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An Extension Study of JR-141-BR21 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: NCT03708965
Recruitment Status : Active, not recruiting
First Posted : October 17, 2018
Last Update Posted : September 17, 2021
Sponsor:
Information provided by (Responsible Party):
JCR Pharmaceuticals Co., Ltd.

Study Description
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Brief Summary:
A Phase II open-label, parallel group, 2 sites (Brazil), designed to evaluate the long term safety and efficacy of study drug for the treatment of the MPS II.

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

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II
Actual Study Start Date : January 1, 2019
Estimated Primary Completion Date : December 31, 2026
Estimated Study Completion Date : December 31, 2026

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Arms and Interventions
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Arm Intervention/treatment
Experimental: JR-141

Subjects will be assigned to 1.0, 2.0 or 4.0 mg of JR-141 per kg of body weight once every week (the same dose taken during the previous study) in the beginning of the study.

During the study, the dose of all subjects will be switched to the selected one.

 Drug: JR-141
IV infusion (lyophilized powder)


Outcome Measures
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Primary Outcome Measures :
  1. Number of participants with Adverse Events [ Time Frame: through study completion, an average of 2 years ]

Secondary Outcome Measures :
  1. Urinary heparan sulfate concentrations [ Time Frame: through study completion, an average of 2 years ]
  2. Urinary dermatan sulfate concentrations [ Time Frame: through study completion, an average of 2 years ]
  3. Serum heparan sulfate concentrations [ Time Frame: through study completion, an average of 2 years ]
  4. Serum dermatan sulfate concentrations [ Time Frame: through study completion, an average of 2 years ]
  5. Liver and spleen volumes (MRI) [ Time Frame: through study completion, an average of 2 years ]
  6. Echocardiography [ Time Frame: through study completion, an average of 2 years ]

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

Inclusion Criteria:

  • Patients who will have completed clinical trial JR-141-BR21.
  • Capable of providing written consent by himself, unless the patient is under the age of 18 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.
  • In the case of a patient who is under the age of 18 years or from whom it is not possible to obtain consent due to his intellectual disabilities associated with MSP 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:

  • Refusal to sign the informed consent form.
  • Unable to perform the study procedures, except for neurocognitive testing.
  • Previous engrafted BMT/HSCT.
  • Judged by the investigator or subinvestigator as being unable to undergo lumbar puncture, including those who have difficulties in taking a position for lumber puncture due to joint contracture or those who are likely to 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 a serious drug allergy or sensitivity.
  • Otherwise judged by the investigator or subinvestigator to be ineligible to participate in the study out of consideration for the subject safety.
Contacts and Locations
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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): NCT03708965


Locations
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Brazil
Grupo de Pesquisa Clínica em Genética Médica - HCPA
Porto Alegre, Brazil
Igeim - Unifesp
São Paulo, Brazil
Sponsors and Collaborators
JCR Pharmaceuticals Co., Ltd.
More Information
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Responsible Party: JCR Pharmaceuticals Co., Ltd.
ClinicalTrials.gov Identifier: NCT03708965    
Other Study ID Numbers: JR-141-BR22
First Posted: October 17, 2018    Key Record Dates
Last Update Posted: September 17, 2021
Last Verified: January 2021

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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