Indication
Mucopolysaccharidosis Type II
4 clinical trials
5 products
Clinical trial
Multicenter, Open-Label, Multi-cohort Study to Evaluate Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Drug Product GNR 055 (JSC "GENERIUM", Russia) in Patients With Mucopolysaccharidosis Type IIStatus: Recruiting, Estimated PCD: 2025-02-01
Product
GNR-055Clinical trial
A Phase 1/2/3 Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects With MPS II (Hunter Syndrome)Status: Active (not recruiting), Estimated PCD: 2024-05-01
Product
RGX-121Clinical trial
A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of RGX-121 in Children 5 Years of Age and Older With MPS II (Hunter Syndrome)Status: Active (not recruiting), Estimated PCD: 2024-05-01
Clinical trial
Open-label Multi-cohort Study of the Tolerability, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers After a Single Intravenous Injection in Increasing DosesStatus: Completed, Estimated PCD: 2023-09-20
Product
Cohort 1Product
Cohort 2Product
Cohort 3