T-regulatory Cells in ALS

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).

Conditions

• ALS (Amyotrophic Lateral Sclerosis)

Interventions

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.

Sponsors & Collaborators

• Massachusetts General Hospital

  collaborator OTHER

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

  collaborator OTHER

• North East Amyotrophic Lateral Sclerosis Consortium

  collaborator NETWORK

• The Methodist Hospital Research Institute

  lead OTHER

Principal Investigators

• Stanley H. Appel, MD · The Methodist Hospital Research Institute

• Jason R. Thonhoff, MD, PhD · The Methodist Hospital Research Institute

• James D. Berry, MD, MPH · Massachusetts General Hospital

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Model

PARALLEL

Eligibility

Min Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2019-08-07

Primary Completion

2022-05-31

Completion

2022-08-31

FDA Drug

Yes

Countries

• United States

Study Locations