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A National Registry on Chinese Patients With Lymphangioleiomyomatosis

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ClinicalTrials.gov Identifier: NCT03193892
Recruitment Status : Recruiting
First Posted : June 21, 2017
Last Update Posted : June 21, 2017
Sponsor:
Collaborators:
Shanghai Zhongshan Hospital
Guangzhou Institute of Respiratory Disease
Central South University
Anhui Provincial Hospital
First Affiliated Hospital of Harbin Medical University
Xiangya Hospital of Central South University
The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
China-Japan Friendship Hospital
Wuxi People's Hospital
Beijing Shijitan Hospital
West China Hospital
Chinese Academy of Medical Sciences, Fuwai Hospital
Fujian Provincial Hospital
Information provided by (Responsible Party):
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.

Condition or disease
Pulmonary Function

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.

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 800 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 3 Years
Official Title: A National Registry on Clinical Manifestations, Genetics, Interventions, and Outcomes in Chinese Patients With Lymphangioleiomyomatosis (LAM-CHINA)
Actual Study Start Date : January 1, 2017
Estimated Primary Completion Date : December 31, 2020
Estimated Study Completion Date : December 31, 2020

Resource links provided by the National Library of Medicine





Primary Outcome Measures :
  1. Change in spirometry (FEV1 and FVC) of lymphangioleiomyomatosis patients. [ Time Frame: 3 years ]
    Spirometry will be evaluated at baseline and through study completion, an average of 3 years.


Secondary Outcome Measures :
  1. Change of diffusing capacity of the lung. [ Time Frame: 3 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: 3 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: 3 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: 3 years ]
    Common Terminology Criteria for Adverse Events (CTCAE) will be used to evaluate adverse drug reactions through study completion, an average of 3 years.


Biospecimen Retention:   Samples With DNA
Biological specimens are preserved in serum, plasma, and leukocytes


Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Female
Gender Based Eligibility:   Yes
Gender Eligibility Description:   Pulmonary lymphangioleiomyomatosis (LAM) affects almost exclusively women.
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Study Population is from across the country of China.
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03193892


Contacts
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Contact: Kai-Feng Xu, M.D. 86-10-69155039 xukf@pumch.cn

Locations
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China, Beijing
Peking Union Medical College Hospital Recruiting
Beijing, Beijing, China, 100730
Contact: Kai-Feng Xu, M.D.    86-10-69155039    xukf@pumch.cn   
Contact: XinLun Tian, M.D.    86-10-69155039    xinlun_t@sina.com   
Sub-Investigator: Xinlun Tian, M.D.         
Principal Investigator: Kai-Feng Xu, M.D.         
Sponsors and Collaborators
Peking Union Medical College Hospital
Shanghai Zhongshan Hospital
Guangzhou Institute of Respiratory Disease
Central South University
Anhui Provincial Hospital
First Affiliated Hospital of Harbin Medical University
Xiangya Hospital of Central South University
The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
China-Japan Friendship Hospital
Wuxi People's Hospital
Beijing Shijitan Hospital
West China Hospital
Chinese Academy of Medical Sciences, Fuwai Hospital
Fujian Provincial Hospital

Additional Information:

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Responsible Party: Peking Union Medical College Hospital
ClinicalTrials.gov Identifier: NCT03193892     History of Changes
Other Study ID Numbers: 2016YFC091502LAM
First Posted: June 21, 2017    Key Record Dates
Last Update Posted: June 21, 2017
Last Verified: June 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Currently, there is no such a plan to share IPD to other researchers

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Peking Union Medical College Hospital:
registry
China
lymphangioleiomyomatosis

Additional relevant MeSH terms:
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Lymphangioleiomyomatosis
Lymphangiomyoma
Lymphatic Vessel Tumors
Neoplasms by Histologic Type
Neoplasms
Perivascular Epithelioid Cell Neoplasms
Neoplasms, Connective and Soft Tissue
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases