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T-cell Therapy
RAPA-501 Therapy for ALS
Phase 2 & 3
Recruiting
Research Sponsored by Rapa Therapeutics LLC
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
No history of abnormal bleeding tendency
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up through 2 years and 30 weeks on-study
Awards & highlights
Study Summary
This trial is testing a new treatment for ALS called RAPA-501 autologous T cells. The trial is open to adults with ALS, and will test different doses of the treatment to see what is safe and effective.
Who is the study for?
This trial is for adults diagnosed with ALS within the last 24 months. They can still take certain ALS medications if doses have been stable for 30 days. Participants need normal kidney function, a specific range of ALSFRS-R scores, and adequate breathing capacity. Exclusions include uncontrolled hypertension, recent heart or pulmonary issues, active infections, positive HIV/Hepatitis tests, pregnancy/breastfeeding, and unwillingness to use contraception.Check my eligibility
What is being tested?
The RAPA-501-ALS study is testing a new therapy using patients' own modified T cells (RAPA-501) to treat ALS. It's in phase 2/3 which means it's looking at both effectiveness and safety in a larger group after initial promising results.See study design
What are the potential side effects?
Potential side effects are not explicitly listed but may include immune reactions due to the introduction of modified T cells into the body. Side effects could also relate to typical risks associated with cell therapies such as infusion reactions or complications from underlying conditions.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I do not have a history of unusual bleeding.
Select...
I have enough T cells for RAPA-501 cell production.
Select...
I have been diagnosed with ALS.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ through 2 years and 30 weeks on-study
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~through 2 years and 30 weeks on-study
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
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).
Secondary outcome measures
Characterize immune system parameters pre- and post-therapy.
Relative to pretreatment values, characterize the potential effect of RAPA-501 therapy on hand grip strength using hand-held dynamometry.
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).
+2 moreOther outcome measures
Cerebrospinal fluid assessment
Neuroinflammation assessment
Trial Design
4Treatment groups
Experimental Treatment
Group I: Phase 2/3 Expansion Cohort, Single-agent RAPA-501 T cellsExperimental Treatment1 Intervention
80 x 10^6 cells per infusion (no host conditioning)
Group II: Phase 1/2 Only, Single-agent RAPA-501 T cells (dose level Arm 1)Experimental Treatment1 Intervention
Dose level 1 is 20 x 10^6 cells/infusion
Group III: Phase 1/2 Only, RAPA-501 + PC Regimen (Arm 3A)Experimental Treatment1 Intervention
RAPA-501 T cell therapy preceded by the 3-day pentostatin-cyclophosphamide (PC) regimen
Group IV: : Phase 1/2 Only, Single-agent RAPA-501 T cells (dose level Arm 2)Experimental Treatment1 Intervention
Dose level 2 is 80 x 10^6 cells/infusion
Find a Location
Who is running the clinical trial?
Rapa Therapeutics LLCLead Sponsor
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50 Total Patients Enrolled
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Massachusetts General HospitalOTHER
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13,198,440 Total Patients Enrolled
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Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- It has been over 2 weeks since my last major surgery or experimental treatment.I have had a blood clot in my lungs within the last 6 months.My ALS symptoms started less than 24 months ago.I have recovered from major side effects of previous treatments.I do not have a history of unusual bleeding.I have had heart surgery or angioplasty and will undergo a heart check.Your bilirubin level should be less than 1.5, unless you have Gilbert's disease.I am of childbearing age and not willing to use contraception.Your ALSFRS-R score is between 34 and 45.Your AST and ALT levels are not more than three times the normal upper limit.Your blood creatinine level is 2.0 mg/dL or lower.I have been on a stable dose of riluzole, edaravone, or sodium phenylbutyrate/taurusodial for at least 30 days.You have tested positive for HIV, hepatitis B, or hepatitis C.My high blood pressure is not well-managed with 3 or fewer medications.Your lung function test shows that you can breathe out a certain amount of air, which is at least 70% of what is expected for someone your age and size.I do not have recent severe heart problems or uncontrolled heart conditions.I have enough T cells for RAPA-501 cell production.I do not have an ongoing infection that isn't responding to treatment.I am 18 years old or older.I have been diagnosed with ALS.
Research Study Groups:
This trial has the following groups:- Group 1: Phase 2/3 Expansion Cohort, Single-agent RAPA-501 T cells
- Group 2: Phase 1/2 Only, Single-agent RAPA-501 T cells (dose level Arm 1)
- Group 3: : Phase 1/2 Only, Single-agent RAPA-501 T cells (dose level Arm 2)
- Group 4: Phase 1/2 Only, RAPA-501 + PC Regimen (Arm 3A)
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Does this research still accept volunteers?
"According to the details found on clinicaltrials.gov, this medical study is presently seeking participants who meet certain criteria. It was initially published on December 15th 2020 and underwent its last amendment on March 1st 2022."
Answered by AI
What is the ultimate aim of this clinical experiment?
"This clinical trial intends to ascertain the maximum tolerated dosage of RAPA-501 T cells over a 30 day period. Secondary objectives include assessing hand grip strength before and after therapy, establishing disease activity scores pre and post treatment, as well as quantifying changes in circulating Th1 cells with TBET expression levels prior to and following intervention (measured by number of positive cells per microliter)."
Answered by AI
How many participants are involved in the experiment?
"That is correct. As per clinicaltrials.gov, this medical trial was initially listed on December 15th 2020 and updated most recently in March 1st 2022. The researchers are aiming to recruit 21 patients from 2 sites."
Answered by AI
Who else is applying?
What site did they apply to?
Massachusetts General Hospital
What portion of applicants met pre-screening criteria?
Met criteria
How many prior treatments have patients received?
2
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