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Clinical Trials in IPF Dr Helen Parfrey Are clinical trials needed ? • Essential to determine if a drug therapy is beneficial • Identify who will benefit the most from the treatment • Side effects associated with the drug therapy • Information about the disease How to determine if a drug treatment is effective? • “Controlled” - compare the drug treatment to a placebo • “Blinded” - the study co-ordinators and patients do not know what treatment has been given • “Endpoints” - markers to indicate if the drug is beneficial • IPF trials use lung function tests (FVC), hospital admissions, survival, quality of life, measures of breathlessness • “Powered” - sufficient number of patients • “Inclusion and exclusion criteria” – how to select patients for a study Have you taken part in a clinical trial for IPF? Exciting time for IPF But, several studies have shown that the treatments have not been effective for IPF • Interferon gamma studies (INSPIRE) • Bosentan studies (BUILD series) • Anti-TNFα studies (Etanercept) • Tyrosine kinase inhibitor (Imatinib) Triple therapy: IFIGENIA study Limitations All patients in the study taking prednisolone and azathioprine Not compare treatment with N-acetylcysteine alone with placebo Demedts et al New Engl J Med 2005 Interim safety analysis: PANTHER-IPF • Phase III trial, double blind placebo controlled to assess effectiveness of NAC • Recruited 238 out of 390 patients with mild to moderate IPF Mortality Hospitalisation SAE Pred/Aza/NAC Placebo 11% 1% 29% 8% 31% 9% • Patient enrolment into Pred/Aza/NAC ceased Anticoagulation therapy for IPF Limitations Small number of patients Not blinded High drop out in treatment group No exclusion of PE as cause for deterioration Kubo et al, Chest 2005 New treatments - Pirfenidone Noble PW et al, Lancet 2011 Cochrane Review of Pirfenidone Studies • First licensed treatment for mild to moderate IPF • FVC≥50%, TLCO≥35%, 6MWD ≥150m • Named patient programme • NICE evaluation currently Clinical Trial • Tyrosine kinase inhibitor BIBF 1120 • Phase II trial, placebo controlled, 432 patients with IPF Richeldi L et al New Engl J Med 2011 How to find out about clinical trials in IPF? • Ask your hospital doctor • Clinicaltrials.gov website • Details of clinical trials in all areas of medicine • Use search function to look for trials related to IPF Current Clinical Trials in IPF 116 studies in IPF 22 therapeutic trials On-going clinical trials • BIBF 1120 • QAX576 • Thalidomide • Sildenafil • Plasma exchange, rituximab and steroids for acute exacerbations • Sirolimus • Losartan • Phase I • AB0024 • PRM-151 • STX-100 • CC930 • IW001 Clinicaltrials.gov Summary • Importance of clinical trials in evaluating new treatments for IPF • Carefully designed • Expotential increase in clinical trials for IPF • Change how IPF is treated Thank you for participating in the studies