Gilenya in Amyotrophic Lateral Sclerosis (ALS)

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01786174
• Date of registration: 04/02/2013
• Prospective Registration: Yes
• Primary sponsor: Massachusetts General Hospital
• Public title: Gilenya in Amyotrophic Lateral Sclerosis (ALS)
• Scientific title: Phase IIa Double-Blind, Placebo-Controlled Study to Evaluate the Safety of Oral Fingolimod in Patients With Amyotrophic Lateral Sclerosis (ALS)
• Date of first enrolment: August 2013
• Target sample size: 30
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01786174
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: James D Berry, MD, MPH
• Affiliation: Massachusetts General Hospital

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Age 18 years or older.

2. Sporadic or familial ALS diagnosed as possible, laboratory-supported probable,
probable, or definite as defined by revised El Escorial criteria (Appendix 1).

3. Onset of weakness or spasticity due to ALS = 2 years (24 months) prior to Baseline
Visit.

4. Slow vital capacity (SVC) measure =65% of predicted for gender, height, and age at the
screening visit.

5. Subjects must not have taken riluzole for at least 30 days, or be on a stable dose of
riluzole for at least 30 days, prior to randomization (riluzole-naïve subjects are
permitted in the study).

6. Subjects must be able to swallow oral medication at the Screening Visit and expected
to be able to swallow the capsule throughout the course of the study.

7. Capable of providing informed consent and following trial procedures.

8. Geographically accessible to the site.

9. Women must not be able to become pregnant (e.g. post menopausal, surgically sterile,
or using adequate birth control methods) for the duration of the study and three
months after study completion. Adequate contraception includes: abstinence, hormonal
contraception (oral contraception, implanted contraception, injected contraception or
other hormonal (patch or contraceptive ring, for example) contraception), intrauterine
device (IUD) in place for = 3 months, barrier method in conjunction with spermicide,
or another adequate method.

10. Subjects must agree not to take live attenuated vaccines (including seasonal flu
vaccine) 30 days before randomization, throughout the duration of the trial and for 60
days following the trial.

Exclusion Criteria:

1. Prior use of fingolimod (Gilenya®).

2. History or presence of cardiac conditions including:

1. Cardiovascular or cerebrovascular disease in the previous 6 months (eg.
myocardial infarction, unstable angina, or stroke)

2. Congestive heart failure

3. First, second- or third-degree atrioventricular block, sick sinus syndrome, or
other serious cardiac rhythm disturbances

4. Any history of Torsades de Pointes

3. Treatment with a prohibited medication within 30 days of the Baseline Visit:

a. Class Ia or III antiarrhythmic medications: i.e., Quinidine, Sotalol Includes
Nuedexta b. QT interval prolonging medications c. Ketoconazole d. Beta-blockers e.
Calcium channel blockers f. Immunosuppressant medication g. Chemotherapeutic
(anti-neoplastic) medications

4. Evidence on examination or ECG of bradycardia (<55 bpm), QTc >450ms for women or >430
msec for men, or 1st degree or higher conduction block.

5. History of unexplained syncope or cardiac syncope.

6. Serum AST and ALT value >2.0 times the upper normal limit.

7. Active infection (acute or chronic).

8. History of diabetes.

9. History of macular edema or uveitis.

10. History of lymphopenia.

11. History of acquired or inherited immune deficiency syndrome, including leukopenia.

12. History of severe untreated chronic obstructive sleep apnea.

13. Exposure to any other agent currently under investigation for the treatment of
patients with ALS (off-label use or investigational) within 30 days of the Baseline
Visit.

14. Presence of tracheostomy.

15. Use of non-invasive ventilation for hypoventilation due to ALS (such as BiPAP).

16. Presence of feeding tube.

17. Presence of diaphragmatic pacing system.

18. The presence of unstable psychiatric disease, cognitive impairment, or dementia that
would impair ability of the subject to provide informed consent, according to PI
judgment, or a history of active substance abuse within the prior year.

19. Clinically significant history of unstable or severe cardiac, oncologic, hepatic, or
renal disease, or other medically significant illness.

20. Pregnant women or women currently breastfeeding.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: Gilenya

Other: Placebo

Primary Outcome(s)

Change in Slow Vital Capacity Score (SVC) [Time Frame: Week 0, Week 2, Week 4 and Week 8]

Forced Expiratory Volume in 1 Second (FEV1) [Time Frame: Screening, Week 0, Week 2, and Week 4]

ALSFRS-R Total Score at Weeks 0, 2, 4 and 8 [Time Frame: Week 0, Week 2, Week 4 and Week 8]

Secondary Outcome(s)

Forced Expiratory Volume in 1 Second (FEV1) / Slow Vital Capacity (SVC) Ratio [Time Frame: Screening, Week 0, Week 2, and Week 4]

Lymphocyte (T-Cell) Subset Trajectories [Time Frame: Week 0, Week 2, and Week 4]

Secondary ID(s)

2013P000313

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

ALS Therapy Development Institute

Results

• Results available: Yes
• Date Posted: 31/12/2015
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01786174