T-regulatory Cells in ALS

• Register: ClinicalTrials.gov
• Last refreshed on: 11 October 2021
• Main ID: NCT04055623
• Date of registration: 12/08/2019
• Prospective Registration: No
• Primary sponsor: The Methodist Hospital Research Institute
• Public title: T-regulatory Cells in ALS Tregs in ALS
• Scientific title: Phase 2a Study of the Expansion and Infusion of Autologous T-Regulatory Cells in Amyotrophic Lateral Sclerosis
• Date of first enrolment: August 7, 2019
• Target sample size: 12
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT04055623
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: James D. Berry, MD, MPH
• Affiliation: Massachusetts General Hospital

• Name: Jason R. Thonhoff, MD, PhD
• Affiliation: The Methodist Hospital Research Institute

• Name: Stanley H. Appel, MD
• Affiliation: The Methodist Hospital Research Institute

Key inclusion & exclusion criteria

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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

ALS (Amyotrophic Lateral Sclerosis)

Intervention(s)

Other: Monthly placebo infusions + 3 times per week placebo injections

Biological: Monthly autologous Treg cells infusions + 3 times per week Interleukin-2 injections

Primary Outcome(s)

Change in Treg suppressive function in the blood from baseline to week 24. [Time Frame: Baseline and week 24.]

Secondary Outcome(s)

Tolerability of ascending doses of Tregs for 6 months of treatment [Time Frame: Baseline to week 24.]

Change in Treg numbers in the blood from baseline to week 24. [Time Frame: Baseline and week 24]

Tolerability of Treg infusions for 6 months of treatment [Time Frame: Baseline to week 24.]

Secondary ID(s)

Pro00022167

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

The Center for Clinical and Translational Sciences (CCTS) Clinical Research Unit at The University of Texas Health Science Center at Houston

North East Amyotrophic Lateral Sclerosis Consortium

Massachusetts General Hospital