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A Plataforma de Ensaios Clínicos para Paralisia Supranuclear Progressiva - Regime B: LM11A-31

4 de novembro de 2026 atualizado por: Adam Boxer

A Plataforma de Ensaios Clínicos de Paralisia Supranuclear Progressiva - 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.

Visão geral do estudo

Status

Inscrevendo-se por convite

Descrição detalhada

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.

Tipo de estudo

Intervencional

Inscrição (Estimado)

147

Estágio

  • Fase 2

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Locais de estudo

    • California
      • San Francisco, California, Estados Unidos, 94158
        • University of California, San Francisco (UCSF), Memory and Aging Center

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

41 anos a 86 anos (Adulto, Adulto mais velho)

Aceita Voluntários Saudáveis

Não

Gêneros Elegíveis para o Estudo

Tudo

Descrição

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.

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

Como o estudo é projetado?

Detalhes do projeto

  • Finalidade Principal: Tratamento
  • Alocação: Randomizado
  • Modelo Intervencional: Atribuição Paralela
  • Mascaramento: Quadruplicar

Armas e Intervenções

Grupo de Participantes / Braço
Intervenção / Tratamento
Comparador de Placebo: Placebo correspondente

Matching placebo is administered orally twice daily.

Experimental: Regime B: LM11A-31

LM11A-31 is administered orally twice daily.

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Disease progression
Prazo: 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

Medidas de resultados secundários

Medida de resultado
Descrição da medida
Prazo
Disease progression
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 52 weeks

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

52 weeks
Neurodegeneration
Prazo: 52 weeks

Change in plasma neurofilament light chain (NfL) concentration.

52 weeks

Colaboradores e Investigadores

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Patrocinador

Investigadores

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

Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Real)

29 de julho de 2026

Conclusão Primária (Estimado)

28 de setembro de 2030

Conclusão do estudo (Estimado)

28 de setembro de 2030

Datas de inscrição no estudo

Enviado pela primeira vez

24 de novembro de 2025

Enviado pela primeira vez que atendeu aos critérios de CQ

24 de novembro de 2025

Primeira postagem (Real)

4 de dezembro de 2025

Atualizações de registro de estudo

Última Atualização Postada (Real)

5 de novembro de 2026

Última atualização enviada que atendeu aos critérios de controle de qualidade

4 de novembro de 2026

Última verificação

1 de novembro de 2026

Mais Informações

Termos relacionados a este estudo

Plano para dados de participantes individuais (IPD)

Planeja compartilhar dados de participantes individuais (IPD)?

SIM

Tipo de informação de suporte de compartilhamento de IPD

  • PROTOCOLO DE ESTUDO

Informações sobre medicamentos e dispositivos, documentos de estudo

Estuda um medicamento regulamentado pela FDA dos EUA

Sim

Estuda um produto de dispositivo regulamentado pela FDA dos EUA

Não

Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .

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