FTD: Clinical trials

AD-Related Dementias Focus

Accelerate the evaluation of novel FTD treatments by developing new clinical trial resources and FTD-specific designs, and by conducting new prevention and treatment trials.

2027 RFA-NS-19-014: Center without Walls for PET Ligand Development for Alzheimer's disease related dementias (ADRDs) (U19) RFA-NS-22-056: Treatments for Lewy Body Dementias and Frontotemporal Dementias--Exploratory Clinical Trial (U01 Clinical Trial Required) 9.S In Progress Develop novel blood/CSF biomarkers or PET ligands for FTLD-tau, -TDP or -FUS during life DN 2019 ADRD Summit: Frontotemporal Lobar Degeneration (FTD), Focus Area 3: Clinical Science, Milestone 5 2022 ADRD Summit: Frontotemporal Lobar Degeneration (FTD), Milestone 3, Priority 3 Biomarkers Center without Walls for PET Ligand Development for Alzheimer's disease related dementias (ADRDs) Veri-T: A phase 1 Placebo-Controlled Trial of Verdiperstat in Semantic Variant Primary Progressive Aphasia Due to Underlying FTLD-TDP Cerebellar TMS for postural instability in progressive supranuclear palsy

  • Initiate at least one sporadic FTD clinical trial in a real-world clinical setting.
  • Develop or leverage an existing research registry for FTD clinical studies that includes underserved and minority group representation reflecting population demographics.
  • Develop standardized protocols for prevention studies of familial FTD syndromes or clinical trials in symptomatic, sporadic, and familial FTD.
  • Initiate at least one clinical trial of novel FTD therapeutics.

Summary of Key Accomplishments

The FTD Disorders Registry is a contact and research registry supported by patient advocacy groups and is open to all with particular interest in including underserved and minority groups and is well-integrated into the large ALLFTD natural history study (AG063911). The development of standardized protocols is proceeding also through the ALLFTD natural history study and collaborating FTD Prevention Initiative (FPI - https://www.thefpi.org/ ) with the refinement of outcome measures and the establishment of which of these outcome measures are most sensitive and useful for which subtype of FTD. The NIH also continues to support clinical trials in FTD, particularly early stage studies looking at novel interventions such as neuromodulation including transcranial direct stimulation and small molecules to target toxic reactive oxygen species.

The key accomplishments summary is current as of July 2022.