Congenital, Hereditary, and Neonatal Diseases and Abnormalities

281 trials 114 recruiting

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Long-term safety and efficacy of CSL312 (garadacimab) in the prophylactic treatment of hereditary angioedema attacks Ended

2024-510777-18-00 Hereditary angioedema 5 2021-06-15
A trial to learn how well darolutamide plus androgen deprivation therapy (ADT) works in comparison to placebo plus ADT in men with prostate … Ended

2022-502244-12-00 Metastatic hormone-sensitive prostate cancer (mHSPC) 5 2021-02-22
Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscu… Ended

2023-509935-23-00 Duchenne Muscular Dystrophy 4 2020-08-21
An extension study of Dasiglucagon in children with CHI Ended

2024-514007-34-00 Children with congenital hyperinsulinism 5 2020-03-08
A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem a… Ended

2024-516067-83-00 Severe sickle cell disease (SCD) 7 2019-05-02
HELIOS-A: A Phase 3 Global Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients with Hereditary Transthyretin Amyloidosis Ended

2023-508365-33-00 Hereditary Transthyretin Amyloidosis 5 2019-04-09
Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3 Ended

2024-510751-34-00 Gaucher's disease type I; Gaucher's disease type III 5 2019-03-26
Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Patients With Fabry… Ended

2024-516735-27-00 Fabry disease (α-galactosidase A deficiency) 5 2019-01-31
A Phase 1/2/3 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progen… Ended

2024-516894-57-00 Transfusion-dependent β-thalassemia (TDT) 5 2018-09-07
Effect of ALlopurinol in addition to hypothermia for hypoxicischemic Brain Injury on Neurocognitive Outcome Ended

2024-511322-31-00 Brain injury termed “hypoxic-ischemic encephalopathy” ( 5 2018-03-26
Venglustat in Combination with Cerezyme in Adult Patients with Gaucher Disease Type 3 with venglustat monotherapy extension Ended

2023-508646-18-00 Gaucher disease type 3 4 2017-09-18
Roll-over study in patients with TSC and refractory seizures who are judged by the Investigator to benefit from continued treatment with eve… Ended

2024-516746-19-00 Refractory seizures associated with tuberous sclerosis 5 2017-08-30
The AGAINST-PLD Study; An investigator-driven, randomized, open label clinical trial assessing efficacy of leuprorelin to halt disease progr… Ended

2023-506637-30-00 polycystic liver disease 5
Effect of dapagliflozin on blood magnesium levels in patients with low magnesium due to HNF1beta-associated kidney disease Ended

2024-518855-43-00 Hypomagnesemia due to HNF1beta mutation 5
Efficacy and safety of the intratracheal administration of budesonide with surfactant in very preterm infants to prevent bronchopulmonary dy… Ended

2024-519980-16-00 Bronchopulmonary dysplasia 5
Trampoline study Ended

2024-512544-27-00 Autosomal Dominant Polycystic Kidney Disease 6
A Phase 3b Multicenter Open-label Trial of the Safety, Tolerability, and Efficacy of Tolvaptan in Infants and Children 28 days to less than … Ended

2023-508217-17-00 Autosomal Recessive Polycystic Kidney Disease 5