Phase 1 Congenital Clinical Trials
18 recruitingPhase 1
What is a Phase 1 trial?
Phase 1 trials test a new treatment in a small group of people for the first time. The primary goal is to evaluate safety, determine a safe dosage range, and identify side effects. These studies typically enroll 20 to 80 participants.
Showing 1–18 of 18 trials
Recruiting
Phase 1
A Safety Assessment of Oral Letermovir in Infants With Symptomatic Congenital Cytomegalovirus
Congenital Cytomegalovirus Infection
National Institute of Allergy and Infectious Diseases (NIAID)12 enrolled10 locationsNCT06118515
Recruiting
Phase 1
A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)
Congenital Myasthenic SyndromeCMS
argenx16 enrolled9 locationsNCT06436742
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 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 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 1
Autologous Induced Pluripotent Stem Cells of Cardiac Lineage for Congenital Heart Disease
Congenital Heart DiseaseHeart Failure NYHA Class IIIHeart Failure NYHA Class IV+1 more
HeartWorks, Inc.50 enrolled1 locationNCT05647213
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 1
Serial Amnioinfusions as Regenerative Therapy for Pulmonary Hypoplasia
Lower Urinary Tract Obstructive SyndromeRenal Failure CongenitalCongenital Renal Anomaly Nos+1 more
Mayo Clinic40 enrolled1 locationNCT03723564
Recruiting
Phase 1
Patients With Congenital Myasthenic Syndrome Will be Treated With Mesenchymal Stem Cell Exosome Solution
Congenital Myasthenic Syndrome
The Foundation for Orthopaedics and Regenerative Medicine20 enrolled3 locationsNCT07226726
Recruiting
Phase 1Phase 2
Propranolol Treatment for Postoperative Chylothorax
Congenital Heart DiseasePostoperative Chylothorax
June Wu50 enrolled1 locationNCT06833320
Recruiting
Phase 1
Implantation Of Vaginal Construct For Patients With Vaginal Aplasia
Congenital Mullerian Duct Anomaly
Wake Forest University Health Sciences10 enrolled1 locationNCT05675722
Recruiting
Phase 1Phase 2
A Phase I/II Clinical Trial with SENS-501 in Children Suffering from Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations
Hearing DisordersDeafnessHearing Loss, Sensorineural+5 more
Sensorion12 enrolled2 locationsNCT06370351
Recruiting
Phase 1Phase 2
Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
Leber Congenital AmaurosisInherited Retinal Diseases Caused by RPE65 Mutations
HuidaGene Therapeutics Co., Ltd.20 enrolled3 locationsNCT05906953
Recruiting
Phase 1
Fluorodopa F 18 in Congenital Hyperinsulinism and Insulinoma
Congenital HyperinsulinismInsulinoma
Cook Children's Health Care System250 enrolled1 locationNCT02021604
Completed
Phase 1
Understanding the blood level of a new coagulant factor VIIa in participants with congenital Haemophilia A
Congenital Hemophilia A in subjects with or without Inhibitors to Factor VIII
LFB USA, Inc.28 enrolled3 locationsACTRN12620001152910
Recruiting
Phase 1
The effect of remote ischaemic preconditioning on lowering postoperative myocardial troponin I release in children with congenital heart disease undergoing cardiopulmonary bypass.
Congenital heart disease witithin neonates
Michael Cheung68 enrolled1 locationACTRN12607000004460