Non-interventional Study of Patients With PN NF1 Starting Selumetinib in Russia

Non-interventional Study of Patients With Plexiform Neurofibromas and Neurofibromatosis Type I Starting Selumetinib in Russia

The purpose of this study is to evaluate the effectiveness, safety of selumetinib and patient's parameters of symptomatic inoperable PN NF1 in real clinical practice in Russia

Study Overview

• Status: Recruiting
• Conditions: Neurofibroma

Detailed Description

This study will be non-interventional retrospective and prospective study. Assessment of parameters will be carried out as if a patient is treated in real-life clinical setting. The patients should be enrolled into this project after evaluation of eligibility criteria by an investigator 10 clinical sites. No additional procedures besides those already used in the routine clinical practice will be applied to the patients. Study population will consist of pediatric patients aged 3 years and older with NF1 and symptomatic inoperable PN who were prescribed with selumetinib

• Study Type: Observational
• Enrollment (Estimated): 150

Contacts and Locations

• Study Contact: Name: AstraZeneca Clinical Study Information Center; Phone Number: 1-877-240-9479; Email: information.center@astrazeneca.com

Study Locations

• Russian Federation
  • Ekaterinburg, Russian Federation
    • Not yet recruiting
    • State Autonomous Healthcare Institution of the Sverdlovsk Region Regional Children Clinical Hospital
    • Principal Investigator: Olga Lvova, Dr
  • Kazan, Russian Federation
    • Not yet recruiting
    • Children Republican Clinical Hospital of the Ministry of Health of the Republic of Tatarstan
    • Principal Investigator: Ilsia Osipova, Dr
  • Novosibirsk, Russian Federation
    • Not yet recruiting
    • Novosibirsk State Medical University
    • Principal Investigator: Yulia Maximova, Dr
  • Saint Petersburg, Russian Federation
    • Not yet recruiting
    • Almazov National Medical Research Centre
    • Principal Investigator: Yulia Dinikina, Dr
  • Saint Petersburg, Russian Federation
    • Not yet recruiting
    • Raisa Gorbacheva Memorial Institute of Children Hematology and Transplantation
    • Principal Investigator: Lyudmila Zubarovskaya, Dr
  • Saint Petersburg, Russian Federation
    • Recruiting
    • St. Petersburg Clinical Scientific and Practical Center of Specialized Types of Medical Care
    • Principal Investigator: Margarita Belogurova, Dr
  • Moskva
    • Moscow, Moskva, Russian Federation, 123112
      • Not yet recruiting
      • GBUZ Morozovskaya Children City Clinical Hospital DZM
      • Principal Investigator: Ella Kumirova, Dr
    • Moscow, Moskva, Russian Federation, 123112
      • Recruiting
      • GUZ MO Children Neuropsychiatric Hospital
      • Principal Investigator: Oleg Lapochkin, Dr
    • Moscow, Moskva, Russian Federation, 123112
      • Not yet recruiting
      • Scientific institute of pediatrics n.a. Veltishchev
      • Principal Investigator: Marina Dorofeeva, Dr
    • Moscow, Moskva, Russian Federation, 123112
      • Not yet recruiting
      • SMIC of children hematology, oncology and immunology n.a. D.Rogachev
      • Principal Investigator: Anastasia Solomatina, Dr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Study population will consist of pediatric patients aged 3 years and older with NF1 and symptomatic inoperable PN who were prescribed with selumetinib

Description

Inclusion Criteria:

• NF1 diagnosed (according to the criteria for evaluating NF1),
• Symptomatic inoperable PN,
• Patients receiving therapy with selumetinib. For newly treated patients - registration on the first day of starting treatment with selumetinib,
• Age at starting of selumetinib treatment ≥ 3 to ≤ 18 years old (included in the study or started the therapy at the age of 18 years old),
• Patients whose parents or guardians have signed a consent form.

Exclusion Criteria:

• Patients currently participating in an interventional study/clinical trial,
• Patients are excluded if there is evidence of an optic glioma, malignant glioma, malignant peripheral nerve sheath tumor, or other cancer, requiring treatment with chemotherapy or radiation therapy,
• Patients who receive other antitumor treatment options on optic nerve glioma, malignant glioma, malignant tumor of the peripheral nerve sheath, or other cancer. If the patient on selumetinib treatment develop optic nerve glioma, malignant glioma, malignant tumor of the peripheral nerve sheath, or other cancer, he can continue selumetinib therapy +\- other treatment options on physician decision,
• Female patients above 12 age who are pregnant /plan to become pregnant or at breast feeding.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
ORR in patients receiving selumetinib according to REiNS criteria	ORR (Overall response rate) in patients receiving selumetinib according to REiNS criteria: ORR is defined as the number of patients with CR (complete response) or cPR (confirmed partial response). CR is defined as disappearance of the target lesion. PR is defined as decrease in the volume of the target PN by 20% or more compared to the baseline and is considered unconfirmed at the first detection, confirmed when observed again within 3-6 months, and durable when the response is maintained for 1 year or longer In this trial ORR could be taken from the source records of the patient retrospectively. In this case CR or cPR counted from the therapy start date of a patient, which could have been before ICF signing.	from the FSI date (21 Mar 2023) till 36 months from FSI
Best objective response on selumetinib treatment	a percentage of subjects who achieved a best response of CR, PR or stable disease during treatment	from the FSI date (21 Mar 2023) till 36 months from FSI
Time to discontinuation of selumetinib		from the FSI date (21 Mar 2023) till 36 months from FSI
Description of diagnostic algorithms for PN NF1 in Russia		from the FSI date (21 Mar 2023) till 36 months from FSI
Reasons for discontinuation of selumetinib		from the FSI date (21 Mar 2023) till 36 months from FSI

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety: adverse events (date, type, severe grade, seriousness criteria, outcome);		from the FSI date (21 Mar 2023) till 36 months from FSI
Clinical profile of patients with PN NF1 in Russia	features of molecular diagnostics, lesion volume and localization of PN (plexiform neurofibroma)	from the FSI date (21 Mar 2023) till 36 months from FSI
Demographic profile of patients with PN NF1 in Russia	age, gender of patients	from the FSI date (21 Mar 2023) till 36 months from FSI

Collaborators and Investigators

• Sponsor: AstraZeneca

Study record dates

Study Major Dates

• Study Start (Actual): March 21, 2023
• Primary Completion (Estimated): March 20, 2026
• Study Completion (Estimated): March 20, 2026

Study Registration Dates

• First Submitted: March 21, 2023
• First Submitted That Met QC Criteria: June 4, 2023
• First Posted (Actual): June 7, 2023

Study Record Updates

• Last Update Posted (Actual): March 22, 2024
• Last Update Submitted That Met QC Criteria: March 20, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: Selumetinib; NON-INTERVENTIONAL
• Additional Relevant MeSH Terms: Nervous System Diseases; Neoplasms by Histologic Type; Neoplasms; Genetic Diseases, Inborn; Neuromuscular Diseases; Neurodegenerative Diseases; Neoplasms, Nerve Tissue; Peripheral Nervous System Diseases; Nervous System Neoplasms; Heredodegenerative Disorders, Nervous System; Neoplastic Syndromes, Hereditary; Nerve Sheath Neoplasms; Neurocutaneous Syndromes; Peripheral Nervous System Neoplasms; Neurofibromatoses; Neurofibroma
• Other Study ID Numbers: D1340R00002

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No