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Mucopolysaccharidosis II clinical trials

View clinical trials related to Mucopolysaccharidosis II.

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NCT ID: NCT04628871 Active, not recruiting - Hemophilia B Clinical Trials

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

LTFU
Start date: November 3, 2020
Phase:
Study type: Observational

Long-term follow-up of subjects who received SB-318, SB-913, or SB-FIX in a previous trial and completed at least 52 weeks post-infusion follow-up in their primary protocol. Enrolled subjects will be followed for a total of up to 10 years following exposure to SB-318, SB-913, or SB-FIX.

NCT ID: NCT04571970 Active, not recruiting - Clinical trials for Mucopolysaccharidosis Type II (MPS II)

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

Start date: March 11, 2021
Phase: Phase 1/Phase 2
Study type: Interventional

RGX-121 is a gene therapy which is designed to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system. This study is a phase I/II study to determine whether RGX-121 is safe, well tolerated, and potentially effective in children five years of age and over who have severe MPS II.

NCT ID: NCT04348136 Active, not recruiting - Clinical trials for Mucopolysaccharidosis II

An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II

Start date: September 1, 2019
Phase: Phase 2/Phase 3
Study type: Interventional

Multicenter, open-label, single-group, designed to evaluate the long term efficacy and safety of study drug for the treatment of the MPS II.

NCT ID: NCT04251026 Active, not recruiting - Clinical trials for Mucopolysaccharidosis II

A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

Start date: July 16, 2020
Phase: Phase 1/Phase 2
Study type: Interventional

This is a multicenter, multiregional, open-label study to assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme replacement therapy (ERT), designed to treat both the peripheral and CNS manifestations of Mucopolysaccharidosis type II (MPS II; Hunter syndrome). Participants, whose physicians feel they are deriving benefit, will have the opportunity to be reconsented into a safety extension and then an open-label extension for continued evaluation.

NCT ID: NCT03708965 Active, not recruiting - Clinical trials for Mucopolysaccharidosis II

An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II

Start date: January 1, 2019
Phase: Phase 2
Study type: Interventional

A Phase II open-label, parallel group, 2 sites (Brazil), designed to evaluate the long term safety and efficacy of study drug for the treatment of the MPS II.

NCT ID: NCT03566043 Active, not recruiting - Clinical trials for Mucopolysaccharidosis Type II (MPS II)

CAMPSIITEā„¢ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

Start date: September 27, 2018
Phase: Phase 2/Phase 3
Study type: Interventional

RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study is a safety and efficacy, dose ranging study to determine whether RGX-121 is safe, effective and well-tolerated by patients with MPS II.

NCT ID: NCT03153319 Active, not recruiting - Clinical trials for Mucopolysaccharidosis II

Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI

Start date: June 5, 2017
Phase: Phase 1/Phase 2
Study type: Interventional

Randomized, double-blind, placebo-controlled, parallel-group, single-center study followed by open-label phase, to evaluate the effects of adalimumab compared to placebo on the change from baseline in joint and skeletal disease in children and adults with mucopolysaccharidosis (MPS) I, II or VI.

NCT ID: NCT02455622 Active, not recruiting - Hunter Syndrome Clinical Trials

Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age

Start date: October 28, 2015
Phase: Phase 4
Study type: Interventional

This long-term study will provide Elaprase treatment to children enrolled in this study and will utilize data from both enrolled patients and Hunter Outcome Survey (HOS) patient registry data to conduct the primary growth analysis to assess changes in height and weight in patients with Mucopolysaccharidosis II (Hunter syndrome) MPS II.