A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)
4. september 2026 oppdatert av: JCR Pharmaceuticals Co., Ltd.
A Global, Open-Label, Phase I/II Trial, to Evaluate Safety, Tolerability, Pharmacodynamic and Preliminary Efficacy of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)
This is a global, open-label, Phase I/II, interventional trial in participants younger than 6 years of age with Mucopolysaccharidosis Type IIIB (MPS IIIB), designed to assess the safety and tolerability of JR-446, determine its pharmacodynamic effects, and explore its potential to demonstrate early clinical effects on disease-relevant outcomes.
Studieoversikt
Status
Har ikke rekruttert ennå
Forhold
Intervensjon / Behandling
Studietype
Intervensjonell
Registrering (Antatt)
12
Fase
- Fase 2
- Fase 1
Kontakter og plasseringer
Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.
Studiekontakt
- Navn: JCR Pharmaceuticals Co., Ltd.
- Telefonnummer: +81-797-32-8582
- E-post: clinical_development@jp.jcrpharm.com
Studiesteder
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California
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Oakland, California, Forente stater, 94609
- Children's Hospital Oakland Research Institution, Attn: 2900 Wing
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Ta kontakt med:
- Irene Chang
- Telefonnummer: 415-476-2757
- E-post: Irene.chang@ucsf.edu
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Pennsylvania
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Pittsburgh, Pennsylvania, Forente stater, 15224
- UPMC Children's Hospital of Pittsburgh
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Ta kontakt med:
- Deepa Rajan
- Telefonnummer: 412-692-6787
- E-post: rajands@upmc.edu
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Virginia
-
Fairfax, Virginia, Forente stater, 22030
- Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)
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Ta kontakt med:
- Ozlem Goker-Alpan
- Telefonnummer: 703-261-6220
- E-post: ogoker-alpan@ldrtc.org
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-
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-
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London, Storbritannia, WC1N 3JH
- Great Ormond Street Hospital for Children
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Ta kontakt med:
- James Davison
- Telefonnummer: 0074 0207 405 9200
- E-post: james.davison@gosh.nhs.uk
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-
-
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Hamburg, Tyskland, 20246
- University Medical Center Hamburg-Eppendorf
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Ta kontakt med:
- Nicole Maria Muschol
- Telefonnummer: +49 (0) 40 7410 53714
- E-post: muschol@uke.de
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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
Tar imot friske frivillige
Nei
Beskrivelse
Inclusion Criteria:
- Patients with confirmed MPS IIIB with a phenotype of MPS IIIB that is not classified as slowly progressive (i.e., not attenuated).
- A chronological age <6 years.
- Ability to complete and achieve a Standard score ≥55, using either Bailey Scale of Infant Development-4 (BSID-4) Cognitive domain or Kaufman Assessment Battery for Children-II (KABC-II) Non-Verbal Index, whichever tool is most appropriate for the participant's chronological age.
Exclusion Criteria:
- Contraindication for lumbar puncture or MRI.
- A participant who has a medical condition or extenuating circumstance that, in the opinion of the principal investigator or sub-investigator, might compromise the participant's ability to comply with protocol requirements, the participant's well-being or safety, or the interpretability of the participant's clinical data.
- A participant who has received any other investigational drug product (including but not limited to, tralesinidase alfa enzyme replacement therapy [TA-ERT], Genistein, KINERET [anakinra], ambroxol, miglustat) within 4 months (or 5 half-lives, whichever is longer) before the time of providing informed consent.
- A participant who has received gene therapy treatment or hematopoietic stem cell transplantation (HSCT) with successful engraftment.
- Serious drug allergy or hypersensitivity to any components of JR-446 or medications likely prescribed during the trial.
- A participant has a 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.
- A patient with recurrent epileptic seizures not adequately controlled with anti-seizure medication, and which, in the clinical judgment of the principal investigator, would preclude safe participation in the trial.
- Serology consistent with human immunodeficiency virus (HIV) exposure or consistent with active hepatitis B (HepB) or hepatitis C (HepC) infection.
- A participant/family, who, in the opinion of the investigator, may not be able to comply with protocol requirements and cooperate fully with the trial assessments, procedures, and scheduling for JR-446 IMP dose administrations.
