Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Authorised, recruiting
Participants planned
586
Countries
11
Sites
43
Migraine
To evaluate the long-term safety and tolerability of atogepant treatment for the prevention of migraine in pediatric participants 6 to 17 years of age.
Key facts
- Sponsor
- Abbvie Deutschland GmbH & Co. KG
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Trial duration
- 22 Dec 2023 → ongoing
- Decision date (initial)
- 2023-08-21
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
- Funding sources
- AbbVie Inc.
External identifiers
- EU CT number
- 2022-501099-24-00
- ClinicalTrials.gov
- NCT05707949
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety
To evaluate the long-term safety and tolerability of atogepant treatment for the prevention of migraine in pediatric participants 6 to 17 years of age.
Conditions and MedDRA coding
Migraine
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 21.1 | LLT | 10066636 | Chronic migraine | 10029205 |
| 20.0 | PT | 10027607 | Migraine with aura | 100000004852 |
| 20.0 | PT | 10027599 | Migraine | 100000004852 |
| 22.0 | LLT | 10082019 | Episodic migraine | 10029205 |
| 20.0 | PT | 10052787 | Migraine without aura | 100000004852 |
Study design 3 periods
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Screening/Baseline Period (De novo migraine subjects only) A 4-week migraine assessment period prior to enrolment for de novo migraine subjects only
|
Not Applicable | None | Screening/Baseline Period: A 4-week migraine assessment period prior to initial study drug administration. | |
| 2 | Open-label Treatment Period A 52-weeks open label treatment period where eligible subjects will be administered an open-label single dose of atogepant to evaluate the safety and tolerability of atogepant in pediatric subjects with episodic (6 to 17 years of age) or chronic (12 to 17 years of age) migraine.
|
2 | None | Atogepant Dose A (12-17 yrs): Participants aged 12-17 will receive oral tablets of atogepant Dose A once a day for up to 52 weeks. Atogepant Dose B (6-11 yrs): Participants aged 6-11 will receive the highest dose of oral tablets of atogepant tested in the lead-in study M21-201. |
|
| 3 | Follow-Up Period A 4-week safety follow-up period following the last study drug administration
|
Not Applicable | None | Follow-up Period: A 4-week safety follow-up period following the last study drug administration |
Regulatory references
- EMA paediatric investigation plan (PIP)
- EMEA-002530-PIP01-18
- Plan to share IPD
- Yes
- IPD plan description
- AbbVie is committed to responsible clinical trial data sharing. This includes access to anonymized, individual and trial-level data (analysis data sets), as well as other information. To learn more about the process, or to submit a request, visit https://www.abbvieclinicaltrials.com/hcp/data-sharing/ For details on when studies are available for sharing, visit https://vivli.org/ourmember/abbvie/
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- Participants must be between 6 and 17 years of age (inclusive), with a history of episodic or chronic migraine.
- Participant must have completed the lead-in Study M21-201, or the pharmacokinetic (PK) substudy in Study M21-201 or the lead-in Study M23-712.
- Weight is >= 20 kg (44 lbs) and < 135 kg (298 lbs).
- A history of migraine with or without aura consistent with a diagnosis according to the International Classification of Headache Disorders, 3rd Edition (ICHD-3; 2018) for at least 6 months.
Exclusion criteria 3
- History of migraine with brainstem aura, hemiplegic migraine, or retinal 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).
- Have any clinically significant hematologic, endocrine, pulmonary, renal, hepatic, gastrointestinal, cardiovascular, or neurologic disease.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 8
- The endpoints of Study M21-199 are the safety and tolerability of atogepant, including: Adverse event (AE) monitoring for severity and incidence including adverse event of special interests (AESIs)
- Clinical laboratory testing (hematology, chemistry, and urinalysis)
- Electrocardiogram (ECG) variables
- Vital sign measurements
- Treatment-emergent suicidal ideations with intent, with or without a plan (i.e., Type 4 or 5 on Columbia-Suicide Severity Rating Scale [C-SSRS]) or any suicidal behaviors
- Menstrual cycle assessments (female subject only)
- Tanner Stage scale
- Behavior Rating Inventory of Executive Function, 2nd Edition (BRIEF2)
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 3
PRD9649618 · Product
- Active substance
- Atogepant
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL
- Max daily dose
- 60 mg milligram(s)
- Max total dose
- 21840 mg milligram(s)
- Max treatment duration
- 52 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- ALLERGAN SALES, LLC (A SUBSIDIARY OF ABBVIE INC.)
- Paediatric formulation
- No
- Orphan designation
- No
PRD9649619 · Product
- Active substance
- Atogepant
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL
- Max daily dose
- 60 mg milligram(s)
- Max total dose
- 21840 mg milligram(s)
- Max treatment duration
- 52 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- ALLERGAN SALES, LLC (A SUBSIDIARY OF ABBVIE INC.)
