Clinical and demographic characteristics of adults with neurofibromatosis in Russia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is an open-label, single-arm, non-interventional multi-center cohort collecting clinical and patient-reported outcomes during routine care for adults with NF1 and confirmed plexiform neurofibromas in Russia. Eligible participants are adults (≥18) with NF1 and symptomatic PN who are naïve to MEK-inhibitor therapy; exclusions include current participation in other interventional trials and active malignant tumors requiring chemotherapy. Data will be gathered at participating research sites in Arkhangelsk, Chelyabinsk, and Krasnoyarsk and compiled to describe demographics, clinical features, and symptom burden. The study is sponsored by AstraZeneca and aims to document real-world patterns of care and outcomes rather than test an experimental treatment.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Age ≥ 18 years at the time of inclusion.
2. Signed and dated written informed consent in accordance with ICH-GCP and local law prior to inclusion in the study.
3. NF1 diagnosed (according to the international consensus criteria for evaluating NF1 \[18\]), see Appendix A.
4. Confirmed PN by clinical assessment, USI, MRI, biopsy; one method is sufficient (histologic confirmation of PN is not necessary in the presence of radiographic findings).
5. Existing of PN-associated symptoms.
6. Adult patients (≥18 years) with newly diagnosed PN or established PN naïve to MEK-inhibitor therapy verified by medical records/histories (e.g., prior prescriptions, hospitalization data).

Exclusion Criteria:

1. The participation in any clinical study currently (patients participating in other non-interventional studies may be included);
2. Patients with the evidence of a malignant glioma, malignant peripheral nerve sheath tumor, or other cancer, requiring treatment with chemotherapy or radiation therapy.
3. In the opinion of the investigator the patient is not able to return for follow-up visits or obtain required follow-up studies.
4. Individuals who are pregnant or breast feeding or who become pregnant while enrolled on this trial, if they are unable to undergo radiographic evaluations or MRI scans requested for research purposes, or other studies which might negatively impact on the pregnancy.
5. Prior receipt of any MEK-inhibitor for PN therapy within 4 months before screening or initiation of therapy prior to age 18 verified by medical records/histories (e.g., prior prescriptions, hospitalization data).
6. Patients who modify their index pathogenetic therapy regimen during the study (for example, pathogenetic antitumor therapy of PN; switching to a different therapy), as determined by the investigator at any protocol-specified visit during the a study, will be excluded from the primary efficacy analysis population from the point of modification onward and will not continue study participation.

Where it's running

• Research Site
  Arkhangelsk, Russia Recruiting
• Research Site
  Chelyabinsk, Russia Recruiting
• Research Site
  Grozny, Russia Recruiting
• Research Site
  Krasnoyarsk, Russia Recruiting
• Research Site
  Kurgan, Russia Recruiting
• Research Site
  Moscow, Russia Recruiting

Study contacts

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

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.