Clinical trial • Phase III • Neurology

EPTINEZUMAB for Chronic migraine

Phase III trial of EPTINEZUMAB for Chronic migraine.

Overview

Trial Therapeutic Area
Neurology
Trial Disease
Chronic migraine
Trial Stage
Phase III
Drug Modality
Monoclonal antibody
Paediatric Trial
Yes

Key dates

Initial CTIS Submission Date
09-08-2024
First CTIS Authorization Date
13-09-2024

Trial design

Randomised, experimental: eptinezumab 300 mg iv single infusion (weight adjusted); experimental: eptinezumab 100 mg iv single infusion (weight adjusted); placebo comparator: nacl 0.9% matching to eptinezumab, single iv infusion.-controlled Phase III trial in Portugal, Poland, Italy and others.

Randomised
Yes
Comparator
Experimental: Eptinezumab 300 mg IV single infusion (weight adjusted); Experimental: Eptinezumab 100 mg IV single infusion (weight adjusted); Placebo comparator: NaCl 0.9% matching to eptinezumab, single IV infusion.
Single Multiple Or Escalation Dose Combined
Yes
Target Sample Size
165
Trial Duration For Participant
84

Eligibility

Recruits 165 paediatric patients.

Vulnerable Population
Vulnerable population: adolescents aged 12-17 are included. Consent/assent handling: parent/legal guardian informed consent is required and documented (Parent/Guardian ICF, Parent_Legal-Rep ICF), and age‑appropriate pediatric assent documents are used (Pediatric Assent 12-15; Assent 12-17 / Assent 13-17 as available). There are also subject information and ICF documents for adult patients (Main ICF / Patient 18 years) and newborn/pregnancy follow-up forms. Materials are provided in multiple languages (Portuguese, Polish, Italian, Spanish, English as per available ICF/assent documents).

Inclusion criteria

  • {"criterion_text":"- The participant has a diagnosis of migraine (with or without aura) as defined by International Classification of Headache Disorders 3 (ICHD-3) guidelines with history of chronic migraine, of at least 6 months prior to the screening visit."}
  • {"criterion_text":"- During the 28-day screening period, the participant must adequately complete the headache eDiary on at least 23 of the 28 days following the screening visit."}
  • {"criterion_text":"- During the 28-day screening period, the participant must have ≥15 to ≤26 headache days, of which at least 8 are migraine days as documented in the eDiary."}

Exclusion criteria

  • {"criterion_text":"- The participant has previously been randomised in this study and exposed to eptinezumab."}
  • {"criterion_text":"- The participant has been exposed to any monoclonal antibody treatment (including exposure in a study) <6 months prior to the screening visit."}
  • {"criterion_text":"- The participant has been exposed to another calcitonin gene-related peptide (CGRP) antibody (including exposure in a study investigating a CGRP antibody) <6 months prior to the screening visit."}
  • {"criterion_text":"- The participant has a history or diagnosis of complicated migraine (ICHD-3 version, 2018), chronic tension-type headache, hypnic headache, cluster headache, hemicrania continua, new daily persistent headache, or unusual migraine subtypes such as hemiplegic migraine (sporadic and familial), migraine with brainstem aura, ophthalmoplegic migraine, or migraine with neurological accompaniments that are not typical of migraine aura (diplopia, altered consciousness, or long duration; for example >60 minutes)."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- \"Change From Baseline in Monthly Migraine Days (MMDs) Averaged Over Weeks 1-12 \"","definition_or_measurement_approach":"Change from baseline in monthly migraine days averaged over Weeks 1-12 as recorded by the participant eDiary (MMDs averaged over Weeks 1-12)."}

