Safety and Tolerability of Perampanel in Amyotrophic Lateral Sclerosis Patients
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|ClinicalTrials.gov Identifier: NCT03377309|
Recruitment Status : Recruiting
First Posted : December 19, 2017
Last Update Posted : December 21, 2017
Amyotrophic lateral sclerosis (ALS), the most common motor neuron disease, is a fatal progressive neurodegenerative disease affecting motor cortex, brainstem and spinal cord leading to motor neuron death. It is a devastating disease of the anterior and lateral corticospinal tracts with approximately 3 years mean duration from symptoms onset to death, one-fifth survival at 5 years and only 10% may make it to 10 years.
Among the neuronal death pathways, excitotoxicity mechanism is considered to be the foremost-involved mechanism. AMPA receptors are thought to be the prime mediator of the fast excitation in spinal motor neurons, where they are expressed ubiquitously. AMPA receptor antagonist was able to prevent this acute degeneration in previous animal studies.
The investigators aim to study the tolerability and safety of the novel AMPA antagonist, perampanel, in patients diagnosed with ALS. Perampanel [2-(2-oxo-1-phenyl-5- pyridin-2-yl-1,2-dihydropyridin-3-yl) benzonitrile] with its selective non-competitive AMPA antagonism, was recently approved for epilepsy. Various long-term trials studying perampanel in epilepsy showed favorable tolerability profile and most common side effects were mainly: dizziness, headache and somnolence. All patients presenting to Neurology clinics at AUBMC diagnosed with Amyotrophic Lateral Sclerosis, will be considered for the study. Investigators will obtain informed consents from all patients who agree to be enrolled in this study in accordance with institutional review board (IRB) requirements. Patients of both genders and over 18 years old who meet the El Escorial criteria for possible, probable or definite ALS and fit the inclusion criteria will be recruited. Subjects should not be started on riluzole for the past 30 days or stable on a dose of riluzole for at least 30 days prior to the screening process.
In titration phase, perampanel dose will be increase by 2mg/day increments every one week to reach a maximum dose of 8 mg/day; reaching the maximum dose in four weeks. Treatment phase will be followed by washout period during which, dose will be tapered by 2mg/day every 5 days (over total of 15 days).
|Condition or disease||Intervention/treatment||Phase|
|Amyotrophic Lateral Sclerosis||Drug: Fycompa||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Safety and Tolerability of Perampanel in Amyotrophic Lateral Sclerosis Patients|
|Actual Study Start Date :||December 1, 2017|
|Estimated Primary Completion Date :||June 1, 2018|
|Estimated Study Completion Date :||July 1, 2018|
Dose will be increased by 2mg/day increments every one week to reach a maximum dose of 8 mg/day. Treatment phase will be stable dose for 12 weeks then followed by washout period over 2 weeks.
Other Name: Perampanel
- Safety and Tolerability: Incidence and severity of drug-related adverse effects [ Time Frame: During study period up to 4 weeks post- study ]Incidence and severity of drug-related adverse effects
- Efficacy: As measured by change in ALSFRS-R score [ Time Frame: During study period up to 4 weeks post- study ]Efficacy as measured by change in ALS Functional Rating Scale Revised -ALSFRS-R score ALSFRS-R score is of 12 items with total score of 48(each item score on a scale of 4); 0 reflects severe disability and 48 is the normal score.
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): NCT03377309
|Contact: Johnny S Salameh, MD||009611350000 ext firstname.lastname@example.org|
|Johnny S. Salameh||Recruiting|
|Beirut, Lebanon, 1107 2020|
|Contact: Johnny S Salameh, MD 01350000 ext 7359 email@example.com|
|Principal Investigator: Achraf Makki, MD|
|Sub-Investigator: Mostafa Hotait, MD|
|Principal Investigator:||Johnny Salameh, MD||American University of Beirut Medical Center|