Hunter Syndrome (MPS II) Clinical Trials

A listing of Hunter Syndrome (MPS II) medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

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Found 15 clinical trials
RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

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 …

  • 12 May, 2022
  • 2 locations
Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

45 years inclusive, with MPS II (Hunter syndrome) currently receiving idursulfase ERT (or the equivalent) are eligible to participate. Participants will be followed for safety and efficacy for 5

  • 28 Jul, 2022
  • 1 location
A Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) (COMPASS)

(CNS)-penetrant enzyme-replacement therapy (ERT) for mucopolysaccharidosis type II (MPS II). Participants may also qualify to enter an open-label treatment phase with DNL310 or idursulfase

  • 08 Aug, 2022
  • 2 locations
RGX-121 Gene Therapy in Patients With MPS II (Hunter Syndrome)

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

  • 12 May, 2022
  • 4 locations
A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India

The main aim of this study is to learn more about the safety profile of Elaprase in Indian children and adults with hunter syndrome. Participants will receive Elaprase once per week over a 3-hour period which can be reduced to 1 hour as determined by the study doctor. Participants will …

  • 14 May, 2022
  • 1 location
A Study of DNL310 in Pediatric Participants With Hunter Syndrome

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

iduronate 2-sulfatase
enzyme replacement therapy
  • 21 Apr, 2022
  • 5 locations
A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II

This is phase 2/3 study to evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of the investigational product GNR-055 in MPS II (Hunter syndrome) patients of different age

  • 24 Mar, 2022
  • 2 locations
Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI

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.

bodily pain
adalimumab injection [humira]
mucopolysaccharidosis type i
  • 30 Jan, 2022
  • 1 location
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 as an adjunctive therapy in patients with inborn errors of metabolism who have evidence of early demyelinating disease in the central nervous system (CNS) who are undergoing standard treatment with unrelated umbilical …

mutation analysis
mps iii
niemann pick disease
  • 23 Dec, 2021
  • 1 location
In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases (IUERT)

The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.

replacement therapy
  • 15 May, 2022
  • 1 location