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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
Must not have
Patients of Subjects of childbearing age, or males who have a partner of childbearing potential, who are unwilling to practice contraception
Hypertension not adequately controlled by ≤ 3 medications
Timeline
Screening 3 weeks
Treatment Varies
Follow Up six months after treatment initiation.
Awards & highlights
No Placebo-Only Group

Summary

This trial tests a new treatment for ALS using a patient's own immune cells that are modified to reduce inflammation. It targets ALS patients who have not progressed beyond certain limits. The goal is to see if this treatment 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.
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.
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 below
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I do not have a history of unusual bleeding.
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I have enough T cells for RAPA-501 cell production.
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I have been diagnosed with ALS.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I am of childbearing age and not willing to use contraception.
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My high blood pressure is not well-managed with 3 or fewer medications.
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I do not have recent severe heart problems or uncontrolled heart conditions.
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I do not have an ongoing infection that isn't responding to treatment.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~six months after treatment initiation.
This trial's timeline: 3 weeks for screening, Varies for treatment, and six months after treatment initiation. for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Characterize immune system parameters pre- and post-therapy.
Other study objectives
Cerebrospinal fluid assessment
Neuroinflammation assessment

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

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
5 Previous Clinical Trials
155 Total Patients Enrolled
1 Trials studying Amyotrophic Lateral Sclerosis
40 Patients Enrolled for Amyotrophic Lateral Sclerosis
Massachusetts General HospitalOTHER
3,026 Previous Clinical Trials
13,413,779 Total Patients Enrolled
38 Trials studying Amyotrophic Lateral Sclerosis
13,819 Patients Enrolled for Amyotrophic Lateral Sclerosis
Hackensack Meridian HealthOTHER
135 Previous Clinical Trials
30,281 Total Patients Enrolled
1 Trials studying Amyotrophic Lateral Sclerosis
40 Patients Enrolled for Amyotrophic Lateral Sclerosis
Daniel Fowler, M.D.Study DirectorRapa Therapeutics LLC
4 Previous Clinical Trials
115 Total Patients Enrolled

Media Library

RAPA-501 (T-cell Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04220190 — Phase 2 & 3
Amyotrophic Lateral Sclerosis Research Study Groups: Phase 2/3 Expansion Cohort, Single-agent RAPA-501 T cells, Phase 1/2 Only, Single-agent RAPA-501 T cells (dose level Arm 1), : Phase 1/2 Only, Single-agent RAPA-501 T cells (dose level Arm 2), Phase 1/2 Only, RAPA-501 + PC Regimen (Arm 3A)
Amyotrophic Lateral Sclerosis Clinical Trial 2023: RAPA-501 Highlights & Side Effects. Trial Name: NCT04220190 — Phase 2 & 3
RAPA-501 (T-cell Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04220190 — Phase 2 & 3
~27 spots leftby Jul 2026
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