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A Study of JR-141 in Patients With Mucopolysaccharidosis 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: NCT03568175
Recruitment Status : Active, not recruiting
First Posted : June 25, 2018
Last Update Posted : January 9, 2019
Sponsor:
Information provided by (Responsible Party):
JCR Pharmaceuticals Co., Ltd.

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.

Condition or disease Intervention/treatment Phase
Mucopolysaccharidosis II Drug: JR-141 Phase 2 Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II/III Study of JR-141 in Patients With Mucopolysaccharidosis II
Actual Study Start Date : August 1, 2018
Estimated Primary Completion Date : March 31, 2020
Estimated Study Completion Date : March 31, 2020


Arm Intervention/treatment
Experimental: JR-141 2.0 mg/kg/week Drug: JR-141
IV infusion, 2.0 mg/kg/week




Primary Outcome Measures :
  1. Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid [ Time Frame: Baseline to 52 weeks ]

Secondary Outcome Measures :
  1. Change From Baseline in Serum Heparan Sulfate Levels. [ Time Frame: Baseline, 24-26 weeks, 50-52 weeks ]
  2. Change From Baseline in Serum Dermatan Sulfate Levels. [ Time Frame: Baseline, 24-26 weeks, 50-52 weeks ]
  3. Change From Baseline in Urinary Heparan Sulfate Levels. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
  4. Change From Baseline in Urinary Dermatan Sulfate Levels. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
  5. Change From Baseline in Liver Volumes. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
  6. Change From Baseline in Spleen Volumes. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
  7. Change From Baseline in Cardiac Function. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
  8. Change From Baseline in 6-minute Walk Test Distance. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
    Item 9 will be administrated only in patients judged by the investigator or subinvestigator to be possible to perform the 6-minutes walk test

  9. Change From Baseline in Joint Range of Motion. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
  10. Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid. [ Time Frame: Baseline to 25 weeks ]
  11. Change From Baseline in Dermatan Sulfate Levels in Cerebrospinal Fluid. [ Time Frame: Baseline, 25 weeks, 52 weeks ]
  12. Change from Baseline in Neurocognitive Testing (Kyoto Scale of Psychological Development 2001) [ Time Frame: 25, 52 weeks ]
  13. Change from Baseline in Adaptive Behavioral Testing ( Vineland Adaptive Behavior Scales Second Edition. ) [ Time Frame: 25, 52 weeks ]
  14. Drug concentration in Cerebrospinal Fluid. [ Time Frame: 25, 52*weeks ]
    *Drug concentration in Cerebrospinal Fluid at 52 Weeks is applicable only for subjects to be enrolled in extension study .



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. 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.)
  2. 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)
  3. 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.
  4. Naïve patients or patients who are receiving stable enzyme replacement therapy with Elaprase for more than 8 weeks before the observational period starts.

Exclusion Criteria:

  1. Previous engrafted HSCT, excluding those who need enzyme replacement therapy even after HSCT.
  2. 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.
  3. Judged by the investigator or subinvestigator to be ineligible to participate in the study due to a history of serious drug allergy or sensitivity.
  4. Patients who have received other investigational product within 4 months before enrollment in the study.
  5. Otherwise judged by the investigator or subinvestigator to be ineligible to participate 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): NCT03568175


Locations
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Japan
Fukui Clinical site
Fukui, Japan, 910-1193
Fukuoka Clinical site 2
Fukuoka, Japan, 813-0017
Fukuoka Clinical site
Fukuoka, Japan, 830-0011
Gifu Clinical site
Gifu, Japan, 501-1194
Hokkaido Clinical site
Hokkaido, Japan, 063-0005
Kanagawa Clinical site
Kanagawa, Japan, 232-8555
Kumamoto Clinical site
Kumamoto, Japan, 860-8556
Okayama Clinical site
Okayama, Japan, 701-1192
Okayama Clinical site 2
Okayama, Japan, 710-8602
Okinawa Clinical site
Okinawa, Japan, 903-0215
Osaka Clinical site 3
Osaka, Japan, 534-0021
Osaka Clinical site 2
Osaka, Japan, 545-8586
Osaka Clinical site
Osaka, Japan, 565-0871
Saitama Clinical site
Saitama, Japan, 330-8777
Shizuoka Clinical site
Shizuoka, Japan, 420-8660
Shizuoka Clinical site 2
Shizuoka, Japan, 426-8677
Tochigi Clinical site
Tochigi, Japan, 329-0498
Tokyo Clinical site
Tokyo, Japan, 157-8535
Tottori Clinical site
Tottori, Japan, 683-8504
Sponsors and Collaborators
JCR Pharmaceuticals Co., Ltd.

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Responsible Party: JCR Pharmaceuticals Co., Ltd.
ClinicalTrials.gov Identifier: NCT03568175     History of Changes
Other Study ID Numbers: JR-141-301
First Posted: June 25, 2018    Key Record Dates
Last Update Posted: January 9, 2019
Last Verified: January 2019

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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