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

Phase 1
Active, not recruiting
Conditions
Mucopolysaccharidosis I
Interventions
Drug: JR-171 (lepunafusp alfa)
Registration Number
NCT04453085
Lead Sponsor
JCR Pharmaceuticals Co., Ltd.
Brief Summary

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

Detailed Description

Patients who have completed the Part2 of JR-171-101 study 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.

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
14
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.
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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
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Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Dose level 1JR-171 (lepunafusp alfa)2.0 mg/kg/week
Dose level 2JR-171 (lepunafusp alfa)4.0 mg/kg/week
Primary Outcome Measures
NameTimeMethod
Incidence of abnormal EKG readings156 weeks
Incidence of abnormal laboratory test results156 weeks

Urinalysis

Number of participants with Adverse Events156 weeks

Infusion associated reaction (IAR)

Incidence of abnormal vital signs156 weeks

Body weight

Secondary Outcome Measures
NameTimeMethod
Change From Baseline in Dermatan Sulfate Levels in Cerebrospinal FluidWeeks 26, 52, 104, 156
Change From Baseline in Liver VolumeWeeks 13, 26, 52, 78, 104, 156
Changes from baseline in outcome of adaptive behavioral functionWeeks 52, 104, 156

Vineland adaptive behavior scales

Change From Baseline in Dermatan Sulfate Levels in UrinaryWeeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Change From Baseline in 6-minute Walk Test Distance.Weeks 13, 26, 52, 78, 104, 156
Changes from baseline in outcome of the Hopkins Verbal Learning Test-RevisedWeeks 13, 26, 52, 78, 104, 130, 156
Changes from baseline in outcome of the Test of Variables of AttentionWeeks 13, 26, 52, 78, 104, 130, 156
Changes from baseline in outcome of the Pediatric Quality of Life Inventory Family Impact Module (PedsQL-FIM)Weeks 13, 26, 52, 104 156
Change From Baseline in Heparan Sulfate Levels in Cerebrospinal FluidWeeks 26, 52, 104, 156
Change From Baseline in Heparan Sulfate Levels in UrinaryWeeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Change From Baseline in Heparan Sulfate Levels in SerumWeeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Changes from baseline in outcome of the Brief Visuospatial Memory Test-RevisedWeeks 13, 26, 52, 78, 104, 130, 156
CSF opening pressureWeeks 26, 52, 104, 156
Change From Baseline in Dermatan Sulfate Levels in SerumWeeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Change From Baseline in Spleen Volume.Weeks 13, 26, 52, 78, 104, 156
Change From Baseline in Echocardiography.Weeks 13, 26, 52, 78, 104, 156

E/A ratio

Changes from baseline in cognitive age equivalent score of neurocognitive testingWeeks 52, 104, 156

Wechsler IQ test or the Bayley scales of infant and toddler development

Trial Locations

Locations (6)

National Hospital Organization Kokura Medical Center

🇯🇵

Kitakyushu, Japan

Osaka Metropolitan University Hospital

🇯🇵

Osaka, Japan

UCSF Benioff Children's Hospital Oakland

🇺🇸

Oakland, California, United States

Hospital de Clínicas de Porto Alegre

🇧🇷

Porto Alegre, Brazil

Fukuoka Children's Hospital

🇯🇵

Fukuoka, Japan

Instituto de Genética e Erros Inatos do Metabolismo - IGEIM

🇧🇷

São Paulo, Brazil

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