- Paediatric formulation
- No
- Orphan designation
- No
PRD10064866 · Product
- Active substance
- Atogepant
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL
- Max daily dose
- 60 mg milligram(s)
- Max total dose
- 21840 mg milligram(s)
- Max treatment duration
- 52 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- ALLERGAN SALES, LLC (A SUBSIDIARY OF ABBVIE INC.)
- Paediatric formulation
- No
- Orphan designation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Abbvie Deutschland GmbH & Co. KG
- Sponsor organisation
- Abbvie Deutschland GmbH & Co. KG
- Address
- Knollstrasse
- City
- Ludwigshafen Am Rhein
- Postcode
- 67061
- Country
- Germany
Scientific contact point
- Organisation
- Abbvie Deutschland GmbH & Co. KG
- Contact name
- Global Clinical Trials Helpdesk
Public contact point
- Organisation
- Abbvie Deutschland GmbH & Co. KG
- Contact name
- Global Clinical Trials Helpdesk
Third parties 8
| Organisation | City, country | Duties |
|---|---|---|
| Labcorp Central Laboratory Services S.a.r.l. ORG-100011524
|
Meyrin, Switzerland | Laboratory analysis |
| Labcorp Endpoint Clinical Inc. ORG-100040567
|
Wakefield, United States | Interactive response technologies (IRT) |
| Cytel Inc. ORG-100042560
|
Waltham, United States | Other |
| Signant Health LLC ORG-100040732
|
Blue Bell, United States | Other, E-data capture |
| Abbvie Bioresearch Center Inc. ORG-100011500
|
North Chicago, United States | Other |
| Medidata Solutions Inc. ORG-100016256
|
New York, United States | E-data capture |
| Syneos Health Inc. ORG-100008382
|
Raleigh, United States | Other |
| eResearchTechnology GmbH ORG-100044103
|
Estenfeld, Germany | Other |
Locations
11 EU/EEA countries · 43 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Belgium | Ongoing, recruiting | 5 | 2 |
| Denmark | Ongoing, recruiting | 20 | 3 |
| France | Ended | 7 | 3 |
| Hungary | Ongoing, recruiting | 11 | 6 |
| Italy | Ongoing, recruiting | 7 | 3 |
| Netherlands | Ongoing, recruiting | 7 | 4 |
| Poland | Ongoing, recruiting | 14 | 6 |
| Portugal | Authorised, recruitment pending | 25 | 5 |
| Romania | Ended | 10 | 3 |
| Spain | Ongoing, recruiting | 11 | 6 |
| Sweden | Ongoing, recruiting | 10 | 2 |
| Rest of world
Israel, Canada, Puerto Rico, United States, United Kingdom, Japan
|
— | 459 | — |
Investigational sites
Antwerp University Hospital
Pediatric Neurology, Drie Eikenstraat 655, 2650, Edegem
Az St-Jan Brugge-Oostende A.V.
Paediatrics - paediatrics neurology, Ruddershove 10, 8000, Brugge
Regionshospitalet Godstrup
Born og Unge, Hospitalsparken 15, 7400, Herning
Aalborg University Hospital
Department of Pediatric and Adolescent, Reberbansgade 15, 9000, Aalborg
Herlev Hospital
Pediatric Department, Borgmester Ib Juuls Vej 1, 2730, Herlev
Centre Hospitalier Universitaire De Toulouse
Pédiatrie - Centre de ressource douleur soins palliatifs pédiatriques, 2 Rue Viguerie, 31300, Toulouse
Centre Hospitalier Universitaire Amiens Picardie
Service de Neurologie pediatrique, 1 Rond Point Du Professeur Christian Cabrol, 80054, Amiens
Centre Hospitalier Intercommunal Creteil
Consultation pediatrique, 40 Avenue De Verdun, 94000, Creteil
S-Medicon Kft.
Neurologia, Madarasz Viktor utca 47-49, 1138, Budapest
Clinexpert Kft.
