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An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I) | Clinical Trials | Clareo Health

COMPLETEDPHASE1/PHASE2

An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I)

An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I

NCT04453085•JCR Pharmaceuticals Co., Ltd.

View on ClinicalTrials.gov

Summary

Phase I/II, open label, multicenter, multinational (Japan, Brazil and the US) extension study of JR-171-101 (NCT04227600) for the treatment of MPS I

Detailed Description

Patients who have completed the Part2 of JR-171-101 study (NCT04227600) and fulfill all eligibility criteria can be enrolled in this JR-171-102 study. In the JR-171-102 study, subjects will receive either 2.0 or 4.0 mg/kg/week of JR-171 intravenously at the same doses received at Week 12 of the JR-171-101 study.

Eligibility

Age

All ages

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Patients who have completed the Part 2 of JR-171-101 study
•A patient from whom written informed consent can be obtained. If the patient is aged under 18 years (20 years in case of Japan) at the time of assent or willingness to participate in the study cannot be confirmed due to MPS I-related intellectual disability, informed permission from the patient's legally acceptable representative (e.g., his/her parents or guardians) need to be obtained instead of his/her consent. Even in this case, written informed consent should be obtained from the patient, wherever possible
•Female patient or male patient whose co-partners is of child-bearing potential agree to use a medically accepted, highly effective method of contraception, such as spermatocidal gel plus condom, an intrauterine device or oral contraceptives until one month after the final administration.

Exclusion Criteria

•A patient who is unable to perform the study procedures, except for 6-minute walk test, neurocognitive testing, BVMT-R, HVLT-R, and T.O.V.A.
•Judged by the principal investigator or subinvestigator as being unable to undergo lumbar puncture, including those who have difficulties in taking a position for lumbar puncture due to joint contracture or those who are likely to experience difficulty breathing during the lumbar puncture process
•Judged by the principal investigator or subinvestigator to be ineligible to participate in the study due to a history of a serious drug allergy or hypersensitivity to any drugs
•Otherwise judged by the principal investigator or subinvestigator to be ineligible to participate in the study in consideration of patient's safety

Locations (6)

UCSF Benioff Children's Hospital Oakland

Oakland, California, United States

Hospital de Clínicas de Porto Alegre

Porto Alegre, Brazil

Instituto de Genética e Erros Inatos do Metabolismo - IGEIM

São Paulo, Brazil

Fukuoka Children's Hospital

Fukuoka, Japan

National Hospital Organization Kokura Medical Center

Kitakyushu, Japan

Osaka Metropolitan University Hospital

Osaka, Japan

Key Dates

Start Date

October 28, 2021

Primary Completion Date

May 2, 2025

Completion Date

May 2, 2025

Last Updated

August 28, 2025

Enrollment

ACTUAL participants

Conditions

Mucopolysaccharidosis I

Interventions

JR-171

DRUG

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: August 28, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I)

Inclusion Criteria

Exclusion Criteria

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Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant

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A Study of JR-171 in Patients With Mucopolysaccharidosis I