Congenital, Hereditary, and Neonatal Diseases and Abnormalities

281 trials 114 recruiting

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Intranasal Sufentanil for Analgesia of Severe Sickle Cell Vaso-occlusive Pain Crisis in the Pediatric Emergency Department Recruiting

2023-504847-15-00 vaso-occlusive crisis due to sickle cell disease 5 2025-07-21
1. A study to see how well nizubaglustat (AZ-3102) works in participants with GM1 gangliosidosis or GM2 gangliosidosis and how safe it is 2.… Recruiting

2024-515778-28-00 Niemann-Pick type C disease and GM1/GM2 gangliosidoses 5 2025-06-30
The use of sildenafil in patients with univentricular heart after Fontan operation - a pilot SINFON-POL study. Recruiting

2023-503913-31-01 Patients with functionally single ventricle (FSV - func 5 2025-06-27
A Trial to Learn if REGV131-LNP1265 is Safe and Works to Help the Body Make Clotting Factor in Adult Patients with Hemophilia B Recruiting

2023-507260-40-00 Hemophilia B 7 2025-06-16
A Multi-center, Double-Blind, Randomized, Two-Arm, Parallel-Group, Placebo Controlled Study to Assess the Efficacy and Safety of ELGN-2112 o… Recruiting

2024-517101-87-00 Intestinal Malabsorption 5 2025-05-26
Long-term study evaluating joint health in people with haemophilia A receiving real-world prophylactic treatment with efanesoctocog alfa Recruiting

2024-517812-31-00 Haemophilia A 6 2025-05-21
A study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia Recruiting

2024-517706-29-00 Ataxia Telangiectasia 5 2025-05-13
Maternal oxygen therapy during pregnancy for unborn babies with underdeveloped structures on the left side of the heart Recruiting

2024-512679-11-00 unborn baby with developmental disorder of the left sid 5 2025-04-25
CHAPTER-4 – A long term trial of deucrictibant treatment in prophylaxis against HAE attacks in adults and adolescents Recruiting

2024-516248-24-00 Hereditary Angioedema 5 2025-03-28
A Phase 3 Efficacy and Safety Study of Pitolisant in Patients with Prader-Willi Syndrome Recruiting

2023-508307-21-00 Prader-Willi syndrome 5 2025-03-19
A pilot study of enalapril in venous malformations Recruiting

2023-510076-31-00 Venous malformation 4 2025-01-30
Long Term Follow-up Study of AAVAnc80-hOTOF Gene Therapy Recruiting

2024-512584-31-00 Otoferlin gene-mediated hearing loss 10 2024-11-11
Safety and efficacy of avalglucosidase alfa in patients with non-classic Pompe disease aged ≥ 5 years. Recruiting

2024-518215-18-00 Glycogen storage disease type II or acid maltase defici 6 2024-10-16
30% or 60% Oxygen at Birth to Improve Neurodevelopmental Outcomes in Very Low Birthweight Infants Recruiting

2023-509780-25-00 Prematurity 5 2024-10-09
A Trial of Lu AG13909 in Adult Participants with Cushing’s Disease Recruiting

2023-504733-53-00 Cushing's Disease 4 2024-09-26
A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients with Angelman Syndrome Recruiting

2024-510917-14-00 Angelman Syndrome 5 2024-09-03
Oral Propranolol for prevention of threshold retinopathy of prematurity Recruiting

2024-511338-10-01 Retinopathy of prematurity 4 2024-07-24
A study to investigate efficacy, safety, and tolerability of mavorixafor in participants with congenital and acquired primary autoimmune and… Recruiting

2023-508482-32-00 Neutropenias 5 2024-07-22
HYDROchlorothiazide to PROTECT polycystic kidney disease patients and improve their quality of life (HYDRO-PROTECT) Recruiting

2022-500210-26-00 Autosomal Dominant Polycystic Kidney Disease (ADPKD) 5 2024-06-28
Dose-finding for dobutamine during transitional circulation in the very preterm infant Recruiting

2023-504915-34-00 Neonates with haemodynamic insufficiency 9 2024-06-20
Long-Term Follow-Up of Subjects Treated With NTLA-2002 Recruiting

2023-507956-56-00 Hereditary Angioedema 9 2024-05-17
A study to extend treatment with JR-141 after completion of Study JR-141-GS31 Recruiting

2022-503142-41-00 Mucopolysaccharidosis type II 5 2024-05-06
Long-Term Follow-Up of Subjects Treated With NTLA-2001 Recruiting

2023-507385-11-00 -Hereditary Transthyretin Amyloidosis with Polyneuropat 10 2024-05-06
Phase I-IIa, randomized, controlled, open-label, single-center clinical trial to evaluate the safety, feasibility, and evidence of efficacy … Recruiting

2023-506913-23-00 Cleft lip and palate 7 2024-04-17