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How pilot studies improve large-scale clinical trials: lessons learned from the COMBINE Study
Stephanie S O'Malley1, Daniel J Martin, James D Hosking
1Department of Psychiatry, Yale University School of Medicine, CMHC-SAC202, 34 Park Street, New Haven, Connecticut 06519, USA. Stephanie.omalley@yale.edu
Journal of Studies on Alcohol. Supplement
|October 15, 2005
Summary
Pilot studies for the COMBINE trial improved the main study design. These trials informed medication safety, feasibility, and participant recruitment for alcohol dependence treatment.
Area of Science:
- Clinical trial design
- Pharmacotherapy research
- Behavioral therapy research
Background:
- Designing large-scale clinical trials involves critical decisions regarding safety, efficacy, measurement, feasibility, and training.
- The COMBINE Study investigates combined medication and behavioral therapies for alcohol dependence.
Purpose of the Study:
- To demonstrate how pilot studies can enhance the design of large-scale clinical trials.
- To inform the main COMBINE trial through pilot investigations of safety, feasibility, and methodology.
Main Methods:
- COMBINE Pilot 1: Assessed safety and tolerability of acamprosate and naltrexone doses in an inpatient setting.
- COMBINE Pilot 2: Evaluated feasibility of research assessments, recruitment, staff training, and medication safety in an outpatient setting.
Main Results:
- Pilot 1 data supported outpatient medication trials and informed dose adjustments for adverse event management in the main trial.
- Pilot 2 results led to a shorter assessment battery, established recruitment procedures, trained staff, and confirmed medication safety in outpatient conditions.
Conclusions:
- Pilot studies are effective in refining clinical trial protocols.
- The COMBINE Study exemplifies the successful use of pilot data to inform the design of a major clinical trial.