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

study id #: NCT04453085

condition: Mucopolysaccharidosis I

status: Recruiting

purpose:

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

intervention: JR-171

results: https://clinicaltrials.gov/ct2/show/results/NCT04453085

last updated: February 26, 2022

start date: October 28, 2021

estimated completion: April 30, 2025

last updated: November 26, 2021

phase of development: Phase 1/Phase 2

size / enrollment: 15

study 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.

primary outcomes:

• Number of participants with Adverse Events
  Adverse events
• 156 weeks
  Incidence of abnormal laboratory test results
  Hematology
• 156 weeks
  Incidence of abnormal laboratory test results
  Biochemistry
• 156 weeks
  Incidence of abnormal laboratory test results
  Serum iron tests
• 156 weeks
  Incidence of abnormal laboratory test results
  Urinalysis
• 156 weeks
  Incidence of abnormal vital signs
  Pulse rate
• 156 weeks
  Incidence of abnormal vital signs
  Body temperature
• 156 weeks
  Incidence of abnormal vital signs
  Blood pressure
• 156 weeks
  Incidence of abnormal vital signs
  Body weight
• 156 weeks
  Incidence of abnormal EKG readings
  
• 156 weeks
  Number of participants with Adverse Events
  Anti-human-α-L-iduronidase antibodies
• 156 weeks
  Number of participants with Adverse Events
  Anti-JR-171 antibodies
• 156 weeks
  Number of participants with Adverse Events
  Infusion associated reaction (IAR)
• 156 weeks
  

secondary outcomes:

• Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid
  Weeks 26, 52, 104, 156
• Change From Baseline in Dermatan Sulfate Levels in Cerebrospinal Fluid
  Weeks 26, 52, 104, 156
• CSF opening pressure
  Weeks 26, 52, 104, 156
• Change From Baseline in Heparan Sulfate Levels in Urinary
  Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
• Change From Baseline in Dermatan Sulfate Levels in Urinary
  Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
• Change From Baseline in Heparan Sulfate Levels in Serum
  Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
• Change From Baseline in Dermatan Sulfate Levels in Serum
  Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
• Change From Baseline in Liver Volume
  Weeks 13, 26, 52, 78, 104, 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
• Change From Baseline in Echocardiography.
  Weeks 13, 26, 52, 78, 104, 156
• Change From Baseline in Echocardiography.
  Weeks 13, 26, 52, 78, 104, 156
• Change From Baseline in Echocardiography.
  Weeks 13, 26, 52, 78, 104, 156
• Change From Baseline in Echocardiography.
  Weeks 13, 26, 52, 78, 104, 156
• Change From Baseline in Echocardiography.
  Weeks 13, 26, 52, 78, 104, 156
• Change From Baseline in 6-minute Walk Test Distance.
  Weeks 13, 26, 52, 78, 104, 156
• Changes from baseline in cognitive age equivalent score of neurocognitive testing
  Weeks 52, 104, 156
• Changes from baseline in outcome of adaptive behavioral function
  Weeks 52, 104, 156
• Changes from baseline in outcome of the Brief Visuospatial Memory Test-Revised
  Weeks 13, 26, 52, 78, 104, 130, 156
• Changes from baseline in outcome of the Hopkins Verbal Learning Test-Revised
  Weeks 13, 26, 52, 78, 104, 130, 156
• Changes from baseline in outcome of the Test of Variables of Attention
  Weeks 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

inclusion 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: 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

sponsor: JCR Pharmaceuticals Co., Ltd.

contacts: JCR Pharmaceuticals Co., Ltd., +81-(0)797-32-8582, kaihatsu@jcrpharm.co.jp

trial center locations: Brazil,Japan

• Brazil
  Hospital de Clínicas de Porto Alegre
  Roberto Giugliani, +5551-3359-6340
  
  
• Japan
  Fukuoka Children’s Hospital
  Ryutaro Kira, +81-92-682-7000
  
  
• Japan
  National Hospital Organization Kokura Medical Center
  Kyoko Watanabe, +81-93-921-8881
  
  
• Japan
  Osaka City University Hospital
  Takashi Hamazaki, +81-6-6645-2121