Congenital Clinical Trials

351 recruitingLast updated: June 18, 2026

There are 351 actively recruiting congenital clinical trials across 55 countries. Studies span Not Applicable, Phase 2, Phase 1, Phase 3, Phase 4, Early Phase 1. Top locations include Boston, Massachusetts, United States, Paris, France, Los Angeles, California, United States. Updated daily from ClinicalTrials.gov.


Congenital Trials at a Glance

351 actively recruiting trials for congenital are listed on ClinicalTrialsFinder across 6 cities in 55 countries. The largest study group is Not Applicable with 115 trials, with the heaviest enrollment activity in Boston, Paris, and Los Angeles. Lead sponsors running congenital studies include Boston Children's Hospital, IRCCS Azienda Ospedaliero-Universitaria di Bologna, and Assistance Publique - Hôpitaux de Paris.

Browse congenital trials by phase

Treatments under study

About Congenital Clinical Trials

Looking for clinical trials for Congenital? There are currently 1 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Congenital trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Congenital clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 120 of 351 trials

Recruiting

Natural History Study of Patients With Excess Androgen

Congenital Adrenal Hyperplasia (CAH)Familial Male-Limited Precocious Puberty (FMPP)
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)3,000 enrolled2 locationsNCT00250159
Recruiting
Phase 2Phase 3

A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)

Congenital Adrenal HyperplasiaClassic Congenital Adrenal Hyperplasia
Crinetics Pharmaceuticals Inc.153 enrolled35 locationsNCT07159841
Recruiting

ARPKD Database Study

Autosomal Recessive Polycystic Kidney DiseaseNephronophthisisCongenital Hepatic Fibrosis+7 more
Children's Hospital of Philadelphia200 enrolled6 locationsNCT01401998
Recruiting

Genome Medical Sequencing for Gene Discovery

Intellectual disabilitiesRare DisordersCongenital Anomaly
National Human Genome Research Institute (NHGRI)2,000 enrolled1 locationNCT01087320
Recruiting

DMCRN-02-001: Assessing Pediatric Endpoints in DM1

Congenital Myotonic DystrophyCDM
Virginia Commonwealth University50 enrolled5 locationsNCT05224778
Recruiting

Congenital Uterine Anomalies: Identifying Cancer Associations and Genetic and Environmental Factors to Improve Clinical Care

Congenital Uterine Anomaly
Yale University300 enrolled1 locationNCT04661072
Recruiting

Investigation of the Genetics of Hematologic Diseases

Diamond-Blackfan AnemiaDyskeratosis CongenitaFanconi Anemia+10 more
St. Jude Children's Research Hospital1,716 enrolled1 locationNCT02720679
Recruiting

Protein Biomarkers and Host RNA Expression Profiles in Congenital Cytomegalovirus Infection

Sensorineural Hearing LossViral InfectionCytomegalovirus Infections+2 more
Rigshospitalet, Denmark630 enrolled2 locationsNCT07635368
Recruiting
Not Applicable

Safety and Efficacy of Fetoscopic Endoluminal Tracheal Occlusion (FETO) in Congenital Diaphragmatic Hernia (CDH)

Congenital Diaphragmatic Hernia
Dr Erin Perrone40 enrolled1 locationNCT07577414
Recruiting
Phase 3

Efficacy of Atenativ in Patients With Congenital Antithrombin Deficiency Undergoing Surgery or Delivery

Congenital Antithrombin Deficiency
Octapharma38 enrolled30 locationsNCT04918173
Recruiting
Phase 3

A Study to Evaluate Atumelnant in Adults With Congenital Adrenal Hyperplasia

Congenital Adrenal HyperplasiaClassic Congenital Adrenal Hyperplasia
Crinetics Pharmaceuticals Inc.150 enrolled45 locationsNCT07144163
Recruiting
Phase 3

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

Sensation DisordersLeber Congenital AmaurosisRetinal Disease+7 more
Laboratoires Thea32 enrolled16 locationsNCT06891443
Recruiting

A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4

Congenital Myasthenic Syndrome
argenx100 enrolled30 locationsNCT06078553
Recruiting

Post-Market Clinical Follow-Up Study on the Safety and Performance of Ennovate® Complex

HyperkyphosisHyperlordosisCongenital Scoliosis+2 more
Aesculap AG200 enrolled2 locationsNCT05281757
Recruiting

Remote Assessments and Genetic Determinants of Congenital and Childhood Myotonic Dystrophy

Myotonic Dystrophy Type 1Congenital DM1Juvenile DM1+1 more
University of Rochester100 enrolled1 locationNCT07630389
Recruiting

Natural History of Noncirrhotic Portal Hypertension

Cystic FibrosisTurner SyndromeImmunologic Deficiency Syndrome+2 more
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)400 enrolled1 locationNCT02417740
Recruiting

Congenital Aniridia Patient Questionnaire

Congenital Aniridia
Assistance Publique - Hôpitaux de Paris100 enrolled1 locationNCT05390801
Recruiting
Not Applicable

Congenital Heart Disease Physical Activity Lifestyle Study V.2

Heart Defects, CongenitalCardiovascular Disease Other
Jamie Jackson144 enrolled1 locationNCT06003023
Recruiting

Natural History in Children up to 16 Years With Mild to Profound Hearing Loss Due to Mutations in GJB2 / OTOF Genes

OTOF Gene MutationDFNB9Congenital Deafness+4 more
Sensorion180 enrolled1 locationNCT05402813
Recruiting
Phase 2

A Clinical Study to Assess Sutacimig in Participants With Congenital Factor VII Deficiency

Congenital Factor VII Deficiency
Hemab ApS18 enrolled1 locationNCT07347249