The Indacaterol in Lymphangioleiomymotosis (LAM) Trial

Not Applicable

• Conditions: ymphangioleiomyomatosis; Lymphangioleiomyomatosis; Respiratory - Other respiratory disorders / diseases

• Registration Number: ACTRN12613001251718
• Lead Sponsor: Deborah H Yates

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2013-11-14
• Last Update Posted: 13 January 2020

Recruitment & Eligibility

• Status: ot yet recruiting
• Sex: Female
• Target Recruitment: 15

Inclusion Criteria

Women:

1.Age: 18-80 inclusive.
1.2.Diagnosed with LAM and classified into possible, probable and definite according to ERS criteria (1).
1.3.Willingness and ability to participate in the study after fully informed consent.
1.4.Both sporadic LAM (sLAM) and TSC-LAM (tuberous sclerosis-related LAM) to be included.
1.5.Women with all severity categories of LAM are eligible to participate in the study but those on continuous home oxygen will be excluded, as well as those not sufficiently well to participate in the requisite tests.

Exclusion Criteria

1. Known sensitivity to indacaterol or the class of long acting beta agonists
2. Patients with severe medical condition(s) which in the view of the investigator prohibit participation in the study (e.g. uncontrolled diabetes, uncontrolled heart failure, severe renal or liver disease, high blood pressure, coronary artery disease, cardiac arrhythmias, thyroid disorders, or lactose intolerance).
3. Use of any other investigational agent within the last 30 days
4. Current smoking or smoking within 6 months of study start.
5. Concurrent respiratory disease including a diagnosis of asthma (provided LAM has not been misdiagnosed as asthma prior to the diagnosis of LAM). Pleural pathology from previous pleurodeses and disease related to LAM are not included in this category.
6. Respiratory tract infection of exacerbation within the previous 4 weeks. Patients can be re-entered into the trial after a 4 week recovery period, provided baseline FEV1 is within 10% of first study period.
7. Treatment with oral glucocorticosteroids within the previous 4 weeks.
8. Women of childbearing potential not using adequate contraceptive method(s) including the oral contraceptive pill, barrier contraception, IUD, as well as women who are breastfeeding.
9. Patients on continuous oxygen therapy.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
1.To evaluate the efficacy of indacaterol in terms of change in trough forced expiratory volume in 1 second (FEV1) versus baseline in women with lymphangioleiomyomatosis (LAM).<br>[2 week treatment period for both active drug and placebo; assessments at baseline and end of treatment periods in each case. Symptom score monitoring during treatment periods.]

Secondary Outcome Measures

Name	Time	Method
Secondary<br>1. To examine the effect of indacaterol on quality of life measurements (QoL as measured by SF-36 and other measures) in women with LAM.<br>[2 week treatment with drug and placebo; QoL and lung function measured at baseline and after treatment periods.];2. To examine the effect of indacaterol on airway inflammation in LAM using non-invasive exhaled breath monitoring (fractional exhaled nitric oxide assessment, electronic nose analysis (ENA)) and some blood based biomarkers including VEGF-D, HGF, ACE, MMP 2 and 9.<br>[Measurements: FeNO, ENA, blood based biomarkers at baseline and after treatment periods.]