Phase 1 C9orf72 Clinical Trials
2 recruitingPhase 1
What is a Phase 1 trial?
Phase 1 trials test a new treatment in a small group of people for the first time. The primary goal is to evaluate safety, determine a safe dosage range, and identify side effects. These studies typically enroll 20 to 80 participants.
Showing 1–2 of 2 trials
Not Yet Recruiting
Phase 1
A study to assess safety, tolerability and pharmacokinetics of RTX-010/101/111 for participants with C9orf72-mediated Amyotrophic Lateral Sclerosis (ALS). Part 1C adults with C9orf72-mediated ALS only.
C9orf72-mediated Amyotrophic Lateral Sclerosis (ALS)
Repeat Therapeutics Pty Ltd16 enrolled1 locationACTRN12626000385347
Recruiting
Phase 1Phase 2
A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 Genes
Frontotemporal DementiaFTDDementia, Frontotemporal+2 more
Passage Bio, Inc.30 enrolled10 locationsNCT04747431