Renee Stapleton 7/07/05
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Transcript of Renee Stapleton 7/07/05
Developing a Phase II RCT:The effects of n-3 fatty acids on lung and systemic inflammation in patients with acute lung injury
Renee Stapleton7/07/05
Outline
• Choosing a research question and selecting study design
• Process of designing the trial• Timeline• Budget• Lessons learned
Choosing a Research Question
1.What types of clinical questions do I enjoy the most? Supportive care mechanisms in the ICU
2.What methods do I want to focus on? Obtain training as a clinical trialist
3.Which particular areas of ICU care need championing? Nutritional delivery and supplementation
Choosing a Research Question
4. Is there a means to do a small trial feasibly? ALI “Clinical Trials Incubator Unit” (CTIU)
5. Under the umbrella of nutrition and ALI, are there any questions that have a high chance of offering me early success? Fish oil in acute lung injury (ALI)
Designing the Trial
• CTIU established for phase II “proof of concept” trials in ALI
• Details to think about– 2 arms versus 3 arms– Endpoints
• Primary and secondary– Sample size calculations
• Accurate pilot data?– Enough patients at Harborview alone?– Inclusion and exclusion criteria
Designing the Trial
• Details to think about (continued)– Intervention
• Where to get the fish oil?• How to deliver it?• Dose?• Need to do independent testing of product• Do we need to submit an IND to the FDA?
– Placebo– Blinding
• How to successfully achieve blinding?
Designing the Trial
• Details to think about (continued)– Identification, enrollment, and
consent of patients• Who will do it?• When?
– Randomization• Who will do it?• When?• What design?
Designing the Trial
• Details to think about (continued)– Adverse effects– “Cointerventions”
• Need to standardize other practices that may affect outcome
– Collecting and managing data• How to ensure accuracy?• Missing data
– Statistical analyses
The Final Design
100 patients with ALI
1/3 fish oil +enteral nutrition
1/3 placebo +enteral nutrition
1/3 Oxepa®
Enrollment and randomization within 48 hours of ALI diagnosis
• Bronchoalveolar lavage and serum sampling at study entry and on days 5 and 10 (primary outcome is BAL IL-8)
• Study drug delivered through day 10, extubation, or death
• Secondary outcomes recorded for use in designing future trial
Timeline
• February 2004 – Idea was conceived• June 2004 – Started writing K23• October 2004 – Submitted K23• March 2005 – K12 funding• July 2005 – Human subjects application• January 2006 – Begin enrollment• December 2007 – End of enrollment?
Budget
• CTIU already pays for – Full time research nurse– Full time laboratory tech– Some laboratory support for assays
• K12 pays for – My salary– Travel to national meeting– Desktop computer and software– Biostatistical consultation
Budget – My Costs
ITEM 5-YEAR COST
0.25 FTE research nurse $87,500
Pharmacy (IDS) costs for fish oil product, packaging, and randomization
$43,345
Lab assays $43,390
Oxepa® enteral feeding formula $23,680
Laptop computer and printer $3,000
Travel to Toronto and Kingston, Ontario $10,000
Sample banking/storage $10,000???
Total $220,915
Lessons Learned
• Start early• Designing the trial and getting
funded is hard…• Rumor is that conducting and
managing the trial is HARDER!• We’ll see
Mmmm, sausage!