NCT04055623

T-regulatory Cells in ALS

Phase 2a Study of the Expansion and Infusion of Autologous T-Regulatory Cells in Amyotrophic Lateral Sclerosis

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: The Methodist Hospital Research Institute · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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).

Detailed 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 \[Group 1 and Group 2\]: 1. The first period is a 6-month, randomized, placebo-controlled part of the study to study the biological activity, safety, and tolerability of the monthly expanded Tregs administered intravenously (IV) OR placebo (saline) administered intravenously plus subcutaneous low-dose Interleukin-2 (IL-2) OR subcutaneous placebo (saline) injections in 12 adults with ALS. IL2 helps regulate the immune system's white blood cells. \[Group 1 only.\] 2. The second period is a 6-month OPEN-LABEL part of the study (no placebo) in which all participants will receive their own expanded Treg cells administered intravenously in combination with subcutaneous low-dose IL-2 at the following schedule: single dose of Treg cells twice (once per month with three IL2 injections per week); followed by double the dose of Treg cells twice (once per month with three IL2 injections per week); followed by triple the dose of Treg cells twice (one per month with three IL2 injection per week); followed by a 1 month follow-up safety visit after the last triple dose of Tregs with was received and the last set of IL2 injections three times per week injections. \[Group 1 and Group 2.\] 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.

Conditions

• ALS (Amyotrophic Lateral Sclerosis)

Interventions

Type	Name	Description
BIOLOGICAL	Monthly autologous Treg cells infusions + 3 times per week Interleukin-2 injections	For the first 6-months of the study: T-regulatory cells taken from a patient, increased in number in a lab, and returned through monthly intravenous (IV) infusions back to same patient + 3 times per week subcutaneous Interleukin-2 injections
OTHER	Monthly placebo infusions + 3 times per week placebo injections	For first 6-months of study: monthly placebo infusions + 3 times per week subcutaneous placebo injections
BIOLOGICAL	Monthly autologous Treg cells infusions + 3 times per week Interleukin-2 injections	For the second 6-months of the study: all participants will receive T-regulatory cells taken from the patient, that have been increased in number in a lab, and returned through monthly intravenous (IV) infusions back to same patient (Treg cell owner) + 3 times per week subcutaneous Interleukin-2 injections.

Timeline

• Start date: 2019-08-07
• Primary completion: 2022-05-31
• Completion: 2022-08-31
• First posted: 2019-08-14
• Last updated: 2021-10-06

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study