This study is accepting new patients by invitation only

An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects

a study on Hunter Syndrome Mucopolysaccharidosis

Summary

Location
at UCSF
Dates
study started
study ends around

Description

Summary

An extension of Global Phase III open-label, multicenter designed to evaluate the Long-term safety and efficacy of study drug for the treatment of the MPS II.

Official Title

An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis Type II (Hunter Syndrome) Subjects

Keywords

Mucopolysaccharidosis II, JR-141 2.0 mg/kg/week

Eligibility

You can join if…

  1. A subject who participated in the Parent Study (JR-141-GS31) and completed the assessments at Week 105 in Cohort A or Week 53 in Cohort B before being administered the study medication of that respective visit, and in the opinion of the principal investigator there are no safety concerns.
  2. A subject from whom an IRB or IEC-approved written informed consent can be obtained, which is voluntarily signed. If the subject is aged under 18 years (aged under 16 years in the UK) at the time of enrollment or willingness to participate in the study cannot be confirmed due to MPS II-related intellectual disability, the subject's legally acceptable representative (e.g., his parents or guardians) may sign the ICF on behalf of the subject. Written informed assent should be obtained from the subject, wherever possible.
  3. Female subject of child bearing potential or male subject whose female partner is of child-bearing potential, i.e., fertile, following menarche and until becoming post-menopausal unless permanently sterile, agrees to use a medically accepted, highly effective method of contraception from the time of signing the ICF. The method of contraception must be used during the study until 90 days for male subjects, and 30 days for female subjects after the final study drug administration.

You CAN'T join if...

  1. A subject who changed treatment from JR-141 to idursulfase during the treatment period in the Parent Study (JR-141-GS31).
  2. A subject who is unable to comply with the protocol (e.g., is unable to return for safety evaluations or is otherwise unlikely to complete the study) as determined by the principal investigator or sub-investigator.
  3. [Only in France] Persons deprived of their liberty by a judicial or administrative decision, according to article L. 1121-6 of the Public Health Code (Code de la santé publique, CSP) adults who are the subject of a measure of legal protection or unable to express their consent according to article L. 1121-8 of the CSP.

Locations

  • UCSF Benioff Children's Hospital Oakland
    Oakland 5378538 California 5332921 94609 United States
  • Children's Hospital of Philadelphia
    Philadelphia 4560349 Pennsylvania 6254927 19104 United States

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
JCR Pharmaceuticals Co., Ltd.
ID
NCT05594992
Phase
Phase 3 research study
Study Type
Interventional
Participants
Expecting 80 study participants
Last Updated