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New directions in clinical trials for frontotemporal lobar degeneration: Methods and outcome measures

Adam L Boxer1, Michael Gold2, Howard Feldman3

  • 1Memory and Aging Center, Department of Neurology, University of California San Francisco, San Francisco, CA, USA.

Alzheimer'S & Dementia : the Journal of the Alzheimer'S Association
|November 1, 2019
PubMed
Abstract

Insights

Frontotemporal lobar degeneration (FTLD) research faces challenges in clinical trials due to patient heterogeneity and rarity. Developing personalized endpoints and advanced data analysis tools is crucial for effective FTLD therapeutic development.

Area of Science:

  • Neurology
  • Neuroscience
  • Clinical Research

Background:

  • Frontotemporal lobar degeneration (FTLD) is the most prevalent dementia in individuals under 60.
  • Numerous therapeutics targeting FTLD syndromes are currently under development.

Purpose of the Study:

  • To address challenges in conducting clinical trials for Frontotemporal lobar degeneration (FTLD).
  • To discuss advances in the clinical science of FTLD.

Main Methods:

  • Convened the Frontotemporal Degeneration Study Group meeting in March 2018.
  • Discussed advances in FTLD clinical science.

Main Results:

  • Clinical trials for FTLD face significant challenges, including syndrome heterogeneity and rare disease patient recruitment.
  • Heterogeneity complicates the measurement of treatment effects.
  • Rarity poses recruitment difficulties.

Conclusions:

  • Developing personalized endpoints that are clinically meaningful is essential.
  • Personalized approaches using MRI, fluid biomarkers, and wearable technology can enhance FTLD trial power.
  • A computational platform for analyzing combined data modalities is critical for future FTLD research.
Keywords:
ARTFLBiomarkerC9orf72Clinical trialFTDFTLDFrontotemporal dementiaFrontotemporal lobar degenerationGRNLEFFTDSMAPTPrimary progressive aphasiaProgressive supranuclear palsy

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