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Found 15 clinical trials
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
  • 03 Dec, 2021
  • 4 locations
China Post-marketing Surveillance (PMS) Study of Aldurazyme®

This is a single treatment arm study that is open-label to be conducted in Chinese participants with MPS I. Trial Objectives are to evaluate the safety and tolerability of Aldurazyme in Chinese MPS I participants, and to evaluate the efficacy of Aldurazyme on the percent change of urinary glycosaminoglycans (uGAGs) …

  • 0 views
  • 12 May, 2022
  • 1 location
China Post-marketing Surveillance (PMS) Study of Aldurazyme®

This is a single treatment arm study that is open-label to be conducted in Chinese participants with MPS I. Trial Objectives are to evaluate the safety and tolerability of Aldurazyme in Chinese MPS I participants, and to evaluate the efficacy of Aldurazyme on the percent change of urinary glycosaminoglycans (uGAGs) …

  • 0 views
  • 06 May, 2022
  • 1 location
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 α-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
  • 21 Mar, 2022
  • 3 locations
A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants

The purpose of this study is to determine if laronidase is present in the breast milk of post-partum women receiving Aldurazyme® (laronidase) and the effects of Aldurazyme (laronidase) on the growth, development, and immunologic response of their breastfed infants.

  • 0 views
  • 01 Apr, 2022
  • 1 location
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.

adalimumab injection [humira]
mucopolysaccharidosis type i
humira
bodily pain
mucopolysaccharidosis
  • 228 views
  • 30 Jan, 2022
  • 1 location
A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants

The purpose of this study is to determine if laronidase is present in the breast milk of post-partum women receiving Aldurazyme® (laronidase) and the effects of Aldurazyme (laronidase) on the growth, development, and immunologic response of their breastfed infants.

laronidase
deficiency
aldurazyme
enzyme activity assay
  • 12 views
  • 22 Apr, 2022
  • 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
  • 11 views
  • 15 May, 2022
  • 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).

hurler syndrome
regimen b
encephalopathy
cald
peroxisomal disorder
  • 267 views
  • 12 Nov, 2021
  • 1 location
Mucopolysaccharidosis I (MPS I) Registry

The Mucopolysaccharidosis I (MPS I) Registry is an ongoing, observational database that tracks the outcomes of patients with MPS I. The data collected by the MPS I Registry will provide information to better characterize the natural history and progression of MPS I as well as the clinical responses of patients …

  • 398 views
  • 23 Mar, 2022
  • 169 locations