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About
This study is a randomized, placebo-controlled, phase 2a trial to study the biological activity, safety, and tolerability of regulatory T Lymphocytes (Tregs) taken and expanded outside of the body and returned back to the same person whose Treg were removed, given back by IV (intravenously) and in combination with low-dose IL-2 in people with Amyotrophic Lateral Sclerosis (ALS).
Full description
Based on data collected in a previous study with a small group of patients, evidence was found to show that interfering with the immune system using Treg cells slowed ALS disease progression. It is known that Treg cell numbers and function are reduced in patients with ALS and in some patients with lower Treg cells, they have a more marked rapid progression of their ALS. For this study, there are two sites (in Houston, Texas and Boston, Massachusetts) in which Tregs will be taken from participants, increased or expanded outside the body, and then re-administered back to the participants from which the Tregs came.
This study has two parts and due to the pandemic two groups [Group 1 and Group 2]:
Groups:
GROUP 1 will go through the double-blind part of the study (receive either Tregs infusions and IL2 injections OR saline infusions and saline injections) for six months and open label part of the study (receive Treg injections and IL2 injections) for six months.
GROUP 2 will receive only the open label (Tregs infusions and IL2 injections) for six months.
This study is studying whether the enhancement of Treg numbers and function will slow disease progression.
In the first study of Tregs, we completed a single-center, open-label phase I study of Tregs from people with ALS. Tregs were increased outside the body and returned back to the individual Treg owners in multiple doses every 2 to 4 weeks. This early study provided evidence in a small group of patients that treatment with autologous Tregs may be effective in slowing ALS progression.
Enrollment
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Inclusion criteria
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:
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.
Primary purpose
Allocation
Interventional model
Masking
12 participants in 3 patient groups, including a placebo group
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Data sourced from clinicaltrials.gov
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