Clinical trials
Hereditary Diseases clinical trials
Below are recruiting hereditary diseases clinical trials, each written for real people, not researchers. We’re tracking 56 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06672237RecruitingPhase 3
Study of NTLA-2001 for hereditary ATTR amyloidosis with nerve damage
This study is testing a new gene-silencing treatment called NTLA-2001 for people with hereditary ATTR amyloidosis that causes nerve damage. It works by stopping the body from making a faulty protein that builds up and harms nerves.
Buenos AiresAges 18–85 - NCT07257289Recruiting
Heart risk study for inherited heart conditions
This study looks at people with inherited heart diseases and their relatives to better predict risks of dangerous heart rhythms or heart failure. It may help find out if you or your family members need closer monitoring.
BordeauxAges 1–100 - NCT02435940Recruiting
Registry for inherited eye vision conditions
This study is a registry, meaning it collects information about people with inherited retinal (retina) diseases. It may help researchers learn how these conditions progress and connect you with future studies or treatments.
Columbia, MarylandAges Any age - NCT06890143RecruitingPhase 3
Dapagliflozin for kids with genetic kidney disease and protein in urine
This trial tests if dapagliflozin, a medication that lowers blood sugar and protects the kidneys, can reduce protein in the urine of children with inherited kidney disease. It may help preserve kidney function when added to standard blood pressure medication.
Shanghai, Shanghai MunicipalityAges 6–18 - NCT06891443RecruitingPhase 3
Study of Sepofarsen for LCA type 10
This study tests an investigational RNA therapy called sepofarsen for people with Leber Congenital Amaurosis (LCA) type 10, a rare inherited eye disease that causes severe vision loss from birth. The goal is to see if it can slow or improve vision loss.
San Francisco, CaliforniaAges 6 years+ - NCT07587242RecruitingPhase 3
Testing a New Therapy for DMD with Exon 44 Mutations
This study tests whether a new medicine called AOC 1044 (delpacibart zotadirsen) can help boys with Duchenne muscular dystrophy (DMD) who have a specific genetic mutation. The medicine works by helping the body skip a damaged part of the gene to make a functional protein.
LeuvenAges 7–16 - NCT05518617Recruiting
Imaging study for inherited Parkinson’s using PET, SPECT, and spinal fluid
This trial tests how well special brain scans (PET/SPECT) and spinal fluid tests can show changes in people with inherited (genetic) Parkinson’s. It may help researchers understand what’s happening in the brain and improve how these conditions are measured.
Exeter, DevonAges 25–80 - 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+ - NCT06573723Recruiting
Rare disease registry at Hospital Italiano
This study collects information from patients with certain rare diseases to better understand them. If you have one of these conditions and receive care at Hospital Italiano de Buenos Aires, you may be able to join.
Buenos Aires, Buenos AiresAges Any age - NCT07608432RecruitingPhase 3
Testing a New DMD Treatment Given Every 4 Weeks
This study tests whether a new medicine called zeleciment rostudirsen can help ambulatory (walking) males with Duchenne muscular dystrophy (DMD). The medicine is given as an intravenous infusion (through a vein) every 4 weeks and works by helping the body produce a protective muscle protein.
Hillsborough, North CarolinaAges 4–18 - NCT05200715Recruiting
Registry study for inherited and inflammatory eye and joint diseases
This study is a registry, meaning it collects your medical information to better understand several inflammatory (auto-inflammatory) conditions. It may help doctors and researchers improve diagnosis and future treatments for people with these disorders.
AlgiersAges Any age - NCT02378805Recruiting
Alport syndrome registry to help delay kidney failure
This is a registry that collects information from people with Alport syndrome to better understand the disease and how to delay kidney failure. It may help researchers learn which factors and treatments are most important over time.
Göttingen, Lower SaxonyAges Any age - NCT03931707Recruiting
Genetic study of newborns in China
This study collects genetic information from very young newborns to better understand early-life health. Your child’s information may help researchers learn about genetics and future research.
Shanghai, Shanghai MunicipalityAges Up to 4 weeks - NCT06553976Recruiting
Research network for hereditary spastic paraplegia
This trial aims to build a research network and gather information from people with two genetic types of hereditary spastic paraplegia (SPG4 and SPG5A). It will help doctors better understand these conditions and improve care.
Miami, FloridaAges Any age - NCT06107400RecruitingEarly Phase 1
RM-004 cell therapy for Hemoglobin H-Constant Spring disease
This early-phase trial tests a new cell therapy called RM-004 for people with Hemoglobin H-Constant Spring disease, a form of alpha thalassemia that requires regular blood transfusions. The goal is to see if the treatment is safe and can reduce or eliminate the need for transfusions.
Nanning, GuangxiAges 12–35 - NCT07356557Recruiting
Medication adherence in inherited metabolic disease patients
This study looks at how well patients with hereditary metabolic diseases take their daily oral medications. It aims to understand if there are challenges with sticking to the treatment plan.
ParisAges 7–20 - NCT05902351Recruiting
Study of the natural course of Charcot-Marie-Tooth disease
This study looks at how Charcot-Marie-Tooth disease (CMT) or related inherited nerve conditions change over time. It may help researchers better understand what to expect and guide future treatments.
New York, New YorkAges Any age - NCT07718971Enrolling by invitation
Whole-genome sequencing for unexplained medical conditions
This study uses whole-genome sequencing (a complete read of your DNA) to try to find a genetic cause for unexplained medical conditions. If you have a puzzling illness without a clear diagnosis, this test might give you answers.
Seattle, WashingtonAges 18–50 - NCT03749980Recruiting
Study following people with von Hippel-Lindau (VHL)
This study follows people who have von Hippel-Lindau (VHL) over time to better understand how the condition changes. It may help doctors plan future treatments by collecting long-term health information.
Boston, MassachusettsAges Any age - NCT04528498Recruiting
Embryo health study for IVF using PGT-A testing
This study looks at embryo health using a DNA test called PGT-A for couples having IVF. It may help researchers better understand what the test can and cannot tell about which embryos are healthiest.
North Brunswick, New JerseyAges 18 years+ - NCT04531696Recruiting
Post-death breast tissue donation study for cancer research
This trial invites adults with certain breast cancer conditions to donate tissue after death. Researchers hope the donated samples will help better understand cancer and improve future treatments.
LeuvenAges 18 years+ - NCT04905537Recruiting
Genetic screening for newborns after a serious pregnancy loss
This trial looks at doing early genetic testing in newborns/early life cases after a serious pregnancy loss, to learn whether genetics can help understand why it happened. If you have the right timing and sample availability, the results may help future “more precise” care planning and research.
Shanghai, Shanghai MunicipalityAges Up to 3 months - 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+ - 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+
Hear when a new Hereditary Diseases 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 hereditary diseases?
- Yes. Clin2 currently lists 56 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.