Congenital, Hereditary, and Neonatal Diseases and Abnormalities

281 trials 114 recruiting

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ADfind Pending

2025-521833-97-00 Moderate-to-severe atopic dermatitis 6
Lidocaine for opioid sparing in vaso-occlusive crisis of Sickle Cell Disease Pending

2024-518437-28-00 Sickle cell disease 5
A study to learn if fenfluramine hydrochloride is safe and works in people with Rett syndrome Pending

2025-523157-34-00 Rett syndrome 5
Improving Heart Scans in Children: A Study Comparing Two Contrast Agents for MRI Pending

2025-524029-41-00 Cardiomyopathy, tetralogy of Fallot, or complex congeni 5
In situ injection of anti-angiogenics in patients with brain arteriovenous malformations not eligible for exclusion treatment: phase I trial… Pending

2023-506991-28-00 Cardiology and vascular disease 3
A phase I/II open label study to assess safety, feasibility and efficacy of ex vivo expanded, autologous haematopoietic stem and progenitor … Pending

2024-518972-30-00 Autosomal recessive osteopetrosis caused by mutations i 7
Treatment of low-flow vascular malformations with bleomycin electrosclerotherapy (BEST) Pending

2025-524123-45-00 VASCULAR MALFORMATION 4
Neonatal Brain Ultrasound With CEUS and Elastography Pending

2024-519611-33-00 Vastasyntyneiden hypoksis-iskeeminen aivovaurio, vastas 6
Administration of a new gene therapy to patients affected by Hereditary Spastic Paraplegia Type 50 (SPG50) Pending

2025-522603-15-00 Hereditary Spastic Paraplegia Type 50 (SPG50) 4
A randomized, placebo-controlled trial to assess the efficacy, tolerability, and pharmacokinetics of clemastine in children and adults with … Pending

2025-524195-29-00 Pitt-Hopkins syndrome 4
A study of Mirdametinib in Infants and Toddlers under 24months of age with Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas… Pending

2025-522000-24-00 Neurofibromatosis Type 1 (NF1) Associated Plexiform Neu 3
Evaluation of the efficacy of iloprost in the management of vaso-occlusive crises in adult patients with sickle cell: a multicentre, randomi… Pending

2025-522005-38-00 sickle cell anaemia with vaso-occlusive crisis 5
Evaluation of the safety, tolerability, and effectiveness of CTH120 in adult males with Fragile X syndrome Recruiting

2025-522972-97-00 Fragile X Syndrome 4 2026-05-21
A Study in Pediatric Participants with Congenital Adrenal Hyperplasia Recruiting

2024-519578-38-00 Classic congenital adrenal hyperplasia 10 2026-03-30
A Long-term Study of Navenibart in Participants with Hereditary Angioedema Recruiting

2025-521142-22-00 Hereditary Angioedema 5 2026-03-27
Pediatric Safety Follow-up Study of Prior Treatment with Romosozumab for Osteogenesis Imperfecta Recruiting

2023-503293-21-00 Osteogenesis Imperfecta 5 2026-03-25
Low dose corticosteroids adjacent to enzyme replacement therapy or chaperon therapy in patients with cardiac manifestation of Fabry disease Recruiting

2025-521408-24-00 Fabry disease 5 2026-03-05
A Study to Evaluate Atumelnant in Adults with Congenital Adrenal Hyperplasia Recruiting

2024-519579-24-00 Classic Congenital Adrenal Hyperplasia 5 2026-02-26
STOP-PKD: SGLT2-inhibition to improve Prognosis in Polycystic Kidney Disease Recruiting

2025-521276-59-00 Autosomal dominant polycystic kidney disease (ADPKD) 5 2025-12-16
A Multi-center, Double-Blind, Randomized, Two-Arm, Parallel-Group, Placebo Controlled Study to Assess the Safety of ELGN-2112 in Populations… Recruiting

2024-517102-29-00 Intestinal Malabsorption 5 2025-11-19
A Study to Assess How Effective and Safe NVD003 is for Treating Patients with Congenital Pseudarthrosis of the Tibia. Recruiting

2025-520998-39-00 Congenital pseudarthrosis of the tibia 5 2025-11-07
Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 Recruiting

2024-518144-20-00 Primary Hyperoxaluria Type 1 (PH1) 7 2025-09-03
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With AS (Aspire) Recruiting

2024-512600-19-00 Angelman Syndrome 5 2025-04-14
Study to provide continued access to treatment for patients completing a previous trial with efanesoctocog alfa Recruiting

2023-506537-29-00 Haemophilia A 5 2025-02-24