A National Registry on Chinese Patients With Lymphangioleiomyomatosis

Pulmonary Function Clinical Trial

Official title:

A National Registry on Clinical Manifestations, Genetics, Interventions, and Outcomes in Chinese Patients With Lymphangioleiomyomatosis (LAM-CHINA)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03193892
• Other study ID #: 2016YFC091502LAM
• Status: Recruiting
• Start date: January 1, 2017
• Est. completion date: October 1, 2026

Study information

• Verified date: September 2021
• Source: Peking Union Medical College Hospital
• Contact: Kai-Feng Xu, M.D.
• Phone: 86-10-69155039
• Email: xukf[@]pumch.cn
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

Pulmonary lymphangioleiomyomatosis (LAM), a disease characterized by diffuse cystic changes in the lung, is a rare disorder that affects almost exclusively women. The main objectives of this study are to accurately evaluate the prevalence of LAM, the status of disease, the diagnosis and treatment, the quality of care, and the health related outcomes in China.

Description:

Pulmonary lymphangioleiomyomatosis (LAM), a disease characterized by diffuse cystic changes in the lung, is a rare disorder that affects almost exclusively women. The main objectives of this study are to accurately evaluate the prevalence of LAM, the status of disease, the diagnosis and treatment, the quality of care, and the health related outcomes in China. This is a register study lasting 4 years, aims to raise 800 subjects. Primary endpoint is the annual change of forced expiratory volume in the first second (FEV1) and forced vital capacity (FVC) in pulmonary function tests.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 1500
• Est. completion date: October 1, 2026
• Est. primary completion date: October 1, 2026
• Accepts healthy volunteers: No
• Gender: Female
• Age group: N/A and older

Eligibility

Inclusion Criteria:

1. Gender: female.

2. Age: no limitation.

3. Diagnosis meets one of the following criteria, (1) definite or probable diagnosis of LAM based on ATS/JRS and ERS criteria. (2) Investigators recommend including of the patient.

Exclusion Criteria:

1. Suspected LAM patients without other supporting evidence for LAM diagnosis.

2. No diffuse cystic lesions in the lung.

3. Patients with bilateral cystic lung lesions but the LAM diagnosis cannot be established.

4. Without signed informed consent.

5. Difficult to follow up.

Related Conditions & MeSH terms

• Lymphangioleiomyomatosis
• Pulmonary Function

Locations

Country	Name	City	State
China	Peking Union Medical College Hospital	Beijing	Beijing

Sponsors (12)

Lead Sponsor

Collaborator

Peking Union Medical College Hospital

Anhui Provincial Hospital, Beijing Shijitan Hospital, Capital Medical University, Central South University, Fujian Provincial Hospital, Guangzhou Institute of Respiratory Disease, Shanghai Zhongshan Hospital, The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School, West China Hospital, Wuhan TongJi Hospital, Wuxi People's Hospital, Xiangya Hospital of Central South University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in spirometry (FEV1 and FVC) of lymphangioleiomyomatosis patients.	Spirometry will be evaluated at baseline and through study completion, an average of 3 years.	10 years
Secondary	Change of diffusing capacity of the lung.	Pulmonary function tests will be evaluated at baseline and through study completion, an average of 3 years.	10 years
Secondary	Annual incidence of major health outcomes: hemoptysis, pneumothorax, chylothorax, and spontaneous hemorrhage of kidney angiomyolipoma (AML).	The number of occurrences will be recorded through study completion, an average of 3 years.	10 years
Secondary	Other health outcomes: pregnancy, malignant tumors except LAM, lung transplantation, and death.	The number of occurrences will be recorded through study completion, an average of 3 years.	10 year
Secondary	The incidence of adverse drug reactions of long-time treatment with rapamycin.	Common Terminology Criteria for Adverse Events (CTCAE) will be used to evaluate adverse drug reactions through study completion, an average of 3 years.	10 years