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Recruiting NCT03193892

A National Registry on Chinese Patients With Lymphangioleiomyomatosis

— Observational Pulmonary Function

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
This is an observational study: the protocol does not assign a study treatment.
Who it may be relevant to
Registry conditions: Pulmonary Function. Basic parameters: No limits · Female.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

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.

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.

Primary outcome measures

  • Change in spirometry (FEV1 and FVC) of lymphangioleiomyomatosis patients. [Time frame: 10 years]
Secondary outcome measures (4)
  • Change of diffusing capacity of the lung. [Time frame: 10 years]
  • Annual incidence of major health outcomes: hemoptysis, pneumothorax, chylothorax, and spontaneous hemorrhage of kidney angiomyolipoma (AML). [Time frame: 10 years]
  • Other health outcomes: pregnancy, malignant tumors except LAM, lung transplantation, and death. [Time frame: 10 year]
  • The incidence of adverse drug reactions of long-time treatment with rapamycin. [Time frame: 10 years]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

China · 1 center
  • Peking Union Medical College Hospital — Beijing

Publications

  • Wang YY, Zou LP, Xu KF, Xu WS, Zhang MN, Lu Q, Tian XL, Pang LY, He W, Wang QH, Gao Y, Liu LY, Chen XQ, Ma SF, Chen HM, Dun S, Yang XY, Luo XM, Huang LL, Li YF. Long-term safety and influence on growth in patients receiving sirolimus: a pooled analysis. Orphanet J Rare Dis. 2024 Aug 15;19(1):299. doi: 10.1186/s13023-024-03243-5. PMID 39148107
  • Xu W, Yang C, Cheng C, Wang Y, Hu D, Huang J, He Y, Wang J, Chen K, Yang L, Zhou W, Zhang T, Liu S, Dai J, Meng S, Li X, Yang Y, Wang ST, Feng R, Zhang W, Zhang H, Wang L, Tian X, Xu KF. Determinants of Progression and Mortality in Lymphangioleiomyomatosis. Chest. 2023 Jul;164(1):137-148. doi: 10.1016/j.chest.2023.02.026. Epub 2023 Feb 18. PMID 36801466

Identifiers

NCT: NCT03193892 · 2016YFC091502LAM

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