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RecruitingPhase 2 / Phase 3interventionalRules rewrite

Phase 2 / Phase 3: testing RAPA-501 Autologous T stem cells for Amyotrophic Lateral Sclerosis

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).

Official registry: https://clinicaltrials.gov/study/NCT04220190 · Rapa Therapeutics LLC · enrollment listed as 41

What this phase means

Phase 2/3: earlier tests looked promising enough to expand. This stage asks, in a larger group, whether the treatment really works and is safe enough to seek approval.

Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.

What the registry says happens

If you join, the listed intervention(s) are RAPA-501 Autologous T stem cells. The registry lists allocation as na. 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).

They plan to enroll about 41 people at Boston. Taking part typically means an infusion or injection of a biologic plus check-ins.

Official title (unrewritten)

Phase 2/3 Trial of Autologous Hybrid TREG/Th2 (RAPA-501) T Stem Cell Therapy for Amyotrophic Lateral Sclerosis

Interventions: RAPA-501 Autologous T stem cells

Primary outcome (registry wording): 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).

Who it is for (from inclusion criteria)

  • Ages 18 Years to any
  • 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

Public study contact (from the registry)

Daniel Fowler, M.D. Chief Medical Officer, RAPA Therapeutics, LLC

(301) 518-3104

dan@rapatherapeutics.com

Published on ClinicalTrials.gov. Contact them as a possible participant. They decide eligibility.

Sites listed on ClinicalTrials.gov

1 listed

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FAQ about this NCT record

Is NCT04220190 still recruiting?

The sponsor posted status “Recruiting” on ClinicalTrials.gov at last ingest. Sites can pause independently. Confirm with the official record and the listed contact.

What phase is NCT04220190?

Phase 2 / Phase 3. Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.

Can PlainTrials tell me if I qualify for NCT04220190?

No. The inclusion and exclusion lists on this page are readable extracts of the public eligibility block. Only the study team can screen you.

How to read a ClinicalTrials.gov record · Questions to ask the coordinator · Glossary