Efficacy and Safety Study of Rimegepant for the Preventative Treatment of Migraine in Pediatric Subjects

Study Overview
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Brief Summary
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The purpose of this study is to compare the efficacy and safety of rimegepant to placebo as a preventative treatment for migraine in children and adolescents ≥ 6 to <18 years with episodic migraine.

Official Title
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A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Rimegepant in Migraine Prevention in Children and Adolescents ≥ 6 to <18 Years of Age

Conditions
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Migraine

Intervention / Treatment
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* Drug: Rimegepant
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subject has at least a 6 month history of migraine (with or without aura) and including the following: 14 or less headache days per month during the 3 month period prior to the Screening Visit 6 or more migraine days during the Observation Period 14 or less headache days during the Observation Period Pediatric Migraine Disability Assessment Scale (PedMIDAS) Disability Score of >10 to ≤50, indicating mild (score of 11 to 30) or moderate (score of 31 to 50) disruption in daily activities, as assessed at the Baseline (Randomization) Visit Ability to verbally distinguish migraine attacks from tension/cluster or other types of headaches Migraine attacks, on average, lasting 4 - 72 hours if untreated Subjects on prophylactic migraine medication are permitted to remain on therapy if the dose has been stable for at least 3 months (12 weeks) prior to the Screening Phase, and the dose is not expected to change during the course of the study. 2) Male and female subjects ≥ 6 to <18 years; subjects must be less than 18 at the time of signing assent / consent. 3)Subjects must have a weight of ≥40 kg (child cohort requirement ≥15 kg) at the Screening Visit. Exclusion Criteria: Subjects with a history of basilar migraine, cluster headaches, or hemiplegic migraine The subject has a continuous migraine (defined as an unrelenting headache) within 1 month prior to Screening Visit. The subject has a history or diagnosis of complications of migraine The subject has a confounding and clinically significant pain syndrome that may interfere with the subject's ability to participate in this study. The subject has any current psychiatric condition that is uncontrolled and/or untreated for a minimum of 6 months prior to the Screening Visit. Subjects with a lifetime history of psychosis and/or mania are excluded. History of suicidal behavior or the subject is at risk of self-harm or harm to others. History of major psychiatric disorder. The subject has a current diagnosis or history of substance abuse The subject has a history of moderate or severe head trauma or other neurological disorder (including seizure disorder) or systemic medical disease that is, in the investigator's opinion, likely to affect central nervous system functioning.

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Prevention
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Rimegepant / BHV3000<br>Rimegepant 75mg or 50mg (2 X 25mg) ODT | Drug: Rimegepant<br>* Rimegepant 75mg or 50mg (2 X 25mg) ODT<br>* Other names: BHV3000;|
| Placebo Comparator: Matching Placebo<br>Matching placebo 75mg or 50mg (2 X 25mg) ODT | Drug: Placebo<br>* Matching placebo 75mg or 50mg (2 X 25mg) ODT<br>* Other names: Matching Placebo;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in the mean number of migraine days per month as measured over the 12-week double-blind phase of the study in adolescents with episodic migraine | Reduction from baseline in mean number of migraine days per month | 3 months (12 weeks) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Achievement of at least a 50% reduction from baseline in mean monthly migraine days over the course of the double-blind phase in adolescents with episodic migraine |  | 3 months (12 weeks) | 
| Change from baseline in the mean number of migraine days per month in the first 4 weeks (Weeks 1 through 4) of the double-blind treatment phase in adolescents with episodic migraine. |  | Weeks 1 through 4 of treatment | 
| The mean change from baseline in the Pediatric Quality of Life (PedsQL™) total score at Week 12 of the double-blind treatment phase in adolescents with episodic migraine. |  | Total score at week 12 | 
| Change from baseline in the mean number of migraine days per month over the entire course of the double-blind treatment phase in children and children and adolescents combined with episodic migraine. |  | 3 months (12 weeks) | 
| Change from baseline on the proportion of subjects that have at least a 50% reduction in the mean number of moderate to severe migraine days per month. | Measured over the entire course of the double-blind treatment phase in children and children and adolescents combined with episodic migraine. | 3 months (12 weeks) | 
| Compare the use of acute migraine-specific medications (triptans) based on the change from baseline in monthly acute migraine specific medication days. | Measured in each month and over the course of the entire double-blind treatment phase in adolescents, children and adolescents and children combined. | 3 months (12 weeks) | 
| Evaluate the safety and tolerability of rimegepant as a preventative treatment for migraine in children and adolescents. | This will be evaluated by the number of subjects with treatment related adverse events by severity. | 72 Weeks | 
| Evaluate the frequency of hepatic-related adverse events in subjects treated with rimegepant. | Measured by discontinuations in treated subjects due to elevated liver function tests. | 72 Weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Migraine, Migraine prevention, Phonophobia, Photophobia, Nausea, Pediatric migraine, Adolescent migraine