RAPA-501 Therapy for ALS

Summary

Participation Requirements

Description

This is an open-label, non-randomized, multi-center Phase 1/2 study evaluating RAPA-501 T cells in subjects with amyotrophic lateral sclerosis. After a subject consents to the study, an apheresis procedure will be performed to collect cells to manufacture the investigational product, RAPA-501 T cell...

This is an open-label, non-randomized, multi-center Phase 1/2 study evaluating RAPA-501 T cells in subjects with amyotrophic lateral sclerosis. After a subject consents to the study, an apheresis procedure will be performed to collect cells to manufacture the investigational product, RAPA-501 T cells. This study consists of three cohorts. Cohorts 1 and 2 will evaluate a 6-month regimen of four (4) cycles of RAPA-501 T cell therapy without the pentostatin-cyclophosphamide host conditioning regimen (PC regimen). Cohorts 1 and 2 will evaluate two different doses: Cohort 1 - 40 x 10^6 cells/infusion and Cohort 2 - 160 x 10^6 cells/infusion. Both cohorts will evaluate a 6-month regimen of four cycles of the RAPA-501 cell therapy. Cohort 3 will evaluate the highest safe dose of RAPA-501 cells (from Cohort 1 and Cohort 2) in combination with the PC regimen. Cohort 3 will evaluate a 6-month regimen of 4 cycles of RAPA-501 cells administered after the PC regimen. The PC regimen will be 7 days in duration and the RAPA-501 cell therapy will take place on Day 8. All subjects who complete active treatment on each cohort will then complete the follow-up portion of the study (approximately 6-months in duration).

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