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

Sponsor
JCR Pharmaceuticals Co., Ltd. (Industry)
Overall Status
Completed
CT.gov ID
NCT03128593
Collaborator
(none)
14
Enrollment
8
Locations
1
Arm
6.2
Actual Duration (Months)
1.8
Patients Per Site
0.3
Patients Per Site Per Month

Study Details

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

Condition or Disease Intervention/Treatment Phase
Phase 1/Phase 2

Study Design

Study Type:
Interventional
Actual Enrollment :
14 participants
Allocation:
N/A
Intervention Model:
Sequential Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase I/II Study of JR-141 in Patients With Mucopolysaccharidosis Type II
Actual Study Start Date :
Mar 30, 2017
Actual Primary Completion Date :
Oct 4, 2017
Actual Study Completion Date :
Oct 4, 2017

Arms and Interventions

Arm Intervention/Treatment
Experimental: Experimental: JR-141

Drug: JR-141
IV infusion (lyophilized powder), 0.01-2.0 mg/kg/week

Outcome Measures

Primary Outcome Measures

  1. Number of participants with Adverse Events [4 weeks]

    Adverse events Laboratory tests Vital signs 12-lead electrocardiogram Antibody Infusion associated reaction

Secondary Outcome Measures

  1. Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]] [4 weeks]

    Plasma concentration of JR-141

  2. Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]] [4 weeks]

    Plasma concentration of JR-141

  3. Urinary and serum heparan sulfate (HS) /dermatan sulfate (DS) [4 weeks]

  4. HS/DS in CSF [4 weeks]

    JR-141 concentration in CSF

  5. Urinary total GAG [4 weeks]

  6. Liver and spleen volumes [4 weeks]

  7. Cardiac function [4 weeks]

Eligibility Criteria

Criteria

Ages Eligible for Study:
6 Years and Older
Sexes Eligible for Study:
Male
Accepts Healthy Volunteers:
No
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.

Contacts and Locations

Locations

Site City State Country Postal Code
1 Gifu Clinical site Gifu Japan 501-1194
2 Fukuoka Clinical site Kurume Japan 830-0011
3 Tokyo clinical site1 Minato Japan 105-8471
4 Osaka Clinical site1 Osaka Japan 545-8586
5 Saitama Clinical site Saitama Japan 330-8777
6 Tokyo clinical site2 Setagaya Japan 157-8535
7 Osaka Clinical site2 Suita Japan 157-8535
8 Tottori Clinical site Yonago Japan 683-8504

Sponsors and Collaborators

  • JCR Pharmaceuticals Co., Ltd.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

Publications

None provided.
Responsible Party:
JCR Pharmaceuticals Co., Ltd.
ClinicalTrials.gov Identifier:
NCT03128593
Other Study ID Numbers:
  • JR-141-101
First Posted:
Apr 25, 2017
Last Update Posted:
Sep 17, 2021
Last Verified:
Jan 1, 2021
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Mucopolysaccharidosis II
Mucopolysaccharidoses

Study Results

No Results Posted as of Sep 17, 2021