Clinical Trials Logo
  1. Home
  2. Neurofibromatosis 1
  3. NCT06379230

A Retrospective Study on Epidemiological Characteristics of Chinese NF1 Patients in Real World (PROMISE)

Neurofibromatosis 1 Clinical Trial

Official title:
A Retrospective Study on Epidemiological Characteristics of Chinese NF1 Patients in Real World (PROMISE)

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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT06379230
Study type Observational
Source Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong University
Contact Zhichao Wang
Phone +86 13816382311
Email shmuwzc@163.com
Status Recruiting
Phase
Start date January 1, 2019
Completion date February 28, 2025
View Details
See also
  Status Clinical Trial Phase
Active, not recruiting NCT01362803 - AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors Phase 1/Phase 2
Completed NCT00352599 - Trial to Evaluate the Safety of Lovastatin in Individuals With Neurofibromatosis Type I (NF1) Phase 1
Completed NCT05005845 - NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF) Phase 2
Completed NCT03531814 - Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1(NF1) on Clinical Treatment Trials N/A
Completed NCT00846430 - Medical Treatment of "High-Risk" Neurofibromas Phase 2
Completed NCT03433183 - SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors Phase 2
Recruiting NCT05331105 - HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas Phase 2
Completed NCT03310996 - Non-invasive Stimulation in Neurofibromatosis Type 1 N/A
Recruiting NCT03406208 - Resiliency Training for Adults With Neurofibromatosis Via Live Videoconferencing N/A
Not yet recruiting NCT05849662 - A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia Phase 1/Phase 2
Recruiting NCT04750928 - Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas Phase 1/Phase 2
Not yet recruiting NCT06262113 - A Decentralized Clinical Trial to Promote Evidence-Based Care for Underserved Patients With Neurofibromatosis 1 N/A
Active, not recruiting NCT04924608 - Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas Phase 3
Recruiting NCT02544022 - Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)
Not yet recruiting NCT06222203 - Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)
Recruiting NCT04941027 - Evaluating Genetic Modifiers of Cutaneous Neurofibromas in Adults With Neurofibromatosis Type 1
Completed NCT02153931 - Internet Support Group for Parents of a Child With Neurofibromatosis Type 1
Completed NCT05196854 - Clinical Hypnosis and Home Blood Pressure Monitoring in Children With Neurofibromatosis Type 1 N/A
Active, not recruiting NCT01650142 - Modifying Genes in Neurofibromatosis 1 N/A
Completed NCT05377008 - Intervention Effectiveness on the Neurocognitive Functioning of Children and Adolescents With Neurofibromatosis Type 1 N/A