- Study participants for whom informed consent is unable to be provided by a parent or legal guardian; or when applicable for a study participant who is unable to provide assent with respect to study participation in conjunction with parental or legal guardian consent for participation on study.
The above information is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial.
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: N/A
- Intervensjonsmodell: Enkeltgruppeoppdrag
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
|
Eksperimentell: JR-446
Arm: 1
|
IV infusjon
|
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
To establish the safety and tolerability of JR-446 in MPS IIIB patients following repeated dose administration
Tidsramme: Up to 1 year (multiple visits)
|
Incidence and severity of treatment-emergent adverse events; Incidence and severity of infusion-associated reactions; Incidence of clinically significant changes/abnormalities in safety laboratory tests, vital signs, and electrocardiogram.
|
Up to 1 year (multiple visits)
|
Sekundære resultatmål
Resultatmål |
Tidsramme |
|---|---|
|
Change From Baseline in Cerebrospinal Fluid Heparan Sulfate Concentration
Tidsramme: Through Week 53 (up to 1 year; multiple visits)
|
Through Week 53 (up to 1 year; multiple visits)
|
|
Absolute Change From Baseline in Serum Heparan Sulfate Concentration
Tidsramme: Through Week 53 (up to 1 year; multiple visits)
|
Through Week 53 (up to 1 year; multiple visits)
|
|
Percent Change From Baseline in Serum Heparan Sulfate Concentration
Tidsramme: Through Week 53 (up to 1 year; multiple visits)
|
Through Week 53 (up to 1 year; multiple visits)
|
|
Absolute Change From Baseline in Urine Heparan Sulfate Concentration
Tidsramme: Through Week 53 (up to 1 year; multiple visits)
|
Through Week 53 (up to 1 year; multiple visits)
|
|
Percent Change From Baseline in Urine Heparan Sulfate Concentration
Tidsramme: Through Week 53 (up to 1 year; multiple visits)
|
Through Week 53 (up to 1 year; multiple visits)
|
|
Absolute Change From Baseline in Serum Neurofilament Light Chain Concentration
Tidsramme: Through Week 53 (up to 1 year; multiple visits)
|
Through Week 53 (up to 1 year; multiple visits)
|
|
Percent Change From Baseline in Serum Neurofilament Light Chain Concentration
Tidsramme: Through Week 53 (up to 1 year; multiple visits)
|
Through Week 53 (up to 1 year; multiple visits)
|
|
Change in Cognitive Function Assessments From Baseline
Tidsramme: Through Week 53 (up to 1 year)
|
Through Week 53 (up to 1 year)
|
|
Change in Brain MRIs From Baseline
Tidsramme: Through Week 53 (up to 1 year)
|
Through Week 53 (up to 1 year)
|
|
Change in Height Standard Deviation Score (SDS) From Baseline (using WHO child growth standards)
Tidsramme: Through Week 53 (up to 1 year)
|
Through Week 53 (up to 1 year)
|
|
Change in Weight SDS From Baseline (using WHO child growth standards)
Tidsramme: Through Week 53 (up to 1 year)
|
Through Week 53 (up to 1 year)
|
|
Change in Body Mass Index (BMI) SDS From Baseline (using WHO child growth standards)
Tidsramme: Through Week 53 (up to 1 year)
|
Through Week 53 (up to 1 year)
|
Samarbeidspartnere og etterforskere
Det er her du vil finne personer og organisasjoner som er involvert i denne studien.
Sponsor
Samarbeidspartnere
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 (Antatt)
9. september 2026
Primær fullføring (Antatt)
30. juni 2028
Studiet fullført (Antatt)
30. september 2028
Datoer for studieregistrering
Først innsendt
14. mai 2026
Først innsendt som oppfylte QC-kriteriene
8. juni 2026
Først lagt ut (Faktiske)
11. juni 2026
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
8. september 2026
Siste oppdatering sendt inn som oppfylte QC-kriteriene
4. september 2026
Sist bekreftet
1. juli 2026
Mer informasjon
Begreper knyttet til denne studien
Andre studie-ID-numre
- JR-446-201
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
NEI
Legemiddel- og utstyrsinformasjon, studiedokumenter
Studerer et amerikansk FDA-regulert medikamentprodukt
Ja
Studerer et amerikansk FDA-regulert enhetsprodukt
Nei
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