Clinical and Demographic Characteristics of Adult Patients with NEurofibromatosis in RUSsia (NEREUS)
Open-label single-arm, non-interventional, multi-center, cohort study for evaluation of clinical and patient reported outcomes in routine care settings
Study Type
OBSERVATIONAL
Enrollment
200
Research Site
Arkhangelsk, Russia
RECRUITINGResearch Site
Chelyabinsk, Russia
RECRUITINGResearch Site
Age (full years) at the inclusion into the study (date of 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.
Time frame: Day 0 (Visit 1)
Age (full years) at the primary NF1 diagnosis
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.
Time frame: Day 0 (Visit 1)
Proportion of men and women
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.
Time frame: Day 0 (Visit 1)
Body mass index (BMI), kg/m2;
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.
Time frame: Day 0 (Visit 1)
Body surface area (BSA), m2
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.
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Krasnoyarsk, Russia
Research Site
Kurgan, Russia
RECRUITINGResearch Site
Moscow, Russia
RECRUITINGResearch Site
Nizhny Novgorod, Russia
RECRUITINGResearch Site
Novosibirsk, Russia
RECRUITINGResearch Site
Orenburg, Russia
RECRUITINGResearch Site
Saint-Peretsburg, Russia
RECRUITINGResearch Site
Samara, Russia
RECRUITING...and 3 more locations
Time frame: Day 0 (Visit 1)
Proportion of patients with each educational status (higher, secondary special, secondary general);
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.
Time frame: Day 0 (Visit 1)
Proportion of patients from each residential region (federal district of Russia);
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.
Time frame: Day 0 (Visit 1)
Other than PN specific NF1 symptoms and manifestations
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)
Time frame: Day 0 (Visit 1)
NF1-associated complications
malignancy, hypertension, vasculopathy, bone symptoms, chronic neurological conditions, neuropathy, and pain
Time frame: Day 0 (Visit 1)
Non-specific NF1-comorbidities, coded by MedDRA system organ class (SOC) and preferred term (PT)
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.
Time frame: Day 0 (Visit 1)
Proportion of patients with various specific and non-specific NF1-comorbidities
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.
Time frame: Day 0 (Visit 1)
Proportion of patients with various PN-associated symptoms
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.
Time frame: Day 0 (Visit 1)
Proportion of patients with various ECOG score
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.
Time frame: Day 0 (Visit 1)
Proportion of patients with various PN locations
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.
Time frame: Day 0 (Visit 1)
PN volume (established by MRI)
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.
Time frame: Day 0 (Visit 1)
Duration of PN symptoms persistence before inclusion into the study
to be calculated between the date when initially symptoms of PN appeared and the date of Visit 1
Time frame: Day 0 (Visit 1)
Duration of PN diagnosis before inclusion into the study
to be calculated between the date of primary diagnosis of PN and the date of Visit 1
Time frame: Day 0 (Visit 1)
Medical history of PN
size, symptoms, previous medical interventions, surgeries or treatments
Time frame: Day 0 (Visit 1)
Previous physical, instrumental and laboratory examination results
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.
Time frame: Day 0 (Visit 1)
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
if applicable, to be calculated in patients with at least one such hospitalization
Time frame: Day 0 (Visit 1)
Degree of disability at baseline
if applicable, to be calculated in patients with presence of disability at Visit 1
Time frame: Day 0 (Visit 1)
Changing in disability degree during the study
if applicable, to be calculated in patients with presence of disability at Visit 1
Time frame: Day 0 (Visit 1)