Clinexpert Obuda Egeszegcentruma, Kaszasdulo Utca 5, 1033, Budapest III
Semmelweis University
Gyermekgyogyaszati Klinika Bokay utcai Reszleg, Bokay Janos Utca 53, 1083, Budapest VIII
Magyarorszagi Reformatus Egyhaz Bethesda Gyermekkorhaza
Gyermekneurlologiai Osztaly, Bethesda Utca 3, 1146, Budapest Xiv
Semmelweis University
Gyermekgyogyaszati Klinika Tuzolto utcai Reszleg, Tuzolto Utca 7-9, 1094, Budapest
MIND Klinika Kft.
not applicable, Lovohaz Utca 39, Kerulet, Budapest II
Bambino Gesu Childrens Hospital
Neuroscienze, Piazza Sant'onofrio 4, 00165, Rome
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
UOSD Neurofisiopatologia, Via Del Vespro 129, 90127, Palermo
The Foundation Of The Carlo Besta Neurological Institute IRCCS
Headache Center Neuroalgology, Via Giovanni Celoria 11, 20133, Milan
Boerhaave Medisch Centrum
Neurologie, Dintelstraat 60, 1078 W, Amsterdam
Haga Hospital
Juliana Kinderziekenhuis, Els Borst-Eilersplein 275, 2545 AA, 's-Gravenhage
ZorgSaam Ziekenhuis
Neurologie, Wielingenlaan 2, 4535 PA, Terneuzen
Canisius Wilhelmina Hospital
Neurologie, Weg Door Jonkerbos 100, 6532 SZ, Nijmegen
Indywidualna Specjalistyczna Praktyka Lekarska
NA, ul. Onyksowa 12 Lubelskie, 20-582, Lublin
MIGRE Polskie Centrum Leczenia Migreny
NA, ul. Lubinowa 12/7 Dolnośląskie, 52-210, Wroclaw
Athleticomed Sp. z o.o.
NA, Ul. Fordonska 144, 85-752, Bydgoszcz
Specjalistyczne Gabinety Sp. z o.o.
NA, Pl. Lasoty 4, 30-539, Cracow
OHA-Med Sp. z o.o.
NA, Lok 5u-80, Ul. Wolnosc 2, Warsaw
Clinical Research Center Sp. z o.o. Medic-R sp.k.
NA, Ul. Feliksa Nowowiejskiego 5, 61-731, Poznan
Unidade Local De Saude De Santa Maria E.P.E.
Pediatric Department, Avenida Professor Egas Moniz, 1649-035, Lisbon
Unidade Local De Saude De Almada-Seixal E.P.E.
Pediatric Department, Avenida Torrado Da Silva, 2805-267, Almada
Unidade Local De Saude De Santo Antonio E.P.E.
Pediatric Department, Largo Da Maternidade De Julio Dinis 45, 4050-651, Porto
Unidade Local De Saude De Coimbra E.P.E.
Pediatric Department, Avenida Afonso Romao, 3000-602, Coimbra
Hospital Da Luz S.A.
Neurology Service, Avenida Lusiada 100, 1500-650, Lisbon
Spitalul Clinic de Urgenta pentru Copii Cluj Napoca
Pediatric Neurology, Str. Victor Babes nr. 43, 400012, Cluj Napoca
Spitalul Clinic de Copii “Dr. Victor Gomoiu”
Pediatric Neurology, Bulevardul Basarabia nr. 21, 022102, Bucuresti
Delta Health Care S.R.L.
Pediatric Neurology Department, Str Nicolae Caramfil Nr 85a Sector 1, 014146, Bucharest
Hospital Universitari Vall D Hebron
Neurology Department, Edificio Materno-Infantil, Passeig De La Vall D'hebron 119-129, Barcelona
Hospital De La Santa Creu I Sant Pau
Neurology Department, Calle De San Antonio Maria Claret 167, 08025, Barcelona
Hospital Clinico San Carlos
Pediatric Department, Calle Del Profesor Martin Lagos S/n, 28040, Madrid
Hospital Universitario 12 De Octubre
Paediactric Neurology, Avenida De Cordoba Sn, 28041, Madrid
Hospital Universitario Y Politecnico La Fe
Neurology Department, Avenida De Fernando Abril Martorell 106, 46026, Valencia
University Hospital Virgen Del Rocio S.L.
