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A Study of JR-171 in Patients With Mucopolysaccharidosis I | Clinical Trials | Clareo Health

COMPLETEDPHASE1/PHASE2

A Study of JR-171 in Patients With Mucopolysaccharidosis I

Phase I/II Study of JR-171 ㏌ Patients With Mucopolysaccharidosis Type I

NCT04227600•JCR Pharmaceuticals Co., Ltd.

Summary

Phase I/II, open-label, multicenter, multinational (Japan, Brazil and US),designed to evaluate the safety, pharmacokinetics and explore the efficacy for the treatment of mucopolysaccharidosis type I (MPS I).

Detailed Description

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 dosages received at Week 12 of the JR-171-101 study.

Eligibility

Age

0 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•A patient aged 18 years or older in Part 1 or any age in Part 2, at the time of informed consent
•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 or assent should be obtained from the patient, wherever possible
•A patient diagnosed with MPS I based on any one of the following criteria:
•Activity of IDUA enzyme below 10% of lower reference level in leucocytes or cultured skin fibroblasts, AND increased age-related urinary levels of GAGs (before enzyme replacement therapy)
•Activity of IDUA enzyme below 10% of lower reference level in leucocytes or cultured skin fibroblasts, AND presence of one pathogenic mutation in each of the alleles of the IDUA gene
•Increased age-related urinary levels of GAGs (before enzyme replacement therapy), AND presence of one pathogenic mutation in each of the alleles of the IDUA gene
•A patient diagnosed as having no or mild MPS I-related intellectual disability (able to report their own subjective symptoms) by the principal investigator or subinvestigator (Part 1 only)
•A patient who has received laronidase continuously for at least 12 weeks and has received laronidase on a stable dosage for 2 weeks immediately before the initial administration of JR-171, except for a laronidase naïve patient or a patient who has previously been treated by HSCT)
•Female patient or male patient whose co-partner is of child-bearing potential agrees 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 received gene therapy treatment
•A patient who, in the opinion of the principal investigator or subinvestigator, cannot undergo lumbar puncture, including those who have a difficulty in taking a position for lumbar puncture due to joint contracture and those who are likely to develop dyspnea during lumbar puncture
•A patient who is pregnant or lactating
•A patient who has developed serious drug allergy or hypersensitivity to any drugs, in the opinion of the principal investigator or subinvestigator, is inappropriate for participation in the study
•A patient who has received another investigational product within 12 months before enrollment in the study
•A patient who, in the opinion of the principal investigator or subinvestigator, is ineligible to participate in the study out of consideration for the participant 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

Kochi Medical School Hospital

Nankoku, Japan

Osaka Metropolitan University Hospital

Osaka, Japan

Key Dates

Start Date

September 1, 2020

Primary Completion Date

August 2, 2022

Completion Date

August 2, 2022

Last Updated

March 19, 2025

Enrollment

ACTUAL participants

Conditions

Mucopolysaccharidosis I

Interventions

JR-171 (lepunafusp alfa)

DRUG

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ACTIVE_NOT_RECRUITINGPHASE1/PHASE2

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

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Mucopolysaccharidosis IH

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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: March 19, 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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A Study of JR-171 in Patients With Mucopolysaccharidosis I

Inclusion Criteria

Exclusion Criteria

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

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

A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ

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