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

Recruiting

• Conditions: Neurofibromatosis

• Registration Number: NCT07088991
• Lead Sponsor: AstraZeneca

Brief Summary

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

Study Timeline

• Study Start: 2025-06-06
• Status Verified: 2025-07
• Study First Submitted: 2025-07-03
• Study First Submitted QC: 2025-07-25
• Last Update Submitted: 2025-07-25
• Study First Posted: 2025-07-28
• Last Update Posted: 2025-07-28
• Primary Completion: 2028-03-31
• Study Completion: 2028-03-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 200

Inclusion Criteria

Exclusion Criteria

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Age (full years) at the inclusion into the study (date of Visit 1)	Day 0 (Visit 1)	To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.
Age (full years) at the primary NF1 diagnosis	Day 0 (Visit 1)	To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.
Proportion of men and women	Day 0 (Visit 1)	To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.
Body mass index (BMI), kg/m2;	Day 0 (Visit 1)	To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.
Proportion of patients with each educational status (higher, secondary special, secondary general);	Day 0 (Visit 1)	To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.
Proportion of patients from each residential region (federal district of Russia);	Day 0 (Visit 1)	To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.
Other than PN specific NF1 symptoms and manifestations	Day 0 (Visit 1)	skin symptoms and manifestations, tumor manifestations (except malignancies), neurological and cognitive manifestations, cardiovascular manifestations, endocrine manifestations, orthopedic manifestations (distinctive osseous lesions), ophthalmological manifestations, NF1 mutation, presence of NF1 diagnostic criteria in one of parents) (frequency distribution at baseline)
NF1-associated complications	Day 0 (Visit 1)	malignancy, hypertension, vasculopathy, bone symptoms, chronic neurological conditions, neuropathy, and pain
Body surface area (BSA), m2	Day 0 (Visit 1)	To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.
Non-specific NF1-comorbidities, coded by MedDRA system organ class (SOC) and preferred term (PT)	Day 0 (Visit 1)	To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.
Proportion of patients with various specific and non-specific NF1-comorbidities	Day 0 (Visit 1)	To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.
Amount and duration of hospitalizations associated with NF1 according to the investigator's opinion, collected from the medical history since the date of NF1 diagnosis	Day 0 (Visit 1)	if applicable, to be calculated in patients with at least one such hospitalization
Degree of disability at baseline	Day 0 (Visit 1)	if applicable, to be calculated in patients with presence of disability at Visit 1
Changing in disability degree during the study	Day 0 (Visit 1)	if applicable, to be calculated in patients with presence of disability at Visit 1
Proportion of patients with various PN-associated symptoms	Day 0 (Visit 1)	To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.
Proportion of patients with various ECOG score	Day 0 (Visit 1)	To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.
Duration of PN diagnosis before inclusion into the study	Day 0 (Visit 1)	to be calculated between the date of primary diagnosis of PN and the date of Visit 1
Proportion of patients with various PN locations	Day 0 (Visit 1)	To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.
PN volume (established by MRI)	Day 0 (Visit 1)	To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.
Duration of PN symptoms persistence before inclusion into the study	Day 0 (Visit 1)	to be calculated between the date when initially symptoms of PN appeared and the date of Visit 1
Previous physical, instrumental and laboratory examination results	Day 0 (Visit 1)	To achieve the primary objectives of the study the following baseline clinical and demographic characteristics of patients will be collected or evaluated. The term 'baseline' means that the data is obtained at the inclusion into the study (Visit 1) during retrospective data collection from patient medical documentation or via interview with a patient on Visit 1, where applicable.
Medical history of PN	Day 0 (Visit 1)	size, symptoms, previous medical interventions, surgeries or treatments

Trial Locations

Locations (1)

Research site; 🇷🇺 Moscow, Russian Federation