Post-Authorisation Safety Study of Paediatric Patients Initiating Selumetinib: A Multiple-Country Prospective Cohort Study

Study identification

• EU PAS number:
  EUPAS45972
• Study ID:
  48033
• Official title and acronym:
  Post-Authorisation Safety Study of Paediatric Patients Initiating Selumetinib: A Multiple-Country Prospective Cohort Study
• DARWIN EU® study:
  No
• Study countries:
  Austria

  France

  Germany

  Israel

  Italy

  Netherlands

  Portugal

  Spain

  Switzerland

  United Kingdom
• Study description:
  This is a cohort study of paediatric patients (aged 3 to <18 years of age) with NF1 with symptomatic, inoperable PNs who begin selumetinib treatment at study sites across several European countries where selumetinib has been marketed for use. Selumetinib treatment will remain a decision of the treating clinicians and is not mandated by this study protocol. All patients prescribed selumetinib at the study sites in the usual manner and according to the terms of the marketing authorisation will be invited to participate in the study. Patients who meet the eligibility criteria, including parental/legal guardian consent to participation, will be enrolled. One hundred and twenty-five patients (approximately 100 patients of which will be in the Nested Prospective Cohort) will be enrolled over a period of up to 3 years and assigned an index date (Day 1) defined as the date of first prescription of selumetinib. Baseline data will be collected at enrolment through retrospective chart abstraction from Day -365 to Day -1 (baseline period).The Nested Prospective Cohort of patients (aged 8 to < 18 years who have not reached Tanner Stage V on the index date) will be followed prospectively to further characterise the safety of selumetinib. Data from this cohort will be collected on the occurrence of the safety outcomes of interest identified in Section 4.6 (Table 1). Enrolment will occur at approximately 52 sites in up to 12 European countries and Israel, after selumetinib is commercially available in the country and patients are able to receive.
• Study status:
  Ongoing

Research institutions and networks

Institutions

Sana Klinikum Duisburg

Kinder & Jugendmedizin

Institut Curie (IC)

France

• Inselspital - Universitaetsklinik Bern für Pneumologie
• Complexo Hospitalario Universitario de Santiago (CHUS) - Hospital Clínico Universitario
• Sana Kliniken Duisburg
• Hôpital Necker - Enfants Malades
• Azienda Ospedaliera Universitaria Citta della Salute e della Scienza di Torino - Ospedale Molinette
• Azienda Ospedaliera di Padova
• Hôpitaux Universitaires de Strasbourg - Hôpital de Hautepierre
• Medizinische Universitaet Wien - Universitaetsklinik fuer Kinder und Jugendheilkunde
• Universitätsklinikum Hamburg-Eppendorf (UKE)
• Instituto Português Oncologia de Lisboa Francisco Gentil, EPE
• Fondazione Policlinico Universitario Agostino Gemelli
• CHU Hopitaux de Bordeaux - Groupe Hospitalier Pellegrin
• Centre Hospitalier Universitaire Vaudois Lausanne (CHUV)
• Universitätsklinikum Tübingen Klinik für Kinder- und Jugendmedizin Kinderheilkunde I
• IRCCS Ospedale Pediatrico Bambino Gesù
• Hospital Sant Joan de Déu Barcelona
• Schneider Children's Medical Center of Israel
• University Children's Hospital Basel
• Hôpital des Enfants - Toulouse
• Istituto Giannina Gaslini
• CHU Amiens Sud
• Centre Oscar Lambret
• Centre Léon Bérard
• Hospital Universitario Vall d'Hebron
• Hospital Universitario 12 de Octubre
• Fondazione IRCCS Policlinico San Matteo
• Great Ormond Street Hospital for Children NHS Foundation Trust - Great Ormond Street Hospital
• Manchester University NHS Foundation Trust - Royal Manchester Children's Hospital
• Hospital Universitario Virgen del Rocío
• Universitätsklinikum Carl Gustav Carus Dresden
• Centre Hospitalier Universitaire (CHU) de Rennes - Hopital Anne de Bretagne (Hopital Sud)
• Dr. von Haunersches Kinderspital
• Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
• CHU Nancy - Hôpital Brabois
• Tel Aviv Sourasky Medical Center
• Azienda Ospedaliero Universitaria Meyer
• Ostschweizer Kinderspital - Sankt Gallen
• Hospital Infantil Universitario Nino Jesus
• Centre Hospitalier Regional et Universitaire de Tours - Hopital Clocheville
• Chaim Sheba Medical Center
• CHU Hôpital de la Timone
• Centre Hospitalier Universitaire d'Angers
• Istituto di Ricovero e Cura a Carattere Scientifico Materno-Infantile - Burlo Garofolo
• The Newcastle upon Tyne Hospitals NHS Foundation Trust - The Great North Children's Hospital

Networks

FINPEDMED

Study timelines

Date when funding contract was signed

Planned:22/02/2022

Actual:22/02/2022

Study start date

Planned:23/05/2022

Actual:23/05/2022

Data analysis start date

Planned:12/07/2028

Date of final study report

Planned:31/03/2029

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

AstraZeneca AB

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 2 (specific obligation of marketing authorisation)

Other study registration identification numbers and links

D1346R00004