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 9 clinical trials
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

  • 2 views
  • 19 Sep, 2023
  • 28 locations
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)

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 …

  • 23 views
  • 23 Oct, 2022
  • 2 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). Participants, whose physicians feel they

iduronate 2-sulfatase
enzyme replacement therapy
mucopolysaccharidosis
  • 255 views
  • 12 Sep, 2023
  • 7 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 …

  • 0 views
  • 16 Sep, 2023
  • 1 location
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)

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.

  • 461 views
  • 23 Oct, 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

  • 11 views
  • 24 Mar, 2022
  • 2 locations
In Utero Enzyme Replacement Therapy (ERT) for Prenatally Diagnosed Lysosomal Storage Disorders (LSDs). (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
  • 11 views
  • 17 Oct, 2022
  • 1 location
A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

This is a four-part prospective, multicenter, multiregional observational study of patients with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, to assess biomarkers

mucopolysaccharidosis
anesthesia
general anesthesia
ids gene
  • 304 views
  • 28 Apr, 2022
  • 7 locations
Longitudinal Study of Neurodegenerative Disorders

The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.

cell transplantation
neurodegenerative disorders
hematopoetic
genetic disorder
genetic disorders
  • 363 views
  • 13 Nov, 2021
  • 1 location