Safety, Tolerability, and Pharmacokinetics of Topical Calcitriol Formulation for Treatment of Chemotherapy-Induced Alopecia in Patients Receiving Taxane-Based Regimen: Final Results

In 2019, a Phase 1 study was conducted to assess the safety and tolerability of BPM31543, a topical calcitriol formulation, in treating chemotherapy-induced alopecia (CIA) in patients undergoing taxane-based chemotherapy. The study used a 3+3 dose-escalation strategy with 3-6 patients at six dose levels (5/10/20/40/60/80 μg/mL). The study included 23 female patients, 22 of whom experienced at least one treatment-emergent adverse event (TEAE). The most common TEAEs were alopecia (60.9%), fatigue (47.8%), nausea (39.1%), peripheral sensory neuropathy (30.4%), and maculopapular rash and increased vitamin D (26.1% each). The study concluded that BPM31543 was safe and well-tolerated, with no dose-limiting toxicity observed up to 80 µg/mL dose and no maximum tolerated dose level reached. There was also a signal of potential efficacy detected at each dose level. A Phase 2/3 trial strategy for clinical development was planned.