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

Part of paid clinical trials in Oakland, California.

Sponsor
JCR Pharmaceuticals Co., Ltd.
Study ID
NCT05594992
Phase
PHASE3
Status
Enrolling By Invitation

Conditions

  • Mucopolysaccharidosis II

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • JR-141 — DRUG
    IV infusion, 2.0 mg/kg/week

Study Details

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.

Key Dates

Start date
Feb 22, 2023
Status verified
Jan 2026
Primary completion
Feb 28, 2028
Completion
Feb 28, 2028

Study Design

Enrollment
80 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: JR-141 2.0 mg/kg/week

Primary Outcome Measure

Change in heparan sulfate concentrations in cerebrospinal fluid from each subject's first administration of JR-141 throughout the Parent Study (JR-141-GS31) or this study to each time point [ Time Frame: [A-1, A-2, A-3: Week 157, 209, 261] [A-4: Week 131, 183, 235, 287] ]

Locations (2)

FacilityCityStateZIPSite coordinators
UCSF Benioff Children's Hospital OaklandOaklandCalifornia94609-
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-

Find similar trials in Oakland, CA

By research site
UCSF Benioff Children's Hospital Oakland· Oakland, CAChildren's Hospital of Philadelphia· Philadelphia, PA

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