RECRUITING

RAPA-501 Therapy for ALS

Conditions

Amyotrophic Lateral Sclerosis Autologous TREG/Th2 cell therapy RAPA-501

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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).

Official Title

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

Quick Facts

Study Start:2024-12-01

Study Completion:2027-06-01

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT04220190

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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.	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.

Inclusion Criteria

Exclusion 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.

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.

Contacts and Locations

Study Contact

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

Principal Investigator

Daniel Fowler, M.D.

STUDY_DIRECTOR

Rapa Therapeutics LLC

Study Locations (Sites)

Massachusetts General Hospital

Boston, Massachusetts, 02114

United States

Collaborators and Investigators

Sponsor: Rapa Therapeutics LLC

Daniel Fowler, M.D., STUDY_DIRECTOR, Rapa Therapeutics LLC

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-12-01

Study Completion Date2027-06-01

Study Record Updates

Study Start Date2024-12-01

Study Completion Date2027-06-01

Terms related to this study

Keywords Provided by Researchers

Amyotrophic Lateral Sclerosis
Autologous TREG/Th2 cell therapy
RAPA-501

Additional Relevant MeSH Terms

Amyotrophic Lateral Sclerosis