An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Recruiting
Phase 1
Phase 2
Interventional Study
Mucopolysaccharidosis III-B
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- Up to 17
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 10 (estimated)
- Sponsor
- JCR Pharmaceuticals Co., Ltd. · Industry
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
Check Your Eligibility
About This Trial
A Phase I/ II, open-label study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIB patients.
Trial Locations
Loading…
Loading trial locations…
Eligibility Criteria
Inclusion Criteria:
* Chronological age of \<18 years
* Confirmed diagnosis of MPS IIIB
Exclusion Criteria:
* 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 si…
Contacts