Pulmonary FunctionNCT03193892
A National Registry on Chinese Patients With Lymphangioleiomyomatosis
An observational study of Pulmonary Function, run by Peking Union Medical College Hospital at 1 site in China.
Trial 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… Read more about this study
- Who is it for?
- Any age, women only
- Conditions the study is for
- Pulmonary Function
- How many people
- 1,500 participants
- Where
- 1 siteChina
- Study dates
- 1 Jan 2017to 1 Oct 2026
- How long will I take part?
- 10 Years
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Women only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Study Population is from across the country of China.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730
Kai-Feng Xu, M.D., 86-10-69155039, xukf@pumch.cn
XinLun Tian, M.D., 86-10-69155039, xinlun_t@sina.com
About this study
Official title: A National Registry on Clinical Manifestations, Genetics, Interventions, and Outcomes in Chinese Patients With Lymphangioleiomyomatosis (LAM-CHINA)
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.
Read the 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.
- Conditions
- Pulmonary Function
- Keywords
- registry, China, lymphangioleiomyomatosis
- Medical subject headings
- Lymphangioleiomyomatosis
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in spirometry (FEV1 and FVC) of lymphangioleiomyomatosis patients.
Spirometry will be evaluated at baseline and through study completion, an average of 3 years.
Measured: 10 years
Secondary outcomes (4)
Change of diffusing capacity of the lung.
Pulmonary function tests will be evaluated at baseline and through study completion, an average of 3 years.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jun 2017
- Study start1 Jan 2017
- Primary completion1 Oct 2026 (estimated)
- Study completion1 Oct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Pulmonary FunctionStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1,500 participants (estimated)
- Target duration
- 10 Years
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Shanghai Zhongshan Hospital, Guangzhou Institute of Respiratory Disease, Central South University, Anhui Provincial Hospital, Xiangya Hospital of Central South University, The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School, Wuxi People's Hospital, Beijing Shijitan Hospital, Capital Medical University, West China Hospital, Fujian Provincial Hospital, Wuhan TongJi Hospital
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03193892
- Sponsor's protocol ID
- 2016YFC091502LAM
- Status last verified
- Sep 2021
- First submitted
- 14 Jun 2017
- Last updated
- 24 Sep 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Currently, there is no such a plan to share IPD to other researchers
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.
PubMed 39148107Xu 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.
PubMed 36801466
Study at a glance
From the registry record, last updated by the sponsor on 24 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A National Registry on Clinical Manifestations, Genetics, Interventions, and Outcomes in Chinese Patients With Lymphangioleiomyomatosis (LAM-CHINA)
- ClinicalTrials.gov ID
- NCT03193892
- Acronym / sponsor ID
- 2016YFC091502LAM
- Conditions
- Pulmonary Function
- Keywords
- registry, China, lymphangioleiomyomatosis
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 1,500 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Collaborators
- Shanghai Zhongshan Hospital, Guangzhou Institute of Respiratory Disease, Central South University, Anhui Provincial Hospital, Xiangya Hospital of Central South University, The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School, Wuxi People's Hospital, Beijing Shijitan Hospital, Capital Medical University, West China Hospital, Fujian Provincial Hospital, Wuhan TongJi Hospital
- Study start
- 1 Jan 2017 (actual)
- Primary completion
- 1 Oct 2026 (estimated)
- Study completion
- 1 Oct 2026 (estimated)
- First posted
- 21 Jun 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 10 Years
Who may qualify
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Study Population is from across the country of China.
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
4 secondary outcomes
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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Sep 2021.
- Derived from this study: PubMed 39148107Wang 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.
- Derived from this study: PubMed 36801466Xu 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.
- Individual participant dataSharing planned: No. Currently, there is no such a plan to share IPD to other researchers
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Pulmonary Function, run by Peking Union Medical College Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 24 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Kai-Feng Xu, M.D. (86-10-69155039, xukf@pumch.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in spirometry (FEV1 and FVC) of lymphangioleiomyomatosis patients. (time frame: 10 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03193892, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Sep 2021.