A National Registry on Chinese Patients With Lymphangioleiomyomatosis

N/ARecruitingNCT03193892

Peking Union Medical College Hospital1,500 enrolled

Overview

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 Type

OBSERVATIONAL

Enrollment

1,500

Conditions

Pulmonary Function

Eligibility

Sex: FEMALE

Medical Language ↔ Plain English

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.

Locations (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, China

RECRUITING

Outcomes

Primary Outcomes

Change in spirometry (FEV1 and FVC) of lymphangioleiomyomatosis patients.

Spirometry will be evaluated at baseline and through study completion, an average of 3 years.

Time frame: 10 years

Secondary Outcomes

Change of diffusing capacity of the lung.

Pulmonary function tests will be evaluated at baseline and through study completion, an average of 3 years.

Time frame: 10 years

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.

Time frame: 10 years

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.

Time frame: 10 year

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.

Time frame: 10 years

Central Contacts

Kai-Feng Xu, M.D.

CONTACT

86-10-69155039 xukf@pumch.cn

Data from ClinicalTrials.gov

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