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Found 21 clinical trials
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Gene Therapy in Patients With Mucopolysaccharidosis Disease

This study investigated the safety and efficacy of gene therapy approaches for Mucopolysaccharidosis type VI disease caused by the deficiency of arylsulfatase B (ARSB) enzyme. The aim of the

  • 3 views
  • 14 Oct, 2021
  • 3 locations
None
A Study of JR-171 in Patients With Mucopolysaccharidosis I

Phase I/II, open-label, multicenter, multinational (Japan, Brazil and US),designed to evaluate the safety, pharmacokinetics and explore the efficacy for the treatment of mucopolysaccharidosis

  • 0 views
  • 27 Sep, 2021
  • 5 locations
None
An Extension Study of JR-171-101 Study in Patients With MPS I

Phase I/II, open label, multicenter, multinational (Japan, Brazil and the US) extension study of JR-171-101 for the treatment of MPS I

  • 0 views
  • 22 Oct, 2021
  • 3 locations
None
RGX-111 Gene Therapy in Patients With MPS I

RGX-111 is a gene therapy which is intended to deliver a functional copy of the -L-iduronidase (IDUA) gene to the central nervous system. This is a safety and dose ranging study to determine whether RGX-111 is safe and tolerated by patients with MPS I.

  • 53 views
  • 15 Jul, 2021
  • 4 locations
None
Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI

Mucopolysaccharidoses (MPS) are multisystemic diseases with significant clinical overlap between their types, with cardiac problems being among the most commonly observed manifestations and are also among the main causes of mortality in these patients. For some of the cardiovascular manifestations, such as aortic root dilation and valve diseases, there is …

  • 0 views
  • 26 Jan, 2021
  • 1 location
None
Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I and II

baseline in joint and skeletal disease in children and adults with mucopolysaccharidosis (MPS) I or II.

mucopolysaccharidosis
adalimumab injection [humira]
bodily pain
humira
mucopolysaccharidosis type i
  • 210 views
  • 17 Feb, 2021
  • 1 location
None
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
mucopolysaccharidosis
iduronate 2-sulfatase
  • 35 views
  • 12 Sep, 2021
  • 4 locations
None
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
None
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).

mucopolysaccharidosis
peroxisomal disorder
fludarabine
preparative regimen
leukodystrophy
  • 137 views
  • 30 May, 2021
  • 1 location
None
Ophthalmic Manifestations of Patients With Mucopolysaccharidosis

Mucopolysaccharidosis (MPSs) are a group of disorders caused by inherited defects in lysosomal enzymes resulting in widespread intra- and extra-cellular accumulation of glycosaminoglycan(1,2

  • 0 views
  • 06 Mar, 2021
  • 1 location