Actively Recruiting
RAPA-501 Therapy for ALS
Led by Rapa Therapeutics LLC · Updated on 2025-06-18
41
Participants Needed
1
Research Sites
125 weeks
Total Duration
On this page
Sponsors
R
Rapa Therapeutics LLC
Lead Sponsor
M
Massachusetts General Hospital
Collaborating Sponsor
AI-Summary
What this Trial Is About
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
Official Title
RAPA-501 Therapy for ALS
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female patients 18 years of age or older
- Diagnosed with sporadic or familial ALS as laboratory-supported possible, probable, or definite by El Escorial Criteria
- Symptom onset within 24 months or less
- Total ALSFRS-R score between 34 and 45
- Peripheral CD3+ T cell count of at least 500 cells per microliter
- May continue stable doses of riluzole, edaravone, or sodium phenylbutyrate/taurursodiol for at least 30 days prior to screening
- At least 2 weeks since major surgery or investigational therapy
- Resolved clinical toxicities to grade 2 or less
- Serum creatinine less than or equal to 2.0 mg/dL
- AST and ALT less than or equal to 3 times the upper limit of normal
- Bilirubin less than or equal to 1.5 (except Gilbert's disease)
- Pulmonary slow vital capacity of at least 70% predicted
- No history of abnormal bleeding tendency
- Voluntary written consent given before any study procedures
You will not qualify if you...
- Active uncontrolled infection
- Hypertension not controlled by three or fewer medications
- History of pulmonary embolus within 6 months
- Significant cardiac problems including recent myocardial infarction, advanced heart failure, uncontrolled angina, severe arrhythmias, or acute ischemia
- History of coronary artery bypass or angioplasty unless cleared by cardiology
- Positive for HIV, hepatitis B, or hepatitis C
- Pregnant or breastfeeding
- Childbearing age patients or males with partners of childbearing potential unwilling to use contraception
- Any condition deemed by the principal investigator to pose unacceptable medical or psychiatric risk
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Actively Recruiting
Research Team
D
Daniel Fowler, M.D. Chief Medical Officer, RAPA Therapeutics, LLC
CONTACT
J
Jennifer Sunga Regulatory Affairs Associate, RAPA Therapeutics, LLC
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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