A Trial of Tocilizumab in ALS Subjects

A Trial of Tocilizumab in ALS Subjects

Quick Info:

Status:
Ongoing, But Not Recruiting
Estimated Enrollment:
22
Phase:
2
Treatment Type:
Drug
Trial Type:
Double blind, Randomized
Sponsor:
Barrow Neurological Institute
Primary Investigator:
Contact Information:

Enrollment Criteria:

Forced Ventilation

Breathing Ability

Percent lung function (FVC) or (SVC)
>60
Months/Onset

Months Since Onset

Number of months since first
symptoms of ALS
<36
BiPap Allowed

Non-Invasive Ventilation (NIV)

Can PALS use a BiPAP in the trial?
Maybe
DPS Allowed

Diaphragm Pacer (DPS)

Can PALS use a DPS in the trial?
No
Edaravone Usage

Edaravone Usage

Can a PALS use edaravone (Radicut/Radicava)
while enrolled in the trial?
Unknown

Update Notes:

4/11/2018Recruitment Status updated
12/12/2017Location updated
8/25/2017No significant updates
12/27/2016Based on enrollment figures, moved proposed completion date ahead full year, from Q3 2017 to Q3 2018.
2/8/2016Recruitment status updated.
12/8/2015Recruitment status updated.
6/12/2015Clinical trial added.

Locations:

Barrow Neurological Institute, Phoenix, 85013
University of Kansas Medical Center, Kansas City, 66160
Wake Forest University School of Medicine, Winston-Salem, 27157
Penn State College of Medicine Milton S. Hershey M, Hershey, 17033
Massachusetts General Hospital, Boston, 02114

Other Information:

Purpose: This research study is being done to find out if tocilizumab, also known as Actemra™, can help with Amyotrophic Lateral Sclerosis (ALS). The investigators also want to find out if tocilizumab is safe to take without causing too many side effects. Currently ALS has no cure and only 1 modestly effective treatment to slow the progression of the disease. Although not the initial cause of ALS, the immune system plays a role in the death of motor neurons. The immune cells that participate in this process are stimulated by a substance called interleukin-6 (IL-6) whose effect is blocked by tocilizumab and thus, may slow the death of motor neurons and slow the disease.
Eligibility: 18 to 75 years old, all genders, No healthy volunteers
Details: This is a multicenter, randomized, double-blind, placebo-controlled 16-week study evaluating the safety and tolerability of tocilizumab in subjects with 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.
Collaborator(s):
News Articles and Summaries:
ALS Forum:
First Published on Clinicaltrials.gov: 6/3/2015
ClinicalTrials.gov ID: NCT02469896
Trial Protocol as Published on Clinicaltrials.gov:
ClinicalTrials.gov