Department of Neurology, Avenida De Manuel Siurot S/n, 41013, Sevilla
Vastra Gotalandsregionen
Molnlycke Pediatric Clinic, Regionens Hus, 462 80, Vänersborg
Soedersjukhuset AB
Sach's Children and Youth Hospital, Sjukhusbacken 10, 118 83, STOCKHOLM
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Belgium | 2024-02-27 | 2024-11-27 | |||
| Denmark | 2024-01-03 | 2024-06-04 | |||
| France | 2024-02-27 | 2026-05-28 | 2024-07-02 | ||
| Hungary | 2024-01-18 | 2024-03-11 | |||
| Italy | 2024-01-09 | 2024-08-30 | |||
| Netherlands | 2024-02-12 | 2024-03-28 | |||
| Poland | 2024-01-16 | 2024-04-02 | |||
| Romania | 2024-03-07 | ||||
| Spain | 2023-12-22 | 2024-04-01 | |||
| Sweden | 2024-02-07 | 2024-03-20 |
Oversight and notifications
Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77
Corrective measures 1 · Art. 77 CTR
Corrective measure CM-HU-0001
- Member state
- Hungary
- Publication date
- 2024-07-05
- Type
- 3
- Reason
- 5
- Immediate action required
- No
- Justification
- associated notification: ad hoc assessment FR-0000000010; Justification: New identified risk
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 186 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_m21199-protocol-redacted | 5.0 |
| Protocol (for publication) | D4_Patient facing documents-BRIEF2 Parent Form-allMS-public | 1 |
| Protocol (for publication) | D4_Patient facing documents-BRIEF2 Self-Report Form-allMS-public | 1 |
| Recruitment arrangements (for publication) | 1_M21-199_ES-Recruitment and ICF Procedures-san public | 2 |
| Recruitment arrangements (for publication) | K1 M21-199 PT Recruitment and ICF Procedures Public | 1 |
| Recruitment arrangements (for publication) | K1_M21-199 BE Recruitment and ICF Procedures_Public redacted | 2 |
| Recruitment arrangements (for publication) | K1_M21-199 FR Recruitment and ICF Procedures_Public | 2 |
| Recruitment arrangements (for publication) | K1_M21-199 NL Recruitment and ICF procedure_Public | 2.0 |
| Recruitment arrangements (for publication) | K1_M21-199 PL Recruitment and ICF Procedures_Public | 2 |
| Recruitment arrangements (for publication) | K1_M21-199 Recruitment and ICF Procedures clean_public redacted | 2.0 |
| Recruitment arrangements (for publication) | K1_M21-199_HU_Recruitment and ICF Procedures_clean_Public | 2.0 |
| Recruitment arrangements (for publication) | K1_M21-199_RO-Recruitment and ICF Procedures-public | 2.0 |
| Recruitment arrangements (for publication) | K1_M21-199_SE-Recruitment and ICF Procedures | 2.0 |
| Recruitment arrangements (for publication) | M21-199_ IT- Participant Letters GP to GP_public | 2 |
| Recruitment arrangements (for publication) | M21-199_BE-Participant Letters GP to Parent Dutch-san public | 2 |
| Recruitment arrangements (for publication) | M21-199_BE-Participant Letters GP to Parent English-san public | 2 |
| Recruitment arrangements (for publication) | M21-199_BE-Participant Letters GP to Parent French-san public | 2 |
| Recruitment arrangements (for publication) | M21-199_BE-Recruitment letters GP to GP Dutch-san public | 2 |
| Recruitment arrangements (for publication) | M21-199_BE-Recruitment letters GP to GP English-san public | 2 |
| Recruitment arrangements (for publication) | M21-199_BE-Recruitment letters GP to GP French-san public | 2 |
| Recruitment arrangements (for publication) | M21-199_DK- Participant Letters GP to Parent-san public | 1 |
| Recruitment arrangements (for publication) | M21-199_DK-Recruitment and ICF Procedures-san public | 3 |
| Recruitment arrangements (for publication) | M21-199_DK-Recruitment letters GP to GP-san public | 2 |
| Recruitment arrangements (for publication) | M21-199_ES-Participant Letters GP to Parent-san public | 1 |
| Recruitment arrangements (for publication) | M21-199_ES-Recruitment letters GP to GP-san public | 2 |
| Recruitment arrangements (for publication) | M21-199_FR-Participant Letter GP to GP French_public | 1 |
| Recruitment arrangements (for publication) | M21-199_FR-Participant Letter GP to Patient_public | 1 |
| Recruitment arrangements (for publication) | M21-199_HU-Participant Letters GP to Parent-san public | 2 |
| Recruitment arrangements (for publication) | M21-199_HU-Recruitment letters GP to GP-san public | 2 |
| Recruitment arrangements (for publication) | M21-199_IT-Participant Letters GP to Patient Italian_public | 3 |
| Recruitment arrangements (for publication) | M21-199_NL-Participant Letters GP to Parent_public | 1 |
| Recruitment arrangements (for publication) | M21-199_PL Recruitment and ICF Procedures_public | 1 |
| Recruitment arrangements (for publication) | M21-199_PL-Participant Letters GP to GP Polish_public | 1 |
| Recruitment arrangements (for publication) | M21-199_PL-Participant Letters GP to Patient_public | 1 |
