Acamprosate in C9orf72 Hexanucleotide Repeat Expansion Amyotrophic Lateral Sclerosis (ACALS)

Running, not enrolling · Phase 1

Conditions studied: Amyotrophic Lateral Sclerosis, Motor Neuron Disease

In brief

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.

Key facts

Study ID

NCT07204977

Run by

National Institute of Neurological Disorders and Stroke (NINDS)

People needed

30

Starts

2026-02-02

Expected to finish

2028-09-30

Last updated by the study team

2026-07-24

Who can join

Age: 18 and older, up to 99. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• An individual who meets any of the following criteria will be excluded from participation in this study:
• 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:
• Active drug abuse or alcoholism.
• Unstable medical condition that, in the opinion of the investigators, makes participation unsafe.
• Renal impairment estimated glomerular filtration rate <=60mL/min/1.73m\^2.
• Unstable psychiatric illness defined as psychosis or untreated major depression within 90 days of the screening visit.
• Safety Laboratory Criteria at the screening visit:
• Estimated glomerular filtration rate <=60mL/min/1.73m\^2.
• Platelet concentration of <100,000/microl.
• PT and PTT >1.2 times the upper limit of normal.
• Hemoglobin <10mg/dL.
• Positive Hepatitis B Surface Antigen and Hepatitis C Virus Antibody
• Participants who are unable to swallow tablets whole, as required by the administration guidelines. This includes individuals with any medical condition or physical limitation that impedes their ability to swallow solid dosage forms.
• Participants who are dependent on a gastrostomy tube for medication administration. Since acamprosate is in tablet form that cannot be crushed or altered, it is unsuitable for administration through a gastrostomy tube.

Where it is running

• National Institutes of Health Clinical Center — Bethesda, Maryland, United States

Full record on ClinicalTrials.gov

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