15 minutes at a time and have some muscle strength left.
Columbia, MissouriAges 18 years+ - NCT07152197Recruiting
Resistance Exercise for Charcot-Marie-Tooth Disease
This trial tests whether resistance exercises (like lifting weights with your legs) can help people with Charcot-Marie-Tooth disease. It is for young adults aged 15 to 29 who can move their ankle enough to do strength training.
Temuco, La AraucaníaAges 15–29 - NCT07206095Recruiting
Better diagnosis for inherited red blood cell diseases
This trial aims to improve how doctors diagnose rare inherited anemias, including sickle cell disease and other conditions that destroy red blood cells. It may help if you have a confirmed or suspected diagnosis but need more clarity through advanced testing.
Barcelona, BarcelonaAges Any age - NCT07314268Recruiting
Study of tiny blood particles in heart amyloidosis
This study looks at tiny particles called extracellular vesicles in the blood of people with a specific type of heart amyloidosis. It aims to find better ways to track the disease and predict outcomes.
São Paulo, São PauloAges 18 years+
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Common questions
- Are there clinical trials for hereditary diseases?
- Yes. Clin2 currently lists 55 recruiting hereditary diseases 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 hereditary diseases 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 hereditary diseases 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.