Medical genetics
Genetic & congenital clinical trials
Inherited and congenital conditions present from birth or written into the genes. We’re tracking 3,762 recruiting studies in this area right now.
By phase
- Not applicable2,682
- Phase 2280
- Phase 1239
- Phase 3209
- Phase 1/Phase 2208
- Phase 495
- Phase 2/Phase 349
Top conditions
- Sickle Cell Disease133
- Atopic Dermatitis124
- Congenital Heart Disease83
- Cystic Fibrosis77
- Down Syndrome51
- Genetic Disease42
- Cystic Fibrosis (CF)37
- Duchenne Muscular Dystrophy36
- Fabry Disease36
- Hemophilia A36
- Cardiac Amyloidosis35
- Retinitis Pigmentosa33
- Huntington Disease31
- Lynch Syndrome31
- Heart Failure30
- Bronchopulmonary Dysplasia29
- Congenital Diaphragmatic Hernia29
- Duchenne Muscular Dystrophy (DMD)29
Leading sponsors
- Assistance Publique - Hôpitaux de Paris57
- Mayo Clinic39
- Boston Children's Hospital38
- National Institute of Allergy and Infectious Diseases (NIAID)38
- IRCCS Azienda Ospedaliero-Universitaria di Bologna32
- Children's Hospital Medical Center, Cincinnati31
- Hospices Civils de Lyon29
- Children's Hospital of Philadelphia23
- National Human Genome Research Institute (NHGRI)23
- St. Jude Children's Research Hospital23
Where studies are running
- California324
- Maryland160
- Alabama133
- Massachusetts107
- Arizona88
- New York84
- Texas70
- Minnesota68
- Pennsylvania64
- Florida59
Recruiting studies3,762 total
- NCT06692426RecruitingPhase 1
Cell therapy for Duchenne muscular dystrophy
This trial tests a new cell-based treatment for Duchenne muscular dystrophy in adults who can no longer walk. The goal is to see if the therapy can help improve muscle function.
Minneapolis, MinnesotaAges 18 years+ - NCT06689800Recruiting
Family communication tool for kids getting transplant or cell therapy
This study looks at a family communication tool for children and teens getting stem cell transplants or cellular therapy. It helps families talk about treatment together.
St Louis, MissouriAges 8 years+ - NCT06691867Recruiting
Integrative training for sickle cell pain in teens
This study tests an integrative training program designed to help adolescents with sickle cell disease manage their pain better. It combines physical and mental techniques to reduce pain and improve quality of life.
Hartford, ConnecticutAges 12–18 - NCT06687733RecruitingPhase 1/Phase 2
Gene therapy for phenylketonuria
This trial tests a new gene therapy, NGGT002, which delivers a healthy gene to help people with phenylketonuria (PKU) manage their phenylalanine levels. It may reduce the need for strict diet control.
Bengbu, AnhuiAges 18–55 - NCT06685536Enrolling by invitation
Long-term follow-up for CS-101 recipients
This study follows up on people who have already received the CS-101 infusion in a previous trial. It aims to check how participants are doing over time. There are no restrictions to join if you already received CS-101.
Nanning, GuangxiAges 6–35 - NCT06401538RecruitingPhase 2
BMB-101 for absence epilepsy and DEE
This trial tests a new drug called BMB-101 for people with absence epilepsy (including Jeavons syndrome) or developmental epileptic encephalopathies (DEE) like Dravet or Lennox-Gastaut. It aims to see if the drug can reduce seizure frequency and is safe.
Randwick, New South WalesAges 18–65 - NCT06042140Recruiting
Umbilical cord patch for spina bifida repair before birth
This study tests whether a patch made from donated umbilical cord can help repair spina bifida in the womb using a minimally invasive fetoscopic approach. If successful, it may reduce the need for more invasive open fetal surgery.
Houston, TexasAges 18 years+ - NCT06676904Recruiting
Platelet transfusion study for very preterm babies
This study looks at the best way to decide when to give platelet transfusions to very premature babies. It aims to help prevent bleeding while avoiding unnecessary transfusions.
Birmingham, AlabamaAges 1 hour–2 days - NCT06675097Recruiting
Gas after abdominal surgery: how it affects recovery
This study looks at how having gas after abdominal surgery might affect your recovery. It aims to understand if passing gas sooner could help you feel better faster and leave the hospital sooner.
Nantes, Loire AtlantiqueAges 18 years+ - NCT06679270RecruitingPhase 3
Long-term safety study of metreleptin for partial lipodystrophy
This study tests the long-term safety of metreleptin for people with partial lipodystrophy. It is for those who already completed a related study and want to continue treatment.
