Efficacy and Safety of Erenumab in Pediatric Participants With Episodic Migraine
NCT ID: NCT03836040
Last Updated: 2025-12-23
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
457 participants
INTERVENTIONAL
2019-07-19
2026-11-15
Brief Summary
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Detailed Description
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The trial consists of four phases: screening (up to 3 weeks of initial screening and a 4-week prospective baseline phase); the DBTP (24 weeks for Group 1 participants; 12-weeks for Group 2 participants) in which participants receive placebo or Erenumab dose 1, dose 2 or dose 3 (based on participant's body weight) via subcutaneous injection once a month; the optional dose level blinded extension phase (40 weeks), in which all participants are assigned to receive dose 1, dose 2 or dose 3 of Erenumab; and a 12 weeks safety follow-up phase (16 weeks after the last dose of investigational drug).
The study intends to enroll 436 participants (376 adolescents and up to 60 children).
Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Dose level 1
Participants will be randomized to one of two doses determined by their body weight at Day 1. Participants who enrolled under the original protocol or protocol amendment 1 will be identified as group 1. Those enrolled under protocol amendment 2 will be identified as group 2.
Erenumab Dose 1
Participants in the low body-weight group at day 1 and who are randomized to Dose Level 1 will receive this dose.
Erenumab Dose 2
Participants in the low body-weight group at day 1 who are randomized to Dose Level 2 and subjects in the high body-weight group at day 1 who are randomized to Dose Level 1 will receive this dose.
Dose level 2
Participants will be randomized to one of two doses determined by their body weight at Day 1. Participants who enrolled under the original protocol or protocol amendment 1 will be identified as group 1. Those enrolled under protocol amendment 2 will be identified as group 2.
Erenumab Dose 2
Participants in the low body-weight group at day 1 who are randomized to Dose Level 2 and subjects in the high body-weight group at day 1 who are randomized to Dose Level 1 will receive this dose.
Erenumab Dose 3
Participants in the high body-weight group at day 1 who are randomized to Dose Level 2 will receive this dose.
Placebo
Participants will be randomized to a placebo comparator.
Placebo
Placebo matching dose for erenumab dose 1, 2 and 3.
Interventions
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Erenumab Dose 1
Participants in the low body-weight group at day 1 and who are randomized to Dose Level 1 will receive this dose.
Erenumab Dose 2
Participants in the low body-weight group at day 1 who are randomized to Dose Level 2 and subjects in the high body-weight group at day 1 who are randomized to Dose Level 1 will receive this dose.
Erenumab Dose 3
Participants in the high body-weight group at day 1 who are randomized to Dose Level 2 will receive this dose.
Placebo
Placebo matching dose for erenumab dose 1, 2 and 3.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Participant's parent or legal representative has provided written informed consent before initiation of any study-specific activities/procedures.
* History of migraine (with or without aura) for greater than or equal to 12 months before screening according to the IHS Classification ICHD-3 (Headache Classification Committee of the International Headache Society, 2013) based on medical records and/or participant self-report or parents' or legal representative's report.
* The following ICHD-3 specifications for pediatric migraine (participants aged less than 18 years), should be considered for the diagnosis of migraine:
* Attacks may last 2 to 72 hours.
* Migraine headache is more often bilateral than in adults; unilateral pain usually emerges in late adolescence or early adult life.
* Migraine headache is usually frontotemporal. Occipital headache in children is rare and calls for diagnostic caution.
* A subset of otherwise typical participants have facial location of pain, which is called 'facial migraine' in the literature; there is no evidence that these participants form a separate subgroup of migraine participants.
* In young children, photophobia and phonophobia may be inferred from their behavior.
* History of less than 15 headache days per month of which greater than or equal to 4 headache days were assessed by the participant as migraine days in each of the 3 months prior to screening (refer to Section 5.6 for definition of migraine day).
