Phase 2 Congenital Clinical Trials
29 recruitingPhase 2
What is a Phase 2 trial?
Phase 2 trials evaluate whether a treatment works for a specific condition and further assess its safety. These studies typically enroll several hundred participants and provide preliminary data on effectiveness.
Showing 1–20 of 29 trials
Recruiting
Phase 2
18FluoroLDOPA PET Imaging for the Detection and Localization of Focal Congenital Hyperinsulinism
Congenital Hyperinsulinism
Miguel Pampaloni50 enrolled1 locationNCT04205604
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 enrolled32 locationsNCT07159841
Recruiting
Phase 1Phase 2
A Clinical Trial of EHT102 Injection in Pediatric Patients With Biallelic hOTOF Mutations
Treatment of Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
Shanghai Euhearing Therapeutics Co., Ltd30 enrolled1 locationNCT07288580
Recruiting
Phase 1Phase 2
Influence of Personalized Lung Volume Optimization Maneuver on Lung Function and Cardiac Performance in Children
Mechanical VentilationCongenital Heart DiseaseChildren+8 more
Charite University, Berlin, Germany80 enrolled1 locationNCT07193719
Recruiting
Phase 2
CMV CTLs in Neonates With CMV Infection
Congenital Cytomegaloviral (CMV) Disease
New York Medical College23 enrolled4 locationsNCT05564598
Recruiting
Phase 1Phase 2
A Study to Investigate the Improvement of Photosensitivity in Terms of Skin Lesions Associated With CEP Following Administration of Oral ATL-001 (Ciclopirox Oral Solution) in Participants Aged >18 Years of Age With CEP
Congenital Erythropoietic Porphyria (CEP)
Atlas Molecular Pharma6 enrolled1 locationNCT07024316
Recruiting
Phase 2
Pharmacokinetics, Safety and Tolerability of Crinecerfont in Participants With Congenital Adrenal Hyperplasia Who Are Less Than 2 Years Old
Congenital Adrenal Hyperplasia
Neurocrine Biosciences6 enrolled3 locationsNCT07187375
Recruiting
Phase 1Phase 2
Zoledronic Acid Treatment in Patients With Congenital Dyserythropoietic Anemia
Congenital Dyserythropoietic Anemia (CDA)
Institute of Hematology & Blood Diseases Hospital, China2 enrolled1 locationNCT07471516
Recruiting
Phase 1Phase 2
A Trial of Lu AG13909 in Participants With Congenital Adrenal Hyperplasia
Congenital Adrenal Hyperplasia
H. Lundbeck A/S42 enrolled17 locationsNCT05669950
Recruiting
Phase 1Phase 2
A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
Regeneron Pharmaceuticals30 enrolled15 locationsNCT05788536
Recruiting
Phase 2
A Study to Evaluate Tabelecleucel in Participants With Epstein Barr Virus (EBV) Associated Diseases
LeiomyosarcomaSolid Organ Transplant ComplicationsLymphoproliferative Disorders+8 more
Pierre Fabre Medicament190 enrolled40 locationsNCT04554914
Recruiting
Phase 2
A Clinical Study to Assess Sutacimig in Participants With Congenital Factor VII Deficiency
Congenital Factor VII Deficiency
Hemab ApS18 enrolled1 locationNCT07347249
Recruiting
Phase 2
Multicenter Trial of ECMO in Children With Severe Cardiac Failure Using the Cardiohelp System
Heart FailureCardiomyopathiesCardiogenic Shock+1 more
Stanford University50 enrolled5 locationsNCT06080074
Recruiting
Phase 2
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
Juvenile Rheumatoid Arthritis (JRA)Primary Immunodeficiency (PID)Congenital Bone Marrow Failure Syndromes+4 more
Paul Szabolcs100 enrolled1 locationNCT01962415
Recruiting
Phase 2
Evaluation of Antimicrobial Prophylaxis to Prevent Syphilis in Pregnancy in Patients at Risk in Rio de Janeiro, Brazil
Congenital Syphilis
University of California, Los Angeles500 enrolled4 locationsNCT07189208
Recruiting
Phase 1Phase 2
Propranolol Treatment for Postoperative Chylothorax
Congenital Heart DiseasePostoperative Chylothorax
June Wu50 enrolled1 locationNCT06833320
Recruiting
Phase 2
An Extension Study to Evaluate Safety and Efficacy in Participants Treated With CRN04894
Congenital Adrenal HyperplasiaClassic Congenital Adrenal Hyperplasia
Crinetics Pharmaceuticals Inc.150 enrolled9 locationsNCT06712823
Recruiting
Phase 2
A 44-week Monocentric Open Study Assessing the Efficacy and Safety of Deucravacitinib in Adults With Inflammatory Genodermatoses
IchthyosisEpidermolysis Bullosa SimplexGenodermatosis+1 more
Centre Hospitalier Universitaire de Nice10 enrolled2 locationsNCT06136403
Recruiting
Phase 2Phase 3
Human Penile Allotransplantation
Wounds and InjuriesAmputationAmputation, Traumatic+6 more
Johns Hopkins University60 enrolled1 locationNCT02395497
Recruiting
Phase 2Phase 3
Fetal Cerebrovascular Autoregulation in Congenital Heart Disease and Association With Neonatal Neurobehavior
HypoxiaNeurodevelopmental DisordersCongenital Heart Disease in Children+1 more
University of Utah150 enrolled5 locationsNCT05767385