| Recruitment arrangements (for publication) | M21-199_RO-Participant Letters GP to Parent-san public | 2 |
| Recruitment arrangements (for publication) | M21-199_RO-Recruitment letters GP to GP-san public | 2 |
| Recruitment arrangements (for publication) | M21-199_SE-Participant Letters GP to Parent_public | 1 |
| Subject information and informed consent form (for publication) | L1 M21-199 IT Assent Roll Over Subjects 12-17 years clean public | 6.0 |
| Subject information and informed consent form (for publication) | L1_M21-199 NL ICF Optional 16 plus_Public | 2.1 |
| Subject information and informed consent form (for publication) | L1_M21-199 NL ICF Optional Assent 12-15_Public | 2.1 |
| Subject information and informed consent form (for publication) | L1_M21-199 NL ICF Optional Parents_Public | 2.1 |
| Subject information and informed consent form (for publication) | L1_M21-199 NL ICF Pregnant Subject_Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_M21-199 PL ICF Assent 13-17 Main Rollover_Public | 6 |
| Subject information and informed consent form (for publication) | L1_M21-199 PL ICF Assent 13-17 Optional Rollover_Public | 2 |
| Subject information and informed consent form (for publication) | L1_M21-199 PL ICF Main_Public | 6 |
| Subject information and informed consent form (for publication) | L1_M21-199 PL ICF Optional_Public | 2 |
| Subject information and informed consent form (for publication) | L1_M21-199 PL ICF Parents Main Rollover_Public | 6 |
| Subject information and informed consent form (for publication) | L1_M21-199 PL ICF Parents Optional Rollover_Public | 2 |
| Subject information and informed consent form (for publication) | L1_M21-199 PT Assent 12-15 Years_Public Redacted | 1 |
| Subject information and informed consent form (for publication) | L1_M21-199 PT Combined ICF Main and Optional_16-17 Years_Public Redacted | 1 |
| Subject information and informed consent form (for publication) | L1_M21-199 PT Combined ICF Main and Optional_Parents_Public Redacted | 1 |
| Subject information and informed consent form (for publication) | L1_M21-199 PT Combined ICF_Main and Optional_Public Redacted | 1 |
| Subject information and informed consent form (for publication) | L1_M21-199 PT ICF Pregnant_Public Redacted | 1 |
| Subject information and informed consent form (for publication) | L1_M21-199_BE_ICF Assent 12-17 rollover Dutch_public | 7.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_BE_ICF Assent 12-17 rollover English_public | 7.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_BE_ICF Assent 12-17 rollover French_public | 7.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_BE_ICF Main Parent and 18 plus rollover French_public_Redacted | 9.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_BE-ICF Main Parent and 18 plus rollover Dutch_public_Redacted | 9.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_BE-ICF Main Parent and 18 plus rollover English_public_Redacted | 9.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_DK_ICF Assent Age 12-14 Roll Over_public | 5 |
| Subject information and informed consent form (for publication) | L1_M21-199_DK_ICF Assent Age 15-17 Roll Over_public | 5 |
| Subject information and informed consent form (for publication) | L1_M21-199_DK_ICF Optional Research Parent and 18 plus_public | 3 |
| Subject information and informed consent form (for publication) | L1_M21-199_DK_ICF Parent and 18 plus Roll Over_public | 5 |
| Subject information and informed consent form (for publication) | L1_M21-199_ES_ICF Assent 12-17 rollover_public | 5.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_ES_ICF Main 18 plus rollover_public | 5.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_ES_ICF Main Parent rollover_public | 5.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_ES-ICF Optional 18 plus rollover_public | 3.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_ES-ICF Optional Parent rollover_public | 3.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_ES-ICF Pregnant Partner_public | 2 |
| Subject information and informed consent form (for publication) | L1_M21-199_FR_ICF Assent 12-17 yo Roll Over_Public | 5.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_FR_ICF Main Roll Over_Public | 5.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_FR_ICF Parent Roll Over_Public | 5.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_HU_List of ICFs_clean_Public | 2.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_HU_PIS_Assent_Rollover 12-17ys_clean_Public | 5.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_HU_PIS_ICF_Rollover over 18ys_clean_Public Redacted | 5.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_HU_PIS_ICF_Rollover Parent_clean_Public Redacted | 5.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_IT_ICF for pregnancy_public | 2.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_IT_Parent Roll Over Subjects clean_public | 6.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_NL_ICF Main Assent Rollover 12-15_Public | 5.2 |
