A Phase III Study of JR-141 in Patients with Mucopolysaccharidosis II (STARLIGHT)
- Conditions
- Mucopolysaccharidosis II
- Interventions
- Registration Number
- NCT04573023
- Lead Sponsor
- JCR Pharmaceuticals Co., Ltd.
- Brief Summary
A Global Phase III multicenter, randomized, assessor-blinded, active-controlled designed to evaluate safety and efficacy of study drug for the treatment of the MPS II.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 80
- A patient who voluntarily signs an Institutional Review Board or Independent Ethics Committee-approved written informed consent form. If the patient is aged under 18 years (aged under 16 years in the UK) at the time of enrollment or willingness to participate in the study cannot be confirmed due to MPS II-related intellectual disability, the patient's legally acceptable representative (e.g., his/her parents or guardians) may sign the informed consent on behalf of the patient. Written informed assent should be obtained from the patient, wherever possible.
- Patients with confirmed diagnosis of MPS II
- Naïve patients or patients who are receiving stable enzyme replacement therapy with idursulfase for more than 12 weeks before starting administration of JR-141 or idursulfase for this study.
- Patients or patients whose partners are of child-bearing potential agree to use a medically accepted, highly effective method of contraception being use of condoms from the time of informed consent.
<Cohort A>
- Patients aged 36-42 months old at the time of ICF signing: patients must have a standard score measured by the BSID-III of 85 or less at screening.
- Patients aged 43-71 months old at the time of ICF signing: patients must EITHER have (1) A DQ measured by BSID-III of 20 to 85 at screening OR (2) A composite standard score on NVI measured by KABC-II of 85 or less at screening (only who can perform KABC-II)
- Patients aged 30-35 months old at the time of randomization and who are judged as having the severe phenotype by the Expert Board.
<Cohort B>
- Patients 6 years of age or older at the time of ICF signing and whose IQ are 70 and higher.
- Enrollment of subjects in Cohort B is contingent on the availability in that country of a validated country-specific version of the test (either WISC-V, WAIS-IV, or T.O.V.A.).
- Attenuated patients with 1 SD deficiency in the omission errors or variability domains of the T.O.V.A..
- A patient with a history of HSCT with successful engraftment.
- A patient who has received gene therapy treatment at any point.
- Unable to undergo lumbar puncture.
- A patient who is enrolled in another clinical study that involves clinical investigations or use of any investigational product (drug or device) within 4 months before obtaining informed consent.
- Unable to comply with the protocol as determined by the principal investigator or subinvestigator.
- Judged by the principal investigator or subinvestigator to be ineligible to participate in the study due to a history of serious drug allergy or sensitivity including anesthesia or hypersensitivity to any component of JR-141.
- A patient who has a known or suspected local or general infection or is at risk of abnormal bleeding due to medical conditions or therapies.
- A patient who has documented mutation of other genes, including loci adjacent to the IDS gene that are known to be associated with developmental delay, seizures, or other significant CNS disorders.
- A patient who has documented loss of activity of sulfatases other than IDS.
- A patient who has had a ventriculoperitoneal shunt placed or any other brain surgery, or has a clinically significant ventriculoperitoneal shunt malfunction within 30 days of screening.
- full time employee of the sponsor or research site personnel directly affiliated with this study or their immediate family members.
- A patient who otherwise is judged by the principle investigator or sub-investigator to be ineligible to participate in the study.
[Only in France]
- Persons deprived of their liberty by a judicial or administrative decision, according to article L.1121-6 the Public Health Code (Code de la santé publique), adults who are the subject of a measure of legal protection or unable to express their consent according to article L. 1121-8 of the Code de la santé publique)
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description JR-141 2.0 mg/kg/week JR-141 - administered as the standard of care: idursulfase (ELAPRASE®) Idursulfase standard of care-controlled study Rescue arm JR-141 or Idursulfase -
- Primary Outcome Measures
Name Time Method Change in levels of cerebrospinal fluid heparan sulfate from baseline (Cohort A) Baseline to Week 53 Change in the raw scores of cognitive testing measured from baseline (BSID-III) (Cohort A) Baseline to Week 105
- Secondary Outcome Measures
Name Time Method Relative change in distance walked using the 6-minute walk test from baseline to Week 53 (Cohort B) Baseline to Week 53 Change in the growth scores of cognitive testing measured from baseline (BSID-III) (Cohort A) Baseline to Week 105 Change in the age equivalent scores of adaptive behavior measured from baseline (VABS-II) (Cohort A) Baseline to Week 105 Relative change in liver volume relative to body weight from baseline (Cohort A and Cohort B) Baseline to Week 53 Relative change in spleen volume relative to body weight from baseline (Cohort A and Cohort B) Baseline to Week 53
Trial Locations
- Locations (27)
The Chaim Sheba Medical Center
🇮🇱Ramat Gan, Israel
UCSF Benioff Children's Hospital Oakland
🇺🇸Oakland, California, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Columbia University
🇺🇸New York City, New York, United States
Phoenix Children's Hospital
🇺🇸Phoenix, Arizona, United States
Ha'Emek Medical Center
🇮🇱Afula, Israel
University of North Carolina at Chapel Hill Medical School Wing E
🇺🇸Chapel Hill, North Carolina, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Hospital Universitario Austral
🇦🇷Buenos Aires, Argentina
Hospital de Clínicas de Porto Alegre
🇧🇷Porto Alegre, Brazil
Instituto de Medicina Integral Prof. Fernando Figueira - Imip
🇧🇷Recife, Brazil
Instituto de Genética e Erros Inatos do Metabolismo
🇧🇷São Paulo, Brazil
Fundación Cardio Infantil - Instituto de Cardiología
🇨🇴Bogotá, Colombia
Hôpital Femme Mère Enfant
🇫🇷Bron cedex, France
Chu De Montpellier Hopital Gui De Chauliac
🇫🇷Montpellier, France
Hôpital Armand Trousseau
🇫🇷Paris, France
Universitätsklinikum Giessen
🇩🇪Giessen, Germany
Universitätsklinikum Hamburg-Eppendorf
🇩🇪Hamburg, Germany
SphinCS GmbH
🇩🇪Hochheim, Germany
Universitätsmedizin Mainz
🇩🇪Mainz, Germany
Osp. Pediatrico Bambino Gesù, IRCCS
🇮🇹Rome, Italy
Uniwersytecki Szpital Dziecięcy
🇵🇱Kraków, Poland
Hospital Sant Joan de Déu
🇪🇸Barcelona, Spain
Hospital Universitario Virgen del Rocio
🇪🇸Seville, Spain
Gazi University Medicine Faculty Hospital
🇹🇷Ankara, Turkey
Great Ormond Street Hospital for Children NHS Trust - Metabolic Medicine
🇬🇧London, United Kingdom