A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)
NCT ID: NCT04573023
Last Updated: 2026-01-21
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
86 participants
INTERVENTIONAL
2022-02-14
2027-10-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
SINGLE
Study Groups
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JR-141 2.0 mg/kg/week
JR-141
IV infusion, 2.0 mg/kg/week
administered as the standard of care: idursulfase (ELAPRASE®)
standard of care-controlled study
Idursulfase
IV infusion
Rescue arm
JR-141 or Idursulfase
The subjects who have achieved the pre-specified criteria\* are able to change the drug.
\*If a subject in Idursulfase group shows decline in their neurocognitive outcome, idursulfase can be switched to JR-141.
If a subject in JR-141 group shows decline in their peripheral outcome, JR-141 will be switched to idursulfase.
Interventions
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JR-141
IV infusion, 2.0 mg/kg/week
Idursulfase
IV infusion
JR-141 or Idursulfase
The subjects who have achieved the pre-specified criteria\* are able to change the drug.
\*If a subject in Idursulfase group shows decline in their neurocognitive outcome, idursulfase can be switched to JR-141.
If a subject in JR-141 group shows decline in their peripheral outcome, JR-141 will be switched to idursulfase.
Eligibility Criteria
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Inclusion Criteria
* 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..
* Patients or patients whose female partners are of child-bearing potential i.e., fertile, following menarche and until becoming post-menopausal unless permanently sterile, agree to use a medically accepted, highly effective method of contraception, from the time of informed consent. The method of contraception must be used during the study until 90 days for male subjects, and 30 days for female subjects after the final study intervention administration.
* For subjects with hearing impairment requiring hearing aid(s), every effort has been made to encourage compliance with the use of functioning hearing aid(s) before baseline neurocognitive assessments, and parent/legally acceptable representative or subject agrees to encourage wearing them during the study and on neurocognitive testing days.
Exclusion Criteria
* A patient who has received gene therapy treatment at any point.
* A patient who is judged by the principal investigator or sub-investigator as being unable to undergo lumbar puncture, including those who have difficulties in taking position for lumbar puncture due to joint contracture or those who are likely to experience breathing difficulties during the lumbar puncture process.
* 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.
* A patient who is 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.
* The subject has a positive pregnancy test or is breastfeeding at screening or randomization.
\[Only in France\]
* 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)
ALL
No
Sponsors
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JCR Pharmaceuticals Co., Ltd.
INDUSTRY
Responsible Party
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Locations
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Phoenix Children's Hospital
Phoenix, Arizona, United States
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, New York, United States
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, , 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
Höchheim, , Germany
Universitätsmedizin Mainz
Mainz, , Germany
Ha'Emek Medical Center
Afula, , Israel
The Chaim Sheba Medical Center
Ramat Gan, , Israel
Fondazione IRCCS San Gerardo dei Tintori
Monza, , Italy
Osp. Pediatrico Bambino Gesù, IRCCS
Rome, , Italy
Uniwersytecki Szpital Dziecięcy
Krakow, , Poland
Hospital Sant Joan de Déu
Barcelona, , Spain
Hospital Universitario Virgen del Rocio
Seville, , Spain
Gazi University Medicine Faculty Hospital
Ankara, , Turkey (Türkiye)
Ege University Children Hospital
Izmir, , Turkey (Türkiye)
Great Ormond Street Hospital for Children NHS Trust - Metabolic Medicine
London, , United Kingdom
Countries
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Other Identifiers
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JR-141-GS31
Identifier Type: -
Identifier Source: org_study_id
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