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An Extension 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: NCT04348136
Recruitment Status : Enrolling by invitation
First Posted : April 16, 2020
Last Update Posted : April 16, 2020
Sponsor:
Information provided by (Responsible Party):
JCR Pharmaceuticals Co., Ltd.

Brief Summary:
Multicenter, open-label, single-group, designed to evaluate the long term 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)
Actual Enrollment : 27 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Extension Study of JR-141-301 in Patients With Mucopolysaccharidosis II
Actual Study Start Date : September 1, 2019
Estimated Primary Completion Date : June 30, 2021
Estimated Study Completion Date : June 30, 2021


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




Primary Outcome Measures :
  1. Number of participants with Adverse Events [ Time Frame: up to approximately 3 years ]
    Adverse events

  2. Number of participants with Adverse Events [ Time Frame: up to approximately 3 years ]
    Antidrug antibodiesLaboratory tests (hematologic test, blood biochemical test, determination of iron-related levels and urine analysis)

  3. Number of participants with Adverse Events [ Time Frame: up to approximately 3 years ]
    Vital signs (pulse rate, body temperature, and blood pressure,Blood pressures in mmHg,Heart rate in beats/minute,Respiratory rate in breaths/minute,Temperature in °C,Presence or absence of abnormalities for physical examination

  4. Number of participants with Adverse Events [ Time Frame: up to approximately 3 years ]
    Presence or absence of abnormalities for 12-lead electrocardiogram

  5. Number of participants with Adverse Events [ Time Frame: up to approximately 3 years ]
    Antibody tests

  6. Number of participants with Adverse Events [ Time Frame: up to approximately 3 years ]
    • Routine laboratory tests in blood (hematology, liver function, renal function, iron-related levels) and urine (urinalysis)Infusion Associated Reaction (IAR)


Secondary Outcome Measures :
  1. Change From the first dose of the previous study (JR-141-301) in Serum Heparan Sulfate Levels. [ Time Frame: 78, 104, 130 weeks,the end of the study* ]
  2. Change From the first dose of the previous study (JR-141-301) in Serum Dermatan Sulfate Levels. [ Time Frame: 78, 104, 130 weeks,the end of the study* ]
  3. Change From the first dose of the previous study (JR-141-301) in Urinary Heparan Sulfate Levels. [ Time Frame: 78, 104, 130 weeks,the end of the study* ]
  4. Change From the first dose of the previous study (JR-141-301) in Urinary Dermatan Sulfate Levels. [ Time Frame: 78, 104, 130 weeks,the end of the study* ]
  5. Change From the first dose of the previous study (JR-141-301) in Liver Volumes. [ Time Frame: 78, 104, 130 weeks,the end of the study* ]
  6. Change From the first dose of the previous study (JR-141-301) in Spleen Volumes. [ Time Frame: 78, 104, 130 weeks,the end of the study* ]
  7. Change From Baseline in Echocardigraphy [ Time Frame: 78, 104, 130 weeks,the end of the study* ]
  8. Change From the first dose of the previous study (JR-141-301) in 6-minute Walk Test Distance. [ Time Frame: 104 weeks, the end of the study* ]
    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 the first dose of the previous study (JR-141-301) in Joint Range of Motion (goniometer) [ Time Frame: 104, 130 weeks, the end of the study* ]
  10. Change From the first dose of the previous study (JR-141-301) in Heparan Sulfate Levels in Cerebrospinal Fluid. [ Time Frame: 104 weeks, the end of the study* ]
  11. Change From the first dose of the previous study (JR-141-301) in Dermatan Sulfate Levels in Cerebrospinal Fluid. [ Time Frame: 104 weeks, the end of the study* ]
  12. Change From the first dose of the previous study (JR-141-301) in Neurocognitive Testing (Kyoto Scale of Psychological Development 2001) [ Time Frame: 104 weeks, the end of the study* ]
  13. Change From the first dose of the previous study (JR-141-301) in Adaptive Behavioral Testing ( Vineland Adaptive Behavior Scales Second Edition. ) [ Time Frame: 104 weeks, the end of the study* ]
  14. Change From the first dose of the previous study (JR-141-301) in Cognitive Testing (Bayley Scales of Infant and Toddler Development, Third Edition. ) [ Time Frame: 104 weeks, the end of the study* ]
  15. Change From the first dose of the previous study (JR-141-301) in Cognitive Testing (Kaufman Assessment Battery for Children, Second Edition) [ Time Frame: 104 weeks, the end of the study* ]
    *Target patients: Patients who haven't conducted the test for more than 26 weeks.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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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. Patients who will have completed clinical trial JR-141-301 and judged by the investigator or subinvestigator to be able to participate in the study from safety aspects.
  2. Capable of providing written consent by himself, unless the patient is under the age of 20 years at the time of informed consent process, or it is not possible to obtain consent from the patient himself due to his intellectual disabilities associated with MPS II.
  3. 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 his 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, wherever possible.

Exclusion Criteria:

  1. Judged by the investigator or subinvestigator to be ineligible to participate in the study due to receive concomitant therapy which affectseffect the clinical evaluation.
  2. Judged by the investigator or subinvestigator to be ineligible to participate in the study due to a history of a serious drug allergy or sensitivity.
  3. 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): NCT04348136


Locations
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Sponsors and Collaborators
JCR Pharmaceuticals Co., Ltd.
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Responsible Party: JCR Pharmaceuticals Co., Ltd.
ClinicalTrials.gov Identifier: NCT04348136    
Other Study ID Numbers: JR-141-302
First Posted: April 16, 2020    Key Record Dates
Last Update Posted: April 16, 2020
Last Verified: April 2020

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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