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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03128593
Recruitment Status : Completed
First Posted : April 25, 2017
Last Update Posted : October 12, 2017
Sponsor:
Information provided by (Responsible Party):
JCR Pharmaceuticals Co., Ltd.

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
Mucopolysaccharidosis II Drug: JR-141 Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
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 : March 30, 2017
Actual Primary Completion Date : March 30, 2017
Actual Study Completion Date : October 4, 2017


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




Primary Outcome Measures :
  1. Number of participants with Adverse Events [ Time Frame: 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]] [ Time Frame: 4 weeks ]
    Plasma concentration of JR-141

  2. Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]] [ Time Frame: 4 weeks ]
    Plasma concentration of JR-141

  3. Urinary and serum heparan sulfate (HS) /dermatan sulfate (DS) [ Time Frame: 4 weeks ]
  4. HS/DS in CSF [ Time Frame: 4 weeks ]
    JR-141 concentration in CSF

  5. Urinary total GAG [ Time Frame: 4 weeks ]
  6. Liver and spleen volumes [ Time Frame: 4 weeks ]
  7. Cardiac function [ Time Frame: 4 weeks ]


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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03128593


Locations
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Japan
Gifu Clinical site
Gifu, Japan, 501-1194
Fukuoka Clinical site
Kurume, Japan, 830-0011
Tokyo clinical site1
Minato, Japan, 105-8471
Osaka Clinical site1
Osaka, Japan, 545-8586
Saitama Clinical site
Saitama, Japan, 330-8777
Tokyo clinical site2
Setagaya, Japan, 157-8535
Osaka Clinical site2
Suita, Japan, 157-8535
Tottori Clinical site
Yonago, Japan, 683-8504
Sponsors and Collaborators
JCR Pharmaceuticals Co., Ltd.

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Responsible Party: JCR Pharmaceuticals Co., Ltd.
ClinicalTrials.gov Identifier: NCT03128593     History of Changes
Other Study ID Numbers: JR-141-101
First Posted: April 25, 2017    Key Record Dates
Last Update Posted: October 12, 2017
Last Verified: October 2017

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Mucopolysaccharidosis II
Mucopolysaccharidoses
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System