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

Status: Recruiting
Location: See all (45) locations...
Study Type: Observational
SUMMARY

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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 3
Maximum Age: 17
Healthy Volunteers: f
View:

• 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

Locations
Other Locations
Austria
Research Site
RECRUITING
Vienna
France
Research Site
RECRUITING
Amiens
Research Site
RECRUITING
Angers
Research Site
RECRUITING
Bordeaux
Research Site
RECRUITING
Lille
Research Site
RECRUITING
Lyon
Research Site
RECRUITING
Marseille
Research Site
RECRUITING
Paris
Research Site
RECRUITING
Rennes
Research Site
RECRUITING
Strasbourg
Research Site
RECRUITING
Toulouse
Research Site
RECRUITING
Tours
Research Site
TERMINATED
Villejuif
Germany
Research Site
RECRUITING
Dresden
Research Site
RECRUITING
Duisburg
Research Site
RECRUITING
Hamburg
Research Site
RECRUITING
München
Research Site
RECRUITING
Tübingen
Israel
Research Site
RECRUITING
Petah Tikva
Research Site
RECRUITING
Ramat Gan
Research Site
RECRUITING
Tel Aviv
Italy
Research Site
RECRUITING
Florence
Research Site
RECRUITING
Genova
Research Site
RECRUITING
Milan
Research Site
RECRUITING
Padua
Research Site
RECRUITING
Pavia
Research Site
RECRUITING
Roma
Research Site
RECRUITING
Torino
Research Site
RECRUITING
Trieste
Netherlands
Research Site
RECRUITING
Rotterdam
Portugal
Research Site
RECRUITING
Lisbon
Research Site
TERMINATED
Porto
Spain
Research Site
RECRUITING
Barcelona
Research Site
RECRUITING
Esplugues De Llobregat
Research Site
RECRUITING
Madrid
Research Site
RECRUITING
Málaga
Research Site
RECRUITING
Santiago De Compostela
Research Site
RECRUITING
Seville
Switzerland
Research Site
RECRUITING
Basel
Research Site
RECRUITING
Bern
Research Site
RECRUITING
Lausanne
Research Site
RECRUITING
Sankt Gallen
United Kingdom
Research Site
RECRUITING
London
Research Site
RECRUITING
Manchester
Research Site
RECRUITING
Newcastle
Contact Information
Primary
AstraZeneca Clinical Study Information Center
information.center@astrazeneca.com
1-877-240-9479
Time Frame
Start Date: 2022-05-23
Estimated Completion Date: 2028-05-23
Participants
Target number of participants: 125
Treatments
Base Cohort
The Base Cohort includes all enrolled patients aged 3 to \< 18 years.
Nested Prospective Cohort
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
Sponsors
Leads: AstraZeneca

This content was sourced from clinicaltrials.gov