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

Neurofibroma Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05891847
• Other study ID #: D1340R00002
• Status: Recruiting
• Start date: March 21, 2023
• Est. completion date: March 20, 2026

Study information

• Verified date: May 2024
• Source: AstraZeneca
• Contact: AstraZeneca Clinical Study Information Center
• Phone: 1-877-240-9479
• Email: information.center[@]astrazeneca.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 150
• Est. completion date: March 20, 2026
• Est. primary completion date: March 20, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Neurofibroma
• Neurofibromatoses

Locations

Country	Name	City	State
Russian Federation	State Autonomous Healthcare Institution of the Sverdlovsk Region Regional Children Clinical Hospital	Ekaterinburg
Russian Federation	Children Republican Clinical Hospital of the Ministry of Health of the Republic of Tatarstan	Kazan
Russian Federation	GBUZ Morozovskaya Children City Clinical Hospital DZM	Moscow	Moskva
Russian Federation	GUZ MO Children Neuropsychiatric Hospital	Moscow	Moskva
Russian Federation	Scientific institute of pediatrics n.a. Veltishchev	Moscow	Moskva
Russian Federation	SMIC of children hematology, oncology and immunology n.a. D.Rogachev	Moscow	Moskva
Russian Federation	Novosibirsk State Medical University	Novosibirsk
Russian Federation	Almazov National Medical Research Centre	Saint Petersburg
Russian Federation	Raisa Gorbacheva Memorial Institute of Children Hematology and Transplantation	Saint Petersburg
Russian Federation	St. Petersburg Clinical Scientific and Practical Center of Specialized Types of Medical Care	Saint Petersburg

Sponsors (1)

Lead Sponsor	Collaborator
AstraZeneca

Country where clinical trial is conducted

Russian Federation, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	Time to discontinuation of selumetinib		from the FSI date (21 Mar 2023) till 36 months from FSI
Primary	Description of diagnostic algorithms for PN NF1 in Russia		from the FSI date (21 Mar 2023) till 36 months from FSI
Primary	Reasons for discontinuation of selumetinib		from the FSI date (21 Mar 2023) till 36 months from FSI
Secondary	Safety: adverse events (date, type, severe grade, seriousness criteria, outcome);		from the FSI date (21 Mar 2023) till 36 months from FSI
Secondary	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
Secondary	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