Back to resultsA Study of JR-141 in Patients With Mucopolysaccharidosis II
Primary Purpose
Mucopolysaccharidosis II
Status
Completed
Phase
Phase 2
Locations
Brazil
Study Type
Interventional
Intervention
JR-141
JR-141
JR-141
Sponsored by
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis II
Eligibility 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:
- 0 to 3 years and 11 months old (6 patients, 2 in each dose)
- 4 years to 7 years and 11 months old (6 patients, 2 in each dose)
- 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.
Sites / Locations
- Grupo de Pesquisa Clínica em Genética Médica - HCPA
- Igeim - Unifesp
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Experimental
Arm Label
JR-141 1.0 mg/kg/week
JR-141 2.0 mg/kg/week
JR-141 4.0 mg/kg/week
Arm Description
Outcomes
Primary Outcome Measures
Number of participants with Adverse Events
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
Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]]
Plasma concentration of JR-141
Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]]
Plasma concentration of JR-141
Liver and spleen volumes (MRI)
Left ventricular mass by a standard 2-dimensional Doppler echocardiogram
Urinary heparan sulfate concentrations
Urinary dermatan sulfate concentrations
Serum heparan sulfate concentrations
Serum dermatan sulfate concentrations
Full Information
NCT ID
NCT03359213
First Posted
November 17, 2017
Last Updated
November 10, 2022
Sponsor
JCR Pharmaceuticals Co., Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT03359213
Brief Title
A Study of JR-141 in Patients With Mucopolysaccharidosis II
Official Title
Phase II Study of JR-141 in Patients With Mucopolysaccharidosis II
Study Type
Interventional
2. Study Status
Record Verification Date
November 2022
Overall Recruitment Status
Completed
Study Start Date
July 26, 2018 (Actual)
Primary Completion Date
October 4, 2019 (Actual)
Study Completion Date
October 4, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
JCR Pharmaceuticals Co., Ltd.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucopolysaccharidosis II
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
18 (Actual)
8. Arms, Groups, and Interventions
Arm Title
JR-141 1.0 mg/kg/week
Arm Type
Experimental
Arm Title
JR-141 2.0 mg/kg/week
Arm Type
Experimental
Arm Title
JR-141 4.0 mg/kg/week
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
JR-141
Intervention Description
IV infusion (lyophilized powder), 1.0 mg/kg/week
Intervention Type
Drug
Intervention Name(s)
JR-141
Intervention Description
IV infusion (lyophilized powder), 2.0 mg/kg/week
Intervention Type
Drug
Intervention Name(s)
JR-141
Intervention Description
IV infusion (lyophilized powder), 4.0 mg/kg/week
Primary Outcome Measure Information:
Title
Number of participants with Adverse Events
Description
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)
Time Frame
26 weeks
Secondary Outcome Measure Information:
Title
Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]]
Description
Plasma concentration of JR-141
Time Frame
21 hours after dosing at the first and last infusions
Title
Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]]
Description
Plasma concentration of JR-141
Time Frame
21 hours after dosing at the first and last infusions
Title
Liver and spleen volumes (MRI)
Time Frame
26 weeks
Title
Left ventricular mass by a standard 2-dimensional Doppler echocardiogram
Time Frame
26 weeks
Title
Urinary heparan sulfate concentrations
Time Frame
26 weeks
Title
Urinary dermatan sulfate concentrations
Time Frame
26 weeks
Title
Serum heparan sulfate concentrations
Time Frame
26 weeks
Title
Serum dermatan sulfate concentrations
Time Frame
26 weeks
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
0 Years
Accepts Healthy Volunteers
No
Eligibility 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:
0 to 3 years and 11 months old (6 patients, 2 in each dose)
4 years to 7 years and 11 months old (6 patients, 2 in each dose)
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.
Facility Information:
Facility Name
Grupo de Pesquisa Clínica em Genética Médica - HCPA
City
Porto Alegre
Country
Brazil
Facility Name
Igeim - Unifesp
City
São Paulo
Country
Brazil
12. IPD Sharing Statement
Learn more about this trial
A Study of JR-141 in Patients With Mucopolysaccharidosis II
We'll reach out to this number within 24 hrs