Intermediate-size Patient Population Expanded Access Protocol
United States
Plain-language summary
The purpose of this Expanded Access Program is to provide tazbentetol to ALS patients who are not eligible to enroll in an ALS clinical trial. This Expanded Access Program will assess safety and tolerability, and clinical efficacy of tazbentelol.
Who can participate
Age range
18 Years – 85 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:
* ALS diagnosis
* Age 18 -85 years at time of signing informed consent form
* Ineligible for other interventional ALS clinical research participation
* Vital Capacity greater than 35% of predicted capacity for age, height, and sex
* If currently taking standard of care treatment for ALS, must be on stable dose for at least 30 days prior to taking tazbentetol.
* Life expectancy of at least 6 months, according to Investigator's judgement
Exclusion Criteria:
* Clinically significant and/or unstable medical condition (other than ALS) that would pose a risk to the patient
* Known ongoing or clinically uncontrolled cardiac disease
* Clinically significant liver disease
* Clinical significant cognitive impairment or neurological disorder, as determined by Investigator judgement
* Concomitant use of another investigational medical product for treatment of ALS
* Unable to reliably and regularly swallow whole oral medications on a daily basis
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.