Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,858 recruiting studies in this area right now.
By phase
- Not applicable2,771
- Phase 2283
- Phase 1235
- Phase 3216
- Phase 1/Phase 2204
- Phase 497
- Phase 2/Phase 352
Top conditions
- Sickle Cell Disease140
- Atopic Dermatitis128
- Congenital Heart Disease86
- Cystic Fibrosis78
- Down Syndrome54
- Genetic Disease42
- Duchenne Muscular Dystrophy39
- Fabry Disease38
- Hemophilia A38
- Cystic Fibrosis (CF)37
- Cardiac Amyloidosis35
- Huntington Disease32
- Lynch Syndrome32
- Heart Failure31
- Retinitis Pigmentosa31
- Bronchopulmonary Dysplasia30
- Congenital Diaphragmatic Hernia29
- Duchenne Muscular Dystrophy (DMD)27
Leading sponsors
- Assistance Publique - Hôpitaux de Paris62
- Boston Children's Hospital42
- National Institute of Allergy and Infectious Diseases (NIAID)38
- Mayo Clinic37
- Children's Hospital Medical Center, Cincinnati33
- Hospices Civils de Lyon32
- IRCCS Azienda Ospedaliero-Universitaria di Bologna32
- Riphah International University26
- Massachusetts General Hospital23
- National Cancer Institute (NCI)23
Where studies are running
- California340
- Maryland161
- Alabama132
- Massachusetts116
- New York84
- Arizona78
- Texas73
- Minnesota67
- Pennsylvania65
- Ohio57
Recruiting studies3,858 total
- NCT07037238RecruitingPhase 2
Everolimus for treating vascular malformations
This study tests a drug called everolimus to see if it helps treat vascular malformations (abnormal clusters of blood vessels). It is for adults aged 18 to 65 who have been diagnosed by MRI and are able to take oral medication.
BeijingAges 18–65 - NCT07037862RecruitingPhase 1/Phase 2
Study of ENTR-601-44 for Duchenne muscular dystrophy (exon 44 skipping)
This trial tests a new medicine called ENTR-601-44 for people with Duchenne muscular dystrophy whose genetic mutation can be treated by skipping a part of the gene called exon 44. The goal is to see if it is safe and helps with muscle function.
GhentAges 4–20 - NCT07038200RecruitingPhase 3
Study of Del-brax (AOC 1020) for People with FSHD
This study tests an experimental medicine called Del-brax (AOC 1020) for people with facioscapulohumeral muscular dystrophy (FSHD). The goal is to see if it can help improve muscle strength and slow the disease. You may be able to join if you have a confirmed FSHD diagnosis and can walk at least 10 meters on your own.
Orange, CaliforniaAges 16–70 - NCT07038239Recruiting
MORC2 gene mutation study for nerve and brain conditions
This study looks at how changes in the MORC2 gene relate to nerve or brain development problems. It may help doctors better understand these conditions and improve care.
BesançonAges 4 years+ - NCT07038824RecruitingPhase 1/Phase 2
A study of ENTR-601-45 for Duchenne MD (exon 45 skipping)
This trial tests an experimental drug called ENTR-601-45 for people with Duchenne muscular dystrophy that can be treated with 'exon 45 skipping.' The goal is to see if it's safe and helps improve muscle function. It's for males between 4 and 20 who can still walk and have enough arm strength.
GhentAges 4–20 - NCT07039084Recruiting
Tablet for helping children with rare genetic conditions communicate
This study tests whether a speech-generating tablet (like an iPad with a communication app) can help children with rare genetic conditions who are minimally verbal (using fewer than 50 words) to communicate better. If your child fits the criteria, they could get a device and training to see if it helps them express themselves more easily.
Melbourne, VictoriaAges 3–12 - NCT07039552Recruiting
Ovarian cancer risk check for family members
This study looks at family members of people with ovarian cancer to build a better tool for predicting their risk. If you have a BRCA1 or BRCA2 gene mutation, you may be able to join.
Beijing, Beijing MunicipalityAges 18 years+ - NCT07039799Recruiting
Virtual reality for arm function in Duchenne
This trial tests whether using virtual reality (VR) games can help improve arm and hand function in people with Duchenne muscular dystrophy. It may be a fun and engaging way to do exercises that strengthen your upper body.
Istanbul, FatihAges 7–18 - NCT07040137RecruitingPhase 3
New treatment for spinocerebellar degeneration (SCD)
This trial tests an experimental drug, KPS-0373, to see if it can help people with spinocerebellar degeneration (a condition that affects coordination and balance). It is for Japanese patients with mild to moderate trouble with movement (ataxia).
Multiple LocationsAges 18 years+ - NCT07040774Recruiting
Study of type 1 interferonopathies in Europe
This study looks at people with a confirmed type 1 interferonopathy, a rare immune condition caused by a gene change. It aims to learn more about how the condition progresses over time.
