Clinical Trials

Clinical Trial
  • Status: Not yet recruiting
  • Phase: Phase 3
  • Intervention Type: Drug
  • Participants: 50
  • Start Date: March 31, 2021
A Phase Ⅲ Study of JR-141 in Patients With Mucopolysaccharidosis II
Clinical Trial
  • Status: Recruiting
  • Intervention Type: Other
  • Participants: 40
  • Start Date: March 2021
A Prospective, Observational Study of Pediatric Patients With Neuronopathic Forms of MPS II (Hunter Syndrome)
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Clinical Trial
  • Status: Recruiting
  • Phase: Phase 1/Phase 2
  • Intervention Type: Genetic
  • Participants: 6
  • Start Date: February 2021
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)
Clinical Trial
  • Status: Enrolling by invitation
  • Intervention Type: Biological
  • Participants: 13
  • Start Date: November 3, 2020
Long-Term Follow-up of Subjects Who Were Treated With SB-318, SB-913, or SB-FIX, for Targeted Genome Editing Into the Albumin Gene in the Liver
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 1/Phase 2
  • Intervention Type: Drug
  • Participants: 16
  • Start Date: July 16, 2020
A Phase 1/2, Multicenter, Open-Label Study to Determine the Safety, Pharmacokinetics, and Pharmacodynamics of DNL310 in Pediatric Subjects With Hunter Syndrome
Clinical Trial
  • Status: Recruiting
  • Intervention Type: Other
  • Participants: 28
  • Start Date: October 23, 2019
A Study of Potential Treatment-Responsive Biomarkers in Hunter Syndrome
Clinical Trial
  • Status: Active, not recruiting
  • Phase: Phase 2/Phase 3
  • Intervention Type: Drug
  • Participants: 27
  • Start Date: September 1, 2019
An Extension Study of JR-141-301 in Patients With Mucopolysaccharidosis II
Clinical Trial
  • Status: Active, not recruiting
  • Phase: Phase 2
  • Intervention Type: Drug
  • Participants: 18
  • Start Date: January 1, 2019
An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 1/Phase 2
  • Intervention Type: Genetic
  • Participants: 12
  • Start Date: September 27, 2018
A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects With MPS II (Hunter Syndrome)
Clinical Trial
  • Status: Recruiting
  • Participants: 1000
  • Start Date: August 20, 2018
Biomarkers for Hunter Syndrome: An International, Multicenter, Observational, Longitudinal Protocol
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 1/Phase 2
  • Intervention Type: Drug
  • Participants: 14
  • Start Date: June 5, 2017
Phase 1/2 Study of the Effect of Adalimumab on Physical Function and Musculoskeletal Disease in Mucopolysaccharidosis Types I and II
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