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
Trial Overview
The trial tests the effects of a drug called istradefylline and low oxygen therapy on breathing in ALS patients compared to placebos. It aims to see if these treatments help patients take deeper breaths without causing discomfort.
How Is the Trial Designed?
Participants enrolled in this study arm will ingest a 20mg tablet containing istradefylline. Four hours later, participants will receive acute intermittent hypoxia (AIH). Breathing and pinch strength will be tested prior to taking the medication, and then immediately before, 60 minutes and 120 minutes after AIH. Participants will breathe 15 episodes/session of acute low oxygen. Air concentrations will be monitored to ensure delivery of 1-minute episodes of low oxygen, with 2 minutes room-air intervals. Respiratory rate, oxygen saturation, heart rate/rhythm, and blood pressure will be monitored throughout the session.
This is a sham counterpart to low oxygen, and a placebo counterpart to the istradefylline drug. Participants enrolled in this study arm will ingest a 20mg tablet containing microcrystalline cellulose. Four hours later, participants will receive SHAM acute intermittent hypoxia (SHAM). Breathing and pinch strength will be tested prior to taking the medication, and then immediately before, 60 minutes and 120 minutes after SHAM. Participants will breathe 15 episodes/session of sham low oxygen, in which normal air is used. One-minute episodes of sham low oxygen are separated by 2 minutes room-air intervals. Respiratory rate, oxygen saturation, heart rate/rhythm, and blood pressure will be monitored throughout the session.
This is a placebo counterpart to the istradefylline drug. Participants enrolled in this study arm will ingest a 20mg tablet containing microcrystalline cellulose. Four hours later, participants will receive acute intermittent hypoxia (AIH). Breathing and pinch strength will be tested prior to taking the medication, and then immediately before, 60 minutes and 120 minutes after AIH. Participants will breathe 15 episodes/session of acute low oxygen. Air concentrations will be monitored to ensure delivery of 1-minute episodes of low oxygen, with 2 minutes room-air intervals. Respiratory rate, oxygen saturation, heart rate/rhythm, and blood pressure will be monitored throughout the session.
This is a sham counterpart to the low oxygen. Participants enrolled in this study arm will ingest a 20mg tablet containing istradefylline. Four hours later, participants will receive SHAM acute intermittent hypoxia (SHAM). Breathing and pinch strength will be tested prior to taking the medication, and then immediately before, 60 minutes and 120 minutes after SHAM. Participants will breathe 15 episodes/session of sham low oxygen, in which normal air is used. One-minute episodes of sham low oxygen are separated by 2 minutes room-air intervals. Respiratory rate, oxygen saturation, heart rate/rhythm, and blood pressure will be monitored throughout the session.
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
Citations
Adenosine 2A Receptor Antagonism and AIH in ALS
The purpose of this research study is to determine the effects of a medication, istradefylline, in conjunction with breathing air with reduced oxygen for ...
Istradefylline (Nourianz™)
There is a lack of safety and efficacy data in the use of istradefylline (Nourianz) in those under the age of 18. III. Motor symptoms in PD affect as many as 77 ...
Istradefylline and AIH for ALS
A recently completed study found that people with ALS took deeper breaths, 60 minutes after using AIH.
Efficacy of Istradefylline, an Adenosine A2A Receptor ...
In this pooled analysis, istradefylline significantly improved OFF time and ON-WoTD relative to placebo and was well-tolerated.
5.
researchgate.net
researchgate.net/publication/10618460_Randomized_trial_of_the_adenosine_A2A_receptor_antagonist_istradefylline_in_advanced_PDRandomized trial of the adenosine A2A receptor antagonist ...
Key findings from this study were that (i) istradefylline showed promise for motor fluctuations, but not dyskinesia, and (ii) doses of 20 and 40 ...
Acute Intermittent Hypoxia and Respiratory Muscle ...
AIH may be a safe way to increase collective inspiratory muscle activity during quiet breathing in ALS patients.
Safety and effectiveness of acute intermittent hypoxia ...
AIH is generally safe and effective for inducing neural plastic responses. · AIH does not impact cognition or cerebral perfusion rates. · AIH acutely results in a ...
NCT03645031 | Acute Intermittent Hypoxia and Breathing ...
Most ALS patients survive less than 5 years after diagnosis, and the main cause of death is respiratory failure. The investigators are interested in the ...
Respiratory therapies for Amyotrophic Lateral Sclerosis
Therapeutic acute intermittent hypoxia is a recently reported non-pharmacological therapy that aims to promote neuroplasticity of breathing ...
10.
journals.physiology.org
journals.physiology.org/doi/full/10.1152/ajpregu.00208.2014?doi=10.1152/ajpregu.00208.2014Therapeutic potential of intermittent hypoxia: a matter of dose
Accumulating evidence suggests that “low dose” IH (modest hypoxia, few episodes) may be a simple, safe, and effective treatment with considerable therapeutic ...
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