The Indacaterol in Lymphangioleiomymotosis (LAM) Trial

INTERVENTION: Double blind placebo controlled cross‐over trial, studying efficacy and safety of inhaled indacaterol 150 micrograms daily in women with lymphangioleiomyomatosis. Treatment duration 2 weeks for active drug and placebo; washout period 2‐6 weeks according to patient convenience. Inhaled medication will be returned at end of treatment periods to monitor adherence. CONDITION: Lymphangioleiomyomatosis PRIMARY OUTCOME: 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). ; SECONDARY OUTCOME: 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. ; Secondary ; 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. ; 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.