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Den progressive supranukleære parese kliniske studieplattform - Regime B: LM11A-31

4. november 2026 oppdatert av: Adam Boxer

Den progressive supranukleære parese kliniske studieplattformen - Regime B: LM11A-31

The Progressive Supranuclear Palsy Clinical Trial Platform (PTP) is a multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of PSP. Regimen B will evaluate the safety and efficacy of a single study drug, LM11A-31, in participants with PSP.

Studieoversikt

Status

Påmelding etter invitasjon

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

The Progressive Supranuclear Palsy Clinical Trial Platform (PTP) is designed as a perpetual platform trial. This means that there is a single Master Protocol dictating the conduct of the trial. The PTP Master Protocol is registered as NCT07173803. Once a participant enrolls into the Master Protocol and meets all eligibility criteria, the participant will be eligible to be randomized into any currently enrolling regimen. All participants will have an equal chance to be randomized to all regimens that are active at the time of screening. If a participant is randomized to Regimen B: LM11A-31, participants will complete a baseline assessment and be randomized in a 3:1 ratio to either active LM11A-31 or matching placebo. Participants must first enroll into the Master Protocol and be eligible to participate in the Master Protocol before being able to be randomly assigned to Regimen B. For a list of enrolling sites, please see the PTP Master Protocol under NCT07173803.

Studietype

Intervensjonell

Registrering (Antatt)

147

Fase

  • Fase 2

Kontakter og plasseringer

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

Studiesteder

    • California
      • San Francisco, California, Forente stater, 94158
        • University of California, San Francisco (UCSF), Memory and Aging Center

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

41 år til 86 år (Voksen, Eldre voksen)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Alle

Beskrivelse

Inclusion Criteria:

  1. Clinical diagnosis of possible or probable PSP Richardson's Syndrome as defined by the 2017 Movement Disorder Society (MDS) criteria.
  2. Presence of PSP symptoms for ≤5 years at screening (based on the best judgment of the site PI).
  3. Mini-Mental State Examination (MMSE) score at screening of ≥25.
  4. Able to walk at least 10 steps with minimal assistance (e.g., one arm for safety, but not postural support).
  5. Stable doses of permitted medications as described per protocol for 30 days prior to screening.
  6. Resides at home or in the community (assisted living is acceptable).
  7. As assessed by the site PI, participant is likely to be able to comply with the protocol for the duration of the study, and has adequate vision, hearing (hearing aid permitted), and literacy (English or Spanish) sufficient for compliance with the required testing procedures.

Exclusion Criteria:

  1. Females who are breastfeeding or pregnant (as documented by a urine pregnancy test) during screening, or plan to become pregnant during the study.
  2. Females of childbearing potential who did not use a highly effective method of contraception within 28 days of screening and/or are not willing to use a highly effective method of contraception for the duration of their participation in the study.
  3. Lacks good venous access such that multiple blood draws would be precluded.
  4. Weighs less than 40kg, or more than 136kg at screening.
  5. Blood transfusion within 4 weeks of screening.
  6. Contraindications to MRI studies, including metal (ferromagnetic) implants, a cardiac pacemaker that is not compatible with MRI, and/or severe claustrophobia.
  7. Screening MRI scan showing structural evidence of alternative pathology not consistent with PSP that could explain a substantial portion of the participant's symptoms as indicated by the central MRI read.
  8. Any unstable and/or clinically significant medical condition likely to hamper the evaluation of safety and/or efficacy of study drug (e.g., clinically significant reduction in serum B12 or folate levels, clinically significant abnormalities of thyroid function, stroke, or other cerebrovascular or cardiovascular conditions), as per the site PI's judgment.
  9. History of severe allergic reaction (e.g., anaphylaxis) including but not limited to: severe allergic reaction to previous vaccines, foods, and/or medications.
  10. Hospitalization within 30 days prior to screening or baseline.
  11. Infections or major surgical procedures within 3 months prior to screening, judged to be clinically significant by the site PI.
  12. Myocardial infarction within 1 year prior to baseline, unstable angina pectoris, symptomatic congestive heart failure.
  13. History of cancer within the past 5 years other than treated skin squamous cell carcinoma, basal cell carcinoma, and melanoma in-situ, localized prostate cancer not requiring treatment, or prostate or breast cancer, which have been fully removed and are considered cured.
  14. History or presence of immunological or inflammatory conditions, including neurological disorders, meningitis or meningoencephalitis.
  15. History or presence of epilepsy requiring ongoing use of antiepileptic medications. Antiepileptic medications are permitted for pain or psychiatric use per the protocol.
  16. DSM-5 criteria for drug or alcohol abuse or dependence currently met within the past 5 years.
  17. Clinically significant abnormal vital signs including sustained sitting blood pressure >160/100 mm Hg.
  18. Diabetes mellitus with hemoglobin A1c (HbA1c) levels of ≥8.0%.
  19. Known history of human immunodeficiency virus (HIV-1 or 2).
  20. Known history of acute/chronic hepatitis B or C unless treated curatively.