Secondary endpoints

  • {"endpoint_text":"- Percentage of Participants With 50% Reduction From Baseline in MMDs Averaged Over Weeks 1-12","definition_or_measurement_approach":"Proportion of participants achieving ≥50% reduction from baseline in monthly migraine days averaged over Weeks 1-12 (based on eDiary-recorded MMDs)."}
  • {"endpoint_text":"- Percentage of Participants With Migraine on the Day After Dosing (Day 1)","definition_or_measurement_approach":"Proportion of participants with migraine on the day after dosing (Day 1) as recorded in the eDiary."}
  • {"endpoint_text":"- Change From Baseline in MMDs With Use of Acute Medication Averaged Over Weeks 1-12","definition_or_measurement_approach":"Change from baseline in monthly migraine days with use of acute medication averaged over Weeks 1-12 (eDiary-recorded)."}
  • {"endpoint_text":"- Percentage of Participants With 75% Reduction From Baseline in MMDs Averaged Over Weeks 1-12","definition_or_measurement_approach":"Proportion of participants achieving ≥75% reduction from baseline in monthly migraine days averaged over Weeks 1-12 (eDiary)."}
  • {"endpoint_text":"- Percentage of Participants With 75% Reduction From Baseline in MMDs Averaged Over Weeks 1-4","definition_or_measurement_approach":"Proportion of participants achieving ≥75% reduction from baseline in monthly migraine days averaged over Weeks 1-4 (eDiary)."}
  • {"endpoint_text":"- Percentage of Participants With 50% Reduction From Baseline in MMDs Averaged Over Weeks 1-4","definition_or_measurement_approach":"Proportion of participants achieving ≥50% reduction from baseline in monthly migraine days averaged over Weeks 1-4 (eDiary)."}
  • {"endpoint_text":"- Change From Baseline in Monthly Headache Days Averaged Over Weeks 1-12","definition_or_measurement_approach":"Change from baseline in monthly headache days averaged over Weeks 1-12 (eDiary-recorded headache days)."}
  • {"endpoint_text":"- Change From Baseline in Rate of Migraines With Severe Pain Intensity Averaged Over Weeks 1-12","definition_or_measurement_approach":"Change from baseline in rate of migraines with severe pain intensity averaged over Weeks 1-12 (severity recorded in eDiary)."}
  • {"endpoint_text":"- Change From Baseline in Days With Use of Acute Medication Averaged Over Weeks 1-12","definition_or_measurement_approach":"Change from baseline in days with use of acute medication averaged over Weeks 1-12 (eDiary-recorded medication use)."}
  • {"endpoint_text":"- Change From Baseline in Pediatric Migraine Disability Assessment Questionnaire (PedMIDAS) Score Averaged Over Weeks 1-12","definition_or_measurement_approach":"Change from baseline in PedMIDAS score averaged over Weeks 1-12 (questionnaire-based assessment)."}
  • {"endpoint_text":"- Free Eptinezumab Plasma Concentration","definition_or_measurement_approach":"Measurement of free eptinezumab plasma concentration (pharmacokinetic assay)."}
  • {"endpoint_text":"- Number of Participants With Specific Anti-Eptinezumab Antibodies (Anti-Drug Antibodies [ADA])","definition_or_measurement_approach":"Number of participants with specific anti-eptinezumab antibodies detected by ADA assays."}
  • {"endpoint_text":"- Number of Participants With Specific Anti-Eptinezumab Antibodies for Neutralizing Activity (NAb)","definition_or_measurement_approach":"Number of participants with anti-eptinezumab antibodies displaying neutralizing activity (NAb assays)."}

Recruitment

Digital Remote Recruitment
Yes
Planned Sample Size
165
Recruitment Window Months
52
Consent Approach
Informed consent and assent: parent/legal guardian provides consent for minors (Parent/Guardian ICF / Parent_Legal-Rep ICF) and adolescents provide age-appropriate assent (Pediatric Assent 12-15; Assent 12-17 / Assent 13-17 documents). Adult participants (18 years) sign Main ICF. Additional forms for newborn/pregnancy follow-up and father/newborn ICFs are available. Some pre-consent telephone procedures (Scout Clinical Pre-ICF Telephone Data Consent) are used. Materials and ICF/assent documents are provided in multiple languages (Portuguese, Polish, Italian, Spanish, English where available).

Methods

  • Use of recruitment materials (posters) - country-specific recruitment posters available (document titles: Recruitment-Poster for PT/PL/IT/ES).
  • GP letters and referral letters - GP-Letter and Referral-Letter documents present for multiple countries.
  • Website materials - country-specific study websites and 'Website' documents (PT/IT/ES) used as recruitment channel.
  • Educational videos and links - 'Link-for-videos' and 'Link-for-update-video' materials referenced in recruitment documents.
  • IA flipcharts and local awareness materials (IA-Flip-Chart) used at sites.
  • Patient-facing explanatory materials ('Understanding-Eptinezumab' for adolescents/adults) in multiple languages.
  • Pre-ICF telephone consent/data collection by Scout Clinical (Scout Clinical Pre-ICF Telephone Data Consent).
  • Patient transportation and reimbursement support (Scout Clinical duties) and site-level recruitment/retention services (Jumo Health duties).

