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 2284
- Phase 1235
- Phase 3216
- Phase 1/Phase 2203
- Phase 497
- Phase 2/Phase 352
Top conditions
- Sickle Cell Disease140
- Atopic Dermatitis128
- Congenital Heart Disease86
- Cystic Fibrosis78
- Down Syndrome55
- 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
- California341
- Maryland161
- Alabama131
- Massachusetts116
- New York84
- Arizona78
- Texas72
- Minnesota67
- Pennsylvania65
- Ohio57
Recruiting studies3,858 total
- NCT06372717RecruitingPhase 1
Testing APL-4098 alone or combined for AML or MDS
This trial tests a new drug called APL-4098, given alone or with another medicine, for people with certain blood cancers (AML or MDS) that have come back or not responded to treatment. The goal is to see if it is safe and works well.
Clayton, VictoriaAges 18 years+ - NCT06373861Recruiting
GALAXY study: Long-term look at sex chromosome conditions
This study is following people with sex chromosome differences over time to learn more about their health and development. It is open to anyone at any age who has a confirmed genetic diagnosis.
Aurora, ColoradoAges birth–100 years - NCT06374238Recruiting
Peer Support for Young People with Sickle Cell Pain
This study is testing a peer support program for teens and young adults (ages 16–30) with sickle cell disease who have chronic pain. You'll connect with a trained peer supporter through your smartphone to learn pain coping skills and get support.
Mobile, AlabamaAges 16–30 - NCT06375239Recruiting
Study of tests for tracking vision in retinal disease
This study will check if certain vision tests work well in people with retinal diseases like retinitis pigmentosa or Stargardt. Results will help design future trials.
Irvine, CaliforniaAges 18 years+ - NCT06380530Recruiting
Computer-assisted surgical planning for limb deformity correction
This trial tests a new computer-assisted planning method to help surgeons better correct bone deformities in the arms or legs. It may lead to more precise surgeries and faster recovery.
Bologna, BOAges 2–40 - NCT06381713Recruiting
Speech therapy for children with cleft palate
This study compares two types of speech therapy for children with cleft palate. It looks at how well each type works and how much it costs, to help find the best way to improve your child's speech.
GhentAges 4–12 - NCT06385054RecruitingPhase 2
Probiotics for infant colic in breastfed babies
This study tests a probiotic (friendly bacteria) to see if it can reduce crying and fussing in babies with colic. It is for exclusively breastfed infants 8 weeks and younger who have colic symptoms.
Bécon-les-GranitsAges Up to 2 months - NCT06385886Recruiting
Study to improve sickle cell care for adults not seeing a specialist
This study helps connect adults with sickle cell disease who haven't seen a specialist in over a year back into care. It tests a program designed to make it easier to get regular medical support and improve your health.
Birmingham, AlabamaAges 18 years+ - NCT06389877RecruitingPhase 1/Phase 2
Testing BEAM-302 for alpha-1 antitrypsin deficiency
This trial tests a new treatment called BEAM-302, which aims to correct the genetic defect that causes alpha-1 antitrypsin deficiency. It may help prevent further lung and liver damage in people with the PiZZ mutation.
Birmingham, AlabamaAges 18–70 - NCT06390696Recruiting
Sand play for eczema: a biodiversity study
This study looks at whether playing in sand with different types of natural materials (to increase your contact with harmless microbes) can improve eczema symptoms. You would stop using your usual eczema creams during the trial to see if the sand play alone helps.
Helsinki, UusimaaAges 2–5 - NCT06390878Recruiting
Study on natural kindergarten yards
This study looks at how bringing nature back into kindergarten yards might affect children's health. It involves children who attend kindergarten and have not used antibiotics recently.
Helsinki, UusimaaAges 1–5 - NCT06391736RecruitingPhase 1/Phase 2
Gene therapy for late-onset Pompe disease
This trial tests a new gene therapy for late-onset Pompe disease. The therapy aims to help your body produce the enzyme needed to break down glycogen, which may improve muscle and breathing function.
