Clinical trial NCT04453085: An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I)
Official title: An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Mucopolysaccharidosis I
Keywords: Mucopolysaccharidosis I
Lead sponsor: JCR Pharmaceuticals Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2020-07-01
Enrollment: 14
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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 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.

Eligibility 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

Primary outcomes:
measure: Number of participants with Adverse Events
timeFrame: 156 weeks
measure: Incidence of abnormal laboratory test results
timeFrame: 156 weeks
measure: Incidence of abnormal laboratory test results
timeFrame: 156 weeks
measure: Incidence of abnormal laboratory test results
timeFrame: 156 weeks
measure: Incidence of abnormal laboratory test results
timeFrame: 156 weeks
measure: Incidence of abnormal vital signs
timeFrame: 156 weeks
measure: Incidence of abnormal vital signs
timeFrame: 156 weeks
measure: Incidence of abnormal vital signs
timeFrame: 156 weeks
measure: Incidence of abnormal vital signs
timeFrame: 156 weeks
measure: Incidence of abnormal EKG readings
timeFrame: 156 weeks
measure: Number of participants with Adverse Events
timeFrame: 156 weeks
measure: Number of participants with Adverse Events
timeFrame: 156 weeks
measure: Number of participants with Adverse Events
timeFrame: 156 weeks
Secondary outcomes:
measure: Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid (CSF)
timeFrame: Weeks 26, 52, 104, 156
measure: Change From Baseline in Dermatan Sulfate Levels in Cerebrospinal Fluid
timeFrame: Weeks 26, 52, 104, 156
measure: CSF opening pressure
timeFrame: Weeks 26, 52, 104, 156
measure: Change From Baseline in Heparan Sulfate Levels in Urinary
timeFrame: Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
measure: Change From Baseline in Dermatan Sulfate Levels in Urinary
timeFrame: Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
measure: Change From Baseline in Heparan Sulfate Levels in Serum
timeFrame: Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
measure: Change From Baseline in Dermatan Sulfate Levels in Serum
timeFrame: Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
measure: Change From Baseline in Liver Volume
timeFrame: Weeks 13, 26, 52, 78, 104, 156
measure: Change From Baseline in Spleen Volume.
timeFrame: Weeks 13, 26, 52, 78, 104, 156
measure: Change From Baseline in Echocardiography.
timeFrame: Weeks 13, 26, 52, 78, 104, 156
measure: Change From Baseline in Echocardiography.
timeFrame: Weeks 13, 26, 52, 78, 104, 156
measure: Change From Baseline in Echocardiography.
timeFrame: Weeks 13, 26, 52, 78, 104, 156
measure: Change From Baseline in Echocardiography.
timeFrame: Weeks 13, 26, 52, 78, 104, 156
measure: Change From Baseline in Echocardiography.
timeFrame: Weeks 13, 26, 52, 78, 104, 156
measure: Change From Baseline in Echocardiography.
timeFrame: Weeks 13, 26, 52, 78, 104, 156
measure: Change From Baseline in 6-minute Walk Test Distance.
timeFrame: Weeks 13, 26, 52, 78, 104, 156
measure: Changes from baseline in cognitive age equivalent score of neurocognitive testing
timeFrame: Weeks 52, 104, 156
measure: Changes from baseline in outcome of adaptive behavioral function
timeFrame: Weeks 52, 104, 156
measure: Changes from baseline in outcome of the Brief Visuospatial Memory Test-Revised
timeFrame: Weeks 13, 26, 52, 78, 104, 130, 156
measure: Changes from baseline in outcome of the Hopkins Verbal Learning Test-Revised
timeFrame: Weeks 13, 26, 52, 78, 104, 130, 156
measure: Changes from baseline in outcome of the Test of Variables of Attention
timeFrame: Weeks 13, 26, 52, 78, 104, 130, 156
measure: Changes from baseline in outcome of the Pediatric Quality of Life Inventory Family Impact Module (PedsQL-FIM)
timeFrame: Weeks 13, 26, 52, 104 156
Other outcomes:
Locations:
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospital Oakland
state: California
city: Porto Alegre
country: Brazil
facility: Hospital de Clínicas de Porto Alegre
city: São Paulo
country: Brazil
facility: Instituto de Genética e Erros Inatos do Metabolismo - IGEIM
city: Fukuoka
country: Japan
facility: Fukuoka Children's Hospital
city: Kitakyushu
country: Japan
facility: National Hospital Organization Kokura Medical Center
city: Osaka
country: Japan
facility: Osaka Metropolitan University Hospital
Study officials:
References: