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
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
Treatments tested in this trial
- RAPA-501 Autologous T stem cells
Treatment groups
41 Participants
are divided into 1 treatment groupSponsors and collaborators
Rapa Therapeutics LLC
Lead sponsor
Massachusetts General Hospital
Collaborator
Source ClinicalTrials.gov