Health TrialsbyHealthPanel

RAPA-501 Therapy for ALS

ConditionAmyotrophic Lateral Sclerosis
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age18+
SponsorRapa Therapeutics LLC

About this trial

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).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

Active uncontrolled infection.

Hypertension not adequately controlled by ≤ 3 medications.

History of documented pulmonary embolus within 6 months of enrollment.

Clinically significant cardiac pathology, as defined by: myocardial infarction within 6 months prior to enrollment, Class III or IV heart failure according to NYHA, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities.

Trial design

Design model

Single group

Treatments tested in this trial

  • RAPA-501 Autologous T stem cells

    Biological/Vaccine

    Experimental: Phase 2/3 Expansion Cohort, Single-agent RAPA-501 T stem cells 80 x 10EE6 cells per infusion (no host conditioning)

Treatment groups

41 Participants
are divided into 1 treatment group
Group A: RAPA-501 Autologous T stem cellsExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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).

Time frame
Through 30 Weeks On-Study

Secondary outcomes

1

Characterize immune system parameters pre- and post-therapy.

Specifically, relative to pretreatment patient peripheral blood values, determine whether study interventions: (a) increase circulating Th2 and TREG cells; (b) reduce circulating Th1 cells; (c) increase T cell expression of programmed death-1 (PD-1); and (d) reduce inflammatory cytokines IL-1-beta, IL-6, and TNF-alpha.

Time frame
Through 30 Weeks On-Study
2

Relative to pretreatment values, characterize the potential effect of RAPA-501 therapy on serum markers of neurodegeneration (neurofilament light, NfL).

Time frame
Through 30 Weeks On-Study
3

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).

Time frame
Through 30 Weeks On-Study
4

Relative to pretreatment values, characterize the potential effect of RAPA-501 therapy on hand grip strength using hand-held dynamometry.

Time frame
Through 30 Weeks On-Study

Other outcomes

Sponsors and contacts

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Rapa Therapeutics LLC

Lead sponsor

Massachusetts General Hospital

Collaborator

Source ClinicalTrials.gov
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