RAPA-501 Therapy for ALS

NCT04220190 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: RAPA-501-ALS
• Study Type: interventional
• Target: 41
• Locations: 1 country
• Sites: 1

Brief Summary

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

Keywords

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

Outcome Measures

Primary Outcomes (1)

• In the expansion cohort enrolling standard-risk pwALS, determine the feasibility and safety of the highest established safe dose of RAPA-501 (80 x 10^6 cells per infusion).

  Through 30 Weeks On-Study

Secondary Outcomes (4)

• Characterize immune system parameters pre- and post-therapy.

  Through 30 Weeks On-Study

• Relative to pretreatment values, characterize the potential effect of RAPA-501 therapy on serum markers of neurodegeneration (neurofilament light, NfL).

  Through 30 Weeks On-Study

• Relative to pretreatment values, characterize the potential effect of RAPA-501 therapy on pulmonary function, as measured by slow vital capacity measurements (SVC, percent of predicted normal).

  Through 30 Weeks On-Study

• Relative to pretreatment values, characterize the potential effect of RAPA-501 therapy on hand grip strength using hand-held dynamometry.

  Through 30 Weeks On-Study

Study Arms (1)

RAPA-501 Autologous T stem cells

EXPERIMENTAL

Phase 2/3 Expansion Cohort, Single-agent RAPA-501 T stem cells 80 x 10EE6 cells per infusion (no host conditioning)

Biological: RAPA-501 Autologous T stem cells

Interventions

RAPA-501 Autologous T stem cells BIOLOGICAL

Experimental: Phase 2/3 Expansion Cohort, Single-agent RAPA-501 T stem cells 80 x 10EE6 cells per infusion (no host conditioning)

Also known as: RAPA-501 cells

RAPA-501 Autologous T stem cells

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 amyotrophic lateral sclerosis (ALS) diagnosed as laboratory-supported possible, probable, or definite according to World Federation of Neurology El Escorial Criteria.
• Less than or equal to 24 months since ALS symptom onset.
• Total ALSFRS-R score between 34 and 45.
• 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 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.
• Patients must be ≥ 2 two weeks removed from major surgery or investigational therapy.
• Patients must have recovered from clinical toxicities (\[resolution of CTCAE(v5) \[version 5\] toxicity to a value of ≤ 2\].).
• 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).
• Pulmonary slow vital capacity (SVC) ≥ 70% of predicted normal.
• 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 participant at any time without prejudice to future medical care.

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.
• 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 Subjects of childbearing age, or males who have a partner of childbearing potential, who are unwilling to practice contraception.
• 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.

Sponsors & Collaborators

• Rapa Therapeutics LLC: lead
• Massachusetts General Hospital: collaborator

Study Sites (1)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

RECRUITING

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 Fowler, M.D.

  Rapa Therapeutics LLC

  STUDY DIRECTOR

Central Study Contacts

Daniel Fowler, M.D. Chief Medical Officer, RAPA Therapeutics, LLC

CONTACT

(301) 518-3104; dan@rapatherapeutics.com

Jennifer Sunga Regulatory Affairs Associate, RAPA Therapeutics, LLC

CONTACT

jsunga@rapatherapeutics.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Masking Details: No masking
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 3, 2020
• First Posted: January 7, 2020
• Study Start: January 2, 2025
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): June 1, 2027
• Last Updated: June 18, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)