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A Study of JR-141 in Patients With Mucopolysaccharidosis Type II

Primary Purpose

Mucopolysaccharidosis II

Status
Completed
Phase
Phase 1
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

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

Inclusion Criteria:

  • Patients aged 6 years or older at the time of informed consent.
  • Patients diagnosed with MPS II.
  • Patients who have received idursulfase (0.5 mg/kg/week) continuously for at least 12 weeks until the initial dose of JR-141.

Exclusion Criteria:

  • Patients with a history of hematopoietic stem cell transplantation, excluding those who need enzyme replacement therapy even after hematopoietic stem cell transplantation.
  • Patients in whom lumbar puncture cannot be performed.
  • Patients who have developed serious drug allergy or hypersensitivity that is inappropriate for participation in the study.
  • Patients who have received other investigational products within 4 months before enrollment in the study.

Sites / Locations

  • Gifu Clinical site
  • Fukuoka Clinical site
  • Tokyo clinical site1
  • Osaka Clinical site1
  • Saitama Clinical site
  • Tokyo clinical site2
  • Osaka Clinical site2
  • Tottori Clinical site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Experimental: JR-141

Arm Description

Outcomes

Primary Outcome Measures

Number of participants with Adverse Events
Adverse events Laboratory tests Vital signs 12-lead electrocardiogram Antibody Infusion associated reaction

Secondary Outcome Measures

Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]]
Plasma concentration of JR-141
Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]]
Plasma concentration of JR-141
Urinary and serum heparan sulfate (HS) /dermatan sulfate (DS)
HS/DS in CSF
JR-141 concentration in CSF
Urinary total GAG
Liver and spleen volumes
Cardiac function

Full Information

First Posted
April 10, 2017
Last Updated
November 10, 2022
Sponsor
JCR Pharmaceuticals Co., Ltd.
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1. Study Identification

Unique Protocol Identification Number
NCT03128593
Brief Title
A Study of JR-141 in Patients With Mucopolysaccharidosis Type II
Official Title
A Phase I/II Study of JR-141 in Patients With Mucopolysaccharidosis Type II
Study Type
Interventional

2. Study Status

Record Verification Date
November 2022
Overall Recruitment Status
Completed
Study Start Date
March 30, 2017 (Actual)
Primary Completion Date
October 4, 2017 (Actual)
Study Completion Date
October 4, 2017 (Actual)

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
The purpose of this study in patients with mucopolysaccharidosis type II (MPS II) is below, to collect the safety information of JR-141 to evaluate the plasma pharmacokinetics of JR-141 to explore the efficacy of JR-141 on MPS II-related central nervous system symptoms and general symptoms

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 1, Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
14 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Experimental: JR-141
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
JR-141
Intervention Description
IV infusion (lyophilized powder), 0.01-2.0 mg/kg/week
Primary Outcome Measure Information:
Title
Number of participants with Adverse Events
Description
Adverse events Laboratory tests Vital signs 12-lead electrocardiogram Antibody Infusion associated reaction
Time Frame
4 weeks
Secondary Outcome Measure Information:
Title
Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]]
Description
Plasma concentration of JR-141
Time Frame
4 weeks
Title
Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]]
Description
Plasma concentration of JR-141
Time Frame
4 weeks
Title
Urinary and serum heparan sulfate (HS) /dermatan sulfate (DS)
Time Frame
4 weeks
Title
HS/DS in CSF
Description
JR-141 concentration in CSF
Time Frame
4 weeks
Title
Urinary total GAG
Time Frame
4 weeks
Title
Liver and spleen volumes
Time Frame
4 weeks
Title
Cardiac function
Time Frame
4 weeks

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients aged 6 years or older at the time of informed consent. Patients diagnosed with MPS II. Patients who have received idursulfase (0.5 mg/kg/week) continuously for at least 12 weeks until the initial dose of JR-141. Exclusion Criteria: Patients with a history of hematopoietic stem cell transplantation, excluding those who need enzyme replacement therapy even after hematopoietic stem cell transplantation. Patients in whom lumbar puncture cannot be performed. Patients who have developed serious drug allergy or hypersensitivity that is inappropriate for participation in the study. Patients who have received other investigational products within 4 months before enrollment in the study.
Facility Information:
Facility Name
Gifu Clinical site
City
Gifu
ZIP/Postal Code
501-1194
Country
Japan
Facility Name
Fukuoka Clinical site
City
Kurume
ZIP/Postal Code
830-0011
Country
Japan
Facility Name
Tokyo clinical site1
City
Minato
ZIP/Postal Code
105-8471
Country
Japan
Facility Name
Osaka Clinical site1
City
Osaka
ZIP/Postal Code
545-8586
Country
Japan
Facility Name
Saitama Clinical site
City
Saitama
ZIP/Postal Code
330-8777
Country
Japan
Facility Name
Tokyo clinical site2
City
Setagaya
ZIP/Postal Code
157-8535
Country
Japan
Facility Name
Osaka Clinical site2
City
Suita
ZIP/Postal Code
157-8535
Country
Japan
Facility Name
Tottori Clinical site
City
Yonago
ZIP/Postal Code
683-8504
Country
Japan

12. IPD Sharing Statement

Citations:
PubMed Identifier
30595526
Citation
Okuyama T, Eto Y, Sakai N, Minami K, Yamamoto T, Sonoda H, Yamaoka M, Tachibana K, Hirato T, Sato Y. Iduronate-2-Sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial. Mol Ther. 2019 Feb 6;27(2):456-464. doi: 10.1016/j.ymthe.2018.12.005. Epub 2018 Dec 8.
Results Reference
derived
Links:
URL
https://pubmed.ncbi.nlm.nih.gov/30595526/
Description
Iduronate-2-Sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial

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A Study of JR-141 in Patients With Mucopolysaccharidosis Type II

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