Filter By
Clear all Advanced
I am/have/had
added new label for I am/have/had
more
I am looking for
added new label for I am looking for
more
Advanced Filters
Found 29 clinical trials
A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II

A Global Phase III multicenter, randomized, assessor-blinded, active-controlled designed to evaluate safety and efficacy of study drug for the treatment of the MPS II.

  • 0 views
  • 25 Jul, 2022
  • 2 locations
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

  • 0 views
  • 08 Aug, 2022
  • 2 locations
Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

Phase 1, open-label, sequential ascending dose-escalation study. Designed to evaluate the safety and efficacy of a single IV infusion of investigational gene therapy HMI-203. Males, ages 18 to 45 years inclusive, with MPS II (Hunter syndrome) currently receiving idursulfase ERT (or the equivalent) are eligible to participate. Participants will be …

  • 5 views
  • 28 Jul, 2022
  • 1 location
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 …

  • 15 views
  • 12 May, 2022
  • 2 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
  • 14 May, 2022
  • 1 location
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 groups.

  • 11 views
  • 24 Mar, 2022
  • 2 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) …

  • 34 views
  • 06 May, 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.

aldurazyme
laronidase
enzyme activity assay
deficiency
  • 12 views
  • 22 Apr, 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.

  • 0 views
  • 01 Apr, 2022
  • 1 location