Phase 3 Rare Disease Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You are 18 years of age or older
- You have been diagnosed with ALS (either sporadic or familial) that meets specific diagnostic standards set by a leading neurology organization (confirm with trial site)
- Your ALS symptoms began no more than 24 months ago
- Your ALS function rating score falls between 34 and 45 on a standard scale used to measure daily functioning (confirm with trial site)
- A blood test shows your immune cell count is high enough to produce the treatment used in this trial (confirm with trial site)
- If you are already taking riluzole, edaravone, or sodium phenylbutyrate/taurursodiol for ALS, you have been on a stable dose for at least 30 days before screening
- It has been at least 2 weeks since any major surgery or participation in another experimental treatment
- Any side effects from previous treatments have mostly resolved (confirm with trial site)
- Your kidney function, liver function, and bilirubin levels are within acceptable ranges based on blood tests (confirm with trial site)
- Your lung breathing capacity is at least 70% of what is considered normal for someone your age and size
- You have no history of unusual or abnormal bleeding
- You are willing and able to give written consent to participate
Who may not be able to join:
- You currently have an active infection that is not under control
- You have high blood pressure that requires more than 3 medications to manage
- You have had a blood clot in your lungs within the past 6 months
- You have significant heart problems, such as a heart attack in the last 6 months, severe heart failure, uncontrolled chest pain, serious irregular heartbeat, or signs of reduced blood flow to the heart
- You have a history of heart bypass surgery or a procedure to open blocked arteries (though you may still be considered on a case-by-case basis after a heart evaluation)
- You have tested positive for HIV, hepatitis B, or hepatitis C
- You are currently pregnant or breastfeeding
- You are able to have children (or have a partner who is able to have children) and are unwilling to use contraception during the trial
- The lead doctor of the trial determines that joining the trial would pose an unacceptable health or mental health risk to you
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Daniel Fowler, M.D., Rapa Therapeutics LLC
Phone: (301) 518-3104
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
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).
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.