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New treatment option for Amyotrophic Lateral Sclerosis
Official title RAPA-501 Therapy for ALS
ClinicalTrials.gov ID: NCT04220190
What this study is testing
What is RAPA-501 Autologous T stem cells?
RAPA-501 Autologous T stem cells is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for amyotrophic lateral sclerosis.
Also referred to as RAPA-501 cells.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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).
- Phase 3: a large, late-stage study
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join 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...
- . 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...
You likely can't join 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...
- Patients with history of coronary artery bypass grafting or angioplasty will receive a cardiology evaluation and be considered on a case-by-case...
- HIV, hepatitis B, or hepatitis C seropositive.
See the full eligibility criteria
- 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...
- 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...
- 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 people of childbearing age, or males who have a partner of childbearing potential, who are unwilling to practice contraception.
- Patients people 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.
The study team makes the final eligibility decision.
Where it's taking place
- Boston, Massachusetts, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Boston, Massachusetts, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.