Trial Parameters
Brief Summary
RAPA-501-ALS is a phase 2/3 expansion cohort study of RAPA-501 autologous hybrid TREG/Th2 cells in patients living with amyotrophic lateral sclerosis (pwALS).
Eligibility Criteria
Inclusion Criteria: 1. Male or female patients ≥ 18 years of age. 2. Patients with sporadic or familial amyotrophic lateral sclerosis (ALS) diagnosed as laboratory-supported possible, probable, or definite according to World Federation of Neurology El Escorial Criteria. 3. . Less than or equal to 24 months since ALS symptom onset. 4. Total ALSFRS-R score between 34 and 45. 5. 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. 6. Patients may continue riluzole (Rilutek®), and/or edaravone (Radicava®), and/or sodium phenylbutyrate/taurusodial (Relyvrio™) if on a stable dose for at least 30 days prior to the screening visit. 7. Patients must be ≥ 2 two weeks removed from major surgery or investigational therapy. 8. Patients must have recovered from clinical toxicities (\[resolution of CTCAE(v5) \[version 5\] toxicity to a value of ≤ 2\].). 9. Serum creatinine ≤ less than or equal t