A National Registry on Chinese Patients With Lymphangioleiomyomatosis

Recruiting

• Conditions: Pulmonary Function

• Registration Number: NCT03193892
• Lead Sponsor: Peking Union Medical College Hospital

Brief 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.

Detailed 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.

Study Timeline

• Study Start: 2017-01-01
• Study First Submitted: 2017-06-14
• Study First Submitted QC: 2017-06-18
• Study First Posted: 2017-06-21
• Status Verified: 2021-09
• Last Update Submitted: 2021-09-18
• Last Update Posted: 2021-09-24
• Primary Completion: 2026-10-01
• Study Completion: 2026-10-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Female
• Target Recruitment: 1500

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Change in spirometry (FEV1 and FVC) of lymphangioleiomyomatosis patients.	10 years	Spirometry will be evaluated at baseline and through study completion, an average of 3 years.

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Peking Union Medical College Hospital; 🇨🇳 Beijing, Beijing, China