Status:
COMPLETED
An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I)
Lead Sponsor:
JCR Pharmaceuticals Co., Ltd.
Conditions:
Mucopolysaccharidosis I
Eligibility:
All Genders
Phase:
PHASE1
PHASE2
Brief Summary
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
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 ei...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
October 28 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 2 2025
Estimated Enrollment :
14 Patients enrolled
Trial Details
Trial ID
NCT04453085
Start Date
October 28 2021
End Date
May 2 2025
Last Update
August 28 2025
Active Locations (6)
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1
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
2
Hospital de Clínicas de Porto Alegre
Porto Alegre, Brazil
3
Instituto de Genética e Erros Inatos do Metabolismo - IGEIM
São Paulo, Brazil
4
Fukuoka Children's Hospital
Fukuoka, Japan