1 / 18

Clinical Trials in IPF

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.

megan
Download Presentation

Clinical Trials in IPF

An Image/Link below is provided (as is) to download presentation Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author. Content is provided to you AS IS for your information and personal use only. Download presentation by click this link. While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server. During download, if you can't get a presentation, the file might be deleted by the publisher.

E N D

Presentation Transcript

  1. Clinical Trials in IPF Dr Helen Parfrey

  2. 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

  3. 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

  4. Have you taken part in a clinical trial for IPF?

  5. Exciting time for IPF

  6. 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)

  7. 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

  8. 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 • Patient enrolment into Pred/Aza/NAC ceased

  9. 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

  10. New treatments - Pirfenidone Noble PW et al, Lancet 2011

  11. 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

  12. 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

  13. 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

  14. Current Clinical Trials in IPF 116 studies in IPF 22 therapeutic trials

  15. 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

  16. 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

  17. Thank you for participating in the studies

More Related