Geography

Total Number Of Sites
22
Total Number Of Participants
174

Portugal

Earliest CTIS Part Ii Submission Date
29-08-2024
Latest Decision Or Authorization Date
13-02-2025
Processing Time Days
168
Number Of Sites
3
Number Of Participants
40

Sites

Site Name
Unidade Local de Saude de Sao Joao E.P.E.
Department Name
Departamento de Pediatria
Principal Investigator Name
Mafalda Sampaio
Principal Investigator Email
mafalda.sampaio@chsj.min-saude.pt
Contact Person Name
Mafalda Sampaio
Site Name
Unidade Local De Saude De Almada-Seixal E.P.E.
Department Name
Pediatria
Principal Investigator Name
João Carvalho
Principal Investigator Email
joao.nuno.carvalho@hgo.min-saude.pt
Contact Person Name
João Carvalho
Site Name
Unidade Local De Saude De Coimbra E.P.E.
Department Name
Neuropediatria
Principal Investigator Name
Filipe Palavra
Principal Investigator Email
filipepalavra@gmail.com
Contact Person Name
Filipe Palavra
Contact Person Email
filipepalavra@gmail.com

Poland

Earliest CTIS Part Ii Submission Date
20-03-2025
Latest Decision Or Authorization Date
17-09-2025
Processing Time Days
181
Number Of Sites
7
Number Of Participants
44

Sites

Site Name
ETG Lublin Sp. z o.o.
Department Name
ETG Lublin Poradnie Specjalistyczne
Principal Investigator Name
Dominika Tomaka
Principal Investigator Email
d.tomaka@etg-network.com
Contact Person Name
Dominika Tomaka
Contact Person Email
d.tomaka@etg-network.com
Site Name
Centrum Medyczne Plejady Magdalena Celinska Loewenhoff Michal Zolnowski sp.k.
Principal Investigator Name
Marta Żołnowska
Principal Investigator Email
trials@plejady.com.pl
Contact Person Name
Marta Żołnowska
Contact Person Email
trials@plejady.com.pl
Site Name
Instytut Zdrowia Dr Boczarska-Jedynak Sp. z o.o. S.K.
Principal Investigator Name
Magdalena Boczarska-Jedynak
Principal Investigator Email
m.boczarska@instytutboczarska.pl
Contact Person Name
Magdalena Boczarska-Jedynak
Site Name
MIGRE Polskie Centrum Leczenia Migreny Anna Gryglas-Dworak
Principal Investigator Name
Anna Gryglas-Dworak
Principal Investigator Email
anna.gryglas@migre.pl
Contact Person Name
Anna Gryglas-Dworak
Contact Person Email
anna.gryglas@migre.pl
Site Name
Athleticomed Sp. z o.o.
Principal Investigator Name
Magdalena Nowaczewska
Principal Investigator Email
m.nowaczewska@athleticomed.pl
Contact Person Name
Magdalena Nowaczewska
Contact Person Email
m.nowaczewska@athleticomed.pl
Site Name
Etg Neuroscience Sp. z o.o.
Principal Investigator Name
Aleksandra Karbowniczek
Principal Investigator Email
a.karbowniczek@neuroscience.com.pl
Contact Person Name
Aleksandra Karbowniczek
Site Name
Mtz Clinical Research Powered By Pratia
Principal Investigator Name
Damian Okruciński
Principal Investigator Email
badacz@pratia.com
Contact Person Name
Damian Okruciński
Contact Person Email
badacz@pratia.com

Italy

Earliest CTIS Part Ii Submission Date
29-08-2024
Latest Decision Or Authorization Date
21-10-2025
Processing Time Days
418
Number Of Sites
7
Number Of Participants
60

