9031 Background: Uveal melanoma (UM) is a rare cancer with a propensity for metastasis. There are no effective systemic therapies for metastatic UM, although dacarbazine is commonly used in practice. Sunitinib is a tyrosine kinase inhibitor with activity against several targets including c-Kit and VEGF receptors, both of which have been implicated in the pathogenesis of UM. Methods: In this randomized multicentre, phase II study (SUAVE), patients (pts) with metastatic UM, ECOG PS 0-2, and no prior systemic therapy for advanced disease, were randomized 1:1 to sunitinib (50mg daily for 28 days, followed by a 14 day break), or dacarbazine (1000 mg/m2once every 21 days). Crossover was permitted on progression. The primary endpoint was PFS; secondary endpoints included response rate and OS. A sample size of 124 was planned, with a power of 0.9 to detect an increase in 3 month PFS from 0.2 to 0.4 (HR: 0.563) and a one-sided alpha of 0.05. A preplanned futility analysis was performed after 50% of events, and recruitment stopped early, due to low conditional power (0.17% under current trend). Presentation of results was approved by DMC. Results: 74 pts from 12 centres were randomized over 24 months. Overall response rates of 0% and 8% were observed in the sunitinib and dacarbazine arms; while stable disease was observed in 24% of pts on sunitinib, and 11% on dacarbazine. PFS and OS were not improved with sunitinib (see Table). 11 pts in the sunitinib arm and 23 in the dacarbazine arm underwent crossover on progression. No unexpected AEs were observed, and no deaths due to toxicity occurred. Conclusions: In these preliminary results sunitinib did not have significant clinical activity in metastatic UM. This trial is one of the largest undertaken in metastatic UM and demonstrates that timely recruitment to collaborative multicentre randomized trials is achievable in this rare disease. Clinical trial information: 75033520. [Table: see text]
The POWER-tool: Recommendations for involving patient representatives in choosing relevant outcome measures during rare disease clinical trial design