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

August 26, 2025 updated by: JCR Pharmaceuticals Co., Ltd.

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

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

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
14

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Brazil
      • Porto Alegre, Brazil
        • Hospital de Clinicas de Porto Alegre
      • São Paulo, Brazil
        • Instituto de Genética e Erros Inatos do Metabolismo - IGEIM
  • Japan
      • Fukuoka, Japan
        • Fukuoka Children's Hospital
      • Kitakyushu, Japan
        • National Hospital Organization Kokura Medical Center
      • Osaka, Japan
        • Osaka Metropolitan University Hospital
  • United States
    • California
      • Oakland, California, United States, 94609
        • UCSF Benioff Children's Hospital Oakland

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Dose level 1
2.0 mg/kg/week
Drug: JR-171
Intravenous (IV) infusion
Other Names:
  • lepunafusp alfa
Experimental: Dose level 2
4.0 mg/kg/week
Drug: JR-171
Intravenous (IV) infusion
Other Names:
  • lepunafusp alfa

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of participants with Adverse Events
Time Frame: 156 weeks
Adverse events
156 weeks
Incidence of abnormal laboratory test results
Time Frame: 156 weeks
Hematology
156 weeks
Incidence of abnormal laboratory test results
Time Frame: 156 weeks
Biochemistry
156 weeks
Incidence of abnormal laboratory test results
Time Frame: 156 weeks
Serum iron tests
156 weeks
Incidence of abnormal laboratory test results
Time Frame: 156 weeks
Urinalysis
156 weeks
Incidence of abnormal vital signs
Time Frame: 156 weeks
Pulse rate
156 weeks
Incidence of abnormal vital signs
Time Frame: 156 weeks
Body temperature
156 weeks
Incidence of abnormal vital signs
Time Frame: 156 weeks
Blood pressure
156 weeks
Incidence of abnormal vital signs
Time Frame: 156 weeks
Body weight
156 weeks
Incidence of abnormal EKG readings
Time Frame: 156 weeks
156 weeks
Number of participants with Adverse Events
Time Frame: 156 weeks
Anti-human-α-L-iduronidase antibodies
156 weeks
Number of participants with Adverse Events
Time Frame: 156 weeks
Anti-JR-171 antibodies
156 weeks
Number of participants with Adverse Events
Time Frame: 156 weeks
Infusion associated reaction (IAR)
156 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change From Baseline in Dermatan Sulfate Levels in Cerebrospinal Fluid
Time Frame: Weeks 26, 52, 104, 156
Weeks 26, 52, 104, 156
CSF opening pressure
Time Frame: Weeks 26, 52, 104, 156
Weeks 26, 52, 104, 156
Change From Baseline in Heparan Sulfate Levels in Urinary
Time Frame: Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Change From Baseline in Dermatan Sulfate Levels in Urinary
Time Frame: Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Change From Baseline in Heparan Sulfate Levels in Serum
Time Frame: Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Change From Baseline in Dermatan Sulfate Levels in Serum
Time Frame: Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Change From Baseline in Liver Volume
Time Frame: Weeks 13, 26, 52, 78, 104, 156
Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Spleen Volume.
Time Frame: Weeks 13, 26, 52, 78, 104, 156
Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Echocardiography.
Time Frame: Weeks 13, 26, 52, 78, 104, 156
Left ventricular posterior wall thickness
Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Echocardiography.
Time Frame: Weeks 13, 26, 52, 78, 104, 156
interventricular septal thickness
Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Echocardiography.
Time Frame: Weeks 13, 26, 52, 78, 104, 156
left ventricular mass index
Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Echocardiography.
Time Frame: Weeks 13, 26, 52, 78, 104, 156
left ventricular ejection fraction
Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Echocardiography.
Time Frame: Weeks 13, 26, 52, 78, 104, 156
left ventricular ejection fraction,
Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Echocardiography.
Time Frame: Weeks 13, 26, 52, 78, 104, 156
E/A ratio
Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in 6-minute Walk Test Distance.
Time Frame: Weeks 13, 26, 52, 78, 104, 156
Weeks 13, 26, 52, 78, 104, 156
Changes from baseline in outcome of adaptive behavioral function
Time Frame: Weeks 52, 104, 156
Vineland adaptive behavior scales
Weeks 52, 104, 156
Changes from baseline in outcome of the Brief Visuospatial Memory Test-Revised
Time Frame: Weeks 13, 26, 52, 78, 104, 130, 156
Weeks 13, 26, 52, 78, 104, 130, 156
Changes from baseline in outcome of the Hopkins Verbal Learning Test-Revised
Time Frame: Weeks 13, 26, 52, 78, 104, 130, 156
Weeks 13, 26, 52, 78, 104, 130, 156
Changes from baseline in outcome of the Test of Variables of Attention
Time Frame: Weeks 13, 26, 52, 78, 104, 130, 156
Weeks 13, 26, 52, 78, 104, 130, 156
Changes from baseline in outcome of the Pediatric Quality of Life Inventory Family Impact Module (PedsQL-FIM)
Time Frame: Weeks 13, 26, 52, 104 156
Weeks 13, 26, 52, 104 156
Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid (CSF)
Time Frame: Weeks 26, 52, 104, 156
Weeks 26, 52, 104, 156
Changes from baseline in cognitive age equivalent score of neurocognitive testing
Time Frame: Weeks 52, 104, 156
Wechsler Intelligence Quotient (IQ) test or the Bayley scales of infant and toddler development
Weeks 52, 104, 156

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
JCR Pharmaceuticals Co., Ltd.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 28, 2021

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
May 2, 2025

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
May 2, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 4, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 26, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 1, 2020

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 28, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 26, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • JR-171-102

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Mucopolysaccharidosis I

Clinical Trials on JR-171

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings