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

Primary Purpose

Mucopolysaccharidosis II

Status
Active
Phase
Phase 2
Locations
Japan
Study Type
Interventional
Intervention
JR-141
Sponsored by
JCR Pharmaceuticals Co., Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Mucopolysaccharidosis II

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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.

Sites / Locations

  • Fukui Clinical site
  • Fukuoka Clinical site 2
  • Fukuoka Clinical site
  • Gifu Clinical site
  • Hiroshima Prefectural Hospital
  • Hokkaido Clinical site
  • Kananagawa Ckinical site
  • Kumamoto Clinical site
  • Okayama Clinical site
  • Okayama Clinical site 2
  • Okinawa Clinical site
  • Osaka Clinical site 3
  • Osaka Clinical site 2
  • Osaka Clinical site
  • Saitama Clinical site
  • Shizuoka Clinical site
  • Shizuoka Clinical site 2
  • Tochigi Clinical site
  • Tokyo Clinical site
  • Tottori Clinical site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

JR-141

Arm Description

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Full Information

First Posted
December 18, 2019
Last Updated
November 30, 2022
Sponsor
JCR Pharmaceuticals Co., Ltd.
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1. Study Identification

Unique Protocol Identification Number
NCT04348136
Brief Title
An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II
Official Title
An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II
Study Type
Interventional

2. Study Status

Record Verification Date
November 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
September 1, 2019 (Actual)
Primary Completion Date
March 31, 2030 (Anticipated)
Study Completion Date
March 31, 2030 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
JCR Pharmaceuticals Co., Ltd.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucopolysaccharidosis II

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
27 (Actual)

8. Arms, Groups, and Interventions

Arm Title
JR-141
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
JR-141
Intervention Description
IV infusion, 2.0 mg/kg/week
Primary Outcome Measure Information:
Title
Occurrence of adverse events
Time Frame
From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years
Title
Occurrence of adverse reactions
Time Frame
From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years
Title
Incidence of abnormal vital signs
Description
Laboratory tests (hematology)
Time Frame
Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Title
Incidence of abnormal vital signs
Description
Laboratory tests (biochemistry)
Time Frame
Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Title
Incidence of abnormal vital signs
Description
Laboratory tests (iron-related tests)
Time Frame
Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Title
Laboratory tests (urinalysis)
Time Frame
Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Title
Vital signs (pulse rate)
Time Frame
Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Title
Vital signs (body temperature)
Time Frame
Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Title
Vital signs (blood pressure)
Time Frame
Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Title
12-lead electrocardiogram
Description
The presence or absence of abnormal findings (if present, specific findings and whether or not they are reported as adverse events)
Time Frame
Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Title
Antibody tests (anti-JR-141 antibodies)
Time Frame
Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Title
IAR
Time Frame
From the start of study (Week 52 of preceding study) up to the end of study, up to approximately 10.6 years
Title
Time course of developmental assessment data (Kyoto Scale of Psychological Development 2001) from initial dosing in the preceding study
Time Frame
Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Title
Time course of developmental assessment data (Vineland-II) from initial dosing in the preceding study
Time Frame
Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Title
Time course of developmental assessment data (Bayley-III or KABC-II) from initial dosing in the preceding study
Time Frame
Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Title
Time course of CSF substrate (HS and DS) concentrations from initial dosing in the preceding study
Time Frame
Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Title
Time course of serum HS and DS concentrations from initial dosing in the preceding study
Time Frame
Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Title
Time course of urinary HS concentration from initial dosing in the preceding study
Time Frame
Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Title
Time course of urinary DS concentration from initial dosing in the preceding study
Time Frame
Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Title
Time course of uronic acid concentration from initial dosing in the preceding study
Time Frame
Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Title
Time course of liver volume (assessed by CT or MRI) from initial dosing in the preceding study
Time Frame
Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Title
Time course of spleen volume (assessed by CT or MRI) from initial dosing in the preceding study
Time Frame
Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Title
Time course of cardiac function (assessed by echocardiography) from initial dosing in the preceding study
Time Frame
Week 78, 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Title
Time course of 6-minute walk test distance from initial dosing in the preceding study
Time Frame
Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years
Title
Time course of joint range of motion from initial dosing in the preceding study
Time Frame
Week 104, an average of 52 weeks after Week 104, up to approximately 10.6 years

10. Eligibility

Sex
Male
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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. 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.) 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.) Male patient whose partner is of child-bearing potential and agrees to use a medically accepted, highly effective method of contraception. Exclusion Criteria: A patient who used any concomitant medication or therapy that could affect study assessments in the opinion of the investigator or subinvestigator. 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. A patient judged to be ineligible by the investigator or subinvestigator for other reasons.
Facility Information:
Facility Name
Fukui Clinical site
City
Fukui
ZIP/Postal Code
910-1193
Country
Japan
Facility Name
Fukuoka Clinical site 2
City
Fukuoka
ZIP/Postal Code
813-0017
Country
Japan
Facility Name
Fukuoka Clinical site
City
Fukuoka
ZIP/Postal Code
830-0011
Country
Japan
Facility Name
Gifu Clinical site
City
Gifu
ZIP/Postal Code
501-1194
Country
Japan
Facility Name
Hiroshima Prefectural Hospital
City
Hiroshima
ZIP/Postal Code
734-8530
Country
Japan
Facility Name
Hokkaido Clinical site
City
Hokkaido
ZIP/Postal Code
063-0005
Country
Japan
Facility Name
Kananagawa Ckinical site
City
Kanagawa
ZIP/Postal Code
232-8555
Country
Japan
Facility Name
Kumamoto Clinical site
City
Kumamoto
ZIP/Postal Code
860-8556
Country
Japan
Facility Name
Okayama Clinical site
City
Okayama
ZIP/Postal Code
701-1192
Country
Japan
Facility Name
Okayama Clinical site 2
City
Okayama
ZIP/Postal Code
710-8602
Country
Japan
Facility Name
Okinawa Clinical site
City
Okinawa
ZIP/Postal Code
903-0215
Country
Japan
Facility Name
Osaka Clinical site 3
City
Osaka
ZIP/Postal Code
534-0021
Country
Japan
Facility Name
Osaka Clinical site 2
City
Osaka
ZIP/Postal Code
545-8586
Country
Japan
Facility Name
Osaka Clinical site
City
Osaka
ZIP/Postal Code
565-0871
Country
Japan
Facility Name
Saitama Clinical site
City
Saitama
ZIP/Postal Code
330-8777
Country
Japan
Facility Name
Shizuoka Clinical site
City
Shizuoka
ZIP/Postal Code
420-8660
Country
Japan
Facility Name
Shizuoka Clinical site 2
City
Shizuoka
ZIP/Postal Code
426-8677
Country
Japan
Facility Name
Tochigi Clinical site
City
Tochigi
ZIP/Postal Code
329-0498
Country
Japan
Facility Name
Tokyo Clinical site
City
Tokyo
ZIP/Postal Code
157-8535
Country
Japan
Facility Name
Tottori Clinical site
City
Tottori
ZIP/Postal Code
683-8504
Country
Japan

12. IPD Sharing Statement

Learn more about this trial

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

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