Health Trials

An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB

ConditionMucopolysaccharidosis III-B
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
AgeUp to 17
SponsorJCR Pharmaceuticals Co., Ltd.

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Chronological age of <18 years

Confirmed diagnosis of MPS IIIB

Disqualifiers

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

Trial design

Design model

Single group

Treatments tested in this trial

  • JR-446

    Drug

    IV infusion

Treatment groups

10 Participants
are divided into 1 treatment group
Group A: JR-446Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

To establish the safety and tolerability of JR-446 in MPSIIIB patients

Time frame
up to 4 years (multiple visits)
2

To determine the pharmacodynamic effects of JR-446 in MPSIIIB patients

Time frame
up to 4 years (multiple visits)

Secondary outcomes

1

To assess PK profile of JR-446 in MPSIIIB patients

Time frame
up to 4 years (multiple visits)
2

To explore the potential efficacy of JR-446 in the treatment of patients with MPS IIIB

Time frame
up to 4 years (multiple visits)

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

JCR Pharmaceuticals Co., Ltd.

Lead sponsor

Source ClinicalTrials.gov
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