Autosomal Clinical Trials in Paris
5 recruitingParis, France
Showing 1–5 of 5 trials
Recruiting
Phase 2
A Trial to Compare Treatment With Surlorian (ARM210, S48168) to Placebo in Effects on Muscle Strength and Safety in Adults With Autosomal Dominant RYR1-related Myopathy
Autosomal Dominant RYR1-Related Myopathy
RyCarma Therapeutics, Inc.28 enrolled9 locationsNCT07560020
Recruiting
Phase 2
Phase 2a Study of VX-407 in Participants With ADPKD Who Have a Subset of PKD1 Gene Variants (AGLOW)
Autosomal Dominant Polycystic Kidney Disease (ADPKD
Vertex Pharmaceuticals Incorporated24 enrolled43 locationsNCT07161037
Recruiting
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Cystic FibrosisHuntington DiseaseSickle Cell Disease+12 more
Assistance Publique - Hôpitaux de Paris550 enrolled1 locationNCT06147414
Recruiting
Phase 3
ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency
Ectonucleotide Pyrophosphatase/phosphodiesterase1 DeficiencyAutosomal Recessive Hypophosphatemic RicketsGeneralized Arterial Calcification of Infancy 1
Inozyme Pharma12 enrolled8 locationsNCT07473973
Recruiting
Phase 2
ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
Arterial calcificationPseudoxanthoma ElasticumEctonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency+2 more
Inozyme Pharma200 enrolled5 locationsNCT06462547