RAPA-501 Therapy for ALS

  • End date
    Dec 15, 2023
  • participants needed
  • sponsor
    Rapa Therapeutics LLC
Updated on 2 August 2021
primary lateral sclerosis
familial amyotrophic lateral sclerosis
tendency to bleed


RAPA-501-ALS is an open-label, dose escalation, Phase 1/2 study of RAPA-501 autologous T cells in adults with amyotrophic lateral sclerosis (ALS).


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).

Condition Myelopathy, Amyotrophic Lateral Sclerosis, Amyotrophic Lateral Sclerosis (ALS), Spinal Cord Disorders, lou gehrig's disease
Treatment RAPA-501 Autologous T cells
Clinical Study IdentifierNCT04220190
SponsorRapa Therapeutics LLC
Last Modified on2 August 2021


Yes No Not Sure

Inclusion Criteria

Male or female patients 18 years of age
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
Must have a source of autologous T cells potentially sufficient to manufacture RAPA-501 cells, as defined by a peripheral CD3+ T cell count 800 cells per l
Patients may continue riluzole (Rilutek) and/or edaravone (Radicava) therapy if on a stable dose for one month prior to study entry
Patients must be two weeks from major surgery, from edaravone therapy, and from participation in investigational trials
Patients must have recovered from clinical toxicities (resolution of CTCAE [version 5] toxicity to a value of 2)
Serum creatinine less than or equal to 2.0 mg/dL
Pulmonary slow vital capacity (SVC) 50% of predicted normal
Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) 3 x upper limit of normal
Bilirubin 1.5 (except if due to Gilbert's disease)
No history of abnormal bleeding tendency
For accrual to Cohort 3, additional eligibility inclusion criteria are as
Voluntary written consent must be given before performance of any study related procedure not part of standard medical care, with the understanding that consent may be withdrawn by the participant at any time without prejudice to future care
\. Ejection fraction by MUGA or 2-D echocardiogram within institution normal
limits (applies only to study participants on cohort)

Exclusion Criteria

Active uncontrolled infection
Hypertension not adequately controlled by 3 medications
History of documented pulmonary embolus within 6 months of enrollment
Clinically significant cardiac pathology, as defined by: myocardial infarction within 6 months prior to enrollment, Class III or IV heart failure according to NYHA, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities
Patients with history of coronary artery bypass grafting or angioplasty will receive a cardiology evaluation and be considered on a case-by-case basis
HIV, hepatitis B, or hepatitis C seropositive
Pregnancy or breastfeeding patients
Patients of childbearing age, or males who have a partner of childbearing potential, who are unwilling to practice contraception
Patients may be excluded at the discretion of the PI or if it is deemed that allowing participation would represent an unacceptable medical or psychiatric risk
For accrual to Cohort 3, additional eligibility exclusion criteria are as
\. Calculated creatinine clearance value of < 70 mL/min/1.73 m2, as
calculated by the Cockcroft-Gault formula
\. Diagnosis of malignancy (active)
\. Urinary obstruction
\. Hypersensitivity to the agents in the PC regimen (pentostatin
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