Innovative design for a phase 1 trial with intra-patient dose escalation: The Crotoxin study

Crotoxin has a broad antitumor activity but has shown frequent neurotoxic toxicity. To induce tolerance and limit this toxicity, we propose a new design with intra-patient dose escalation.

A new Dose Limiting Toxicity definition was used. The concept of Target Ceiling Dose was introduced.

Dose Limiting Toxicity was the inability to dose escalate twice. Target Ceiling Dose was the highest planned dose to be administered to a patient and could change for patients along time. Recommended Dose was defined similarly as in a (3 + 3) conventional design.

This innovant design was used and the clinical trial is now closed for inclusions. Results will be presented later.