A National Registry on Chinese Patients With Lymphangioleiomyomatosis

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Observational [Patient Registry]
Eligible Ages N/A and Over
Gender Female
More Inclusion & Exclusion Criteria

Inclusion Criteria:

1. Gender: female. 2. Age: no limitation. 3. Diagnosis: to meet one of the following criteria,
  • (1) definite or probable diagnosis of LAM by ERS 2010 criteria.
  • (2) with clinical features of LAM and blood VEGF-D≥ 800 pg/ml.
  • (3) clinical diagnosed LAM plus TSC2 gene mutations in blood or tissue specimens.
4. LAM diagnosed based on other documented supporting evidence that is not listed in the current diagnosis criteria. These supporting evidence needs to be recorded for further analysis.

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.

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT03193892
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Peking Union Medical College Hospital
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

N/A
Principal Investigator Affiliation N/A
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

OtherOtherOtherOtherOtherOtherOtherOtherOtherOtherOtherOtherOtherOther
Overall Status Recruiting
Countries China
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Pulmonary Function
Additional Details

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.

Contact a Trial Team

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International Sites

Peking Union Medical College Hospital, Beijing, Beijing, China

Status

Recruiting

Address

Peking Union Medical College Hospital

Beijing, Beijing, 100730

Site Contact

Kai-Feng Xu, M.D.

xukf@pumch.cn

86-10-69155039

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