Intermediate-Size Expanded Access Trial of Autologous Hybrid TREG /Th2 T Stem Cell Therapy (RAPA-501) of Amyotrophic Lateral Sclerosis

Trial ID: NCT06169176

Interventional - Phase Unknown - Temporarily not available

RAPA-501-ALS is an Intermediate-Size Expanded Access Trial of RAPA-501 autologous hybrid TREG/Th2 T stem 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 ALS diagnosed as laboratory-supported possible, probable, or definite according to World Federation of Neurology El Escorial Criteria.
• 3. 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.
• 4. 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.
• 5. 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.
• 6. Patients must be ≥ two (2) weeks removed from major surgery, or investigational therapy.
• 7. Patients must have no ongoing, unstable serious illness other than ALS, as determined by the Site Investigator.
• 8. Serum creatinine less than or equal to 2.0 mg/dL.
• 9. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 x upper limit of normal (ULN).
• 10. Bilirubin ≤ 1.5 (except if due to Gilbert's disease).
• 11. No history of abnormal bleeding tendency.
• 12. 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of any study related procedure not part of standard medical care, with the understanding that consent may be withdrawn by the patient at any time without prejudice to future care.
• 13. Not enrolled in another interventional clinical trial or expanded access protocol and must have stopped taking other experimental drug(s) at least 2 weeks prior to screening.

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. HIV, hepatitis B, or hepatitis C seropositive.
• 6. Pregnant or breastfeeding subjects.
• 7. Subjects of childbearing age, or males who have a partner of childbearing potential, who are unwilling to practice contraception.
• 8. Subjects may be excluded at the Principal Investigator discretion or if it is deemed that allowing participation would represent an unacceptable medical or psychiatric risk.

Sponsor

Rapa Therapeutics LLC, Massachusetts General Hospital, National Institute of Neurological Disorders and Stroke (NINDS)

Source: ClinicalTrials.gov

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