InnsbruckAges Any age - NCT07042126RecruitingPhase 3
Testing 611 for Teen Eczema (moderate to severe)
This trial tests a new injectable medicine (611) for teenagers with moderate to severe eczema that hasn't been helped enough by creams. It aims to see if the drug can safely reduce itching and skin symptoms.
Beijing, Beijing MunicipalityAges 12–17 - NCT07046806RecruitingPhase 1/Phase 2
Oral deucrictibant for hereditary angioedema attacks
This trial tests an oral medication called deucrictibant to prevent and treat swelling attacks in people with hereditary angioedema. It may help reduce the number and severity of attacks.
Wheaton, MarylandAges 18–75 - NCT07047144RecruitingPhase 2
Apitegromab for infants with SMA under 2 years old
This study tests a new medicine called apitegromab for babies under 2 years old with spinal muscular atrophy (SMA). It looks at whether the drug can help with muscle strength and reaching motor milestones like sitting or rolling.
Phoenix, ArizonaAges Up to 2 years - NCT07048106Recruiting
Study on eye changes in people with PXE
This study uses special eye imaging to track changes caused by PXE, a rare genetic condition. It may help researchers understand how PXE affects the eyes over time.
Bonn, North Rhine-WestphaliaAges Any age - NCT07048418Recruiting
Nose job results in adults born with a cleft lip and palate
This trial asks adults who had a nose job as teens or children, after being born with a cleft lip and palate, to rate how they look and feel about their nose. Their answers will help doctors understand how these surgeries affect patients long-term.
MontpellierAges 18–30 - NCT07049588Recruiting
Finding early signs of Charcot-Marie-Tooth 1A
This trial looks for new markers of Charcot-Marie-Tooth 1A (CMT1A) early in the disease. Researchers hope to find signs that can help diagnose and track the disease better, which could lead to better treatments in the future.
MarseilleAges 10–30 - NCT07049900Recruiting
Abdominal binder study for premature babies
This trial is testing whether a soft abdominal binder (a wrap around the belly) helps premature babies who need breathing support through a mask or prongs. It aims to make breathing easier and more comfortable for these tiny infants.
Minneapolis, MinnesotaAges 6 months–7 months - NCT07050381Recruiting
Risk factors for ruptured aneurysms in brain AVM
This study looks at why certain aneurysms tied to a brain AVM tear more often. If you have a brain AVM with aneurysms that bled, your data may help doctors understand how to prevent future ruptures.
Nanjing, JiangsuAges Any age - NCT07051902Recruiting
Microbiome and Atopy in Mali
This study is looking at how the natural bacteria on our skin and in our gut relate to eczema and asthma in people living in Mali. It will compare people with these conditions to healthy volunteers to learn more about what might cause them.
BamakoAges 3 years+ - NCT07052266Recruiting
Combined genetic screening for pregnancy and cancer risk
This trial offers a combined genetic test for both common inherited conditions and hereditary cancer risks to pregnant women or those planning pregnancy. It aims to see if adding cancer screening to routine pregnancy testing is helpful.
Brooklyn, New YorkAges 18–55 - NCT07053969Recruiting
Does a protein gel thicken gum tissue around implants?
This study tests whether a protein-based gel (enamel matrix derivative) can make the gum tissue thicker during a second-stage implant procedure. It may help people getting dental implants achieve better healing and gum support.
San Antonio, TexasAges 18–18 - NCT07056569Recruiting
Testing a dermatitis cream for adults and children with eczema
This study tests a medical cream for people with atopic or contact dermatitis (eczema). It aims to see how well the cream works and if it is safe for both adults and children over 6 months old.
MilanAges 6 months–65 years - NCT07059689Recruiting
Comparing breathing support after heart surgery in children
This trial compares two types of breathing support (high-flow nasal cannula vs. non-invasive ventilation) in children who have recently had heart surgery and are at high risk of having trouble breathing after their breathing tube is removed. The goal is to see which method works better to help them breathe on their own.
Jakarta, DKI JakartaAges Up to 18 years - NCT07061509Recruiting
Exercise habits and health risks in gym goers
This study looks at how exercise habits can become unhealthy and what health risks might come with too much exercise. It may help you understand if your workout routine is balanced and safe.
Istanbul, ÜsküdarAges 18–85
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,858 recruiting medical genetics studies from the U.S. registry right now, out of 5,779 we cover in this area in total.
- What does “Genetic & congenital” cover here?
- Inherited and congenital conditions present from birth or written into the genes. We group each study into genetic & congenital based on the conditions it studies, so you can scan the whole therapeutic area at once and drill into a specific condition.
- How do I find a genetic & congenital trial near me?
- Use the search to filter genetic & congenital studies by location and eligibility. Every Clin2 trial page explains the criteria in plain English and offers a short, optional pre-screen for a fit read — the study team makes the final decision.
Other specialties
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov; specialty groupings are derived from each study’s conditions. Talk with your doctor about whether a specific trial is right for you.