Trial Outcomes & Findings for Letrozole for Lymphangioleiomyomatosis (NCT NCT01353209)
NCT ID: NCT01353209
Last Updated: 2024-04-17
Results Overview
FEV1 values reported are in liters or milliliters. There are no definite minimum and maximum values of FEV1 as it is a physiological measure of lung function and varies from individual to individual. Higher FEV1 scores indicate better lung function.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
17 participants
Primary outcome timeframe
12 months
Results posted on
2024-04-17
Participant Flow
Patients were eligible for inclusion in the study if they were postmenopausal women with a definite diagnosis of pulmonary LAM and abnormal pulmonary function. Patients on sirolimus were required to wait three months before beginning this trial.
Participant milestones
| Measure |
Letrozole
Letrozole: 2.5 mg daily for twelve months
|
Placebo
Placebo: sugar pill given daily for twelve months
|
|---|---|---|
|
Overall Study
STARTED
|
9
|
8
|
|
Overall Study
COMPLETED
|
8
|
7
|
|
Overall Study
NOT COMPLETED
|
1
|
1
|
Reasons for withdrawal
| Measure |
Letrozole
Letrozole: 2.5 mg daily for twelve months
|
Placebo
Placebo: sugar pill given daily for twelve months
|
|---|---|---|
|
Overall Study
Adverse Event
|
1
|
1
|
Baseline Characteristics
Letrozole for Lymphangioleiomyomatosis
Baseline characteristics by cohort
| Measure |
Letrozole
n=9 Participants
Letrozole: 2.5 mg daily for twelve months
|
Placebo
n=8 Participants
Placebo: sugar pill given daily for twelve months
|
Total
n=17 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
8 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Age, Continuous
|
57 years
n=5 Participants
|
59 years
n=7 Participants
|
58 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
7 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
9 participants
n=5 Participants
|
8 participants
n=7 Participants
|
17 participants
n=5 Participants
|
|
LAM subtyped
Sporadic LAM
|
6 participants
n=5 Participants
|
8 participants
n=7 Participants
|
14 participants
n=5 Participants
|
|
LAM subtyped
TSC LAM
|
3 participants
n=5 Participants
|
0 participants
n=7 Participants
|
3 participants
n=5 Participants
|
|
Sirolimus status
On sirolimus
|
5 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Sirolimus status
Not on sirolimus
|
4 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 12 monthsFEV1 values reported are in liters or milliliters. There are no definite minimum and maximum values of FEV1 as it is a physiological measure of lung function and varies from individual to individual. Higher FEV1 scores indicate better lung function.
Outcome measures
| Measure |
Letrozole
n=8 Participants
Letrozole: 2.5 mg daily for twelve months
|
Placebo
n=7 Participants
Placebo: sugar pill given daily for twelve months
|
|---|---|---|
|
Rate of Change in Forced Expiratory Volume in 1 Second in ml/Month
|
1.04 milliliters per month
Interval 0.56 to 2.02
|
1.66 milliliters per month
Interval 0.77 to 2.63
|
SECONDARY outcome
Timeframe: twelve monthsPost-bronchodilator FVC in milliliters
Outcome measures
| Measure |
Letrozole
n=8 Participants
Letrozole: 2.5 mg daily for twelve months
|
Placebo
n=7 Participants
Placebo: sugar pill given daily for twelve months
|
|---|---|---|
|
Post-bronchodilator FVC
|
2.58 milliliters per month
Interval 2.07 to 3.44
|
2.46 milliliters per month
Interval 1.26 to 3.21
|
SECONDARY outcome
Timeframe: twelve monthsQuality of Life scale for respiratory symptoms. This is a disease-specific instrument designed to measure impact on overall health, daily life, and perceived well-being in patients with obstructive airways disease. Scores range from 0 to 100, with higher scores indicating worse quality of life.
