Disease Observational Study-Rare Disease Registry-Cross-Registry Analysis September 25, 2024 Sanofi Genzyme Rare Disease Registry - A Multi-Center, International, Longitudinal, Observational program designed to track natural history and outcomes of patients with Gaucher, Fabry, MPSI, and...
Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program (LC-FAOD DMP)-UX007-CL401 September 25, 2024 Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program ClinicalTrials.gov ID NCT04632953 Sponsor Ultragenyx Pharmaceutical Inc Information provided by Ultragenyx...
A Phase I/II, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects with Fabry Disease June 05, 2020 A Phase I/II, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects with Fabry...
An observation, longitudinal, prospective, long-team registry of patients with hypophosphatasia-Alexion HPP Registry ALX-HPP-501 June 05, 2020 An Observational, Longitudinal, Prospective, Long-Term Registry of Patients with hypophosphatasia ClinicalTrials.gov ID NCT02306720 Sponsor Alexion Pharmaceuticals, Inc. Information provided by...
A Phase 3 Multi-Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase in Adolescent Subjects (Ages 12-17) with Phenylketonuria Featuring an Open-Label Randomized Two-Arm (Active vs Diet-Only Control) Design/BMN 165-306 June 05, 2020 A Phase 3 Multi-Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase in Adolescent Subjects (Ages 12-17) with Phenylketonuria Featuring an Open-Label Randomized...
A Non-Interventional Post-Authorization Safety Study (PASS) of Carbaglu® for the Treatment of Hyperammonemia Due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA) in Adult and Pediatric Patient Populations June 02, 2020 A Non-Interventional Post-Authorization Safety Study (PASS) of Carbaglu® for the Treatment ofHyperammonemia Due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA)in Adult and Pediatric...
A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects with Phenylketonuria and Plasma Phe Levels > 600 μmol June 02, 2020 A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects...
A Phase 1, First-In-Human, Multiple Part, Single Ascending and Multiple Dose Study of JNT-517 in Healthy Participants and in Participants with Phenylketonuria June 02, 2020 A Phase 1, First-In-Human, Multiple Part, Single Ascending and Multiple Dose Study of JNT-517 in Healthy Participants and in Participants with Phenylketonuria ClinicalTrials.gov ID NCT05781399 Spons...
