Mobile health technologies in an interventional hybrid study on actinic keratosis: Results from an early phase randomized controlled trial investigating the safety and efficacy of a cytosolic phospholipase A2 inhibitor gel in photodamaged skin

AbstractHybrid trials are a new trend in dermatological research that leverage mobile health technologies to decentralize a subset of clinical trial elements and thereby reduce the number of in‐clinic visits. In a Phase I/IIa randomized controlled hybrid trial, the safety and efficacy of an anti‐proliferative and anti‐inflammatory drug inhibiting cytosolic phospholipase A2 (AVX001) was tested using 1%, 3% or vehicle gel in 60 patients with actinic keratosis (AK) and assessed in‐clinic as well as remotely. Over the course of 12 weeks, patients were assessed in‐clinic at baseline, end of treatment (EOT) and end of study (EOS), as well as 9 times remotely on a weekly to biweekly basis. Safety outcomes comprising local skin reactions (LSR; 0–5), adverse events (AE) and cosmesis, were graded in‐clinic and remotely using patient‐obtained smartphone photographs (PSPs) and questionnaires; efficacy was assessed in‐clinic based on clinically visible clearance of AK target area of >50%. A total of 55 participants (91.7%) completed the treatment course. The average submission rate of PSPs was high (≥85%), of which 93% were of sufficient quality. No serious AE were reported and only two experienced temporary LSR >2 (scale 0–4) and cosmesis remained stable throughout the study. Based on the mild AE and LSR profile, daily application of AVX001 gel for 1 month appears safe, tolerable, and cosmetically acceptable for use in patients with AK. At EOT, AVX001 achieved a subtle treatment response with clearance of AK target area of >50% in 18% of patients. Remote and in‐clinic assessments of LSRs were in high agreement, suggesting that the use of mobile health technologies in early‐phase hybrid studies of AK does not compromise patient safety.