Istradefylline and AIH for ALS
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores how a medication called istradefylline, combined with breathing low-oxygen air for short periods (acute intermittent hypoxia or AIH), affects breathing in people with amyotrophic lateral sclerosis (ALS) and healthy adults. Istradefylline typically helps individuals with movement issues in other neuromuscular conditions. The researchers aim to determine if this combination can improve breathing for people with ALS, especially since a recent study showed that AIH alone helped ALS patients take deeper breaths. Ideal participants are adults with ALS who have breathing difficulties but can still breathe deeply enough on their own. As a Phase 1, Phase 2 trial, this research focuses on understanding how the treatment works and measuring its effectiveness in an initial, smaller group of people.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but you must be on a stable dose of riluzole and/or edaravone for more than 30 days if you are taking them. You cannot participate if you use certain medications like CYP3A4 inducers or those that may suppress breathing.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that istradefylline has been safely used in people with Parkinson's disease. In studies involving 734 patients, most tolerated it well, though some experienced side effects like dizziness and constipation. The FDA's approval of istradefylline for Parkinson's indicates its general safety.
Regarding acute intermittent hypoxia (AIH), research has found it to be a safe method for strengthening breathing muscles in people with ALS. AIH does not affect brain function or blood flow and is considered safe and effective for promoting positive changes in the nervous system.
Both treatments, istradefylline and AIH, have demonstrated safety in past research. However, individual health conditions should be considered when deciding to join a trial.12345Why are researchers excited about this trial's treatments for ALS?
Istradefylline and its combination with acute intermittent hypoxia (AIH) offer a fresh approach to tackling ALS. Unlike typical ALS treatments like riluzole and edaravone, which primarily aim to slow disease progression, istradefylline works by targeting adenosine A2A receptors, potentially altering disease pathways more directly. AIH, on the other hand, employs brief, controlled exposure to low oxygen levels to stimulate the body's adaptive responses, which might enhance motor function and breathing. Researchers are excited about this combination because it offers a dual mechanism of action that could potentially improve symptoms and quality of life for ALS patients more effectively than current treatments.
What evidence suggests that this trial's treatments could be effective for ALS?
Research suggests that istradefylline, a medication used to aid movement in other nerve and muscle conditions, might also enhance breathing in people with ALS. In this trial, one group will receive istradefylline along with a low-oxygen technique called acute intermittent hypoxia (AIH). A study found that people with ALS took deeper breaths 60 minutes after using AIH. AIH alone, tested in another group of this trial, has shown promise in improving breathing and aiding brain adaptation in ALS patients. Istradefylline blocks certain brain receptors and has improved movement symptoms in other conditions. While more research is needed, these findings suggest that combining istradefylline with AIH might enhance breathing in ALS.678910
Who Is on the Research Team?
Barbara Smith
Principal Investigator
University of Florida
Are You a Good Fit for This Trial?
Adults aged 21-75 with ALS, non-smokers, having a vital capacity over 60% of predicted value and an ALSFRS-R score above 30. They must not have used antibiotics recently or have certain respiratory or cardiovascular issues. Stable doses of riluzole/edaravone for over 30 days are required if in use.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants undergo 4 individual study visits with different conditions: AIH + istradefylline, sham-AIH + istradefylline, AIH + placebo, and sham-AIH + placebo.
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Istradefylline
- Low Oxygen therapy
- Placebo counterpart to the istradefylline drug
- SHAM counterpart to low oxygen therapy
Istradefylline is already approved in United States, Japan for the following indications:
- Parkinson's disease
- Parkinson's disease
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of Florida
Lead Sponsor
ALS Association
Collaborator