Ipsen has reported positive results from the CELESTIAL trial, a Phase III study of cabozantinib against placebo to treat patients with advanced hepatocellular carcinoma (HCC), the most common form of liver cancer in adults.
The randomised, double-blind, placebo-controlled global trial enrolled HCC patients who have been previously treated with sorafenib and may have received up to two prior systemic cancer therapies for HCC and had adequate liver function.
The trial was carried out at more than 100 sites in 19 countries.
As part of the CELESTIAL study, 773 patients were randomised in 2:1 ratio to receive 60mg of cabozantinib once daily or placebo.
Patients were arranged in a number of study arms based on etiology of the disease, geographic region, as well as the presence of extrahepatic spread and/or macrovascular invasion.
There was no crossover between the study arms.
The trial’s primary endpoint was overall survival (OS), while its secondary endpoints included objective response rate and progression-free survival.
Its exploratory endpoints comprised patient-reported outcomes, biomarkers and safety.
The CELESTIAL trial met its primary endpoint of OS after cabozantinib provided a statistically significant and clinically meaningful improvement in median OS compared to placebo in the enrolled patients.
Its median OS was 10.2 months with cabozantinib versus eight months with placebo.
In addition, the median progression-free survival (PFS) more than doubled, at 5.2 months with cabozantinib and 1.9 months with placebo.
Objective response rates measured per response evaluation criteria in solid tumours (RECIST) 1.1 were 4% with cabozantinib and 0.4% with placebo.
The trial also reported a 64% disease control response in patients treated with cabozantinib compared with 33% of patients in the placebo group.
Its safety data were reported to be consistent with the established profile of cabozantinib.
The trial’s independent data monitoring committee suggested Ipsen to stop trial for efficacy following review of the second planned interim analysis.