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

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ClinicalTrials.gov Identifier: NCT04453085
Recruitment Status : Recruiting
First Posted : July 1, 2020
Last Update Posted : May 11, 2022
Sponsor:
Information provided by (Responsible Party):
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

Condition or disease Intervention/treatment Phase
Mucopolysaccharidosis I Drug: JR-171 Phase 1 Phase 2

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. Until the dose determination in the JR-171-102 study, subjects will intravenously receive either low dose or high dose of JR-171 at the same doses received at Week 12 of the JR-171-101 study. Thereafter, all subjects will be transitioned to the optimal dose determined based on the results of JR-171-101 study.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I
Actual Study Start Date : October 28, 2021
Estimated Primary Completion Date : April 30, 2025
Estimated Study Completion Date : April 30, 2025


Arm Intervention/treatment
Experimental: JR-171
Until the dose determination, subjects will intravenously receive either the low dose or high dose of JR-171 (the same dose as at Week 12 of the JR-171-101 study). Thereafter, all subjects will receive the optimal dose of JR-171 determined based on the results of JR-171-101 study.
Drug: JR-171
JR-171 IV infusion




Primary Outcome Measures :
  1. Number of participants with Adverse Events [ Time Frame: 156 weeks ]
    Adverse events

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

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

  4. Incidence of abnormal laboratory test results [ Time Frame: 156 weeks ]
    Serum iron tests

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

  6. Incidence of abnormal vital signs [ Time Frame: 156 weeks ]
    Pulse rate

  7. Incidence of abnormal vital signs [ Time Frame: 156 weeks ]
    Body temperature

  8. Incidence of abnormal vital signs [ Time Frame: 156 weeks ]
    Blood pressure

  9. Incidence of abnormal vital signs [ Time Frame: 156 weeks ]
    Body weight

  10. Incidence of abnormal EKG readings [ Time Frame: 156 weeks ]
  11. Number of participants with Adverse Events [ Time Frame: 156 weeks ]
    Anti-human-α-L-iduronidase antibodies

  12. Number of participants with Adverse Events [ Time Frame: 156 weeks ]
    Anti-JR-171 antibodies

  13. Number of participants with Adverse Events [ Time Frame: 156 weeks ]
    Infusion associated reaction (IAR)


Secondary Outcome Measures :
  1. Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid [ Time Frame: Weeks 26, 52, 104, 156 ]
  2. Change From Baseline in Dermatan Sulfate Levels in Cerebrospinal Fluid [ Time Frame: Weeks 26, 52, 104, 156 ]
  3. CSF opening pressure [ Time Frame: Weeks 26, 52, 104, 156 ]
  4. Change From Baseline in Heparan Sulfate Levels in Urinary [ Time Frame: Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156 ]
  5. Change From Baseline in Dermatan Sulfate Levels in Urinary [ Time Frame: Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156 ]
  6. Change From Baseline in Heparan Sulfate Levels in Serum [ Time Frame: Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156 ]
  7. Change From Baseline in Dermatan Sulfate Levels in Serum [ Time Frame: Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156 ]
  8. Change From Baseline in Liver Volume [ Time Frame: Weeks 13, 26, 52, 78, 104, 156 ]
  9. Change From Baseline in Spleen Volume. [ Time Frame: Weeks 13, 26, 52, 78, 104, 156 ]
  10. Change From Baseline in Echocardiography. [ Time Frame: Weeks 13, 26, 52, 78, 104, 156 ]
    Left ventricular posterior wall thickness

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

  12. Change From Baseline in Echocardiography. [ Time Frame: Weeks 13, 26, 52, 78, 104, 156 ]
    left ventricular mass index

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

  14. Change From Baseline in Echocardiography. [ Time Frame: Weeks 13, 26, 52, 78, 104, 156 ]
    left ventricular ejection fraction,

  15. Change From Baseline in Echocardiography. [ Time Frame: Weeks 13, 26, 52, 78, 104, 156 ]
    E/A ratio

  16. Change From Baseline in 6-minute Walk Test Distance. [ Time Frame: Weeks 13, 26, 52, 78, 104, 156 ]
  17. Changes from baseline in cognitive age equivalent score of neurocognitive testing [ Time Frame: Weeks 52, 104, 156 ]
    Wechsler IQ test or the Bayley scales of infant and toddler development

  18. Changes from baseline in outcome of adaptive behavioral function [ Time Frame: Weeks 52, 104, 156 ]
    Vineland adaptive behavior scales

  19. Changes from baseline in outcome of the Brief Visuospatial Memory Test-Revised [ Time Frame: Weeks 13, 26, 52, 78, 104, 130, 156 ]
  20. Changes from baseline in outcome of the Hopkins Verbal Learning Test-Revised [ Time Frame: Weeks 13, 26, 52, 78, 104, 130, 156 ]
  21. Changes from baseline in outcome of the Test of Variables of Attention [ Time Frame: Weeks 13, 26, 52, 78, 104, 130, 156 ]
  22. 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 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04453085


Contacts
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Contact: JCR Pharmaceuticals Co., Ltd. +81-(0)797-32-8582 clinical_development@jp.jcrpharm.com

Locations
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United States, California
UCSF Benioff Children's Hospital Oakland Recruiting
Oakland, California, United States, 94609
Contact: Paul Harmatz         
Contact    +1-510-428-3058      
Brazil
Hospital de Clínicas de Porto Alegre Recruiting
Porto Alegre, Brazil
Contact: Roberto Giugliani    +5551-3359-6340      
Japan
Fukuoka Children's Hospital Recruiting
Fukuoka, Japan
Contact: Ryutaro Kira    +81-92-682-7000      
National Hospital Organization Kokura Medical Center Recruiting
Kitakyushu, Japan
Contact: Kyoko Watanabe    +81-93-921-8881      
Osaka City University Hospital Recruiting
Osaka, Japan
Contact: Takashi Hamazaki    +81-6-6645-2121      
Sponsors and Collaborators
JCR Pharmaceuticals Co., Ltd.
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Responsible Party: JCR Pharmaceuticals Co., Ltd.
ClinicalTrials.gov Identifier: NCT04453085    
Other Study ID Numbers: JR-171-102
First Posted: July 1, 2020    Key Record Dates
Last Update Posted: May 11, 2022
Last Verified: May 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by JCR Pharmaceuticals Co., Ltd.:
Mucopolysaccharidosis I
Additional relevant MeSH terms:
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Mucopolysaccharidoses
Mucopolysaccharidosis I
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases