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Completed Phase 2 Interventional

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

ClinicalTrials.gov ID: NCT04993755

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.

ClinicalTrials.gov public records Last synced Oct 24, 2026, 10:48 PM EDT

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)

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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).

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

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
Oct 1, 2021 (Actual)
Primary completion
Aug 17, 2023 (Actual)
Completion
Sep 1, 2023 (Actual)
Last update posted
Apr 3, 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 3, 2026 · Synced Oct 24, 2026

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Open the official record

The complete public registry record, listed eligibility criteria, and available contact information for NCT04993755 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →