- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03128593
A Study of JR-141 in Patients With Mucopolysaccharidosis Type II
November 10, 2022 updated by: JCR Pharmaceuticals Co., Ltd.
A Phase I/II Study of JR-141 in Patients With Mucopolysaccharidosis Type II
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
Study Overview
Study Type
Interventional
Enrollment (Actual)
14
Phase
- Phase 2
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
-
Gifu, Japan, 501-1194
- Gifu Clinical site
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Kurume, Japan, 830-0011
- Fukuoka Clinical site
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Minato, Japan, 105-8471
- Tokyo Clinical Site1
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Osaka, Japan, 545-8586
- Osaka Clinical Site1
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Saitama, Japan, 330-8777
- Saitama Clinical site
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Setagaya, Japan, 157-8535
- Tokyo Clinical Site2
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Suita, Japan, 157-8535
- Osaka Clinical Site2
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Yonago, Japan, 683-8504
- Tottori Clinical site
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
6 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
Male
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Sequential Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Experimental: JR-141
|
IV infusion (lyophilized powder), 0.01-2.0
mg/kg/week
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of participants with Adverse Events
Time Frame: 4 weeks
|
|
4 weeks
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]]
Time Frame: 4 weeks
|
Plasma concentration of JR-141
|
4 weeks
|
Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]]
Time Frame: 4 weeks
|
Plasma concentration of JR-141
|
4 weeks
|
Urinary and serum heparan sulfate (HS) /dermatan sulfate (DS)
Time Frame: 4 weeks
|
4 weeks
|
|
HS/DS in CSF
Time Frame: 4 weeks
|
JR-141 concentration in CSF
|
4 weeks
|
Urinary total GAG
Time Frame: 4 weeks
|
4 weeks
|
|
Liver and spleen volumes
Time Frame: 4 weeks
|
4 weeks
|
|
Cardiac function
Time Frame: 4 weeks
|
4 weeks
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
March 30, 2017
Primary Completion (Actual)
October 4, 2017
Study Completion (Actual)
October 4, 2017
Study Registration Dates
First Submitted
April 10, 2017
First Submitted That Met QC Criteria
April 20, 2017
First Posted (Actual)
April 25, 2017
Study Record Updates
Last Update Posted (Actual)
November 14, 2022
Last Update Submitted That Met QC Criteria
November 10, 2022
Last Verified
November 1, 2022
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-141-101
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Mucopolysaccharidosis II
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University of ChicagoNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedKrabbe Disease | Mucopolysaccharidosis Type II (MPS II) | Mucopolysaccharidosis Type I (MPS I) | Mucopolysaccharidosis Type III (MPS III) | Mucopolysaccharidosis Type VI (MPS VI)United States
-
University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedMucopolysaccharidosis Type I | Mucopolysaccharidosis Type II | Mucopolysaccharidosis Type VI | Mucopolysaccharidosis Type IV | Mucopolysaccharidosis Type VIIUnited States, Canada
-
REGENXBIO Inc.Active, not recruitingMucopolysaccharidosis Type II (MPS II)United States, Brazil
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REGENXBIO Inc.Active, not recruitingMucopolysaccharidosis Type II (MPS II)United States, Canada
-
Lundquist Institute for Biomedical Innovation at...CompletedMucopolysaccharidosis Type I | Mucopolysaccharidosis Type II | Mucopolysaccharidosis Type VIUnited States
-
University Hospital HeidelbergCompletedMucopolysaccharidosis Type I | Mucopolysaccharidosis Type II | Coping Behavior | Mucopolysaccharidosis Type III | Behavior DisordersGermany
-
TakedaCompletedMucopolysaccharidosis (MPS)Brazil
-
JCR Pharmaceuticals Co., Ltd.Active, not recruitingMucopolysaccharidosis IIJapan
-
AO GENERIUMRecruitingMetabolic Diseases | Mucopolysaccharidosis Type IIRussian Federation
-
Denali Therapeutics Inc.Enrolling by invitationMucopolysaccharidosis IIUnited States, Canada, Belgium
Clinical Trials on JR-141
-
JCR Pharmaceuticals Co., Ltd.RecruitingMucopolysaccharidosis IIUnited States, Germany, Spain, France, Brazil, Argentina, Turkey, United Kingdom, Poland, Italy
-
JCR Pharmaceuticals Co., Ltd.Active, not recruitingMucopolysaccharidosis IIJapan
-
JCR Pharmaceuticals Co., Ltd.Completed
-
JCR Pharmaceuticals Co., Ltd.Active, not recruitingMucopolysaccharidosis IIBrazil
-
JCR Pharmaceuticals Co., Ltd.Enrolling by invitationMucopolysaccharidosis IIUnited States
-
JCR Pharmaceuticals Co., Ltd.Completed
-
AbbVieRecruiting
-
TetraLogic PharmaceuticalsPPD; The Leukemia and Lymphoma Society; Veristat, Inc.; Therapeutics, Inc.CompletedLymphoma, T-Cell, CutaneousUnited States
-
JCR Pharmaceuticals Co., Ltd.RecruitingMucopolysaccharidosis III-AGermany
-
Kissei Pharmaceutical Co., Ltd.JCR Pharmaceuticals Co., Ltd.Completed