Phase 3 Recessive Clinical Trials
3 recruitingPhase 3
What is a Phase 3 trial?
Phase 3 trials compare the new treatment against the current standard of care in large groups, often hundreds to thousands of participants. Successful Phase 3 results are typically required for regulatory approval.
Showing 1–3 of 3 trials
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 3
A Study Comparing KB803 and Matched Placebo in Patients With Dystrophic Epidermolysis Bullosa
Recessive dystrophic epidermolysis bullosaDystrophic Epidermolysis BullosaDEB - Dystrophic Epidermolysis Bullosa+1 more
Krystal Biotech, Inc.16 enrolled5 locationsNCT07016750
Recruiting
Phase 3
A Study to See Iftolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old withAutosomal Recessive Polycystic Kidney Disease (ARPKD)
Autosomal Recessive Polycystic Kidney (ARPKD)
Otsuka Pharmaceutical Development & Commercialization, Inc.20 enrolled23 locationsNCT04782258