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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05891847
Other study ID # D1340R00002
Secondary ID
Status Recruiting
Phase
First received
Last updated
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
Health authority
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.

Study Design


Related Conditions & MeSH terms


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
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