An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II

Multicenter, open-label, single-group, designed to evaluate the long term efficacy and safety of study drug for the treatment of the MPS II.

Study Overview

• Status: Active, not recruiting
• Conditions: Mucopolysaccharidosis II
• Intervention / Treatment: Drug: JR-141

• Study Type: Interventional
• Enrollment (Actual): 27
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• Japan
  • Fukui, Japan, 910-1193
    • Fukui Clinical site
  • Fukuoka, Japan, 813-0017
    • Fukuoka Clinical site 2
  • Fukuoka, Japan, 830-0011
    • Fukuoka Clinical site
  • Gifu, Japan, 501-1194
    • Gifu Clinical site
  • Hiroshima, Japan, 734-8530
    • Hiroshima Prefectural Hospital
  • Hokkaido, Japan, 063-0005
    • Hokkaido Clinical site
  • Kanagawa, Japan, 232-8555
    • Kananagawa Ckinical site
  • Kumamoto, Japan, 860-8556
    • Kumamoto Clinical site
  • Okayama, Japan, 701-1192
    • Okayama Clinical site
  • Okayama, Japan, 710-8602
    • Okayama Clinical site 2
  • Okinawa, Japan, 903-0215
    • Okinawa Clinical site
  • Osaka, Japan, 534-0021
    • Osaka Clinical site 3
  • Osaka, Japan, 545-8586
    • Osaka Clinical Site 2
  • Osaka, Japan, 565-0871
    • Osaka Clinical site
  • Saitama, Japan, 330-8777
    • Saitama Clinical site
  • Shizuoka, Japan, 420-8660
    • Shizuoka Clinical site
  • Shizuoka, Japan, 426-8677
    • Shizuoka Clinical site 2
  • Tochigi, Japan, 329-0498
    • Tochigi Clinical site
  • Tokyo, Japan, 157-8535
    • Tokyo Clinical site
  • Tottori, Japan, 683-8504
    • Tottori Clinical site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. A patient who participated in the preceding Study JR-141-301 and completed the Week 52 visit, and has no safety concerns to enter this study in the opinion of the investigator or subinvestigator.
2. A patient capable of providing written informed consent in person (However, this is not required for patients aged younger than 20 years at the time of consent or patients with intellectual disability associated with MPS II whose willingness cannot be verified.)
3. For patients aged younger than 20 years at the time of consent or patients with intellectual disability associated with MPS II whose willingness cannot be verified, written consent must be obtained from the legally acceptable representative. (Wherever possible, written consent of the patient should be obtained.)
4. Male patient whose partner is of child-bearing potential and agrees to use a medically accepted, highly effective method of contraception.

Exclusion Criteria:

1. A patient who used any concomitant medication or therapy that could affect study assessments in the opinion of the investigator or subinvestigator.
2. A patient with a history of serious drug allergy or hypersensitivity that precludes participation in this study in the opinion of the investigator or subinvestigator.
3. A patient judged to be ineligible by the investigator or subinvestigator for other reasons.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: JR-141	Drug: JR-141; IV infusion, 2.0 mg/kg/week

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Occurrence of adverse events		From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years
Occurrence of adverse reactions		From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years
Incidence of abnormal vital signs	Laboratory tests (hematology)	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Incidence of abnormal vital signs	Laboratory tests (biochemistry)	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Incidence of abnormal vital signs	Laboratory tests (iron-related tests)	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Laboratory tests (urinalysis)		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Vital signs (pulse rate)		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Vital signs (body temperature)		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Vital signs (blood pressure)		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
12-lead electrocardiogram	The presence or absence of abnormal findings (if present, specific findings and whether or not they are reported as adverse events)	Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Antibody tests (anti-JR-141 antibodies)		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
IAR		From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years
Time course of developmental assessment data (Kyoto Scale of Psychological Development 2001) from initial dosing in the preceding study		Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of developmental assessment data (Vineland-II) from initial dosing in the preceding study		Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of developmental assessment data (Bayley-III or KABC-II) from initial dosing in the preceding study		Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of CSF substrate (HS and DS) concentrations from initial dosing in the preceding study		Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of serum HS and DS concentrations from initial dosing in the preceding study		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of urinary HS concentration from initial dosing in the preceding study		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of urinary DS concentration from initial dosing in the preceding study		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of uronic acid concentration from initial dosing in the preceding study		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of liver volume (assessed by CT or MRI) from initial dosing in the preceding study		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of spleen volume (assessed by CT or MRI) from initial dosing in the preceding study		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of cardiac function (assessed by echocardiography) from initial dosing in the preceding study		Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of 6-minute walk test distance from initial dosing in the preceding study		Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Time course of joint range of motion from initial dosing in the preceding study		Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

Collaborators and Investigators

• Sponsor: JCR Pharmaceuticals Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): September 1, 2019
• Primary Completion (Estimated): March 31, 2030
• Study Completion (Estimated): March 31, 2030

Study Registration Dates

• First Submitted: December 18, 2019
• First Submitted That Met QC Criteria: April 13, 2020
• First Posted (Actual): April 16, 2020

Study Record Updates

• Last Update Posted (Actual): March 15, 2024
• Last Update Submitted That Met QC Criteria: March 13, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Connective Tissue Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Mental Retardation, X-Linked; Intellectual Disability; Heredodegenerative Disorders, Nervous System; Mucopolysaccharidosis II; Mucopolysaccharidoses
• Other Study ID Numbers: JR-141-302

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No