Efficacy and Safety of Sonidegib in Adult Patients with Nevoid Basal Cell Carcinoma Syndrome (Gorlin Syndrome): Results from a Phase 2, Double-Blind, Randomized Trial

Figures & data

Table 1 Efficacy and Safety Outcomes

Efficacy	Sonidegib	Placebo
	n = 7^a	n = 2
Patients with clinical clearance of target BCC at week 16^b
Complete (100% improvement)	3 (43)	0
Marked (76%–99% improvement)	3 (43)	0
Moderate (26%–75% improvement)	1 (14)	0
Slight (1%–25% improvement)	0	1 (50)
Worsening	0	1 (50)
Patients with histological clearance of target BCC at week 16	4 (57)	0
Number of total BCCs
Baseline	566	510
Week 12	341	571
Week 16	309	619
Safety	n = 8	n = 2
Any AEs (through long-term follow-up period)	7 (88)	1 (50)
Mild	3 (38)	0
Moderate	3 (38)	0
Severe	1 (13)	1 (50)
Related to treatment	6 (75)	1 (50)
Leading to discontinuation	0	1 (50)
Serious	1 (13)	1 (50)
AEs in ≥20% of patients (core study period)
Muscle spasms	3 (38)	1 (50)
Alopecia	2 (25)	0
CK increased	2 (25)	0
Fatigue	2 (25)	0
Headache	2 (25)	0
Nasopharyngitis	2 (25)	0
Nausea	2 (25)	0

Notes: Data presented as n (%) of total patients in treatment arm unless otherwise indicated. ^aOne patient was excluded from efficacy analysis due to receipt of placebo in 5 of 13 doses. ^bOne target BCC was counted per patient and assigned to 1 of 5 listed clearance categories.

Abbreviations: AE, adverse event; BCC, basal cell carcinoma; CK, creatine kinase.

Figure 1 Patient disposition.

Notes: ^aPatients were still included in the analysis and enrolled in long-term follow-up due to completion of most of the treatment period. ^bOne patient was excluded from efficacy analysis due to receipt of placebo in 5 of 13 doses.
Abbreviation: BCC, basal cell carcinoma.