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En fase III-studie av JR-141 hos pasienter med mukopolysakkaridose II

29. september 2026 oppdatert av: 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.

Studieoversikt

Status

Aktiv, ikke rekrutterende

Forhold

Intervensjon / Behandling

Studietype

Intervensjonell

Registrering (Faktiske)

86

Fase

  • Fase 3

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

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

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

  • Barn
  • Voksen
  • Eldre voksen

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

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

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Randomisert
  • Intervensjonsmodell: Parallell tildeling
  • Masking: Enkelt

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: JR-141 2,0 mg/kg/uke
Legemiddel: JR-141

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

standard of care-controlled study
Legemiddel: Idursulfase

IV infusion

Hva måler studien?

Primære resultatmål

Resultatmål
Tidsramme
Endring i nivåer av cerebrospinalvæske heparansulfat fra baseline (Kohort A)
Tidsramme: Grunnlinje til uke 53
Grunnlinje til uke 53
Endring i råskårene for kognitiv testing målt fra baseline (BSID-III) (Kohort A)
Tidsramme: Grunnlinje til uke 105
Grunnlinje til uke 105

Sekundære resultatmål

Resultatmål
Tidsramme
For andre regioner enn oss: Endring i aldersekvivalent score for kognitiv testing målt fra baseline (BSID-III eller KABC-II) (kohort a)
Tidsramme: Baseline til uke 105
Baseline til uke 105
For andre regioner enn oss: Endring i aldersekvivalent score til adaptiv atferd målt fra baseline (VABS-II) (kohort a)
Tidsramme: Baseline til uke 105
Baseline til uke 105
For USA: Endring i rå score til adaptiv atferd målt fra baseline (VABS-II) (kohort a)
Tidsramme: Baseline til uke 105
Baseline til uke 105
For alle regioner: Relativ endring i levervolum i forhold til kroppsvekt fra baseline (kohort A og kohort b)
Tidsramme: Baseline til uke 53
Baseline til uke 53
For alle regioner: Relativ endring i miltvolum i forhold til kroppsvekt fra baseline (kohort A og kohort b)
Tidsramme: Baseline til uke 53
Baseline til uke 53

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

JCR Pharmaceuticals Co., Ltd.

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

14. februar 2022

Primær fullføring (Antatt)

31. oktober 2027

Studiet fullført (Antatt)

31. oktober 2027

Datoer for studieregistrering

Først innsendt

17. september 2020

Først innsendt som oppfylte QC-kriteriene

25. september 2020

Først lagt ut (Faktiske)

5. oktober 2020

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

2. oktober 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

29. september 2026

Sist bekreftet

1. juni 2026

Mer informasjon

Begreper knyttet til denne studien

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • JR-141-GS31

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Ja

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

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