A Phase 2a Study of TPN-101 in Patients With C9ORF72 ALS/FTD
Public ClinicalTrials.gov record NCT04993755. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 2a Study 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)
Study identification
- NCT ID
- NCT04993755
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Lead sponsor
- Transposon Therapeutics, Inc.
- Industry
- Enrollment
- 42 participants
Conditions and interventions
Interventions
- Placebo Drug
- TPN-101, 400 mg/day Drug
Drug
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Sep 30, 2021
- Primary completion
- Aug 16, 2023
- Completion
- Aug 31, 2023
- Last update posted
- Apr 2, 2026
2021 – 2023
United States locations
- U.S. sites
- 10
- U.S. states
- 6
- U.S. cities
- 8
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California San Diego | La Jolla | California | 92037 | — |
| University of California Irvine - ALS & Neuromuscular Center | Orange | California | 92868 | — |
| UCSF Neurosciences Clinical Research Unit (NCRU) | San Francisco | California | 94158 | — |
| John Hopkins University | Baltimore | Maryland | 21287 | — |
| Johns Hopkins Outpatient Center | Baltimore | Maryland | 21287 | — |
| Massachusetts General Hospital (MGH) - Amyotrophic Lateral Sclerosis (ALS) Multidisciplinary Clinic | Boston | Massachusetts | 02114 | — |
| Mayo Family Clinic Northwest | Rochester | Minnesota | 55905 | — |
| Hospital for Special Surgery | New York | New York | 10021 | — |
| Columbia University Medical Center - The Neurological Institute of New York | New York | New York | 10032 | — |
| The University of North Carolina at Chapel Hill, Department of Neurology | Chapel Hill | North Carolina | 27599 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 9 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04993755, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Apr 2, 2026 · Synced May 20, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04993755 live on ClinicalTrials.gov.