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A Phase 2a study of TPN-101 in patients with C9ORF72 ALS/FTD

Transposon Therapeutics, Inc.

Information about the drug that will be tested in the study


Oral capsules once daily

Is the drug already on the market for another medical condition?


Will all participants receive the same drug?

Participants will be selected at random to either receive one of the following options:

  • An oral capsule of TPN-101 (400mg) for 24 weeks
  • An oral capsule of placebo (also called a dummy treatment which is an inactive substance identical in appearance to the drug being tested with no active therapeutic effect) for 24 weeks

Neither the participant nor his/her doctor will know if the person is receiving the investigational drug or the placebo.

After this 24-week treatment period, all participant will enter an open-label treatment period to receive oral capsules of TPN-101 (400mg) for 24 weeks.

Information about participating in the trial

What are the researchers trying to find out?

The purpose of the study is to evaluate the safety and tolerability of TPN-101 in people with Amyotrophic Lateral Sclerosis and/or Frontotemporal Dementia associated with Hexanucleotide Repeat Expansion in the C9orf72 gene (C9ORF72 ALS/FTD).

How long will the treatment last?

The treatment lasts 48 weeks.

The study includes a 6-week screening period, followed by a 24-week treatment period and finally a 24-week open-label treatment period. A follow-up visit is scheduled 4 weeks post-treatment.

What your involvement will entail?
  • During the study, participants will be asked to complete some laboratory/biological tests to evaluate the emergent adverse effects (unfavourable signs, symptoms or diseases temporally associated with the use of the drug tested in the study)
  • Participants will be asked to perform lumbar punctures (CSF) to see changes in biomarkers in the brainy
  • Participants will complete a test that will assess memory, orientation, judgment and problem solving, personal care and community affairs (this is a test called CDR).

Further information on the procedures, tests and number of visits can be obtained from the study team.

Who can participate in this study?

Who can participate in the study?

To take part in the study (part A), participants with prodomal Alzheimer's disease must:

  • Have 18 years and older
  • Have a mutation in the C9orf72 gene (documentation proving a clinical genetic test demonstrating a hexanucleotide repeat expansion (HRE) in the C9orf72 gene).

To take part in the study, participants with Frontotemporal Dementia must:

  • Have a study partner who has a sufficient contact with the participant, is willing to participate in study procedures throughout the study duration
  • Have a gradual, progressive decline in behaviour, language, or motor function consistent with mild cognitive impairment, mild behavioural impairment, mild cognitive/behavioural impairment, behavioural variant with Frontotemporal Dementia, primary progressive aphasia, or amnestic syndrome
  • Have a score of 0.5-2 in the CDR plus NACC FTLD test.

To take part in the study, participants with Amyotrophic Lateral Sclerosis (with or without with Frontotemporal Dementia) must:

  • Have a diagnosis of Amyotrophic Lateral Sclerosis according to the World Federation of Neurology revised E1 Escorial criteria
  • Have an onset of weakness within 3 years
  • Have a score above 30 in the Functional Rating Scale-Revised (ALSFRS-R) test and a score of 3 or 4 on Item #3 (swallowing). This test is used to measure the severity of Amyotrophic Lateral Sclerosis.
Who cannot participate in the study?

People cannot take part in the study if they have or have experienced:

  • Presence of other significant neurological or psychiatric disorders
  • History of clinically significant brain abnormality
  • Clinically significant medical illness
  • Tracheostomy or diaphragmatic pacing
  • Autoimmune disease requiring treatment or management (e.g. quiescent rheumatoid arthritis, psoriasis, or controlled Type 1 diabetes are acceptable)
  • History of human immunodeficiency virus (HIV) or hepatitis B infection.

The above list is not exhaustive. It includes the most common conditions and diseases that might exclude people from the study.

Information for your doctor

EudraCT Number

2021-002251-11 identifier


Study contact information

Link to full text

The information contained in this document is based on information available on public registries (e.g. website) on November 2022.

Click here to download the accessible easy-to-read version of this document


Alzheimer Europe's Clinical Trials Watch database was developed as part of the 2018 Work Plan which received funding under an operating grant from the European Union’s Health Programme (2014–2020).

Alzheimer Europe gratefully acknowledges the support of its corporate sponsors for the update and maintenance of the database.