Status:
ACTIVE_NOT_RECRUITING
An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II
Lead Sponsor:
JCR Pharmaceuticals Co., Ltd.
Conditions:
Mucopolysaccharidosis II
Eligibility:
MALE
Phase:
PHASE2
PHASE3
Brief Summary
Multicenter, open-label, single-group, designed to evaluate the long term efficacy and safety of study drug for the treatment of the MPS II.
Eligibility Criteria
Inclusion
- A patient who participated in the preceding Study JR-141-301 and completed the Week 52 visit, and has no safety concerns to enter this study in the opinion of the investigator or subinvestigator.
- A patient capable of providing written informed consent in person (However, this is not required for patients aged younger than 20 years at the time of consent or patients with intellectual disability associated with MPS II whose willingness cannot be verified.)
- For patients aged younger than 20 years at the time of consent or patients with intellectual disability associated with MPS II whose willingness cannot be verified, written consent must be obtained from the legally acceptable representative. (Wherever possible, written consent of the patient should be obtained.)
- Male patient whose partner is of child-bearing potential and agrees to use a medically accepted, highly effective method of contraception.
Exclusion
- A patient who used any concomitant medication or therapy that could affect study assessments in the opinion of the investigator or subinvestigator.
- A patient with a history of serious drug allergy or hypersensitivity that precludes participation in this study in the opinion of the investigator or subinvestigator.
- A patient judged to be ineligible by the investigator or subinvestigator for other reasons.
Key Trial Info
Start Date :
September 1 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 31 2030
Estimated Enrollment :
27 Patients enrolled
Trial Details
Trial ID
NCT04348136
Start Date
September 1 2019
End Date
March 31 2030
Last Update
July 24 2025
Active Locations (20)
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1
Fukui Clinical site
Fukui, Japan, 910-1193
2
Fukuoka Clinical site 2
Fukuoka, Japan, 813-0017
3
Fukuoka Clinical site
Fukuoka, Japan, 830-0011
4
Gifu Clinical site
Gifu, Japan, 501-1194