A Phase 2 Randomized, Placebo Controlled Trial of Tocilizumab in ALS Subjects
Study Purpose:
The purpose of the study is to evaluate the safety and tolerability of tocilizumab (Actemra) in subjects with sporadic ALS.
Disease:
Amyotrophic Lateral Sclerosis (ALS), Familial ALS, Sporadic ALSStudy Type:
Interventional TrialStudy Category:
Drug TrialStudy Status:
Not enrollingPhase:
Phase IIStudy Chair(s)/Principal Investigator(s):
Shafeeq Ladha, MD, Barrow Neurological Institute
Clinicaltrials.gov ID (11 digit #):
NCT02469896Neals Affiliated?
YesCoordinating Center Contact Information
Mass General HospitalMichelle (Shelley) McGovern / .(JavaScript must be enabled to view this email address) / 617-643-1618
Armineuza Evora / .(JavaScript must be enabled to view this email address) 165 Cambridge Street
6th Floor, Room 653A
Boston, Massachusetts 02114 United States
Full Study Summary:
This is a multicenter, randomized, double-blind, placebo-controlled 16-week study evaluating the safety and tolerability of tocilizumab in subjects with sporadic ALS.
The primary objective of the study is to determine the safety and tolerability of intravenous administration of 8 mg/kg of tocilizumab every 4 weeks vs. matched intravenous placebo administered every 4 weeks over an 8 week period.
The secondary objectives of the study are to describe the expression of pro-inflammatory genes in Peripheral Blood Mononuclear Cells (PBMCs) of sporadic ALS patients, to assess the ability of tocilizumab to reduce the expression of pro-inflammatory genes in PBMCs and pro-inflammatory cytokines in the cerebrospinal fluid (CSF) of patients with sporadic ALS and to assess the CSF penetration of tocilizumab.
Approximately 4 Northeast ALS Consortium (NEALS) Centers in the US will participate in the study. Twenty-four subjects will be randomized in the study.
This study will be conducted in subjects who meet the El Escorial criteria of possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS. At screening, eligible subjects must be at least 18 years old, must have a slow vital capacity (SVC) ≥ 60% of predicted capacity for age, height and gender, and must provide written informed consent prior to screening. Subjects on a stable dose of riluzole and those not taking riluzole, and women of child-bearing age at screening are eligible for inclusion as long as they meet specific protocol requirements. Detailed criteria are described in the body of the protocol.
Subjects will be randomly assigned in a 2:1 ratio to intravenous tocilizumab 8 mg/kg or matching placebo every 4 weeks over an 8 week period.
This research study protocol allows the subject to receive up to 3 infusions of Tocilizumab. Even if the treatment is shown to be of benefit, additional infusions of Tocilizumab beyond that allowed in the protocol cannot be given to the subject while she/he is participating in this study.
Subjects will remain on randomized, placebo-controlled, double-blind treatment until the Week 8 visit. Each randomized subject will also have a Week 12 Follow-up visit and Week 16 End-of-Study visit to assess for adverse events (AEs), changes in concomitant medications, to administer the ALSFRS-R and selected study procedures.
Study Sponsor:
ALS Association, Barrow Neurological Foundation Massachusetts General Hospital, Genentech, Inc.Participant Duration:
16-weeks