Necker-Enfants Malades Hospital | ClinicalTrials HQ

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Trial	Conditions	Status	Phase	Updated	More info	NCT ID
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)	Abnormalities, Multiple, Angelman syndrome, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Imprinting Disorders, central nervous system disorder, chromosomal disorder, hereditary disease, movement disorder, nervous system disorder	Active, not recruiting	Phase 2	2026-09-09		NCT07157254
ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency	Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Skin Abnormalities, Skin and Connective Tissue Diseases, arterial calcification of infancy, autosomal recessive inherited pseudoxanthoma elasticum, cardiovascular disorder, connective tissue disorder, hematologic disorder, hemorrhagic disease, hereditary disease, hereditary skin disorder, hypophosphatemic rickets, autosomal recessive, 2, skin disorder, vascular disorder, vascular hemostatic disease	Active, not recruiting	Phase 2	2026-08-28		NCT06462547
HYDROchlorothiazide to PROTECT Polycystic Kidney Disease Patients and Improve Their Quality of Life	Abnormalities, Multiple, Ciliopathies, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Pathological Conditions, Signs and Symptoms, Polyuria, Signs and Symptoms, Urination Disorders, Urogenital Diseases, Urological Manifestations, autosomal dominant polycystic kidney disease, cystic kidney disease, hereditary disease, kidney disorder, polycystic kidney disease, urinary system disorder	Recruiting	Phase 3	2026-08-25		NCT05373264