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

Official title

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

Overview

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.

Interventions

• Drug Rimegepant
  Rimegepant 75mg or 50mg (2 X 25mg) ODT
• Drug Placebo
  Matching placebo 75mg or 50mg (2 X 25mg) ODT

Primary outcome measures

• 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 [Time frame: 3 months (12 weeks)]

Secondary outcome measures (8)

• 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 [Time frame: 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. [Time frame: 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. [Time frame: 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. [Time frame: 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. [Time frame: 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. [Time frame: 3 months (12 weeks)]
• Evaluate the safety and tolerability of rimegepant as a preventative treatment for migraine in children and adolescents. [Time frame: 72 Weeks]
• Evaluate the frequency of hepatic-related adverse events in subjects treated with rimegepant. [Time frame: 72 Weeks]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Sequential

Masking

Quadruple blind

Primary purpose

Prevention

Study locations

United States · 53 centers

• The Belinga Clinic — Fort Smith
• Advanced Investigative Medicine, Inc. — Hawthorne
• Velocity Clinical Research San Diego — La Mesa
• Colorado Springs Neurological Associates — Colorado Springs
• Advanced Neurosciences Research, LLC — Fort Collins
• Velocity Clinical Research, Washington DC — Washington D.C.
• Accel Research Sites Network - Edgewater Clinical Research Unit — Edgewater
• Direct Helpers Research Center — Hialeah
• … and 45 more centers

Japan · 13 centers

• Jinnouchi Neurosurgical Clinic — Kasuga-shi
• Hikita Pediatric Clinic — Kiryu-shi
• Konan Medical Center — Higashinada-ku Kobe-shi
• Yamaguchi Clinic — Nishinomiya-shi
• Mito Kyodo General Hospital — Mito
• Tanaka Neurosurgery & Headache Clinic — Kagoshima
• Atago Hospital — Kochi
• Sendai Headache and Neurology Clinic — Taihaku-ku Sendai-shi
• … and 5 more centers

Poland · 8 centers

• Clinical Research Center Sp. z o.o. MEDIC-R Sp. k. — Poznan
• Wojewodzki Specjalistyczny Szpital Dzieciecy — Krakow
• MIGRE Polskie Centrum Leczenia Migreny Anna Gryglas-Dworak — Wroclaw
• NZOZ Neuromed M. i M. Nastaj Sp. P. — Lublin
• … and 4 more centers

Italy · 6 centers

• UOSVD Centro Epilessia ed EEG dellEta Evolutiva — Bari
• Sapienza Universita di Roma Polo Pontino - ICOT, Headache clinic — Latina
• IRCCS San Raffaele — Rome
• Istituto Neurologico "Carlo Besta" Fondazione IRCCS — Milan
• Divisione di Neurologia, Ospedale Pediatrico Bambino Gesu — Rome
• ASST Spedali Civili di Brescia — Brescia

Spain · 6 centers

Center list to be confirmed — check the primary protocol.

France · 3 centers

• Centre Hospitalier Intercommunal de Creteil — Créteil
• Centre Hospitalier Universitaire de Montpellier — Montpellier
• Hopital des Enfants — Toulouse

Canada · 1 center

• Children's and Women's Health Centre of British Columbia — Vancouver

Publications

• Shin L, Mentz O, Gelfand AA, de Prado BM, Shapiro H, Oh A, Onifade OO, Saunders J, Raj N, Szperka CL. Gepants for headache prevention in children and adolescents: A multicenter chart review study. Headache. 2026 Jan;66(1):239-248. doi: 10.1111/head.15087. Epub 2025 Nov 14. PMID 41235617

Identifiers

NCT: NCT05156398 · BHV3000-315 · C4951009 · 2021-005246-15 · 2021-005246-15 · 2024-512382-13-00

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