Gilenya in Amyotrophic Lateral Sclerosis (ALS)

Quick Info:
Status:
Ongoing, But Not Recruiting
Estimated Enrollment:
30
Phase:
II
Treatment Type:
orally available
Trial Type:
Double-Blind, Placebo-Controlled
Sponsor:
Massachusetts General Hospital
Primary Investigator:
James D Berry, MD, MPH Massachusetts General Hospital
Contact Information:
Enrollment Criteria:
  • Forced Ventilation
    Breathing Ability
    Percent lung function (FVC) or (SVC)
    >65%
  • Months/Onset
    Months Since Onset
    Number of months since first symptoms of ALS
    <24
  • BiPap Allowed
    Non-Invasive Ventilation (NIV)
    Can PALS use a BiPAP in the trial?
    No
  • DPS Allowed
    Diaphragm Pacer (DPS)
    Can PALS use a DPS in the trial?
    No
Update Notes:
12/9/2014Recruitment status updated.
4/14/2014No significant updates.
2/3/2014No significant updates.
1/24/2014Recruitment status updated
11/13/2013All recruitment sites currently recruiting
10/11/2013Location recruitment status updated
8/21/2013GA location and contact information updated
8/9/2013This study has one site currently recruiting.
3/27/2013Enrollment criteria added.
2/7/2013Trial added.
Locations: (Available Locations: 4) Recruiting?
United States, GA, Augusta, Georgia Regents UniversityNO
United States, MA, Boston, Massachusetts General HospitalNO
United States, TX, Houston, Methodist Neurological InstituteNO
United States, CA, Orange, University of California, IrvineNO
Other Information:
Purpose: The purpose of this study is to determine whether Gilenya, also known as fingolimod, is safe and tolerable in patients with Amyotrophic Lateral Sclerosis (ALS).
Eligibility: 18 years and older. Both genders. No healthy volunteers.
Details: The primary objective of the study is to determine the acute safety and tolerability of oral administration of Gilenya (fingolimod) 0.5mg daily vs. matched oral placebo administered daily. The primary outcome measure will be safety and tolerability; safety will be assessed by the occurrence of adverse events and clinically meaningful changes in vital signs, ophthalmologic examination, physical examination, electrocardiogram and standard clinical laboratory blood tests, and tolerability will be defined as the ability of subjects to complete the entire 4-week study. The secondary outcome measure will be the measured effect of the treatment on circulating lymphocyte populations in patients with ALS. Exploratory outcome measures will include the rate of decline of the ALS Functional Rating Scale (Revised) (ALSFRS-R) and Slow Vital Capacity (VC) during the course of treatment. 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 an SVC ≥ 65% 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. Subjects will remain on randomized, placebo-controlled, double-blind treatment until the Week 4 visit. Each randomized subject will also have a Week 8 Follow-up Telephone Interview to assess for adverse events (AEs), changes in concomitant medications and to administer the ALSFRS-R.
Collaborator(s):
ALS Forum:
First Published on Clinicaltrials.gov: 2/4/2013
ClinicalTrials.gov ID: NCT01786174
Trial Protocol as Published on Clinicaltrials.gov:
ClinicalTrials.gov