* Criteria to be assessed prospectively during the 4-week baseline phase and confirmed before randomizing the participant into the DBTP:
* Migraine frequency: greater than or equal 4 and less than 15 migraine days based on the eDiary data during the last 28 days of the baseline phase if greater than 28 days in duration
* Headache frequency: less than 15 headache days based on the eDiary data during the last 28 days of the baseline phase if greater than 28 days in duration.
* Demonstrated at least 80% compliance with the eDiary based on the last 28 days of the baseline period, if greater than 28 days in duration (eg, completing eDiary items for at least 23 out of the last 28 days of the baseline phase).
Exclusion Criteria
* No therapeutic response with greater than 2 of the following 10 medication categories for prophylactic treatment of migraine after an adequate therapeutic trial. These medication categories are:
* Category 1: beta blockers (eg, propranolol, atenolol, bisoprolol, metoprolol, nadolol, nebivolol, pindolol, timolol)
* Category 2: tricyclic antidepressants (eg, amitriptyline, nortriptyline, protriptyline)
* Category 3: topiramate
* Category 4: divalproex sodium, sodium valproate
* Category 5: serotonin-norepinephrine reuptake inhibitors (eg, venlafaxine, desvenlafaxine, duloxetine, milnacipran)
* Category 6: cyproheptadine
* Category 7: flunarizine, cinnarizine
* Category 8: botulinum toxin
* Category 9: lisinopril/candesartan
* Category 10: medications targeting the CGRP pathway.
* No therapeutic response is defined as no reduction in headache frequency, duration, or severity after administration of the medication for at least 6 weeks at the generally-accepted therapeutic dose(s) based on the investigator's assessment.
* The following scenarios do not constitute lack of therapeutic response:
* Lack of sustained response to a medication.
* Partial, suboptimal response to a medication.
* Failure to tolerate a therapeutic dose.
* Evidence of drug or alcohol abuse or dependence within 12 months before screening, based on medical records, participant self-report, or positive urine drug test performed during screening (with the exception of prescribed medications such as opioids or barbiturates).
* Human immunodeficiency virus (HIV) infection by history.
* History of seizure disorder or other significant neurological disorder other than migraine. Note: a single childhood febrile seizure is not exclusionary.
* History of major psychiatric disorder (such as schizophrenia, schizoaffective disorder, bipolar disorder, obsessive-compulsive disorder, or pervasive developmental disorder), or current evidence of major depressive disorder based on a patient health questionnaire-9 modified for adolescents (PHQ-A) score greater than or equal to 10 at screening. Participants with anxiety disorder and/or mild major depressive disorder (with PHQ-A score ≤ 9) are permitted in the study if they are considered by the investigator to be stable and are taking no more than 1 medication for each disorder. Participant must have been on a stable dose within the 3 months before the start of the baseline phase.
* Use of prohibited medication within 1 month before the start of the baseline phase and/or during the baseline phase.
* Use of prohibited devices (such as stimulation devices) or procedures (such as acupuncture, biofeedback, relaxation techniques, or psychotherapy) with the goal of preventing migraines, within 3 months before the start of the baseline phase and/or during the baseline phase.
* Participants receiving Cognitive Behavioral Therapy (CBT) are excluded unless they are on a stable, maintenance phase of a CBT program for migraine for at least 3 months before the start of the baseline phase. Participants undergoing CBT are considered on a stable, maintenance phase if they have undergone greater than or equal 6 weekly or biweekly sessions of CBT administered by adequately trained psychologists and who, for at least 3 months before the start of the baseline phase, only follow "booster" CBT sessions at a monthly, bimonthly or quarterly frequency. Note: Participants who have discontinued CBT within 3 months prior to the start of the baseline phase are eligible for the study provided that there is evidence of CBT failure/lack of efficacy prior to initial screening (per medical records or investigator's assessment).
* Received botulinum toxin in the head and/or neck region within 4 months before the start of the baseline phase or during the baseline phase.
