Phase 2/3 Trial of Autologous Hybrid TREG/Th2 (RAPA-501) T Stem Cell Therapy for Amyotrophic Lateral Sclerosis

Trial ID: NCT04220190

Interventional - Phase 2/3 - Recruiting

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).

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

• RAPA-501 Autologous T stem cells

Eligibility

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 to 2.0 mg/dL.
• 10. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 x upper limit of normal (ULN).
• 11. Bilirubin ≤ 1.5 (except if due to Gilbert's disease).
• 12. Pulmonary slow vital capacity (SVC) ≥ 70% of predicted normal.
• 13. No history of abnormal bleeding tendency.
• 14. 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of any study related procedure not part of standard medical care, with the understanding that consent may be withdrawn by the patient participant at any time without prejudice to future medical care.

Exclusion criteria

• 1. Active uncontrolled infection.
• 2. Hypertension not adequately controlled by ≤ 3 medications.
• 3. History of documented pulmonary embolus within 6 months of enrollment.
• 4. 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.
• 5. Patients with history of coronary artery bypass grafting or angioplasty will receive a cardiology evaluation and be considered on a case-by-case basis.
• 6. HIV, hepatitis B, or hepatitis C seropositive.
• 7. Pregnancy or breastfeeding patients.
• 8. Patients of Subjects of childbearing age, or males who have a partner of childbearing potential, who are unwilling to practice contraception.
• 9. Patients Subjects may be excluded at the Principal Investigator discretion of the PI or if it is deemed that allowing participation would represent an unacceptable medical or psychiatric risk.

Sponsor

Rapa Therapeutics LLC, Massachusetts General Hospital

Source: ClinicalTrials.gov

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