| Subject information and informed consent form (for publication) | L1_M21-199_NL_ICF Main Rollover 16 plus_Public | 5.2 |
| Subject information and informed consent form (for publication) | L1_M21-199_NL_ICF Main Rollover and DeNovo Parents_Public | 5.2 |
| Subject information and informed consent form (for publication) | L1_M21-199_PL_ICF Pregnancy_Public | 3 |
| Subject information and informed consent form (for publication) | L1_M21-199_RO_ICF Parent and 18 plus rollover_public | 3.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_RO_ICF_Assent 12-17ys_Rollover_clean_Public | 3.1 |
| Subject information and informed consent form (for publication) | L1_M21-199_SE_ICF Assent 12-17 roll-over_Public | 5.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_SE_ICF Parent-Guardian-Adult roll-over_Public | 5.0 |
| Subject information and informed consent form (for publication) | L1_M21-199_SE-ICF Assent 6-11 roll-over_public | 2.0 |
| Subject information and informed consent form (for publication) | L2_M21-199 FR Lettre Informations Patient_Public | 1 |
| Subject information and informed consent form (for publication) | L2_M21-199_DK_Dine rettigheder som forsgsperson i forsg med medicin | 2 |
| Subject information and informed consent form (for publication) | M21-199 _DK -ICF Assent De novo Age 15-17_public | 1 |
| Subject information and informed consent form (for publication) | M21-199 _FR-ICF Main Combined subject -Adult De Novo_public | 1 |
| Subject information and informed consent form (for publication) | M21-199 DK -ICF Other Danish_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_BE-ICF Assent 12-17 de novo Dutch_public | 2 |
| Subject information and informed consent form (for publication) | M21-199_BE-ICF Assent 12-17 de novo English_public | 2 |
| Subject information and informed consent form (for publication) | M21-199_BE-ICF Assent 12-17 de novo French_public | 2 |
| Subject information and informed consent form (for publication) | M21-199_BE-ICF Assent 6-11 de novo Dutch_public | 2 |
| Subject information and informed consent form (for publication) | M21-199_BE-ICF Assent 6-11 de novo English_public | 2 |
| Subject information and informed consent form (for publication) | M21-199_BE-ICF Assent 6-11 de novo French_public | 2 |
| Subject information and informed consent form (for publication) | M21-199_BE-ICF Assent 6-11 rollover English_public | 2 |
| Subject information and informed consent form (for publication) | M21-199_BE-ICF Assent 6-11 rollover Dutch_public | 2 |
| Subject information and informed consent form (for publication) | M21-199_BE-ICF Assent 6-11 rollover French_public | 2 |
| Subject information and informed consent form (for publication) | M21-199_BE-ICF Main Parent and 18 plus de novo Dutch_public_Redacted | 4 |
| Subject information and informed consent form (for publication) | M21-199_BE-ICF Main Parent and 18 plus de novo English_public_Redacted | 4 |
| Subject information and informed consent form (for publication) | M21-199_BE-ICF Main Parent and 18 plus de novo French_public_Redacted | 4 |
| Subject information and informed consent form (for publication) | M21-199_BE-ICF Optional de novo Parent and 18 plus Dutch_public_Redacted | 2 |
| Subject information and informed consent form (for publication) | M21-199_BE-ICF Optional de novo Parent and 18 plus English_public_Redacted | 2 |
| Subject information and informed consent form (for publication) | M21-199_BE-ICF Optional de novo Parent and 18 plus French_public_Redacted | 2 |
| Subject information and informed consent form (for publication) | M21-199_BE-ICF Optional rollover Parent and 18 plus Dutch_public_Redacted | 2 |
| Subject information and informed consent form (for publication) | M21-199_BE-ICF Optional rollover Parent and 18 plus English_public_Redacted | 2 |
| Subject information and informed consent form (for publication) | M21-199_BE-ICF Optional rollover Parent and 18 plus French_public_Redacted | 2 |
| Subject information and informed consent form (for publication) | M21-199_BE-ICF Pregnant Partner Dutch_public | 2 |
| Subject information and informed consent form (for publication) | M21-199_BE-ICF Pregnant Partner English_public | 2 |
| Subject information and informed consent form (for publication) | M21-199_BE-ICF Pregnant Partner French_public | 2 |
| Subject information and informed consent form (for publication) | M21-199_DK-ICF Assent 12-14 de novo_public | 1.1 |
| Subject information and informed consent form (for publication) | M21-199_DK-ICF Assent 6-11 de novo_public | 1.1 |
| Subject information and informed consent form (for publication) | M21-199_DK-ICF Assent 6-11 rollover_public | 1.1 |
