Mucopolysaccharidosis Type II Clinical Trials

16 clinical trials for Mucopolysaccharidosis Type II across international registries, including 4 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II — RECRUITING · PHASE2
• EFFECT OF ADALIMUMAB ON MUSCULOSKELETAL DISEASE IN MUCOPOLYSACCHARIDOSIS TYPES I, II, IVA AND VI — RECRUITING
• AN OPEN-LABEL EXTENSION TO INVESTIGATE THE LONG-TERM SAFETY, TOLERABILITY, AND EFFICACY OF DNL310 IN PATIENTS WITH MUCOPOLYSACCHARIDOSIS TYPE II (MPS II) FROM STUDY DNLI-E-0002 OR STUDY DNLI-E-0007 — RECRUITING
• A PHASE 2/3, MULTICENTER, DOUBLE-BLIND, RANDOMIZED STUDY TO DETERMINE THE EFFICACY AND SAFETY OF DNL310 VS IDURSULFASE IN PEDIATRIC PARTICIPANTS WITH NEURONOPATHIC OR NON-NEURONOPATHIC MUCOPOLYSACCHARIDOSIS TYPE II — RECRUITING · PHASE2
• A Multi-Center, Open-Label Study Evaluating Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Enzyme Replacement Therapy — COMPLETED · PHASE4
• RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome) — COMPLETED · PHASE1
• CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome) — ACTIVE_NOT_RECRUITING · PHASE2
• A Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers — COMPLETED · PHASE1
• Core Outcome Set for Head, Neck and Respiratory Disease in Mucopolysaccharidosis II — COMPLETED
• 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) — COMPLETED · PHASE1
• A PHASE 1 OPEN-LABEL DOSE ESCALATION STUDY TO EVALUATE THE SAFETY AND EFFICACY OF HMI-203 IN ENZYME REPLACEMENT THERAPY (ERT)-TREATED ADULTS WITH MUCOPOLYSACCHARIDOSIS TYPE II (MPS II) (JUMPSTART TRIAL) — COMPLETED · PHASE1
• A Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers — COMPLETED · PHASE1
• Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children — COMPLETED
• Longitudinal Studies of Brain Structure and Function in MPS Disorders — COMPLETED
• Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III — COMPLETED
• Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI — COMPLETED · PHASE1

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