None listed
Conditions
Brief summary
Lymphangioleiomyomatosis (LAM) is a rare lung disease restricted to adult women. This may occur either sporadically (s-LAM) or in association with tuberous sclerosis (TSC-LAM). The lungs of patients with LAM are infiltrated by smooth muscle cells (LAM cells).There is a very limited evidence base for treatment of LAM and it is only recently that the first randomised trials have been initiated. The LAM clinic at St Vincent’s follows the largest number of women with LAM in Australia, currently approximately 40 patients. The first ever trial into the efficacy of doxycycline in LAM was completed at St Vincent’s LAM clinic, and this new study aims to assist the breathlessness associated with this disease and evaluate the mechanisms underlying this. Indacaterol is a novel, inhaled, once-daily, ultra-long-acting beta 2-agonist for the treatment of chronic obstructive pulmonary disease (COPD). Bronchodilator agents are frequently prescribed in LAM but have never been the subject of a clinical trial. Long acting beta2-agonists have been described as having anti-proliferative effects on smooth muscle in vitro as well as in vitro, but these agents have not yet been investigated in LAM. It is theoretically possible that women with LAM will respond clinically, and it is also possible that indacaterol might have a beneficial effect within the airway. This trial will evaluate indacaterol in LAM using a classical study design ie. a double blind placebo controlled trial, in 15 women with LAM.
Interventions
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.
Sponsors
Deborah H Yates
Study design
Allocation
Randomised controlled trial
Intervention model
Crossover
Primary purpose
Treatment
Masking
Blinded (masking used) (Subject, Caregiver)
Eligibility
Sex/Gender
All
Age
18 Years to 80 Years
Healthy volunteers
No
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
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.
Outcome results
None listed