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Recruiting NCT07088991

Clinical and Demographic Characteristics of Adult p Atients With NEurofibromatosis in RUSsia

— Observational Neurofibromatosis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
This is an observational study: the protocol does not assign a study treatment.
Who it may be relevant to
Registry conditions: Neurofibromatosis. Basic parameters: No limits · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Russia
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

Clinical and Demographic Characteristics of Adult Patients With NEurofibromatosis in RUSsia

Overview

Clinical and Demographic Characteristics of Adult Patients with NEurofibromatosis in RUSsia (NEREUS)

Detailed description

Open-label single-arm, non-interventional, multi-center, cohort study for evaluation of clinical and patient reported outcomes in routine care settings

Primary outcome measures

  • Age (full years) at the inclusion into the study (date of Visit 1) [Time frame: Day 0 (Visit 1)]
  • Age (full years) at the primary NF1 diagnosis [Time frame: Day 0 (Visit 1)]
  • Proportion of men and women [Time frame: Day 0 (Visit 1)]
  • Body mass index (BMI), kg/m2; [Time frame: Day 0 (Visit 1)]
  • Body surface area (BSA), m2 [Time frame: Day 0 (Visit 1)]
  • Proportion of patients with each educational status (higher, secondary special, secondary general); [Time frame: Day 0 (Visit 1)]
  • Proportion of patients from each residential region (federal district of Russia); [Time frame: Day 0 (Visit 1)]
  • Other than PN specific NF1 symptoms and manifestations [Time frame: Day 0 (Visit 1)]
  • NF1-associated complications [Time frame: Day 0 (Visit 1)]
  • Non-specific NF1-comorbidities, coded by MedDRA system organ class (SOC) and preferred term (PT) [Time frame: Day 0 (Visit 1)]

Eligibility criteria

Inclusion criteria

  • Age ≥ 18 years at the time of inclusion.
  • Signed and dated written informed consent in accordance with ICH-GCP and local law prior to inclusion in the study.
  • NF1 diagnosed (according to the international consensus criteria for evaluating NF1 \[18\]), see Appendix A.
  • 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).
  • Existing of PN-associated symptoms.
  • 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

  • The participation in any clinical study currently (patients participating in other non-interventional studies may be included);
  • Patients with the evidence of a malignant glioma, malignant peripheral nerve sheath tumor, or other cancer, requiring treatment with chemotherapy or radiation therapy.
  • In the opinion of the investigator the patient is not able to return for follow-up visits or obtain required follow-up studies.
  • 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.
  • 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).
  • 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

Russia · 16 centers
  • Research Site — Arkhangelsk
  • Research Site — Chelyabinsk
  • Research Site — Grozny
  • Research Site — Krasnoyarsk
  • Research Site — Kurgan
  • Research Site — Moscow
  • Research Site — Nizhny Novgorod
  • Research Site — Novosibirsk
  • … and 8 more centers

Identifiers

NCT: NCT07088991 · D134BR00007

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