Birmingham, AlabamaAges 13 years+ - 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 - NCT06679881Enrolling by invitationPhase 3
Daily oral tablet to prevent hereditary angioedema attacks
This study tests a daily oral tablet called deucrictibant to see if it can prevent swelling attacks in people with hereditary angioedema (HAE). If you join, you will take the study drug for a longer period and track your symptoms in a diary.
Birmingham, AlabamaAges 12 years+ - NCT06674811Recruiting
Childhood cancer surgery recovery study
This study looks at how children and young adults recover after surgery for solid tumors. It also includes their caregivers to understand how resilience affects recovery.
Rochester, MinnesotaAges 3–25 - NCT06679790Recruiting
Understanding changes in Tourette Syndrome over time
This study tracks how tics and related symptoms change over time in people with Tourette Syndrome. It aims to better understand the condition and could help develop better treatments.
Minneapolis, MinnesotaAges 12–17 - NCT06679972Enrolling by invitation
Impact of pregnancy liver condition on baby's breathing
This study looks at how a pregnancy liver condition (intrahepatic cholestasis) affects the baby's breathing after birth. It compares newborns whose mothers had this condition (and took medication for it) with newborns whose mothers did not have any liver issues during pregnancy.
Bucharest, BucharestAges 1 hour–4 weeks - NCT06674109RecruitingPhase 4
Testing a diabetes drug for gout prevention
This study tests if a type 2 diabetes medication (SGLT2 inhibitor) can help people with frequent gout flares. It aims to lower uric acid levels and reduce flare-ups.
Boston, MassachusettsAges 18–80 - NCT06669949Recruiting
Studying the natural history of SPLIS disease
This study collects health information from people with SPLIS to better understand the condition over time. It may include your siblings if they also have SPLIS, and data from patients who have passed away.
San Francisco, CaliforniaAges Any age - NCT06670781Recruiting
Comparing two ways to do the Valsalva maneuver during a heart ultrasound
This trial compares two ways of doing a breathing maneuver (Valsalva) during a heart ultrasound to see if it better detects a common hole in the heart (PFO). If you are already scheduled for this test, the study will help find the best method for you.
Ottawa, OntarioAges 18 years+ - NCT06678737RecruitingPhase 2
TMS plus behavioral therapy for teens with tics
This study tests whether combining a brain stimulation technique called TMS with behavioral therapy can reduce tics in adolescents and young adults. If you have had tics for at least a year and they are moderate or severe, this trial may be a good fit.
Minneapolis, MinnesotaAges 12–21 - NCT05643209Recruiting
Mapping and ablation to treat abnormal heart electrical signals
This study uses detailed 3D heart mapping to find where abnormal electrical activity starts in Brugada syndrome and then performs ablation (a targeted scar or block). It’s mainly for adults who have had concerning symptoms and already have an implanted defibrillator (ICD).
Cotignola, RavennaAges 18 years+ - NCT06657859Enrolling by invitationPhase 2
Extended study of GLM101 for PMM2-CDG patients
This study is for people who already completed a previous trial of GLM101 for PMM2-CDG. It offers continued access to the treatment while doctors monitor safety and effects.
Minneapolis, MinnesotaAges 2 years+ - NCT06667141RecruitingPhase 1/Phase 2
Study of ACR-2316 for advanced solid tumors
This trial is testing an experimental drug called ACR-2316 for people with certain advanced solid tumors that have not responded to standard treatments. The study aims to see if the drug can shrink tumors or slow their growth.
Phoenix, ArizonaAges 18 years+ - NCT06666413RecruitingPhase 4
Study of Nexviazyme in children with infantile Pompe disease
This study tests the safety and effects of the enzyme replacement therapy avalglucosidase alfa (Nexviazyme) in children with infantile-onset Pompe disease. It is for children who have had Pompe symptoms since infancy and heart problems at diagnosis.
ShanghaiAges Up to 17 years - NCT06663358Recruiting
Safety and effectiveness of Elfabrio in Fabry disease patients
This study is for people with Fabry disease who are taking or planning to take the medication Elfabrio (pegunigalsidase alfa). It will look at how safe and effective the treatment is in real-world use.
Birmingham, AlabamaAges 18 years+
Common questions
- How many genetic & congenital clinical trials are recruiting?
- Clin2 is tracking 3,762 recruiting medical genetics studies from the U.S. registry right now, out of 5,482 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.