NCT03193892

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1,500

participants targeted

Target at P75+ for all trials

Timeline
5mo left

Started Jan 2017

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress96%
Jan 2017Oct 2026

Study Start

First participant enrolled

January 1, 2017

Completed
5 months until next milestone

First Submitted

Initial submission to the registry

June 14, 2017

Completed
7 days until next milestone

First Posted

Study publicly available on registry

June 21, 2017

Completed
9.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2026

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2026

Last Updated

September 24, 2021

Status Verified

September 1, 2021

Enrollment Period

9.8 years

First QC Date

June 14, 2017

Last Update Submit

September 18, 2021

Conditions

Pulmonary Function

Keywords

registryChinalymphangioleiomyomatosis

Outcome Measures

Primary Outcomes (1)

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

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

    10 years

Secondary Outcomes (4)

  • Change of diffusing capacity of the lung.

    10 years

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

    10 years

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

    10 year

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

    10 years

Eligibility Criteria

Sexfemale(Gender-based eligibility)
Gender Eligibility DetailsPulmonary lymphangioleiomyomatosis (LAM) affects almost exclusively women.
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodProbability Sample
Study Population

Study Population is from across the country of China.

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

RECRUITING

Related Publications (2)

  • 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: 39148107DERIVED

  • 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: 36801466DERIVED

Biospecimen

Retention: SAMPLES WITH DNA

Biological specimens are preserved in serum, plasma, and leukocytes

MeSH Terms

Conditions

Lymphangioleiomyomatosis

Condition Hierarchy (Ancestors)

LymphangiomyomaNeoplasm, Lymphatic TissueNeoplasms by Histologic TypeNeoplasmsPerivascular Epithelioid Cell NeoplasmsNeoplasms, Connective and Soft TissueLymphoproliferative DisordersLymphatic DiseasesHemic and Lymphatic DiseasesImmunoproliferative DisordersImmune System Diseases

Central Study Contacts

Kai-Feng Xu, M.D.

CONTACT

86-10-69155039xukf@pumch.cn

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Target Duration
10 Years
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 14, 2017

First Posted

June 21, 2017

Study Start

January 1, 2017

Primary Completion (Estimated)

October 1, 2026

Study Completion (Estimated)

October 1, 2026

Last Updated

September 24, 2021

Record last verified: 2021-09

Data Sharing

IPD Sharing
Will not share

Currently, there is no such a plan to share IPD to other researchers

Locations

CN(1)