Acamprosate for ALS
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests the safety of acamprosate, a pill, in people with ALS—a disease that weakens muscles and affects physical function—who have a specific genetic mutation in the C9orf72 gene. The study will help researchers determine if this treatment is safe and how it might affect ALS symptoms. Participants must attend some in-person visits and complete others by phone over 32 weeks. This trial suits those with ALS and the C9orf72 gene mutation whose symptoms began less than two years ago. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.
Do I need to stop taking my current medications to join the trial?
If you are taking riluzole, edaravone, or phenylbutyrate/TUDCA, you must be on a stable dose for at least 30 days before the screening visit or have stopped taking them at least 30 days before the baseline visit.
Is there any evidence suggesting that acamprosate is likely to be safe for humans?
Previous studies have shown that acamprosate is safe. The FDA has approved it for other uses, indicating it is generally well-tolerated. These studies reported no major safety concerns. Participants took acamprosate without significant side effects. Although acamprosate is now being tested specifically for ALS, its past use in other conditions offers some reassurance about its safety.12345
Why do researchers think this study treatment might be promising?
Most treatments for ALS, like Riluzole and Edaravone, aim to slow disease progression by reducing nerve damage. But Acamprosate works differently, targeting neurotransmitter balance in the brain. Researchers are excited about this because it has a unique mechanism that might offer a new way to protect nerve cells. This could potentially enhance the quality of life for people with ALS by offering a fresh approach to managing symptoms.
What evidence suggests that acamprosate might be an effective treatment for ALS?
Research suggests that acamprosate might help treat ALS, particularly for individuals with the C9orf72 gene mutation. Studies have found that acamprosate can protect motor neurons, the nerve cells responsible for muscle movement. This protective effect appeared in several cell samples from ALS patients. Although acamprosate typically treats alcohol dependence, its ability to protect nerve cells makes it a promising option for ALS treatment. However, further research is necessary to fully understand its impact on ALS symptoms. Participants in this trial will receive acamprosate to evaluate its safety profile throughout the study.12356
Who Is on the Research Team?
Justin Y Kwan, M.D.
Principal Investigator
National Institute of Neurological Disorders and Stroke (NINDS)
Are You a Good Fit for This Trial?
This trial is for adults over 18 with ALS who have a specific genetic mutation (C9orf72). Participants will need to attend multiple clinic visits and phone check-ins, undergo various tests including blood work, imaging scans, breathing assessments, and a lumbar puncture.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Baseline
Participants undergo baseline assessments including physical exams, imaging scans, and neurological exams
Treatment
Participants take acamprosate pills 3 times a day for 24 weeks, with clinic and phone visits to monitor progress
Follow-up
Baseline tests are repeated during follow-up clinic visits to monitor safety and effectiveness
What Are the Treatments Tested in This Trial?
Interventions
- Acamprosate
Trial Overview
The study is testing the effectiveness of Acamprosate calcium in treating ALS. Over 32 weeks, participants will take two pills three times daily with meals and track their intake. The impact on symptoms will be monitored through repeated tests at follow-up visits.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
This is a single arm study, capturing the safety profile of acamprosate over the course of the study.
Find a Clinic Near You
Who Is Running the Clinical Trial?
National Institute of Neurological Disorders and Stroke (NINDS)
Lead Sponsor
Citations
Study Details | NCT07204977 | Acamprosate in C9orf72 ...
This study will evaluate the safety and clinical effects of acamprosate in patients diagnosed with amyotrophic lateral sclerosis who have a hexanucleotide ...
Combination of acamprosate and baclofen (PXT864) as a ...
These results demonstrate the value of PXT864 as a promising therapeutic strategy for the treatment of ALS. Keywords: amyotrophic lateral sclerosis, ...
Mechanism-free repurposing of drugs for C9orf72-related ...
We confirmed the efficacy of acamprosate at protecting motor neurons in additional cell lines (five ALS patients carrying the C9orf72 repeat ...
Current potential therapeutics of amyotrophic lateral sclerosis
The results show that Edaravone demonstrated efficacy in a small group of ALS patients who met the screening criteria for the post hoc analysis, ...
5.
alsnewstoday.com
alsnewstoday.com/news/pharnexts-pxt864-shows-promising-neuroprotective-effects-in-als-early-study-shows/Pharnext's PXT864 Shows Neuroprotective Effects in ...
Results showed that PXT864 effectively protected neuromuscular junctions, motor neurons, and cortical neurons against glutamate‐induced injury, ...
Newly posted trial for C9orf72: ## Acamprosate in ...
A drug repurposing evaluation using genomic data from C9orf72 genetic mutation carriers showed that acamprosate may have a neuroprotective ...
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