* Received medication targeting the CGRP pathway within 4 months before the start of the baseline phase or during the baseline phase.
* Taken the following for any indication in any month during the 2 months before the start of the baseline phase, or during the baseline phase:
* Ergotamines or triptans on greater than or equal 10 days per month.
* Simple analgesics (nonsteroidal anti-inflammatory drugs \[NSAIDs\], acetaminophen) on greater than or equal 15 days per month.
* Opioid or butalbital-containing analgesics on greater than or equal 4 days per month.
* Currently receiving treatment in another investigational device or drug study, or less than 90 days since ending treatment on another investigational device or drug study(ies). Other investigational procedures while participating in this study are excluded.
* Participant has clinically significant vital signs, laboratory results, or ECG abnormality during screening that, in the opinion of the investigator, could pose a risk to participant safety or interfere with the study evaluation.
* Hepatic disease by history or total bilirubin (TBL) greater than or equal 2.0 x upper limit of normal (ULN) or alanine transaminase (ALT) or aspartate aminotransferase (AST) greater than or equal 3.0 x ULN, as assessed by the central laboratory at initial screening.
* Female participant is pregnant or breastfeeding or planning to become pregnant or breastfeed during the study and for an additional 16 weeks after the last dose of investigational product. (Females of childbearing potential should only be included in the study after a confirmed menstrual period and a negative highly sensitive urine and serum pregnancy test.)
* Female participants of childbearing potential unwilling to use an acceptable method of effective contraception during treatment and for an additional 16 weeks after the last dose of investigational product.
* Participant has known sensitivity to any of the products or components to be administered during dosing.
* Participant likely to not be available to complete all protocol-required study visits or procedures, and/or to comply with all required study procedures to the best of the participant's legal representative and investigator's knowledge.
* History or evidence of any other clinically significant disorder, condition or disease (with the exception of those outlined above) that, in the opinion of the investigator or Amgen physician, if consulted, would pose a risk to participant safety or interfere with the study evaluation, procedures or completion.
6 Years
17 Years
ALL
No
Sponsors
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Novartis
INDUSTRY
Amgen
INDUSTRY
Responsible Party
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Principal Investigators
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MD
Role: STUDY_DIRECTOR
Amgen
Locations
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Paradigm Clinical Research Center Inc
San Diego, California, United States
Childrens Hospital Colorado
Aurora, Colorado, United States
Colorado Springs Neurological Associates
Colorado Springs, Colorado, United States
New England Institute for Clinical Research
Stamford, Connecticut, United States
Childrens National Health System
Washington D.C., District of Columbia, United States
Northwest Florida Clinical Research Group Limited Liability Company
Gulf Breeze, Florida, United States
Nicklaus Childrens Hospital
Miami, Florida, United States
Pediatric Epilepsy and Neurology Specialists
Tampa, Florida, United States
TrueBlue Clinical Research