| Subject information and informed consent form (for publication) | M21-199_DK-ICF Parent and 18 plus de novo_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_DK-ICF Pregnant Partner_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_DK-Informeret Samtykke Optional Research Forldre S6_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_DK-Informeret Samtykke Optional Research over 18 ar S4_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_DK-POA Main 18 plus_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_DK-POA Main Parent_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_DK-POA Parent to Parent_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_ES-ICF Assent 12-17 de novo_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_ES-ICF Main 18 plus de novo_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_ES-ICF Main Parent de novo_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_ES-ICF Optional 18 plus de novo_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_ES-ICF Optional Parent de novo_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_FR-ICF Assent 12-17 years-De Novo subject__public | 1.1 |
| Subject information and informed consent form (for publication) | M21-199_FR-ICF Assent 6 to11 years_Roll Over_public | 1.1 |
| Subject information and informed consent form (for publication) | M21-199_FR-ICF Assent De Novo 6-11 years old_public | 1.1 |
| Subject information and informed consent form (for publication) | M21-199_FR-ICF Parent-Guardian De Novo_Parental Consent main and optional_public | 1.1 |
| Subject information and informed consent form (for publication) | M21-199_FR-ICF PregPart_public | 1.1 |
| Subject information and informed consent form (for publication) | M21-199_HU-Description of submitted ICFs_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_HU-ICF Assent 12-17 de novo_public | 1.1 |
| Subject information and informed consent form (for publication) | M21-199_HU-ICF Assent 6-11 de novo_public | 1.1 |
| Subject information and informed consent form (for publication) | M21-199_HU-ICF Assent 6-11 rollover_public | 1.1 |
| Subject information and informed consent form (for publication) | M21-199_HU-ICF Main 18 plus de novo_public | 1.1 |
| Subject information and informed consent form (for publication) | M21-199_HU-ICF Main Parent de novo_public | 1.1 |
| Subject information and informed consent form (for publication) | M21-199_HU-ICF Optional PharmacoGenetic 18 plus_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_HU-ICF Optional PharmacoGenetic Assent 12-17_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_HU-ICF Optional PharmacoGenetic Assent 6-11_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_HU-ICF Optional PharmacoGenetic Parent_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_HU-ICF Pregnant Partner_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_HU-PIS Optional PharmacoGenetic 18 plus_public_Redacted | 1 |
| Subject information and informed consent form (for publication) | M21-199_HU-PIS Optional PharmacoGenetic Assent 12-17_public_Redacted | 1 |
| Subject information and informed consent form (for publication) | M21-199_HU-PIS Optional PharmacoGenetic Assent 6-11_public_Redacted | 1 |
| Subject information and informed consent form (for publication) | M21-199_HU-PIS Optional PharmacoGenetic Parent_public_Redacted | 1 |
| Subject information and informed consent form (for publication) | M21-199_HU-Subject Participation Card _public | 1.1 |
| Subject information and informed consent form (for publication) | M21-199_IT-ICF Assent De Novo 12-17 years_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_IT-ICF Assent De novo 6-11 years_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_IT-ICF Assent Roll Over 6-11 years_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_IT-ICF Parent-Guardian De novo_public | 2 |
| Subject information and informed consent form (for publication) | M21-199_NL-ICF Assent De Novo 12-15_public | 1.3 |
| Subject information and informed consent form (for publication) | M21-199_NL-ICF Assent Rollover 6-11_public | 1.1 |
| Subject information and informed consent form (for publication) | M21-199_NL-ICF De Novo 6-11_public | 1.1 |
| Subject information and informed consent form (for publication) | M21-199_NL-ICF Main De Novo 16 plus_public | 1.3 |
| Subject information and informed consent form (for publication) | M21-199_PL-ICF Assent Child under 13 years old de novo_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_PL-ICF Assent Main for Minors age 13-17 de novo_public | 2 |
