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NCT Number: NCT03193892

A National Registry on Chinese Patients With Lymphangioleiomyomatosis

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.

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Key information

Conditions

Pulmonary Function Hemic and Lymphatic Diseases Immune System Diseases Immunoproliferative Disorders Lymphangioleiomyomatosis Lymphangiomyoma Lymphatic Diseases Lymphoproliferative Disorders Neoplasm, Lymphatic Tissue Neoplasms Neoplasms by Histologic Type Neoplasms, Connective and Soft Tissue Perivascular Epithelioid Cell Neoplasms

Sex eligibility

Female

Study type

Observational

Primary location

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

Location status: Recruiting

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Gender: female.
  • Age: no limitation.
  • 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

  • Suspected LAM patients without other supporting evidence for LAM diagnosis.
  • No diffuse cystic lesions in the lung.
  • Patients with bilateral cystic lung lesions but the LAM diagnosis cannot be established.
  • Without signed informed consent.
  • Difficult to follow up.

Treatment and study plan

Primary outcomes

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

    Time frame: 10 years

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

Secondary outcomes

  1. Change of diffusing capacity of the lung.

    Time frame: 10 years

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

  2. Annual incidence of major health outcomes: hemoptysis, pneumothorax, chylothorax, and spontaneous hemorrhage of kidney angiomyolipoma (AML).

    Time frame: 10 years

    The number of occurrences will be recorded through study completion, an average of 3 years.

  3. Other health outcomes: pregnancy, malignant tumors except LAM, lung transplantation, and death.

    Time frame: 10 year

    The number of occurrences will be recorded through study completion, an average of 3 years.

  4. The incidence of adverse drug reactions of long-time treatment with rapamycin.

    Time frame: 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.

Interested in participating?

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Interested in participating?

Request Info

Sponsors and collaborators

Lead sponsor

Peking Union Medical College Hospital

Other

Collaborators

  • 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

Registry information

Official study title

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

Important dates

Study start
2017
Primary completion
2026
Study completion
2026
First posted
Jun 21, 2017
Registry last updated
Sep 24, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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