A Study of JR-141 in Patients With Mucopolysaccharidosis Type II
A Phase I/II Study of JR-141 in Patients With Mucopolysaccharidosis Type II
1 other identifier
interventional
14
1 country
8
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_1
Started Mar 2017
Shorter than P25 for phase_1
8 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
March 30, 2017
CompletedFirst Submitted
Initial submission to the registry
April 10, 2017
CompletedFirst Posted
Study publicly available on registry
April 25, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 4, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
October 4, 2017
CompletedNovember 14, 2022
November 1, 2022
6 months
April 10, 2017
November 10, 2022
Conditions
Outcome Measures
Primary Outcomes (1)
Number of participants with Adverse Events
* Adverse events * Laboratory tests * Vital signs * 12-lead electrocardiogram * Antibody * Infusion associated reaction
4 weeks
Secondary Outcomes (7)
Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]]
4 weeks
Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]]
4 weeks
Urinary and serum heparan sulfate (HS) /dermatan sulfate (DS)
4 weeks
HS/DS in CSF
4 weeks
Urinary total GAG
4 weeks
- +2 more secondary outcomes
Study Arms (1)
Experimental: JR-141
EXPERIMENTALInterventions
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (8)
Gifu Clinical site
Gifu, 501-1194, Japan
Fukuoka Clinical site
Kurume, 830-0011, Japan
Tokyo clinical site1
Minato, 105-8471, Japan
Osaka Clinical site1
Osaka, 545-8586, Japan
Saitama Clinical site
Saitama, 330-8777, Japan
Tokyo clinical site2
Setagaya City, 157-8535, Japan
Osaka Clinical site2
Suita, 157-8535, Japan
Tottori Clinical site
Yonago, 683-8504, Japan
Related Publications (1)
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.
PMID: 30595526DERIVED
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 10, 2017
First Posted
April 25, 2017
Study Start
March 30, 2017
Primary Completion
October 4, 2017
Study Completion
October 4, 2017
Last Updated
November 14, 2022
Record last verified: 2022-11