Sites

Site Name
Ospedale Pediatrico Bambino Gesu
Department Name
Neurologist
Principal Investigator Name
Laura Papetti
Principal Investigator Email
laura.papetti@opbg.net
Contact Person Name
Laura Papetti
Contact Person Email
laura.papetti@opbg.net
Site Name
IRCCS Istituto Giannina Gaslini
Department Name
Neurologist
Principal Investigator Name
Edoardo Canale
Principal Investigator Email
edoardocanale@gaslini.org
Contact Person Name
Edoardo Canale
Contact Person Email
edoardocanale@gaslini.org
Site Name
IRCCS Foundation Istituto Neurologico Carlo Besta
Department Name
Neurologist
Principal Investigator Name
Licia Grazzi
Principal Investigator Email
licia.grazzi@istituto-besta.it
Contact Person Name
Licia Grazzi
Contact Person Email
licia.grazzi@istituto-besta.it
Site Name
Azienda Ospedaliera Universitaria Meyer IRCCS
Department Name
Neurologist
Principal Investigator Name
Renzo Guerrini
Principal Investigator Email
renzo.guerrini@meyer.it
Contact Person Name
Renzo Guerrini
Contact Person Email
renzo.guerrini@meyer.it
Site Name
Azienda Socio Sanitaria Locale N. 8 Di Cagliari
Department Name
Neurologist
Principal Investigator Name
Maria Giuseppa Ledda
Principal Investigator Email
mariagiuseppinaledda@gmail.com
Contact Person Name
Maria Giuseppa Ledda
Contact Person Email
mariagiuseppinaledda@gmail.com
Site Name
Fondazione Istituto Neurologico Nazionale Casimiro Mondino
Department Name
Neurologist
Principal Investigator Name
Valentina De Giorgis
Principal Investigator Email
valentina.degiorgis@mondino.it
Contact Person Name
Valentina De Giorgis
Contact Person Email
valentina.degiorgis@mondino.it
Site Name
Ospedale San Raffaele S.r.l.
Department Name
Neurologist
Principal Investigator Name
Massimo Filippi
Principal Investigator Email
filippi.massimo@hsr.it
Contact Person Name
Massimo Filippi
Contact Person Email
filippi.massimo@hsr.it

Spain

Earliest CTIS Part Ii Submission Date
29-08-2024
Latest Decision Or Authorization Date
15-05-2026
Processing Time Days
624
Number Of Sites
5
Number Of Participants
30

Sites

Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Headache Unit
Principal Investigator Name
Samuel Díaz Insa
Principal Investigator Email
sdiazinsa@yahoo.es
Contact Person Name
Samuel Díaz Insa
Contact Person Email
sdiazinsa@yahoo.es
Site Name
Hospital Clinico San Carlos
Department Name
Pediatrics
Principal Investigator Name
Eva Arias Vivas
Principal Investigator Email
eva.arias.vivas@gmail.com
Contact Person Name
Eva Arias Vivas
Contact Person Email
eva.arias.vivas@gmail.com
Site Name
Hospital Universitari Vall D Hebron
Department Name
Neurology
Principal Investigator Name
Patricia Pozo-Rosich
Principal Investigator Email
patricia.pozo@vhir.org
Contact Person Name
Patricia Pozo-Rosich
Contact Person Email
patricia.pozo@vhir.org
Site Name
Hospital Alvaro Cunqueiro
Department Name
Pediatrics
Principal Investigator Name
Manuel Oscar Blanco Barca
Principal Investigator Email
droblanco@hotmail.com
Contact Person Name
Manuel Oscar Blanco Barca
Contact Person Email
droblanco@hotmail.com
Site Name
University Hospital Virgen Del Rocio S.L.
Department Name
Neurology Department
Principal Investigator Name
Carmen Gonzalez Oria
Principal Investigator Email
carmengoria@hotmail.com
Contact Person Name
Carmen Gonzalez Oria
Contact Person Email
carmengoria@hotmail.com

Sponsor

Primary sponsor

Full Name
H. Lundbeck A/S
Organisation Type
Pharmaceutical company
Country Of Registered Address
Denmark

Contract research organisations

Name
PPD International Holdings LLC
Responsibilities
Supplies for sample collection and testing of safety labs
Name
Pharmaceutical Product Development LLC
Responsibilities
Supplies for sample collection and testing of safety labs
Name
PPD Development LP
Name
Eresearchtechnology Inc.
Responsibilities
Cardiac Services, eCOA
Name
Medidata Solutions Inc.

Third parties

  • {"country":"United States","full_name":"Jumo Health USA Inc.","duties_or_roles":"Patient recruitment and retention materials","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Patient transportation and patient reimbursement","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"Belgium","full_name":"PPD International Holdings LLC","duties_or_roles":"Supplies for sample collection and testing of safety labs","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"Supplies for sample collection and testing of safety labs","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Cardiac Services, eCOA","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
VYEPTI 100 mg concentrate for solution for infusion
Active Substance
EPTINEZUMAB
Modality
Monoclonal antibody
Routes Of Administration
INTRAVENOUS USE
Route
Intravenous
Authorisation Status
Authorised (EU MA: EU/1/21/1599/001)
Starting Dose
100 mg (one of the study doses; weight adjusted)
Dose Levels
100 mg; 300 mg
Frequency
Single IV infusion
Maximum Dose
300 mg
Investigational Product Name
Nacl 0.9%
Modality
Other
Routes Of Administration
Intravenous (placebo matching eptinezumab)
Route
Intravenous
Authorisation Status
Not authorised / N/A
Starting Dose
Placebo matching single IV infusion
Frequency
Single IV infusion

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