A Study of JR-141 in Patients With Mucopolysaccharidosis II

NCT03568175 | Completed Phase 2

• 1 other identifier: JR-141-301
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 19

Brief Summary

A Phase II/ III multicenter, open-label, single-group, designed to evaluate the efficacy and safety of study drug for the treatment of the MPS II.

Conditions

Mucopolysaccharidosis II

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid

  Baseline to 52 weeks

Secondary Outcomes (14)

• Change From Baseline in Serum Heparan Sulfate Levels.

  Baseline, 24-26 weeks, 50-52 weeks

• Change From Baseline in Serum Dermatan Sulfate Levels.

  Baseline, 24-26 weeks, 50-52 weeks

• Change From Baseline in Urinary Heparan Sulfate Levels.

  Baseline, 25 weeks, 52 weeks

• Change From Baseline in Urinary Dermatan Sulfate Levels.

  Baseline, 25 weeks, 52 weeks

• Change From Baseline in Liver Volumes.

  Baseline, 25 weeks, 52 weeks

Study Arms (1)

JR-141 2.0 mg/kg/week

EXPERIMENTAL

Drug: JR-141

Interventions

JR-141 DRUG

IV infusion, 2.0 mg/kg/week

JR-141 2.0 mg/kg/week

Eligibility Criteria

• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Capable of providing written consent by himself (not mandatory for those aged under 20 years at the time of informed consent process, or those who is impossible to obtain consent from the patient himself due to intellectual disabilities associated with MPS II.)
• In the case of a patient who is under the age of 20 years or from whom it is not possible to obtain consent due to intellectual disabilities associated with MPS II, he may be included if written consent can be provided by legal representative (however, written consent should be obtained from the patient himself too, whenever possible)
• Males with confirmed diagnosis of MPS II, based on deficient activity of iduronate-2-sulfatase (IDS) in leucocytes, plasma or fibroblasts and/or pathogenic mutations identified in the IDS gene, etc.
• Naïve patients or patients who are receiving stable enzyme replacement therapy with Elaprase for more than 8 weeks before the observational period starts.

You may not qualify if:

• Previous engrafted HSCT, excluding those who need enzyme replacement therapy even after HSCT.
• Judged by the investigator or subinvestigator as being unable to undergo lumbar puncture, including those who have difficulties in taking position for lumbar puncture due to joint contracture or those who are likely experience difficulty breathing during the lumbar puncture process.
• Judged by the investigator or subinvestigator to be ineligible to participate in the study due to a history of serious drug allergy or sensitivity.
• Patients who have received other investigational product within 4 months before enrollment in the study.
• Otherwise judged by the investigator or subinvestigator to be ineligible to participate in the study.

Sponsors & Collaborators

• JCR Pharmaceuticals Co., Ltd.: lead

Study Sites (19)

Fukui Clinical site

Fukui, 910-1193, Japan

Location

Fukuoka Clinical site 2

Fukuoka, 813-0017, Japan

Location

Fukuoka Clinical site

Fukuoka, 830-0011, Japan

Location

Gifu Clinical site

Gifu, 501-1194, Japan

Location

Hokkaido Clinical site

Hokkaido, 063-0005, Japan

Location

Kanagawa Clinical site

Kanagawa, 232-8555, Japan

Location

Kumamoto Clinical site

Kumamoto, 860-8556, Japan

Location

Okayama Clinical site

Okayama, 701-1192, Japan

Location

Okayama Clinical site 2

Okayama, 710-8602, Japan

Location

Okinawa Clinical site

Okinawa, 903-0215, Japan

Location

Osaka Clinical site 3

Osaka, 534-0021, Japan

Location

Osaka Clinical site 2

Osaka, 545-8586, Japan

Location

Osaka Clinical site

Osaka, 565-0871, Japan

Location

Saitama Clinical site

Saitama, 330-8777, Japan

Location

Shizuoka Clinical site

Shizuoka, 420-8660, Japan

Location

Shizuoka Clinical site 2

Shizuoka, 426-8677, Japan

Location

Tochigi Clinical site

Tochigi, 329-0498, Japan

Location

Tokyo Clinical site

Tokyo, 157-8535, Japan

Location

Tottori Clinical site

Tottori, 683-8504, Japan

Location

Related Links

• A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II

MeSH Terms

Conditions

Mucopolysaccharidosis II

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 1, 2018
• First Posted: June 26, 2018
• Study Start: August 1, 2018
• Primary Completion: February 20, 2020
• Study Completion: February 20, 2020
• Last Updated: November 14, 2022

Record last verified: 2022-11

Locations

JP (19)