A Study to Assess the Adverse Events and Change in Disease Activity of Oral Atogepant Tablets in Pediatric Participants (6-17 Years of Age) With Episodic Migraine

Official title

A Phase 3, Multicenter, 12-Week, Double Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Atogepant for the Preventive Treatment of Episodic Migraine in Pediatric Subjects 6-17 Years of Age.

Overview

A migraine is a moderate to severe headache on one side of the head. A migraine attack is a headache that may be accompanied by throbbing, nausea, vomiting, sensitivity to light and sound, or other symptoms. A number of treatments are available for adults with migraine but there are limited approved treatments available for pediatric participants. The main goal of the study is to evaluate the safety and efficacy (how well treatment works) of a low-dose and high-dose of atogepant in pediatric participants between the ages of 6 and 17. Atogepant is a medicine currently approved to treat adults with migraine (0 to 14 migraine days per month) and is being studied in pediatric participants between the ages of 6 and 17 with a history of episodic migraine. This is a Phase 3, randomized, double-blind study of atogepant in participants with a history of episodic migraine with an open-label pharmacokinetic substudy. Eligible participants will be randomized into 6 different groups. Participants between the ages of 12 and 17 will be randomized to receive placebo, low-dose atogepant, or high-dose atogepant for 12 weeks. Participants between the ages of 6 and 11 will also be randomized to receive placebo, low-dose atogepant, or high-dose atogepant for 12 weeks. The specific atogepant doses to be used in participants between the ages of 6 and 11 will be determined after the PK substudy is complete. Around 450 participants will be enrolled in approximately 100 sites worldwide. Placebo, low-dose atogepant, and high-dose atogepant are given as a tablet to take by mouth once a day. At the end of Week 12, participants will either undergo a follow-up visit 4 weeks after last study treatment or join an extension study where they can continue to receive atogepant for another 52 weeks. There may be a bigger responsibility for participants in this study. Participants will attend regular visits during the study at a hospital or clinic. The effects of treatment will be checked by medical assessments, blood tests, checking for side effects, and completing questionnaires.

Interventions

• Drug Atogepant
  Oral Tablet
• Drug Placebo-Matching Atogepant
  Oral Tablet

Primary outcome measures

• Change from Baseline in Mean Monthly Migraine Days [Time frame: Baseline (Week 0) through Week 12]
• Number of Participants Experiencing Adverse Events [Time frame: Baseline (Week 0) through Week 16]

Secondary outcome measures (6)

• Change from Baseline in Mean Monthly Headache Days [Time frame: Baseline (Week 0) through Week 12]
• Change from Baseline in Mean Monthly Headache Days of at Least Moderate Severity [Time frame: Baseline (Week 0) through Week 12]
• Change from Baseline in Mean Monthly Acute Medication Use Days [Time frame: Baseline (Week 0) through Week 12]
• Percentage of Participants who Achieve at least a 50% Reduction in Average of Monthly Migraine Days [Time frame: Baseline (Week 0) to 3 Months]
• Change from Baseline in the Pediatric Quality of Life Inventory (PedsQL) total score [Time frame: Baseline (Week 0) through Week 12]
• Change from baseline in the Pediatric Migraine Disability Assessment (PedMIDAS) total score [Time frame: Baseline (Week 0) through Week 12]

Eligibility criteria

Inclusion criteria

• Weight is >= 20 kg (44 lbs) and < 135 kg (298 lbs).
• History of episodic migraine with or without aura consistent with a diagnosis according to the International Classification of Headache Disorders (ICHD) -3 (2018) for at least 6 months.
• Participant has to have 4 to 14 migraine days and < 15 headache days in the 28-day baseline period per eDiary.
• To be eligible for the PK substudy, participants must be 6 to 11 years of age (inclusive), with a history of migraine (consistent with a diagnosis according to the ICHD-3 \[2018\]) and per investigator judgment is appropriate to receive preventive treatment for migraine.

Exclusion criteria

• History of migraine brainstem aura, hemiplegic migraine, or retinal migraine as defined by ICHD-3 (2018).
• Have a current diagnosis of chronic migraine as defined by ICHD-3 (2018).
• Have a current diagnosis of new daily persistent headache, trigeminal autonomic cephalgia (e.g., cluster headache), or painful cranial neuropathy as defined by ICHD-3 (2018).

