Clinical Trials

Presently, there are no approved disease-modifying treatments for FTD, though a number of clinical trials are underway. These clinical trials are led by various drug sponsors, and a subset of FPI clinical sites are enrolling patients into these studies. The active treatment trials targeting genetic forms of FTD and/or C9orf72-related ALS are summarized below:

	Sponsor	Drug	Phase	Status	ClinicalTrials.gov ID
*C9ORF72*
Clinical presentation of FTD or FTD-ALS
	Passage Bio, Inc.	PBFT02	Phase 1/2	Active, not recruiting	NCT04747431
	University of Florida	Metformin	Phase 2	Active, not recruiting	NCT04220021
	Alector Inc.	AL001	Phase 2	Completed	NCT03987295
	Transposon Therapeutics, Inc.	TPN-101	Phase 2	Completed	NCT04993755
	Wave Life Sciences USA, Inc.	WVE-004	Phase 1/2	Terminated	NCT04931862
Clinical presentaiton of ALS only
	NINDS	Acamprosate	Phase 1	Active, not recruiting	NCT07204977
	QurAlis Corporation	QRL-201	Phase 1	Active, not recruiting	NCT05633459
	Alector Inc.	AL001	Phase 2	Terminated	NCT05053035
	Biogen	BIIB078	Phase 1	Completed	NCT03626012
	OrphAI Therapeutics	LAM-002A	Phase 2	Completed	NCT05163886
*GRN*
	AviadoBio Ltd	AVB-101	Phase 1/2	Recruiting	NCT06064890
	Denali Therapeutics, Inc.	DNL593	Phase 1/2	Active, not recruiting	NCT05262023
	Passage Bio, Inc.	PBFT02	Phase 1/2	Active, not recruiting	NCT04747431
	Prevail Therapeutics	LY3884963	Phase 1/2	Active, not recruiting	NCT04408625
	Alector Inc.	AL001	Phase 1	Completed	NCT03636204
	Alector Inc.	AL001	Phase 2	Completed	NCT03987295
	Alector Inc.	AL001	Phase 3	Terminated	NCT04374136
	University of California, San Francisco	Nimodipine	Phase 1	Completed	NCT01835665
	Vesper Biotechnologies ApS	VES001	Phase 1	Completed	NCT06226064
	Vesper Biotechnologies ApS	VES001	Phase 1	Completed	NCT06705192
	FORUM Pharmaceuticals Inc.	FRM-0334	Phase 2	Completed	NCT02149160
*MAPT*
	There are currently no trials with enrollment specifically targeting MAPT mutation carriers. This table will be updated as opportunities become available.
	University of California, San Francisco	Gosuranemab	Phase 1	Terminated	NCT03658135

1) This table only includes interventional trials in which recruitment specifically targeted individuals with C9ORF72, GRN, or MAPT
mutations. For a complete list of FTD-related trials that recruit sporadic forms, individuals with specific FTD-related clinical diagnoses, individuals with specific FTD-related pathologies, or other mutation types, please refer to clinicaltrials.gov.
2) This table does not include extension studies.
3) Information included in this table was generated from clinicaltrials.gov and is current as of August 27, 2026. For the most recent updates, please visit the clinicaltrials.gov ID links provided in the table.