Acamprosate in C9orf72 Hexanucleotide Repeat Expansion Amyotrophic Lateral Sclerosis (ACALS)
United States30 participantsStarted 2026-02-02
Plain-language summary
Background:
Amyotrophic lateral sclerosis (ALS) is a disorder that damages nerve cells in the brain and spinal cord. It can cause muscle weakness, paralysis, and loss of movement. The symptoms grow worse over time. Half of all people with ALS live only 3 to 5 years after diagnosis. Current drug treatments can slow the progress of the disease, but they cannot stop or reverse it.
Objective:
To test a study drug (acamprosate) in people with ALS with a mutation in the C9orf72 gene.
Eligibility:
People aged 18 years and older with ALS. They must have a mutation in the C9orf72 gene.
Design:
Participants will have 13 visits over 32 weeks. Five visits will be at the clinic, and 8 visits will be by phone.
Participants will have a baseline visit of up to 3 days. They will have a physical exam with blood tests. They will have imaging scans and tests of their breathing ability. Their memory, thinking, and behavior will be assessed. They will have a neurologic exam to check their reflexes, strength, balance, eyes, and coordination. They will complete questionnaires about their daily life. They will have a lumbar puncture to collect fluid from the area around the spinal cord.
Acamprosate is a pill taken by mouth. Participants will take 2 pills by mouth 3 times a day with meals for 24 weeks. They will record their doses and any missed doses in a diary.
Baseline tests will be repeated during follow-up clinic visits. These tests may be spread out over 3 days.
During phone visits, participants will talk about how they are doing. They will review their diary with researchers.
Who can participate
Age range
18 Years – 99 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Aged 18 years or older at the time of the screening visit.
. Able to provide informed consent and comply with study procedures and availability for the duration of the study. If a participant lacks capacity to give consent, a Legally Authorized Representative (LAR) will be authorized to give consent on behalf of the individual.
. Diagnosis of ALS (possible, probable, or definite according to the World Federation of Neurology El Escorial revised criteria or the Gold Coast Criteria) with or without mild cognitive impairment, mild behavioral impairment, or frontotemporal dementia (FTD).
. CLIA-certified genetic testing showing a pathogenic hexanucleotide repeat expansion in the C9orf72 gene.
. Symptom duration less than 2 years, or if greater than 2 years, disease progression at a rate that, in the judgement of the investigator, would allow for completion of the study.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Safety of acamprosate in patients with ALS and mutation in C9orf72.
Timeframe: Six months
Trial details
NCT IDNCT07204977
SponsorNational Institute of Neurological Disorders and Stroke (NINDS)
. If taking riluzole, edaravone, or phenylbutyrate/TUDCA, the participant must be on a stable dose for at least 30 days prior to the screening visit, or have stopped taking riluzole, edaravone, or phenylbutyrate/TUDCA at least 30 days prior to the baseline visit.
. Participant must be competent to self-administer the medication as deemed by study team. Alternatively, participant must have a competent caregiver who can and will be responsible for administering the study drug. If there is no caregiver, another qualified individual must be available to administer the study drug.
. Participant has established care with a neurologist and will maintain this clinical care throughout the study.
Exclusion criteria
. Dependence on daytime mechanical ventilation (invasive or non-invasive, including Continuous Positive Airway Pressure (CPAP) or Bilevel Positive Airway Pressure (BiPAP) at the time of the screening visit.
. Participation in any other investigational drug trial or using investigational drug(s) (within 4 weeks prior to the Day 0 visit and thereafter).
. Participants must not become pregnant or breastfeed for the duration of the study. Participants of childbearing potential must have a negative pregnancy test at screening and be non-lactating.
. Men who are trying to become fathers or donate sperm.
. History of positive test or positive result at screening for HIV.
. History of severe sulfite allergy (i.e., anaphylaxis).
. Presence of any of the following clinical conditions at the time of screening: