Informations générales (source: ClinicalTrials.gov)
Post-Authorisation Safety Study of Paediatric Patients Initiating Selumetinib: A Multiple-Country Prospective Cohort Study.
Observational
AstraZeneca (Voir sur ClinicalTrials)
mai 2022
mai 2028
14 décembre 2024
Neurofibromatosis type 1 (NF1) is a rare, autosomal dominant genetic disorder that is
caused by germline mutations in the NF1 tumour suppressor gene, which encodes the tumour
suppressor protein neurofibromin 1. Plexiform neurofibromas (PN) are histologically
benign nerve sheath tumours, which typically grow along large nerves and plexi.
On 5 March 2020, a centralised Marketing Authorisation Application was submitted to the
European Medicines Agency (EMA), Marketing Authorisation in EU was granted on 17 Jun
2021.
As part of the approval process, a Risk Management Plan (RMP) was developed and submitted
to the EMA to summarise the safety concerns emerging from the clinical development
program. The RMP included additional pharmacovigilance plans for a noninterventional
Post-authorisation Safety Study (PASS) to further characterise the safety of selumetinib
in paediatric patients with NF1-related PN in routine clinical practice.
The planned non-interventional PASS will address gaps in knowledge identified by the RMP,
including the important identified risk and some of the potential risks and missing
information on long-term developmental toxicity in children, by characterising the safety
profile associated with selumetinib use among paediatric patients (age d 8 to < 18 years
old) with a diagnosis of NF1 with symptomatic, inoperable PN.
This study is a specific obligation in the context of a conditional marketing
authorisation for selumetinib (ie, Category 2 PASS). Study results will contribute to
updating the safety profile of selumetinib in a relatively large population of patients
with different personal characteristics across multiple health care systems and patterns
of real-world clinical practice in European countries and Israel.
The study will enrol 2 cohorts:
1. The Base Cohort includes all enrolled patients aged 3 to < 18 years.
2. The Nested Prospective Cohort will include the subset of Base Cohort patients aged 8
to < 18 years who have not reached Tanner Stage V on the index date.
Etablissements
| Les établissements d'Île-de-France ayant mis à jour leurs données Origine et niveau de fiabilité des données | |||||
|---|---|---|---|---|---|
| CLCC INSTITUT CURIE | 09/01/2025 11:32:20 | Contact (sur clinicalTrials) | |||
| Les établissements sans correspondance certaine dans le répertoire FINESS dont les données sont issues de ClinicalTrials.gov Origine et niveau de fiabilité des données | |||||
| Research Site - Amiens - France | Contact (sur clinicalTrials) | ||||
| Research Site - Angers - France | Contact (sur clinicalTrials) | ||||
| Research Site - Bordeaux - France | Contact (sur clinicalTrials) | ||||
| Research Site - Lille - France | Contact (sur clinicalTrials) | ||||
| Research Site - Lyon - France | Contact (sur clinicalTrials) | ||||
| Research Site - Marseille - France | Contact (sur clinicalTrials) | ||||
| Research Site - Paris - France | Contact (sur clinicalTrials) | ||||
| Research Site - Rennes - France | Contact (sur clinicalTrials) | ||||
| Research Site - Strasbourg - France | Contact (sur clinicalTrials) | ||||
| Research Site - Toulouse - France | Contact (sur clinicalTrials) | ||||
| Research Site - Tours - France | Contact (sur clinicalTrials) | ||||
| Research Site - Villejuif Cedex - France | Contact (sur clinicalTrials) | ||||
Critères
Tous
Inclusion Criteria:
- Have been diagnosed with NF1 with symptomatic, inoperable PN
- Have initial treatment with selumetinib up to 6 months (i.e.182 days)prior to
enrolment into the study (i.e. signature of the ICF)
- Are aged 3 years and above, and are < 18 years of age on the index date
- Parent or legal guardian, as required by country-specific regulation, have provided
informed consent (unless a country-specific waiver is obtained) Additional Criteria
for Nested Prospective Cohort
- Are at least 8 years old and
- Are prior to attainment of Tanner Stage V on the index date
- Have been diagnosed with NF1 with symptomatic, inoperable PN
- Have initial treatment with selumetinib up to 6 months (i.e.182 days)prior to
enrolment into the study (i.e. signature of the ICF)
- Are aged 3 years and above, and are < 18 years of age on the index date
- Parent or legal guardian, as required by country-specific regulation, have provided
informed consent (unless a country-specific waiver is obtained) Additional Criteria
for Nested Prospective Cohort
- Are at least 8 years old and
- Are prior to attainment of Tanner Stage V on the index date
- Have received treatment with a mitogen-activated protein kinase inhibitor before the
index date
- Are participating in an interventional study at index date