| Subject information and informed consent form (for publication) | M21-199_PL-ICF Child Assent under 13 years old rollover_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_PL-ICF Optional for Minors age 13-17 de novo_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_PL-ICF Optional Parents de novo_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_PL-ICF Parent-Guardian-Parents Main de novo_public | 2 |
| Subject information and informed consent form (for publication) | M21-199_RO-ICF Assent 12-17 de novo _public | 1 |
| Subject information and informed consent form (for publication) | M21-199_RO-ICF Assent 6-11 de novo_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_RO-ICF Assent 6-11 rollover_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_RO-ICF Parent and 18 plus de novo_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_RO-ICF Pregnant Partner_public | 1 |
| Subject information and informed consent form (for publication) | M21-199_SE-ICF Assent 12-17 de-novo_public | 1.1 |
| Subject information and informed consent form (for publication) | M21-199_SE-ICF Assent 6-11 de-novo_public | 1.1 |
| Subject information and informed consent form (for publication) | M21-199_SE-ICF Parent Guardian de-novo_public | 1.1 |
| Subject information and informed consent form (for publication) | M21-199_SE-ICF PregPart_public | 1 |
| Subject information and informed consent form (for publication) | Pharma be ICF Template Rationale Letter_public_Redacted | 1 |
| Synopsis of the protocol (for publication) | D1_m21199-protocol-synopsis | 5.0 |
| Synopsis of the protocol (for publication) | D1_m21199-protocol-synopsis-ES-ES | 5.0 |
| Synopsis of the protocol (for publication) | D1_m21199-protocol-synopsis-FR-FR | 5.0 |
| Synopsis of the protocol (for publication) | D1_m21199-protocol-synopsis-HU-HU | 5.0 |
| Synopsis of the protocol (for publication) | D1_m21199-protocol-synopsis-IT-IT | 5.0 |
| Synopsis of the protocol (for publication) | D1_m21199-protocol-synopsis-lay | 2 |
| Synopsis of the protocol (for publication) | D1_m21199-protocol-synopsis-lay-DE-BE | 2 |
| Synopsis of the protocol (for publication) | D1_m21199-protocol-synopsis-lay-FR-BE | 2 |
| Synopsis of the protocol (for publication) | D1_m21199-protocol-synopsis-lay-NL-BE | 2 |
| Synopsis of the protocol (for publication) | D1_m21199-protocol-synopsis-lay-NL-NL | 2 |
| Synopsis of the protocol (for publication) | D1_m21199-protocol-synopsis-lay-RO-RO | 2 |
| Synopsis of the protocol (for publication) | D1_m21199-protocol-synopsis-lay-SV-SE | 2 |
| Synopsis of the protocol (for publication) | D1_m21199-protocol-synopsis-PL-PL | 5.0 |
Application history
26 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-04-28 | Hungary | Acceptable with conditions 2023-08-21
|
2023-08-21 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2023-09-19 | Hungary | Acceptable with conditions 2023-08-21
|
2023-09-19 |
| 3 | SUBSTANTIAL MODIFICATION | SM-1 | 2023-10-05 | Acceptable with conditions | 2023-12-11 | |
| 4 | NON SUBSTANTIAL MODIFICATION | NSM-2 | 2024-01-11 | 2024-01-11 | ||
| 5 | SUBSTANTIAL MODIFICATION | SM-2 | 2024-08-09 | Hungary | Acceptable 2024-11-12
|
2024-11-12 |
| 6 | SUBSTANTIAL MODIFICATION | SM-3 | 2024-12-03 | Acceptable | 2024-12-05 | |
| 7 | SUBSTANTIAL MODIFICATION | SM-10 | 2025-03-25 | Hungary | Acceptable 2025-06-30
|
2025-06-30 |
| 8 | SUBSTANTIAL MODIFICATION | SM-11 | 2025-09-05 | Acceptable | 2025-10-02 | |
| 9 | SUBSTANTIAL MODIFICATION | SM-12 | 2025-09-08 | Acceptable | 2025-10-06 | |
| 10 | SUBSTANTIAL MODIFICATION | SM-15 | 2025-09-08 | Acceptable | 2025-11-25 | |
| 11 | SUBSTANTIAL MODIFICATION | SM-18 | 2025-09-09 | Acceptable | 2025-11-05 | |
| 12 | SUBSTANTIAL MODIFICATION | SM-17 | 2025-09-10 | Acceptable | 2025-10-22 | |
| 13 | SUBSTANTIAL MODIFICATION | SM-19 | 2025-09-11 | Acceptable | 2025-09-24 | |
| 14 | SUBSTANTIAL MODIFICATION | SM-16 | 2025-09-12 | Acceptable | 2025-11-25 | |
| 15 | SUBSTANTIAL MODIFICATION | SM-14 | 2025-09-15 | Hungary | Acceptable | 2025-12-05 |
| 16 | SUBSTANTIAL MODIFICATION | SM-20 | 2025-10-08 | Acceptable | 2025-10-10 | |
| 17 | SUBSTANTIAL MODIFICATION | SM-23 | 2025-11-12 | Acceptable | 2025-12-09 | |
| 18 | SUBSTANTIAL MODIFICATION | SM-22 | 2025-11-13 | Acceptable | 2025-11-24 | |
| 19 | SUBSTANTIAL MODIFICATION | SM-21 | 2025-11-21 | Acceptable | 2025-12-03 | |
| 20 | SUBSTANTIAL MODIFICATION | SM-24 | 2025-11-24 | Acceptable | 2025-12-09 | |
| 21 | SUBSTANTIAL MODIFICATION | SM-25 | 2025-11-24 | Acceptable | 2026-01-15 | |
| 22 | SUBSTANTIAL MODIFICATION | SM-26 | 2025-12-11 | Acceptable | 2026-02-10 | |
| 23 | SUBSTANTIAL MODIFICATION | SM-28 | 2025-12-16 | Acceptable | 2026-02-06 | |
| 24 | SUBSTANTIAL MODIFICATION | SM-30 | 2025-12-19 | Acceptable | 2026-02-06 | |
| 25 | SUBSTANTIAL MODIFICATION | SM-29 | 2026-01-23 | Hungary | Acceptable | 2026-02-25 |
| 26 | SUBSEQUENT ADDITION OF MSC | APP-26 | 2026-02-20 | Acceptable with conditions 2023-08-21
|
2026-05-08 |