Gilenya in Amyotrophic Lateral Sclerosis (ALS)
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|ClinicalTrials.gov Identifier: NCT01786174|
Recruitment Status : Completed
First Posted : February 7, 2013
Results First Posted : December 31, 2015
Last Update Posted : July 1, 2016
|Condition or disease||Intervention/treatment||Phase|
|Amyotrophic Lateral Sclerosis||Drug: Gilenya Other: Placebo||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||30 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||Phase IIa Double-Blind, Placebo-Controlled Study to Evaluate the Safety of Oral Fingolimod in Patients With Amyotrophic Lateral Sclerosis (ALS)|
|Study Start Date :||August 2013|
|Actual Primary Completion Date :||September 2014|
|Actual Study Completion Date :||May 2015|
Experimental: Gilenya (fingolimod)
0.5mg Gilenya (fingolimod) orally once daily for 28 days +/- 3 days
0.5mg Gilenya orally by mouth once daily for approximately 28 days
Other Name: fingolimod
Placebo Comparator: Placebo
0.5mg placebo (sugar pill) orally once daily for 28 days +/- 3 days
0.5mg placebo (sugar pill) orally by mouth once daily for approximately 28 days
- ALSFRS-R Total Score at Weeks 0, 2, 4 and 8 [ Time Frame: Week 0, Week 2, Week 4 and Week 8 ]The ALSFRS-R is a quickly administered (5 minutes) ordinal rating scale (ratings 0-4) used to determine subjects' assessment of their capability and independence in 12 functional activities. All 12 activities are relevant in ALS. Initial validity was established by documenting that in ALS patients, change in ALSFRS-R scores correlated with change in strength over time, was closely associated with quality of life measures, and predicted survival.
- Change in Slow Vital Capacity Score (SVC) [ Time Frame: Week 0, Week 2, Week 4 and Week 8 ]The vital capacity (VC) (percent of predicted normal) was determined using the slow VC method. Vital Capacity is the maximum amount of air a person can expel from the lungs after a maximum inhalation. A subject's VC depends on their age, sex and height. The value is recorded as a percent of predicted normal.
- Forced Expiratory Volume in 1 Second (FEV1) [ Time Frame: Screening, Week 0, Week 2, and Week 4 ]Forced Expiratory Volume (FEV1): Forced Expiratory Volume (FEV1) is the amount of air which can be forcibly exhaled from the lungs in the first second of a forced exhalation.
- Lymphocyte (T-Cell) Subset Trajectories [ Time Frame: Week 0, Week 2, and Week 4 ]Gilenya (fingolimod) has been shown to successfully reduce circulating lymphocytes (a type of white blood cell) by blocking their egress (exit) from the lymph nodes. A secondary objective of the study is to quantify the effect of the treatment on circulating lymphocyte populations in patients with ALS.
- Forced Expiratory Volume in 1 Second (FEV1) / Slow Vital Capacity (SVC) Ratio [ Time Frame: Screening, Week 0, Week 2, and Week 4 ]
Forced Expiratory Volume (FEV1): Forced Expiratory Volume (FEV1) is the amount of air which can be forcibly exhaled from the lungs in the first second of a forced exhalation.
Slow Vital Capacity (SVC): Vital Capacity is the maximum amount of air a person can expel from the lungs after a maximum inhalation. A subject's VC depends on their age, sex and height. The value is recorded as a percent of predicted normal.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01786174
|United States, California|
|University of California, Irvine|
|Orange, California, United States, 92868|
|United States, Georgia|
|Georgia Regents University|
|Augusta, Georgia, United States, 30912|
|United States, Massachusetts|
|Massachusetts General Hospital|
|Boston, Massachusetts, United States, 02114|
|United States, Texas|
|Methodist Neurological Institute|
|Houston, Texas, United States, 77030|
|Principal Investigator:||James D Berry, MD, MPH||Massachusetts General Hospital|