BeijingAges 6 years+ - NCT06392906RecruitingPhase 3
Radiation for Graves' Eye Disease: 1 vs 2 Weeks
This trial tests if a shorter course of radiation (1 week) works as well as a standard longer course (2 weeks) for treating active thyroid eye disease that did not improve enough with steroid medicine. It may be an option if you still have eye symptoms after steroid treatment.
Bangkok NoiAges 18–70 - NCT06394453Recruiting
Feeding during cooling for brain injury in newborns
This study looks at whether it is safe to give small amounts of milk to babies who are being cooled for brain injury after a difficult birth. It may help doctors decide if feeding during cooling can improve outcomes.
CracowAges Up to 6 hours - NCT06395285RecruitingPhase 1
Testing DF-003 for ROSAH syndrome with eye inflammation
This trial tests an oral medication called DF-003 for people with ROSAH syndrome who have eye inflammation (uveitis). The goal is to see if the drug is safe and tolerable. It might help reduce eye inflammation and other symptoms.
Bethesda, MarylandAges 18–65 - NCT06396078RecruitingPhase 4
Antibiotics for Early Water Breaking Before Delivery
This study tests whether giving antibiotics to pregnant people with early water breaking (PPROM) before delivery improves outcomes. It aims to prevent infections and protect both parent and baby while waiting for delivery.
Columbus, OhioAges 18 years+ - NCT06397911RecruitingPhase 2
Testing FB825 for moderate to severe eczema
This trial tests a new injectable medicine called FB825 for people with moderate to severe eczema that hasn't improved enough with standard creams. The study aims to see if FB825 can reduce itching and skin symptoms safely.
Fountain Valley, CaliforniaAges 18–65 - NCT06399107RecruitingPhase 1/Phase 2
Gene therapy trial for sickle cell disease using BAH243
This trial tests a gene therapy that uses a modified virus to deliver a healthy gene to your blood stem cells. It aims to help people with sickle cell disease produce normal red blood cells and reduce painful crises.
Beijing, Beijing MunicipalityAges 2–90 - NCT06402942Recruiting
Game-based occupational therapy for teens with Duchenne
This trial tests a fun, game-based occupational therapy program for teens with Duchenne muscular dystrophy. It aims to help you build skills and stay engaged through activities you can do at home on a computer or tablet.
AnkaraAges 13–18 - NCT06406452Recruiting
Resorbable airway splint trial for severe tracheobronchomalacia in children
This trial tests a new bioresorbable airway splint for babies and young children with severe tracheobronchomalacia (a weak, collapsing windpipe). It aims to help them breathe without a machine or tracheostomy and avoid major surgery.
Ann Arbor, MichiganAges 1 week–3 years - NCT06408376Recruiting
Gentle cord clamping for babies with diaphragmatic hernia
This study compares a gentle way of clamping the umbilical cord after birth in babies with a diaphragmatic hernia. The goal is to see if this approach improves the baby's oxygen levels and reduces the need for breathing support.
Buenos AiresAges Any age - NCT06408337RecruitingPhase 1/Phase 2
Biocleft for cleft palate repair in children
This trial tests a new treatment called BIOCLEFT, made from your child's own cheek cells, to help repair a complete cleft palate on one side. It aims to see if the treatment is safe and works better than standard surgery.
Granada, Spain/GranadaAges 10 months–1.2 years - NCT06408701Recruiting
Lab-grown eye cells from people with eye problems
This study uses stem cells from people with eye malformations to grow tiny 3D eye organ-like structures in the lab. The goal is to better understand how eye problems develop, which could lead to new treatments in the future.
Clermont-FerrandAges 6 years+ - NCT06408662Recruiting
Remote mindfulness program for tics (Tourette or tic disorder)
This study tests a mindfulness program delivered remotely (via phone or tablet) to help people with moderate to severe tics. It aims to see if mindfulness can reduce tic severity and improve quality of life.
Baltimore, MarylandAges 18 years+
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 5,764 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.