RAPA-501 Therapy of ALS Expanded Access Protocol

Inclusion criteria

• . Male or female patients ≥ 18 years of age.
• . Patients with sporadic or familial ALS diagnosed as laboratory-supported possible, probable, or definite according to World Federation of Neurology El Escorial Criteria.
• . Pulmonary slow vital capacity (SVC) \< 50% of predicted normal (as measured within three months prior to screening or at the time of screening; inability to measure an SVC value at the time of screening that is due to severe reduction in respiratory function will also fulfill this eligibility criterion #3). However, SVC values ≥50% are acceptable if an EAP recipient of RAPA-501 is re-enrolled to the study.
• . Must have a source of autologous T cells potentially sufficient to manufacture RAPA-501 cells, as defined by a peripheral CD3+ T cell count ≥ 500 cells per μl.
• . Patients who are taking riluzole (Rilutek®), edaravone (Radicava®), and/or sodium phenylbutyrate/taurursodial (Relyvrio™) are eligible if taking the drug for at least 30 days prior to the screening visit.
• . Patients must be ≥ two (2) weeks removed from major surgery, or investigational therapy.
• . Patients must have no ongoing, unstable serious illness other than ALS, as determined by the Site Investigator.
• . Serum creatinine less than or equal to 2.0 mg/dL.

Exclusion criteria