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Study identifier: NCT04993755 Synced from ClinicalTrials.gov · July 28, 2026
● Completed

A Phase 2a Study of TPN-101 in Patients With C9ORF72 ALS/FTD

Condition: Amyotrophic Lateral Sclerosis · Frontotemporal Dementia  ·  Sponsor: Transposon Therapeutics, Inc.

PhasePhase 2
Planned participants42
Who can joinAll sexes, 18 Years to no upper limit
Healthy volunteersNo

About this study

This is a Phase 2a study to assess the the safety and tolerability of TPN-101 in patients with Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD) Associated with Hexanucleotide Repeat Expansion in the C9orf72 gene (C9ORF72 ALS/FTD).

This description comes directly from the study's public registry record.

Talk to the study team

Contact details are listed per location below or on the registry record.

Always discuss trial participation with your own doctor first.

Locations (19)

University of California San DiegoLa Jolla, California, United States
University of California Irvine - ALS & Neuromuscular CenterOrange, California, United States
UCSF Neurosciences Clinical Research Unit (NCRU)San Francisco, California, United States
John Hopkins UniversityBaltimore, Maryland, United States
Johns Hopkins Outpatient CenterBaltimore, Maryland, United States
Massachusetts General Hospital (MGH) - Amyotrophic Lateral Sclerosis (ALS) Multidisciplinary ClinicBoston, Massachusetts, United States
Mayo Family Clinic NorthwestRochester, Minnesota, United States
Hospital for Special SurgeryNew York, New York, United States
Columbia University Medical Center - The Neurological Institute of New YorkNew York, New York, United States
The University of North Carolina at Chapel Hill, Department of NeurologyChapel Hill, North Carolina, United States
VIB-KU Leuven Center for Brain & Disease ResearchLeuven, Flemish Brabankt, Belgium
CHU Lille - CMRR Hôpital Roger SalengroLille, France

+ 7 more locations — full list on the registry record.

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Source record: clinicaltrials.gov/study/NCT04993755