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

Phase 1

• Conditions: Mucopolysaccharidosis I

• Registration Number: JPRN-jRCT2051210040
• Lead Sponsor: Kawashima Satoshi

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-06-16
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 14

Inclusion Criteria

1. Patients who have completed the Part 2 of JR-171-101 study.
2. A patient from whom written informed consent can be obtained. If the patient is aged under 20 years 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) needs to be obtained instead of his/her consent. Even in this case, written informed consent should be obtained from the patient, wherever possible.
3. Female patient or male patient whose co-partners is of child-bearing potential agree to use a medically accepted.

Exclusion Criteria

1. 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.
2. A patient who is 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.
3. A patient who is 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.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
- Frequency and severity of adverse events (AEs) and their relationship to JR-171<br>- Changes in laboratory parameters (hematology, biochemistry, serum iron tests and urinalysis)<br>- Changes in vital signs (pulse rate, body temperature, blood pressure, respiratory rate and percutaneous oxygen saturation)<br>- Clinically meaningful changes in 12-lead electrocardiogram<br>- Anti-drug antibody production (anti-human alpha-L-iduronidase (anti-human IDUA) and anti-JR-171 antibodies)<br>- Frequency of Infusion associated reactions (IARs)