Clinical trial NCT05891847: Non-interventional Study of Patients With PN NF1 Starting Selumetinib in Russia
Official title: Non-interventional Study of Patients With Plexiform Neurofibromas and Neurofibromatosis Type I Starting Selumetinib in Russia
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Neurofibroma
Keywords: NON-INTERVENTIONAL
Selumetinib
Lead sponsor: AstraZeneca
Sponsor class: INDUSTRY
First posted: 2023-06-07
Enrollment: 150
Sex: ALL
Minimum age (years): 3
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: CASE_CONTROL
Time perspective: OTHER
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

Brief summary: 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

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

Eligibility criteria: 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.

Primary outcomes:
measure: ORR in patients receiving selumetinib according to REiNS criteria
timeFrame: from the FSI date (21 Mar 2023) till 36 months from FSI
measure: Best objective response on selumetinib treatment
timeFrame: from the FSI date (21 Mar 2023) till 36 months from FSI
measure: Time to discontinuation of selumetinib
timeFrame: from the FSI date (21 Mar 2023) till 36 months from FSI
measure: Description of diagnostic algorithms for PN NF1 in Russia
timeFrame: from the FSI date (21 Mar 2023) till 36 months from FSI
measure: Reasons for discontinuation of selumetinib
timeFrame: from the FSI date (21 Mar 2023) till 36 months from FSI
Secondary outcomes:
measure: Safety: adverse events (date, type, severe grade, seriousness criteria, outcome);
timeFrame: from the FSI date (21 Mar 2023) till 36 months from FSI
measure: Clinical profile of patients with PN NF1 in Russia
timeFrame: from the FSI date (21 Mar 2023) till 36 months from FSI
measure: Demographic profile of patients with PN NF1 in Russia
timeFrame: from the FSI date (21 Mar 2023) till 36 months from FSI
measure: Clinical outcomes of selumetinib treatment
timeFrame: From FSI date (21 Mar 2023) till 36 months from FSI
Other outcomes:
Locations:
city: Moscow
country: Russian Federation
facility: Research Site
state: Moskva
city: Ekaterinburg
country: Russian Federation
facility: Research Site
city: Moscow
country: Russian Federation
facility: Research Site
city: Novosibirsk
country: Russian Federation
facility: Research Site
city: Saint-Petersburg
country: Russian Federation
facility: Research Site
Study officials:
References: