Overview
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.
Description
This is a multi-site, intermediate-size patient population expanded access protocol developed to provide compassionate use access to tazbentetol to adult patients with ALS who have progressed following available standard of care.
Eligibility
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