RAPA-501 Therapy of ALS Expanded Access Protocol

NCT06169176 | Unknown

• 2 other identifiers: RAPA-501-ALS-EAP1U01NS136020-01
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 10

Brief Summary

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

Keywords

Amyotrophic Lateral Sclerosis; RAPA-501; ALS; Autologous TREG/Th2 T stem cell therapy; Rare Disease; Orphan Disease

Interventions

RAPA-501 Autologous T stem cells OTHER

Experimental: Intermediate size expanded access cohort. Single-agent RAPA-501 T stem cells, (80 x 10EE6 cells per infusion; acceptable dose range of RAPA-501 T stem cells is 20-to-80 x 10EE6 cells per infusion.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female patients ≥ 18 years of age.
• Patients with sporadic or familial ALS diagnosed as laboratory-supported possible, probable, or definite according to World Federation of Neurology El Escorial Criteria.
• 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.
• 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.
• 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.
• Patients must be ≥ two (2) weeks removed from major surgery, or investigational therapy.
• Patients must have no ongoing, unstable serious illness other than ALS, as determined by the Site Investigator.
• Serum creatinine less than or equal to 2.0 mg/dL.
• Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 x upper limit of normal (ULN).
• Bilirubin ≤ 1.5 (except if due to Gilbert's disease).
• No history of abnormal bleeding tendency.
• 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.
• 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.

You may not qualify if:

• 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.
• HIV, hepatitis B, or hepatitis C seropositive.
• Pregnant or breastfeeding subjects.
• Subjects of childbearing age, or males who have a partner of childbearing potential, who are unwilling to practice contraception.
• 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.

Sponsors & Collaborators

• Rapa Therapeutics LLC: lead
• Massachusetts General Hospital: collaborator
• National Institute of Neurological Disorders and Stroke (NINDS): collaborator

Study Sites (10)

Barrow Neurological Institute

Phoenix, Arizona, 85013, United States

Location

Mayo Clinic Hospital Phoenix

Scottsdale, Arizona, 85259, United States

Location

University of California Irvine Health

Irvine, California, 92868, United States

Location

University of California, San Francisco

San Francisco, California, 94143, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

University of Iowa Health Care

Iowa City, Iowa, 52242, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

Location

Providence Portland Medical Center

Portland, Oregon, 97213, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Rare Diseases

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Daniel H Fowler, M.D.

  Rapa Therapeutics LLC

  STUDY DIRECTOR

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 4, 2023
• First Posted: December 13, 2023
• Last Updated: February 12, 2026

Record last verified: 2026-02

Locations

US (10)