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En fase III undersøgelse af JR-141 i patienter med mucopolysaccharidosis II

29. september 2026 opdateret af: JCR Pharmaceuticals Co., Ltd.

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.

Studieoversigt

Status

Aktiv, ikke rekrutterende

Betingelser

Intervention / Behandling

Undersøgelsestype

Interventionel

Tilmelding (Faktiske)

86

Fase

  • Fase 3

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiesteder

  • Argentina
      • Buenos Aires, Argentina
        • Hospital Universitario Austral
  • Brasilien
      • Porto Alegre, Brasilien
        • Hospital de Clínicas de Porto Alegre
      • São Paulo, Brasilien
        • Instituto de Genética e Erros Inatos do Metabolismo
  • Colombia
      • Bogotá, Colombia
        • Fundación Cardio Infantil - Instituto de Cardiología
  • Det Forenede Kongerige
      • London, Det Forenede Kongerige
        • Great Ormond Street Hospital for Children NHS Trust - Metabolic Medicine
  • Forenede Stater
    • Arizona
      • Phoenix, Arizona, Forenede Stater, 85016
        • Phoenix Children's Hospital
    • California
      • Oakland, California, Forenede Stater, 94609
        • UCSF Benioff Children's Hospital Oakland
    • Illinois
      • Chicago, Illinois, Forenede Stater, 60611
        • Ann & Robert H. Lurie Children's Hospital of Chicago
    • Minnesota
      • Minneapolis, Minnesota, Forenede Stater, 55455
        • University of Minnesota
    • New York
      • New York, New York, Forenede Stater, 10032
        • Columbia University Irving Medical Center
    • North Carolina
      • Chapel Hill, North Carolina, Forenede Stater, 27514
        • University of North Carolina at Chapel Hill Medical School Wing E
    • Pennsylvania
      • Philadelphia, Pennsylvania, Forenede Stater, 19104
        • Children's Hospital of Philadelphia
  • Frankrig
      • Lyon, Frankrig
        • Hôpital Femme Mère Enfant
      • Montpellier, Frankrig
        • Chu De Montpellier Hopital Gui De Chauliac
      • Paris, Frankrig
        • Hôpital Armand Trousseau
  • Israel
      • Afula, Israel, 1834111
        • Clalit Health Services Ha'Emek Medical Center
      • Ramat Gan, Israel, 6265601
        • Sheba Medical Center
  • Italien
      • Rome, Italien
        • Osp. Pediatrico Bambino Gesù, IRCCS
  • Polen
      • Krakow, Polen
        • Uniwersytecki Szpital Dziecięcy
  • Spanien
      • Barcelona, Spanien
        • Hospital Sant Joan de Déu
  • Tyrkiet (Türkiye)
      • Ankara, Tyrkiet (Türkiye)
        • Gazi University Medicine Faculty Hospital
      • Izmir, Tyrkiet (Türkiye)
        • Ege University Children Hospital
  • Tyskland
      • Giessen, Tyskland
        • Universitätsklinikum Giessen
      • Hamburg, Tyskland
        • Universitätsklinikum Hamburg-Eppendorf
      • Höchheim, Tyskland
        • SphinCS GmbH
      • Mainz, Tyskland
        • Universitätsmedizin Mainz

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Barn
  • Voksen
  • Ældre voksen

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

Alle

Beskrivelse

Inclusion Criteria:

  • 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..
  • 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 with a history of HSCT with successful engraftment.
  • 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)

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Enkelt

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: JR-141 2,0 mg/kg/uge
Medicin: JR-141

IV infusion, 2.0 mg/kg/week
Andet: Redningsarm
Medicin: 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.
Andet: administered as the standard of care: idursulfase (ELAPRASE®)

standard of care-controlled study
Medicin: Idursulfase

IV infusion

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Tidsramme
Ændring i niveauer af cerebrospinalvæske heparansulfat fra baseline (kohorte A)
Tidsramme: Baseline til uge 53
Baseline til uge 53
Ændring i de rå scorer for kognitive tests målt fra baseline (BSID-III) (Kohorte A)
Tidsramme: Baseline til uge 105
Baseline til uge 105

Sekundære resultatmål

Resultatmål
Tidsramme
For andre regioner end os: Ændring i aldersækvivalente scoringer af kognitiv test målt fra baseline (BSID-III eller KABC-II) (kohort A)
Tidsramme: Baseline til uge 105
Baseline til uge 105
For andre regioner end os: Ændring i aldersækvivalente scoringer af adaptiv adfærd målt fra baseline (VABS-II) (kohort A)
Tidsramme: Baseline til uge 105
Baseline til uge 105
For USA: Ændring i de rå scoringer af adaptiv adfærd målt fra baseline (VABS-II) (kohort A)
Tidsramme: Baseline til uge 105
Baseline til uge 105
For alle regioner: Relativ ændring i levervolumen i forhold til kropsvægt fra baseline (kohort A og kohort B)
Tidsramme: Baseline til uge 53
Baseline til uge 53
For alle regioner: Relativ ændring i miltvolumen i forhold til kropsvægt fra baseline (kohort A og kohort B)
Tidsramme: Baseline til uge 53
Baseline til uge 53

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

JCR Pharmaceuticals Co., Ltd.

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

14. februar 2022

Primær færdiggørelse (Anslået)

31. oktober 2027

Studieafslutning (Anslået)

31. oktober 2027

Datoer for studieregistrering

Først indsendt

17. september 2020

Først indsendt, der opfyldte QC-kriterier

25. september 2020

Først opslået (Faktiske)

5. oktober 2020

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

2. oktober 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

29. september 2026

Sidst verificeret

1. juni 2026

Mere information

Begreber relateret til denne undersøgelse

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • JR-141-GS31

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

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