T-regulatory Cells in ALS (Tregs in ALS)

This is an interventional treatment trial for ALS (Amyotrophic Lateral Sclerosis) focused on measuring Lou Gehrig's Disease Read More

Inclusion Criteria:

• ALS meeting El Escorial criteria for possible, probable, lab-supported probable, or definite ALS.
• At least 18 years old.
• Provided informed consent and authorized use of protected health information (PHI) in accordance with national and local patient privacy regulations.
• Capable of complying with all study procedures, including the study drug delivery procedure, in the Investigator's opinion.
• On a stable regimen of riluzole for at least 30 days at the time of screening. If not on riluzole at the time of study entry, willing to refrain from initiation of the agent for the duration of the trial.
• Patients on edaravone willing to refrain from taking edaravone on the same day as they will receive the Tregs infusion for the duration of the trial. If not on edaravone at the time of study entry, willing to refrain from initiation of the agent for the duration of the trial.
• Medical record documentation of a decline in ALSFRS-R total score of at least two points in the 90 days prior to screening or at least four points over the 180 days prior to screening.
• Forced vital capacity (FVC) ≥65% of predicted capacity for age, height, and gender at screening.
• Patient able and willing to undergo leukapheresis.

Exclusion Criteria:

• Presence of any of the following clinical conditions that would interfere with the safe conduct of the study, as determined by the Investigator:

  • Unstable neurological, cardiovascular, cerebrovascular, pulmonary, renal, hepatic, endocrine, or hematologic disease; active malignancy or infectious disease; or other medical illness.
  • Unstable psychiatric illness defined as psychosis (hallucinations or delusions), unstable major depression or substance abuse within 180 days prior to screening.
  • Persistent asthma, prior history of acute systemic reactions involving immunoglobulin E (IgE)-dependent mechanisms, history of angioedema, or history of anaphylactic reactions to any medication.
• Serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 3 times the upper limit of normal (ULN) at screening.
• Serum creatinine greater than 1.8 mg/dL or creatinine clearance less than 40 mL/min at screening.
• History of or positive test result for human immunodeficiency virus (HIV), hepatitis C virus, or hepatitis B virus (i.e., positive for both hepatitis B surface antigen and hepatitis B core antibody) at screening.
• Tracheostomy.
• If female, breastfeeding, known to be pregnant, planning to become pregnant during the study, or unwilling to use effective contraception for the duration of the trial and for 90 days after treatment.
• If male of reproductive capacity, unwilling to use effective contraception for the duration of the trial and for 90 days after treatment.
• Enrollment in any other interventional study.
• Treatment with another investigational drug, biological agent, or device within 30 days or 5 half-lives of screening, whichever is longer. Patient participation in an observational/non-interventional clinical study is to be discussed with the Medical Monitor.
• Prior gene or cell therapy treatments for ALS.