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

NCT05891847 | Active Not Recruiting

• 1 other identifier: D1340R00002
• Study Type: observational
• Target: 150
• Locations: 1 country
• Sites: 5

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

Conditions

Neurofibroma

Keywords

NON-INTERVENTIONAL; Selumetinib

Outcome Measures

Primary Outcomes (5)

• 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 Outcomes (4)

• 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

  from the FSI date (21 Mar 2023) till 36 months from FSI

• Demographic profile of patients with PN NF1 in Russia

  from the FSI date (21 Mar 2023) till 36 months from FSI

• Clinical outcomes of selumetinib treatment

  From FSI date (21 Mar 2023) till 36 months from FSI

Eligibility Criteria

• Age: 3 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• 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

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• AstraZeneca: lead

Study Sites (5)

Research Site

Moscow, Moscow, 123112, Russia

Location

Research Site

Moscow, Russia

Location

Research Site

Novosibirsk, Russia

Location

Research Site

Saint Petersburg, Russia

Location

Research Site

Yekaterinburg, Russia

Location

Biospecimen

Retention: NONE RETAINED

n\\a, as all procedures will be performed as routine clinical practice

MeSH Terms

Conditions

Neurofibroma

Condition Hierarchy (Ancestors)

Nerve Sheath Neoplasms; Neoplasms, Nerve Tissue; Neoplasms by Histologic Type; Neoplasms; Peripheral Nervous System Neoplasms; Nervous System Neoplasms; Nervous System Diseases; Peripheral Nervous System Diseases; Neuromuscular Diseases

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 21, 2023
• First Posted: June 7, 2023
• Study Start: March 21, 2023
• Primary Completion: March 20, 2026
• Study Completion: March 20, 2026
• Last Updated: June 10, 2025

Record last verified: 2025-06

Locations

RU (5)