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: Firemannsrom

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Placebo komparator: Matchende placebo

Matching placebo is administered orally twice daily.

Eksperimentell: Regimen B: LM11A-31

LM11A-31 is administered orally twice daily.

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Disease progression
Tidsramme: 52 weeks

Change in disease severity as measured by the 15-item modified Progressive Supranuclear Palsy Rating Scale (mPSPRS-15) in which the minimum score is 0 and the maximum score is 52, with higher scores indicating a worse outcome.

52 weeks

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Disease progression
Tidsramme: 52 weeks

Change in disease severity as measured by the 10-item modified Progressive Supranuclear Palsy Rating Scale (mPSPRS-10) in which the minimum score is 0 and the maximum score is 30, with higher scores indicating a worse outcome.

52 weeks
Disease progression
Tidsramme: 52 weeks

Change in disease severity as measured by the 28-item Progressive Supranuclear Palsy Rating Scale (28-item PSPRS) in which the minimum score is 0 and the maximum score is 100, with higher scores indicating a worse outcome.

52 weeks
Experiences of daily living
Tidsramme: 52 weeks

Change in experiences of daily living over time as measured by the Cortical Basal ganglia Functional Scale (CBFS) in which the minimum score is 0 and the maximum score is 124, with higher scores indicating a worse outcome.

52 weeks
Activities of daily living
Tidsramme: 52 weeks

Change in activities of daily living over time as measured by the Schwab and England Activities of Daily Living Scale (SE-ADL) in which the minimum score is 0% and the maximum score is 100%, with lower scores indicating a worse outcome.

52 weeks
Disease severity
Tidsramme: 52 weeks

Change in disease severity over time as measured by the Clinical Global Impression of disease severity (CGIds) using a 7-point scale, ranging from 1 (normal, not ill) to 7 (extremely ill), with a higher score indicating a worse outcome.

52 weeks
Disease progression
Tidsramme: 52 weeks

Change in disease severity over time as measured by the Clinical Global Impression of Change (CGIC) using a 7-point scale, ranging from 1 (very much improved) to 7 (very much worse), with a score of 4 indicating no change.

52 weeks
Health-related quality of life
Tidsramme: 52 weeks

Change in quality of life over time as measured by the EuroQoL 5 Dimension - 5 Level (EQ-5D-5L) questionnaire in which the minimum score is 0 and the maximum score is 1, with lower scores indicating a worse outcome.

52 weeks
Brain volume
Tidsramme: 52 weeks

Change in volume in midbrain, pontine and other regions over time as measured by volumetric MRI.

52 weeks
Neurodegeneration
Tidsramme: 52 weeks

Change in plasma neurofilament light chain (NfL) concentration.

52 weeks

Samarbeidspartnere og etterforskere

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

Sponsor

Samarbeidspartnere

Etterforskere

  • Hovedetterforsker: Adam Boxer, MD, PhD, University of California, San Francisco
  • Hovedetterforsker: Anne-Marie Wills, MD, Massachusetts General Hospital
  • Hovedetterforsker: Irene Litvan, MD, University of California, San Diego
  • Hovedetterforsker: Julio Rojas-Martinez, MD, PhD, University of California, San Francisco

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)

29. juli 2026

Primær fullføring (Antatt)

28. september 2030

Studiet fullført (Antatt)

28. september 2030

Datoer for studieregistrering

Først innsendt

24. november 2025

Først innsendt som oppfylte QC-kriteriene

24. november 2025

Først lagt ut (Faktiske)

4. desember 2025

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

5. november 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

4. november 2026

Sist bekreftet

1. november 2026

Mer informasjon

Begreper knyttet til denne studien

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

JA

IPD-deling Støtteinformasjonstype

  • STUDY_PROTOCOL

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. .