Back to resultsPhase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA
Primary Purpose
Mucopolysaccharidosis III-A
Status
Recruiting
Phase
Phase 1
Locations
Germany
Study Type
Interventional
Intervention
JR-441
JR-441
Sponsored by
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis III-A
Eligibility Criteria
Inclusion Criteria: Chronological age of ≥1 year and ≤18 years. Confirmed diagnosis of MPS IIIA. Body weight ≥ 10 kg. Exclusion Criteria: Prior experience to gene therapy or HSCT with successful engraftment. Past use of another investigational drug or product in last 4 months or 5 half-lives (whichever is longer) before signing ICF. Current participation in a clinical trial or past participation (within 30 days of enrolment into this study) in a study involving invasive procedures. Past use of Genistein or Kineret (anakinra) within 4 months before signing ICF. Serious drug allergy or hypersensitivity. Contraindication for lumbar puncture or MRI. History of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following lumbar puncture. The above information is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial.
Sites / Locations
- Universitätsklinikum Hamburg-EppendorfRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
JR-441 low dose
JR-441 high dose
Arm Description
Outcomes
Primary Outcome Measures
To evaluate the tolerability of JR-441 in MPSIIIA patients
Adverse events will be reported and graded, laboratory tests will be conducted and vital signs will be monitored
To assess the safety of JR-441 in MPSIII-A patients
Number and severity of infusion-associated reactions, including anaphylaxis
Secondary Outcome Measures
Plasma drug concentration
Plasma PK parameters
Change from baseline in heparan sulfate levels in cerebrospinal fluid (CSF), serum and urine
Change from baseline in cognitive function
Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) and/or Kaufman Assessment Battery for Children, Second Edition (KABC-II)
Full Information
NCT ID
NCT06095388
First Posted
September 13, 2023
Last Updated
October 18, 2023
Sponsor
JCR Pharmaceuticals Co., Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT06095388
Brief Title
Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA
Official Title
Phase I/II Study of Weekly Infusions of JR-441 in Patients With Mucopolysaccharidosis Type IIIA
Study Type
Interventional
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
October 4, 2023 (Actual)
Primary Completion Date
October 31, 2029 (Anticipated)
Study Completion Date
October 31, 2029 (Anticipated)
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 I/ II, open-label, randomized, 2-arm study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIA patients.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucopolysaccharidosis III-A
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
12 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
JR-441 low dose
Arm Type
Experimental
Arm Title
JR-441 high dose
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
JR-441
Intervention Description
IV infusion
Intervention Type
Drug
Intervention Name(s)
JR-441
Intervention Description
IV infusion
Primary Outcome Measure Information:
Title
To evaluate the tolerability of JR-441 in MPSIIIA patients
Description
Adverse events will be reported and graded, laboratory tests will be conducted and vital signs will be monitored
Time Frame
up to 5 years (multiple visits)
Title
To assess the safety of JR-441 in MPSIII-A patients
Description
Number and severity of infusion-associated reactions, including anaphylaxis
Time Frame
up to 5 years (multiple visits)
Secondary Outcome Measure Information:
Title
Plasma drug concentration
Time Frame
up to 5 years (multiple visits)
Title
Plasma PK parameters
Time Frame
up to 5 years (multiple visits)
Title
Change from baseline in heparan sulfate levels in cerebrospinal fluid (CSF), serum and urine
Time Frame
up to 5 years (multiple visits)
Title
Change from baseline in cognitive function
Description
Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) and/or Kaufman Assessment Battery for Children, Second Edition (KABC-II)
Time Frame
up to 5 years (multiple visits)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Chronological age of ≥1 year and ≤18 years.
Confirmed diagnosis of MPS IIIA.
Body weight ≥ 10 kg.
Exclusion Criteria:
Prior experience to gene therapy or HSCT with successful engraftment.
Past use of another investigational drug or product in last 4 months or 5 half-lives (whichever is longer) before signing ICF.
Current participation in a clinical trial or past participation (within 30 days of enrolment into this study) in a study involving invasive procedures.
Past use of Genistein or Kineret (anakinra) within 4 months before signing ICF.
Serious drug allergy or hypersensitivity.
Contraindication for lumbar puncture or MRI.
History of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following lumbar puncture.
The above information is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
JCR Pharmaceuticals Co., Ltd.
Phone
+81-(0)797-32-8582
Email
clinical_development@jp.jcrpharm.com
Facility Information:
Facility Name
Universitätsklinikum Hamburg-Eppendorf
City
Hamburg
Country
Germany
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Nichole Muschol
Phone
+49 40 74100
12. IPD Sharing Statement
Learn more about this trial
Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA
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