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

• Register: ClinicalTrials.gov
• Last refreshed on: 26 August 2024
• Main ID: NCT04453085
• Date of registration: 04/06/2020
• Prospective Registration: Yes
• Primary sponsor: JCR Pharmaceuticals Co., Ltd.
• Public title: An Extension Study of JR-171-101 Study in Patients With MPS I
• Scientific title: An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I
• Date of first enrolment: October 28, 2021
• Target sample size: 14
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT04453085
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

Brazil, Japan, United States

Key inclusion & exclusion criteria

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


Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Mucopolysaccharidosis I

Intervention(s)

Drug: JR-171 (lepunafusp alfa)

Primary Outcome(s)

Incidence of abnormal EKG readings [Time Frame: 156 weeks]

Incidence of abnormal vital signs [Time Frame: 156 weeks]

Number of participants with Adverse Events [Time Frame: 156 weeks]

Incidence of abnormal laboratory test results [Time Frame: 156 weeks]

Secondary Outcome(s)

Change From Baseline in Heparan Sulfate Levels in Urinary [Time Frame: Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156]

Changes from baseline in cognitive age equivalent score of neurocognitive testing [Time Frame: Weeks 52, 104, 156]

Changes from baseline in outcome of adaptive behavioral function [Time Frame: Weeks 52, 104, 156]

Change From Baseline in Dermatan Sulfate Levels in Urinary [Time Frame: 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]

Change From Baseline in Dermatan Sulfate Levels in Cerebrospinal Fluid [Time Frame: Weeks 26, 52, 104, 156]

Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid [Time Frame: Weeks 26, 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]

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]

Change From Baseline in Liver Volume [Time Frame: Weeks 13, 26, 52, 78, 104, 156]

Changes from baseline in outcome of the Test of Variables of Attention [Time Frame: Weeks 13, 26, 52, 78, 104, 130, 156]

Change From Baseline in Echocardiography. [Time Frame: Weeks 13, 26, 52, 78, 104, 156]

Changes from baseline in outcome of the Hopkins Verbal Learning Test-Revised [Time Frame: Weeks 13, 26, 52, 78, 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]

Change From Baseline in Spleen Volume. [Time Frame: 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]

CSF opening pressure [Time Frame: Weeks 26, 52, 104, 156]

Secondary ID(s)

JR-171-102

Source(s) of Monetary Support

Please refer to primary and secondary sponsors