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PASS Study Information Marketing Authorisation Holder(S) BIOPROJET June 21, 2016 Protocol 15-11/ PASS / Wakix® Version 2.0 PASS Study Information Title A 5-year multi-center, observational post-authorization safety study to document the utilisation of Wakix® in the treatment of narcolepsy with or without cataplexy and to collect information on its long-term safety when used in routine medical practice Protocol version identifier Version 2.0 Date of last version of protocol June 21, 2016 EU PAS register number ENCEPP/SDPP/13818 Active substance Pitolisant - ACT code: N07XX11 Medicinal product Wakix® Product reference EU/1/15/1068/001 EU/1/15/1068/002 Procedure number EMA/H/C/002616 Marketing Authorisation Holder BIOPROJET Pharma 9 rue Rameau 75002 Paris - France Ph: +33 (0)1 47 03 66 33 - Fax: +33 (0)1 47 03 66 30 Joint PASS No Research question and objectives To collect information on the long-term safety of Wakix® when used in a real-life setting; To document the drug utilisation patterns of Wakix® in routine medical practice. Country(-ies) of study France; UK; Italy; Germany Author Dr Isabelle Lecomte Bioprojet Pharma 9 rue Rameau 75002 Paris - France Tel: +33 1 47 03 66 38 - Email: [email protected] Marketing authorisation holder(s) Marketing Authorisation Holder BIOPROJET Pharma (sponsor) 9 rue Rameau 75002 Paris - France MAH Contact person Pascale Vernade Bioprojet Pharma 9 rue Rameau – 75002 Paris – France Tel: +33 1 47 03 66 52 Email: [email protected] 1 BIOPROJET June 21, 2016 Protocol 15-11/ PASS / Wakix® Version 2.0 Signature’s Page PROTOCOL P15-11 - ENCEPP/SDPP/13818 / WAKIX® VERSION 2.0 “PASS” A 5-YEAR MULTI-CENTER, OBSERVATIONAL POST-AUTHORIZATION SAFETY STUDY TO DOCUMENT THE UTILIZATION OF WAKIX® IN THE TREATMENT OF NARCOLEPSY WITH AND WITHOUT CATAPLEXY AND TO COLLECT INFORMATION ON ITS LONG-TERM SAFETY WHEN USED IN ROUTINE MEDICAL PRACTICE The study will be conducted in compliance with the following protocol and will be carried in accordance with The European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP). Guide on Methodological Standards in Pharmacoepidemiology (Revision 4). EMA/95098/2010 and local applicable regulatory requirements in the participating countries. I give my complete agreement on this protocol which gives all necessary information to conduct this study. I hereby accept to coordinate this study: The Coordinator Date: __________________ Medical expert Signature: ______________ The Sponsor Dr Jeanne-Marie Lecomte Date: __________________ Bioprojet Responsible Pharmacist Signature: ______________ Medical Project Manager Dr Isabelle Lecomte Date: __________________ Bioprojet Signature: ______________ Qualified Person for Pharmacovigilance Dr Yves Joulin Date: __________________ Bioprojet Signature: ______________ 2 BIOPROJET June 21, 2016 Protocol 15-11/ PASS / Wakix® Version 2.0 Signature’s Page PROTOCOL P15-11 ENCEPP/SDPP/13818 / WAKIX® WAKIX® POST AUTHORIZATION SAFETY STUDY (PASS) A 5-YEAR MULTI-CENTER, OBSERVATIONAL POST-AUTHORIZATION SAFETY STUDY TO DOCUMENT THE TILISATION OF WAKIX® IN THE TREATMENT OF NARCOLEPSY WITH AND WITHOUT CATAPLEXY AND TO COLLECT INFORMATION ON ITS LONG-TERM SAFETY WHEN USED IN ROUTINE MEDICAL PRACTICE The signature below constitutes the approval of this P15-11 PASS Protocol and the attachments, and provides the necessary assurances that this trial will be conducted according all stipulations of the protocol, including all the statements regarding confidentiality, and according to local legal and The European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP). Guide on Methodological Standards in Pharmacoepidemiology (Revision 4). EMA/95098/2010 The site Investigator: Name: ………………………………………………………………………… Hospital: ……………………………………………………………………… Address: ……………………………………………………………………… …………………………………………………………………….. Date: __________________ Signature:______________ 3 BIOPROJET June 21, 2016 Protocol 15-11/ PASS / Wakix® Version 2.0 1 TABLE OF CONTENTS Signature’s Page ............................................................................................................................................... 3 1 TABLE OF CONTENTS ..................................................................................................................................... 4 2 LIST OF ABBREVIATIONS ................................................................................................................................. 7 3 RESPONSIBLE PARTIES ................................................................................................................................... 8 4 ABSTRACT - PROTOCOL P 15-11/ WAKIX® POST AUTHORIZATION SAFETY STUDY (PASS) ................................... 9 5 Amendments and updates .................................................................................................................... 12 6 Milestones ............................................................................................................................................. 12 7 RATIONALE AND BACKGROUND ..................................................................................................................... 13 7.1 Narcolepsy ............................................................................................................................................ 13 7.2 Pitolisant (Wakix®) ............................................................................................................................... 14 7.3 Rational for the Post Marketing Safety Study with Pitolisant .............................................................. 16 8 RESEARCH QUESTION AND OBJECTIVES ........................................................................................................... 17 9 RESEARCH METHODS................................................................................................................................... 18 9.1 Study design .................................................................................................................................. 18 9.1.1 General design .............................................................................................................................. 18 9.1.2 Endpoints ...................................................................................................................................... 19 9.2 Setting ........................................................................................................................................... 21 9.2.1 Selection criteria: .......................................................................................................................... 21 9.2.2 Study visits .................................................................................................................................... 22 V1 – Baseline- Start of a treatment with Wakix® and inclusion of the patient in the study ......... 22 Follow-up visits: at least yearly interim visits (V2, V3, V4, V5 …) ................................................... 23 End of Study Visit (EOS) (V6) after 5 years or in case of Wakix® permanently stop ...................... 24 9.3 Variables ........................................................................................................................................ 24 9.4 Data sources .................................................................................................................................. 26 9.5 Study size ...................................................................................................................................... 26 4 BIOPROJET June 21, 2016 Protocol 15-11/ PASS / Wakix® Version 2.0 9.6 Data management ........................................................................................................................ 28 9.6.1 Identification of any data to be recorded in the CRF and to be considered as source data .. 28 9.6.2 Data Handling and Record keeping ........................................................................................ 28 9.7 Data analysis ................................................................................................................................. 30 9.7.1 Statistical Methods ....................................................................................................................... 30 9.7.2 Primary Endpoints ........................................................................................................................ 31 9.7.3 Secondary endpoints .................................................................................................................... 31 9.7.4 Covariates ..................................................................................................................................... 32 9.7.5 External References ...................................................................................................................... 32 9.7.6 Report ........................................................................................................................................... 33 9.8 Quality control .............................................................................................................................. 33 QUALITY CONTROL AND AUDIT .................................................................................................................... 34 9.9 Limitations of the research methods ............................................................................................ 34 9.10 Other aspect: Confidentiality of study
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