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Den Progressive Supranukleære Parese Kliniske Forsøgsplatform - Regime B: LM11A-31

4. november 2026 opdateret af: Adam Boxer

Det Progressive Supranukleære Parese Kliniske Forsøgsplatform - 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.

Studieoversigt

Status

Tilmelding efter invitation

Detaljeret 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.

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

147

Fase

  • Fase 2

Kontakter og lokationer

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

Studiesteder

    • California
      • San Francisco, California, Forenede Stater, 94158
        • University of California, San Francisco (UCSF), Memory and Aging Center

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

41 år til 86 år (Voksen, Ældre voksen)

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

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

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: Firedobbelt

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Placebo komparator: Matchende placebo

Matching placebo is administered orally twice daily.

Eksperimentel: Regimen B: LM11A-31

LM11A-31 is administered orally twice daily.

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
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
Foranstaltningsbeskrivelse
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

Samarbejdspartnere og efterforskere

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

Sponsor

Efterforskere

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

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)

29. juli 2026

Primær færdiggørelse (Anslået)

28. september 2030

Studieafslutning (Anslået)

28. september 2030

Datoer for studieregistrering

Først indsendt

24. november 2025

Først indsendt, der opfyldte QC-kriterier

24. november 2025

Først opslået (Faktiske)

4. december 2025

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

5. november 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

4. november 2026

Sidst verificeret

1. november 2026

Mere information

Begreber relateret til denne undersøgelse

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

JA

IPD-deling Understøttende informationstype

  • STUDY_PROTOCOL

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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