Tampa, Florida, United States
Premiere Research Institute
West Palm Beach, Florida, United States
Rare Disease Research Center Pediatrics
Atlanta, Georgia, United States
CenExel iResearch, LLC
Savannah, Georgia, United States
Northwestern University
Chicago, Illinois, United States
Chicago Headache Center and Research Institute
Chicago, Illinois, United States
Josephson Wallack Munshower Neurology
Indianapolis, Indiana, United States
University of Maryland, Baltimore
Baltimore, Maryland, United States
New England Regional Headache Center Inc
Worcester, Massachusetts, United States
Michigan Head Pain and Neurological Institute
Ann Arbor, Michigan, United States
Clinical Research Institute Inc
Minneapolis, Minnesota, United States
Childrens Mercy Hospital and Clinics
Kansas City, Missouri, United States
Mercy Research
St Louis, Missouri, United States
Velocity Clinical Research, Inc
Grand Island, Nebraska, United States
Dent Neurosciences Research Center
Amherst, New York, United States
Modern Migraine MD
New York, New York, United States
Columbia University Irving Medical Center
New York, New York, United States
Cincinnati Childrens Hospital Medical Center
Cincinnati, Ohio, United States
Cleveland Clinic Foundation
Cleveland, Ohio, United States
Nationwide Childrens Hospital
Columbus, Ohio, United States
Oregon Health and Science University
Portland, Oregon, United States
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Preferred Primary Care Physicians, Inc
Pittsburgh, Pennsylvania, United States
Palmetto Gastroenterology Clinical Research, LLC
Summerville, South Carolina, United States
Child Neurology Consultants of Austin
Austin, Texas, United States
Helios Clinical Research Inc
Burleson, Texas, United States
Stryde Consulting LLC
Frisco, Texas, United States
Childrens Specialty Group
Norfolk, Virginia, United States
Vaught Neurological Services
Crab Orchard, West Virginia, United States
Marshfield Clinic
Marshfield, Wisconsin, United States
Universitair Ziekenhuis Brussel
Brussels, , Belgium
Algemeen Ziekenhuis Sint-Maarten
Mechelen, , Belgium
Docteur Simona Sava
Saint-Nicolas, , Belgium
Stollery Childrens Hospital
Edmonton, Alberta, Canada
London Health Sciences Centre
London, Ontario, Canada
Childrens Hospital of Eastern Ontario
Ottawa, Ontario, Canada
The Hospital For Sick Children
Toronto, Ontario, Canada
Fundacion Centro de Investigacion Clinica
Medellín, Antioquia, Colombia
Servicios de Salud Ips Suramericana Sas - Ips Sura Industriales
Medellín, Antioquia, Colombia
Institucion Prestadora de Servicios de Salud Sociedad Médica Rionegro SA Somer SA
Rionegro, Antioquia, Colombia
Solano y Terront Servicios Medicos SAS - Unidad Integral de Endocrinologia Uniendo
Bogota, Cundinamarca, Colombia
Cafam
Bogota, Cundinamarca, Colombia
Fundacion Hospital Infantil Universitario De San Jose
Bogota, Cundinamarca, Colombia
Fundacion cardiovascular de Colombia
Bucaramanga, Santander Department, Colombia
Terveystalo Pulssi
Turku, , Finland
Charite - Universitaetsmedizin Berlin, Campus Virchow
Berlin, , Germany
Universitaetsklinikum Essen
Essen, , Germany
Universitaetsklinikum Greifswald
Greifswald, , Germany
Schmerzklinik Kiel
Kiel, , Germany
Arzneimittelforschung Leipzig GmbH
Leipzig, , Germany
Dr Kenessey Albert Korhaz - Rendelointezet
Balassagyarmat, , Hungary
Dr Altmann Anna egyeni vallalkozo
Budapest, , Hungary
High Tech Medical Kft
Budapest, , Hungary
Semmelweis Egyetem
Budapest, , Hungary
Debreceni Egyetem Klinikai Kozpont
Debrecen, , Hungary
Borsod-Abauj-Zemplen Varmegyei Kozponti Korhaz es Egyetemi Oktatokorhaz