Outcome measures
| Measure |
Letrozole
n=8 Participants
Letrozole: 2.5 mg daily for twelve months
|
Placebo
n=7 Participants
Placebo: sugar pill given daily for twelve months
|
|---|---|---|
|
St George Respiratory Questionnaire
|
38 score on a scale
Interval 27.0 to 53.0
|
62 score on a scale
Interval 38.0 to 82.0
|
SECONDARY outcome
Timeframe: twelve monthsVEGF-D values represent serum VEGF-D levels in pg/ml. Higher levels of VEGF-D are associated with Lymphangioleiomyomatosis. A serum VEGF-D greater than 400 pg/ml is a diagnostic biomarker for LAM.
Outcome measures
| Measure |
Letrozole
n=8 Participants
Letrozole: 2.5 mg daily for twelve months
|
Placebo
n=7 Participants
Placebo: sugar pill given daily for twelve months
|
|---|---|---|
|
Serum VEGF-D
|
572 pg/ml
Interval 294.0 to 1816.0
|
944 pg/ml
Interval 350.0 to 1598.0
|
Adverse Events
Letrozole
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Placebo
Serious events: 1 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Letrozole
n=9 participants at risk
Letrozole: 2.5 mg daily for twelve months
|
Placebo
n=8 participants at risk
Placebo: sugar pill given daily for twelve months
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
worsening dyspnea
|
0.00%
0/9 • 1 year
Adverse effects were tabulated using the National Cancer Institute's Common Terminology Criteria for Adverse Events version 4.
|
12.5%
1/8 • Number of events 2 • 1 year
Adverse effects were tabulated using the National Cancer Institute's Common Terminology Criteria for Adverse Events version 4.
|
Other adverse events
| Measure |
Letrozole
n=9 participants at risk
Letrozole: 2.5 mg daily for twelve months
|
Placebo
n=8 participants at risk
Placebo: sugar pill given daily for twelve months
|
|---|---|---|
|
Blood and lymphatic system disorders
Swollen lymph nodes
|
11.1%
1/9 • Number of events 1 • 1 year
Adverse effects were tabulated using the National Cancer Institute's Common Terminology Criteria for Adverse Events version 4.
|
12.5%
1/8 • Number of events 1 • 1 year
Adverse effects were tabulated using the National Cancer Institute's Common Terminology Criteria for Adverse Events version 4.
|
|
Cardiac disorders
Chest pain
|
0.00%
0/9 • 1 year
Adverse effects were tabulated using the National Cancer Institute's Common Terminology Criteria for Adverse Events version 4.
|
12.5%
1/8 • Number of events 1 • 1 year
Adverse effects were tabulated using the National Cancer Institute's Common Terminology Criteria for Adverse Events version 4.
|
|
Endocrine disorders
Endocrine disorder
|
11.1%
1/9 • Number of events 1 • 1 year
Adverse effects were tabulated using the National Cancer Institute's Common Terminology Criteria for Adverse Events version 4.
|
0.00%
0/8 • 1 year
Adverse effects were tabulated using the National Cancer Institute's Common Terminology Criteria for Adverse Events version 4.
|
|
Eye disorders
Blurry vision
|
11.1%
1/9 • Number of events 1 • 1 year
Adverse effects were tabulated using the National Cancer Institute's Common Terminology Criteria for Adverse Events version 4.
|
12.5%
1/8 • Number of events 1 • 1 year
Adverse effects were tabulated using the National Cancer Institute's Common Terminology Criteria for Adverse Events version 4.
|
|
Gastrointestinal disorders
abdominal pain; nausea
|
11.1%
1/9 • Number of events 4 • 1 year
Adverse effects were tabulated using the National Cancer Institute's Common Terminology Criteria for Adverse Events version 4.
|
12.5%
1/8 • Number of events 10 • 1 year
Adverse effects were tabulated using the National Cancer Institute's Common Terminology Criteria for Adverse Events version 4.
|
Additional Information
Susan McMahan, Clinical Research Manager
University of Cincinnati
Phone: 513-558-4831
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place