Ignite Creation Date:
2024-05-06 @ 8:24 PM
Last Modification Date:
2024-10-26 @ 3:27 PM
Study NCT ID:
NCT06379230
Status:
RECRUITING
Last Update Posted:
2024-04-23
First Post:
2024-04-18
Brief Title:
A Retrospective Study on Epidemiological Characteristics of Chinese NF1 Patients in Real World PROMISE
Sponsor:
Shanghai Ninth Peoples Hospital Affiliated to Shanghai Jiao Tong University
Organization:
Shanghai Ninth Peoples Hospital Affiliated to Shanghai Jiao Tong University
Study Overview
Official Title:
A Retrospective Study on Epidemiological Characteristics of Chinese NF1 Patients in Real World PROMISE
Status:
RECRUITING
Status Verified Date:
2024-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
BackgroundRationale Neurofibromatosis type 1 NF1 affects about 1 in every 3000 people worldwide Globally 3050 NF1 patients will develop plexiform neurofibromas PNs which grow rapidly in early childhood and can cause disfigurement motor dysfunction pain airway dysfunction visual impairment and bladder and bowel dysfunction This systemic disease imposes a heavy psychosomatic and financial burden on patients and their caregivers In NF1 patients the lifetime risk of MPNST developed from PN is 8 to 13 The mean age for NF1-associated death was approximately 20 years lower than that for the general population Limited epidemiological and clinical data of Chinese NF1 patients is available to date And the treatment pattern of Chinese NF1-PN patients is also unknown
Objectives and Hypotheses It is a descriptive study without formal hypothesis The primary objective of this study is determining the percentage of NF1 patients who develop PN The secondary objectives of this study include describing the clinical characteristics tumor progression and treatment pattern of NF1-PN The exploratory objective is exploring the epidemiological characteristics of other NF1 manifestations
Methods
Study design The study is a retrospective multi-center chart review study Data Sources All the data will be collected by CRF from inpatient and outpatient electronic medical records in every study site from January 1 2019 to December 31 2022
Study Population Patients who attended the study sites between January 1 2019 - December 31 2022 and were diagnosed with NF1 were included in this study Statistical Analysis This study is purely descriptive without any formal hypotheses
Missing data for baseline characteristics will be assessed and addressed as a categorical variable with a level for missingness All reported measures will be summarized in the study tables Point estimates and their 95 CIs will be presented in the final analyses
Objectives and Hypotheses It is a descriptive study without formal hypothesis The primary objective of this study is determining the percentage of NF1 patients who develop PN The secondary objectives of this study include describing the clinical characteristics tumor progression and treatment pattern of NF1-PN The exploratory objective is exploring the epidemiological characteristics of other NF1 manifestations
Methods
Study design The study is a retrospective multi-center chart review study Data Sources All the data will be collected by CRF from inpatient and outpatient electronic medical records in every study site from January 1 2019 to December 31 2022
Study Population Patients who attended the study sites between January 1 2019 - December 31 2022 and were diagnosed with NF1 were included in this study Statistical Analysis This study is purely descriptive without any formal hypotheses
Missing data for baseline characteristics will be assessed and addressed as a categorical variable with a level for missingness All reported measures will be summarized in the study tables Point estimates and their 95 CIs will be presented in the final analyses
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None