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A Study of JR-141 in Patients With Mucopolysaccharidosis II

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ClinicalTrials.gov Identifier: NCT03359213
Recruitment Status : Not yet recruiting
First Posted : December 2, 2017
Last Update Posted : December 2, 2017
Sponsor:
Information provided by (Responsible Party):
JCR Pharmaceuticals Co., Ltd.

Brief Summary:
A Phase II open-label, randomized, parallel group, 2 sites (Brazil), designed to evaluate the safety and efficacy of 3 doses of study drug for the treatment of the MPS II.

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

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Study of JR-141 in Patients With Mucopolysaccharidosis II
Estimated Study Start Date : February 1, 2018
Estimated Primary Completion Date : December 31, 2018
Estimated Study Completion Date : December 31, 2018


Arm Intervention/treatment
Experimental: JR-141 1.0 mg/kg/week Drug: JR-141
IV infusion (lyophilized powder), 1.0 mg/kg/week

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

Experimental: JR-141 4.0 mg/kg/week Drug: JR-141
IV infusion (lyophilized powder), 4.0 mg/kg/week




Primary Outcome Measures :
  1. Number of participants with Adverse Events [ Time Frame: 26 weeks ]
    • Adverse events
    • Antidrug antibodies
    • 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
    • Presence or absence of abnormalities for 12-lead electrocardiogram
    • Routine laboratory tests in blood (hematology, liver function, renal function, iron-related levels) and urine (urinalysis)


Secondary Outcome Measures :
  1. Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]] [ Time Frame: 21 hours after dosing at the first and last infusions ]
    Plasma concentration of JR-141

  2. Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]] [ Time Frame: 21 hours after dosing at the first and last infusions ]
    Plasma concentration of JR-141

  3. Liver and spleen volumes (MRI) [ Time Frame: 26 weeks ]
  4. Left ventricular mass by a standard 2-dimensional Doppler echocardiogram [ Time Frame: 26 weeks ]
  5. Urinary heparan sulfate concentrations [ Time Frame: 26 weeks ]
  6. Urinary dermatan sulfate concentrations [ Time Frame: 26 weeks ]
  7. Serum heparan sulfate concentrations [ Time Frame: 26 weeks ]
  8. Serum dermatan sulfate concentrations [ Time Frame: 26 weeks ]


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

Inclusion Criteria:

  • Males with confirmed diagnosis of MPS II, based on deficient activity of IDS in leucocytes or fibroblasts and/or pathogenic mutations identified in the IDS gene (if enzyme diagnosis was in dried blood spots or plasma, molecular genetics confirmation is mandatory).
  • One of the following age groups:

    1. 0 to 3 years and 11 months old (6 patients, 2 in each dose)
    2. 4 years to 7 years and 11 months old (6 patients, 2 in each dose)
    3. 8 years or older (6 patients, 2 in each dose)
  • 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.
  • Naïve patients or patients who are receiving enzyme replacement therapy with idursulfase could be included if provided treatment has been stable in the last 6 months and agree to interrupt the treatment at least one week before the first study drug infusion, and agree in suspending this treatment for the duration of the trial.

Exclusion Criteria:

  • Refusal to sign the informed consent form.
  • Unable to perform the study procedures, except for neurocognitive testing.
  • Previous engrafted BMT/HSCT.
  • Surgical or other major medical intervention planned to occur before week 26.
  • Participation in a clinical trial with an investigational drug in the last 12 months.
  • 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.

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): NCT03359213


Contacts
Contact: JCR Pharmaceuticals Co., Ltd. +81-(0)797-32-8582 kaihatsu@jcrpharm.co.jp

Locations
Brazil
Grupo de Pesquisa Clínica em Genética Médica - HCPA
Porto Alegre, Brazil
Igeim - Unifesp
São Paulo, Brazil
Sponsors and Collaborators
JCR Pharmaceuticals Co., Ltd.

Responsible Party: JCR Pharmaceuticals Co., Ltd.
ClinicalTrials.gov Identifier: NCT03359213     History of Changes
Other Study ID Numbers: JR-141-BR21
First Posted: December 2, 2017    Key Record Dates
Last Update Posted: December 2, 2017
Last Verified: November 2017

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

Additional relevant MeSH terms:
Mucopolysaccharidoses
Mucopolysaccharidosis II
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