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

Completed · Phase 1/Phase 2

Conditions studied: Mucopolysaccharidosis I

In brief

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

Key facts

Study ID: NCT04453085
Run by: JCR Pharmaceuticals Co., Ltd.
People needed: 14
Starts: 2021-10-28
Expected to finish: 2025-05-02
Last updated by the study team: 2025-08-28

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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.

You may not qualify if…

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

Where it is running

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

Full record on ClinicalTrials.gov

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