Cytel President and co-founder Cyrus Mehta has co-authored a paper on Infantile Hemangioma, recently published in the New England Journal of Medicine. The successful study was designed as an adaptive confirmatory dose-response which confirmed that 3mg per kilogram per day of propranolol for 6 months is an effective resolution for hemangioma.
What is an Adaptive Confirmatory Dose-Response Study?
An adaptive confirmatory dose-response clinical trial, (sometimes called a combined Phase 2/3 or seamless Phase 2/3 study), is a confirmatory study with one interim look. The interim look is used to determine the correct dose, which is then confirmed during the remainder of the trial.
A popular design begins with a multi-arm trial, which becomes a 2-arm trial after interim analysis by continuing with the best candidate dose. Cytel designed such a trial for Fulyzaq, the first 2-stage adaptive trial to receive FDA approval. Fulyzaq was a trial for HIV related GI-distress. However, the adoption of this design by a recent pediatric study shows that such trials are increasing in popularity.
The rising popularity of this design stems in large part from the fact that these trials shorten trial length by reducing patient enrollment. Information from patients randomized during dose-response can be used during the confirmatory elements of a trial, significantly reducing the sample size.
Fulyzaq was the first Adaptive Confirmatory Dose-Response Trial to Receive FDA Approval
Related Items of Interest
[1] A randomized, controlled trial of oral propranolol in infantile hemangioma (abstract).
[2] www.cytel.com/pdfs/Case-2-two-stage-Napo-Fulyzaq-.pdf
[3] Operationally Seamless & Inferentially Seamless Adaptive Designs