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

Triple blind

Primary purpose

Treatment

Study locations

United States · 42 centers

• Rehabilitation & Neurological Services /ID# 248517 — Huntsville
• The Center for Clinical Trials - Saraland /ID# 271604 — Saraland
• Preferred Research Partners /ID# 249729 — Little Rock
• Preferred Research Partners /ID# 270406 — Little Rock
• Advanced Research Center /ID# 251381 — Anaheim
• Alliance for Research Alliance for Wellness /ID# 248521 — Long Beach
• Excell Research, Inc /ID# 247532 — Oceanside
• Lumos Clinical Research Center /ID# 249731 — San Jose
• … and 34 more centers

Japan · 7 centers

• Konan Medical Center /ID# 254457 — Kobe
• Yamaguchi Clinic /ID# 254753 — Nishinomiya
• Umenotsuji Clinic /ID# 254453 — Kochi
• Sendai Headache and Neurology Clinic Medical Corporation /ID# 254211 — Sendai
• Tominaga Clinic - Osaka /ID# 254450 — Osaka
• Tokyo Medical University Hospital /ID# 254459 — Shinjuku-ku
• Tatsuoka Neurology Clinic /ID# 254455 — Kyoto

Poland · 6 centers

• Clinical Research Center Medic-R /ID# 247383 — Poznan
• Athleticomed Sp. z o.o /ID# 248789 — Bydgoszcz
• Specjalistyczne Gabinety Sp. z o.o. /ID# 247384 — Krakow
• Indywidualna Praktyka Lekarska dr hab. med. Anna Szczepanska-Szerej /ID# 247382 — Lublin
• Oha-Med Sp. Z O.O /ID# 248614 — Warsaw
• MIGRE Polskie Centrum Leczenia Migreny Anna Gryglas-Dworak /ID# 247381 — Wroclaw

Israel · 5 centers

• Shamir Medical Center /ID# 256857 — Beer Ya'akov
• Hillel Yaffe Medical Center /ID# 246747 — Hadera
• The Chaim Sheba Medical Center /ID# 246746 — Ramat Gan
• Tel Aviv Sourasky Medical Center /ID# 247466 — Tel Aviv
• Bnai Zion Medical Center /ID# 247469 — Haifa

Puerto Rico · 5 centers

• Clinical Research Investigator Group /ID# 261254 — Bayamón
• … and 4 more centers

Spain · 5 centers

Center list to be confirmed — check the primary protocol.

United Kingdom · 4 centers

Center list to be confirmed — check the primary protocol.

Canada · 3 centers

• Stollery Children's Hospital /ID# 249672 — Edmonton
• London Health Sciences Center- University Hospital /ID# 252979 — London
• McGill University Health Centre - Glen Site /ID# 247271 — Montreal

Denmark · 3 centers

• Herlev Hospital /ID# 247724 — Herlev
• Regionshospitalet Godstrup /ID# 247902 — Herning
• Aalborg Sygehus Nord /ID# 247428 — Aalborg

France · 3 centers

• CHU Amiens-Picardie Site Sud /ID# 248833 — Amiens
• Centre Hosp Intercommunal de Creteil /ID# 248832 — Créteil
• CHU Toulouse - Hôpital des enfants /ID# 247553 — Toulouse

Italy · 3 centers

• Fondazione IRCCS Istituto Neurologico Carlo Besta /ID# 247581 — Milan
• Ospedale Pediatrico Bambino Gesù /ID# 247647 — Rome
• Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone /ID# 247638 — Palermo

Netherlands · 3 centers

• Canisius-Wilhelmina Ziekenhuis /ID# 253065 — Nijmegen
• HagaZiekenhuis /ID# 247318 — The Hague
• ZorgSaam Zorggroep Zeeuws-Vlaanderen /ID# 247301 — Terneuzen

Romania · 3 centers

Center list to be confirmed — check the primary protocol.

Belgium · 2 centers

• Uza /Id# 247339 — Edegem
• Algemeen Ziekenhuis Sint-Jan Brugge /ID# 247201 — Bruges

Hungary · 2 centers

• MIND Clinic /ID# 248687 — Budapest
• Semmelweis Egyetem /ID# 248360 — Budapest

Sweden · 2 centers

Center list to be confirmed — check the primary protocol.

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: NCT05711394 · M21-201 · 2022-501100-94-00

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