Miskolc, , Hungary
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, , Italy
Azienda di Rilievo Nazionale e Alta Specializzazione Civico Di Cristina Benfratelli
Palermo, , Italy
Fondazione Istituto Neurologico Nazionale C Mondino IRCCS
Pavia, , Italy
IRCCS Ospedale Pediatrico Bambino Gesu
Roma, , Italy
Josai Kids Clinic
Nagoya, Aichi-ken, Japan
Medical Corporation Seikokai Takanoko Hospital
Matsuyama, Ehime, Japan
Hiroshima City Hiroshima Citizens Hospital
Hiroshima, Hiroshima, Japan
Kitami Clinic
Sapporo, Hokkaido, Japan
Konan Medical Center
Kobe, Hyōgo, Japan
Umenotsuji Clinic
Kochi, Kochi, Japan
Kumamoto City Hospital
Kumamoto, Kumamoto, Japan
Tatsuoka Neurology Clinic
Kyoto, Kyoto, Japan
Japanese Red Cross Kyoto Daiichi Hospital
Kyoto, Kyoto, Japan
Ishikawa Clinic
Kyoto, Kyoto, Japan
Sendai Headache and Neurology Clinic
Sendai, Miyagi, Japan
Tominaga Hospital
Osaka, Osaka, Japan
Saitama Neuropsychiatric Institute
Saitama-shi, Saitama, Japan
Tokyo Medical University Hospital
Shinjuku-ku, Tokyo, Japan
Keio University Hospital
Shinjuku-ku, Tokyo, Japan
Nagamitsu Clinic
Hofu-shi, Yamaguchi, Japan
Nagaseki Headache Clinic
Kai-shi, Yamanashi, Japan
Uniwersytecki Dzieciecy Szpital Kliniczny im Ludwika Zamenhofa w Bialymstoku
Bialystok, , Poland
AthleticoMed
Bydgoszcz, , Poland
Uniwersyteckie Centrum Kliniczne
Gdansk, , Poland
Instytut Centrum Zdrowia Matki Polki
Lodz, , Poland
Centrum Medyczne Luxmed Spzoo
Lublin, , Poland
Centrum Medyczne Hope Clinic Sebastian Szklener
Lublin, , Poland
Uniwersytecki Szpital Kliniczny w Poznaniu
Poznan, , Poland
Clinical Research Center Spzoo Medic-R Spolka Komandytowa
Poznan, , Poland
Dr Sekowska Leczenie Bolu
Warsaw, , Poland
Next Stage Spzoo
Warsaw, , Poland
Migre Polskie Centrum Leczenia Migreny Anna Gryglas-Dworak
Wroclaw, , Poland
Unidade Local de Saude de Coimbra, EPE - Hospital Pediatrico de Coimbra
Coimbra, , Portugal
Unidade Local de Saude de Sao Jose, EPE - Hospital Dona Estefania
Lisbon, , Portugal
Hospital da Luz, SA
Lisbon, , Portugal
Unidade Local de Saude de Santa Maria, EPE - Hospital de Santa Maria
Lisbon, , Portugal
Puerto Rico Health and Wellness Institute
Dorado, , Puerto Rico
FSBI Russian Children Clinical Hospital of the MoH RF
Moscow, , Russia
LLC clinic Chaika
Moscow, , Russia
LLC Sibneyromed
Novosibirsk, , Russia
LLC Medical Technologies
Saint Petersburg, , Russia
Hospital Universitario Virgen del Rocio
Seville, Andalusia, Spain
Hospital Universitari Vall d Hebron
Barcelona, Catalonia, Spain
Hospital de la Santa Creu i Sant Pau
Barcelona, Catalonia, Spain
Clinica Universidad de Navarra
Pamplona, Navarre, Spain
Hospital Universitari i Politecnic La Fe
Valencia, Valencia, Spain
Universitaets-Kinderspital beider Basel
Basel, , Switzerland
Kopfwehzentrum Hirslanden
Zollikon, , Switzerland
Noahs Ark Childrens Hospital for Wales
Cardiff, , United Kingdom
Royal Hospital for Children
Glasgow, , United Kingdom
Alder Hey Childrens Hospital
Liverpool, , United Kingdom
Evelina Childrens Hospital
London, , United Kingdom
Great Ormond Street Hospital for Children
London, , United Kingdom
4 Medical Clinical Solutions Manchester
Manchester, , United Kingdom
Oxford Childrens Hospital
Oxford, , United Kingdom
Countries
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Related Links
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AmgenTrials clinical trials website
Other Identifiers
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2023-504930-23
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
20150125
Identifier Type: -
Identifier Source: org_study_id