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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT06379230
Other study ID # SH9H-2023-T378-1
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date January 1, 2019
Est. completion date February 28, 2025

Study information

Verified date April 2024
Source Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong University
Contact Zhichao Wang
Phone +86 13816382311
Email shmuwzc@163.com
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

Background/Rationale: Neurofibromatosis type 1 (NF1) affects about 1 in every 3000 people worldwide. Globally, 30~50% 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 Source(s): 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.


Recruitment information / eligibility

Status Recruiting
Enrollment 2000
Est. completion date February 28, 2025
Est. primary completion date December 31, 2022
Accepts healthy volunteers No
Gender All
Age group N/A and older
Eligibility Inclusion Criteria: 1. Patients who attended the study sites between January 1, 2019, and December 31, 2022. 2. Patients who were diagnosed with NF1(recorded with the text of type I neurofibromatosis) based on National Institutes of Health (NIH) NF1 consensus. Exclusion Criteria: 1. Patients combined with other malignant tumors

Study Design


Intervention

Other:
This study is purely descriptive study.
This study is purely descriptive study.

Locations

Country Name City State
China Xuanwu Hospital, Capital Medical University Beijing Beijing
China West China Hospital, Sichuan University Chengdu Sichuan
China The First Affiliated Hospital of Guangxi Medical University Nanning Guangxi
China Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine Shanghai Shanghai
China The First Affiliated Hospital of Zhengzhou University Zhengzhou Henan

Sponsors (1)

Lead Sponsor Collaborator
Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong University

Country where clinical trial is conducted

China, 

Outcome

Type Measure Description Time frame Safety issue
Other The exploratory endpoints: Epidemiological characteristics of other NF1 manifestations:
Describe the percentage of NF1 patients with cNF at the baseline.
2022.12.31
Primary Determine the percentage of NF1 patients developed PN at the baseline. The clinical diagnosis of PN mainly depends on the clinical manifestations and imaging of NF1 patients. Superficial PN has a clear clinical manifestation. It refers to a proliferation of cells in the nerve sheath which involves multiple nerve fascicles, forming a large pendulous mass with skin pigmentation on the surface. PN in vivo often has no significant clinical features and requires imaging examination, like MRI. Patients diagnosed with PN are recorded with the text of plexiform neurofibromas in the chart.
The percentage of patients diagnosed with PN with imaging and the percentage of patients diagnosed with PN without imaging will be calculated.
2022.12.31
Secondary Clinical characteristics of NF1-PN: Describe the percentage of symptomatic NF1-PN at the baseline. 2022.12.31
Secondary Tumor progression of NF1-PN Describe the 1-year/2-year/3-year progression-free survival (PFS) rate of NF1- PN. 2022.12.31
Secondary Treatment pattern of NF1-PN: Describe the percentage of NF1-PN patients underwent surgeries after index date. 2022.12.31
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