Status:
ACTIVE_NOT_RECRUITING
Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA
Lead Sponsor:
JCR Pharmaceuticals Co., Ltd.
Conditions:
Mucopolysaccharidosis III-A
Eligibility:
All Genders
1-18 years
Phase:
PHASE1
PHASE2
Brief Summary
A Phase I/ II, open-label, randomized, 2-arm study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIA patients.
Eligibility Criteria
Inclusion
- Chronological age of ≥1 year and ≤18 years.
- Confirmed diagnosis of MPS IIIA.
- Body weight ≥ 10 kg.
Exclusion
- Prior experience to gene therapy or HSCT with successful engraftment.
- Past use of another investigational drug or product in last 4 months or 5 half-lives (whichever is longer) before signing ICF.
- Current participation in a clinical trial or past participation (within 30 days of enrolment into this study) in a study involving invasive procedures.
- Past use of Genistein or Kineret (anakinra) within 4 months before signing ICF.
- Serious drug allergy or hypersensitivity.
- Contraindication for lumbar puncture or MRI.
- History of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following lumbar puncture.
- The above information is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial.
Key Trial Info
Start Date :
October 4 2023
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 31 2029
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT06095388
Start Date
October 4 2023
End Date
October 31 2029
Last Update
August 26 2025
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
Universitätsklinikum Hamburg-Eppendorf
Hamburg, Germany