Status:
RECRUITING
PASS of Paediatric Patients Initiating Selumetinib
Lead Sponsor:
AstraZeneca
Conditions:
Neurofibromatosis Type 1
Eligibility:
All Genders
3-17 years
Brief 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 neurof...
Detailed Description
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 neurof...
Eligibility Criteria
Inclusion
- 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
Exclusion
- Have received treatment with a mitogen-activated protein kinase inhibitor before the index date
- Are participating in an interventional study at index date
Key Trial Info
Start Date :
May 23 2022
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
May 23 2028
Estimated Enrollment :
125 Patients enrolled
Trial Details
Trial ID
NCT05388370
Start Date
May 23 2022
End Date
May 23 2028
Last Update
June 3 2025
Active Locations (45)
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1
Research Site
Vienna, Austria
2
Research Site
Amiens, France
3
Research Site
Angers, France
4
Research Site
Bordeaux, France