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 12 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 …

  • 0 views
  • 14 Jun, 2021
  • 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.

  • 274 views
  • 15 Jun, 2021
  • 5 locations
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). The study has three cohorts:Cohort A will

enzyme replacement therapy
iduronate 2-sulfatase
mucopolysaccharidosis
  • 35 views
  • 22 May, 2021
  • 4 locations
Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I and II

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 or II.

mucopolysaccharidosis
mucopolysaccharidosis type i
adalimumab injection [humira]
bodily pain
humira
  • 210 views
  • 17 Feb, 2021
  • 1 location
In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases

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.

  • 0 views
  • 18 Apr, 2021
  • 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 …

seizure
niemann pick disease
pulmonary function tests
diffusion tensor imaging
mutation analysis
  • 153 views
  • 28 Jan, 2021
  • 1 location
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe osteopetrosis (OP).

niemann pick disease
mucopolysaccharidosis
mngie
deficiency
gm1 gangliosidosis
  • 137 views
  • 30 May, 2021
  • 1 location
The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients

The objective of this study is to evaluate the long term safety and efficacy of once weekly dosing of idurasulfase-beta 0.5mg/kg administered in Hunter Syndrome(Mucopolysaccharidosis II

idursulfase
mucopolysaccharidosis
  • 106 views
  • 07 Nov, 2020
A Study of Potential Treatment-Responsive Biomarkers in Hunter Syndrome

This is an observational, Pre-phase 1 study of biomarkers in patients with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.

anesthesia
general anesthesia
ids gene
mucopolysaccharidosis
  • 202 views
  • 31 Mar, 2021
  • 8 locations
Hunter Outcome Survey (HOS)

The purpose of this study is to collect data that will increase understanding of Hunter syndrome. The data from HOS may provide guidance to healthcare professionals about disease treatment options.

  • 222 views
  • 28 Jun, 2021
  • 1 location