Phase III results announced of PETIT2 study of eltrombopag (Promacta™/Revolade™) in paediatric patients with chronic immune thrombocytopen
GlaxoSmithKline (GSK) plc has announced the results from the Phase III PETIT2 study evaluating the efficacy of eltrombopag vs. placebo in paediatric patients with chronic immune (idiopathic) thrombocytopenic purpura (cITP). Eltrombopag - marketed as PromactaTM in the U.S. and as RevoladeTM in Europe and other countries across the world - met its primary endpoint, achieving a statistically significant improvement in platelet counts with almost 40 percent of patients treated with eltrombopag attaining a consistent platelet response for 6 of 8 weeks compared to placebo (39.7 percent vs. 3.4 percent, respectively, p<0.001).
The PETIT2 study results were highlighted today as part of a Press Briefing and Oral Presentation at the European Haematology Association Annual Congress, 12-15 June in Milan, Italy.
"The PETIT2 study results presented today show an increase in platelet response rate with eltrombopag treatment - an important result given that these children had failed other standard therapies," said Dr. Rafael Amado, Head of Oncology R&D, GlaxoSmithKline. "We look forward to continuing to assess the potential of eltrombopag in these patients and to moving forward with planned regulatory submissions for a paediatric indication in cITP later this year."
Efficacy results for PETIT2 were consistent across age cohorts. The safety profile was consistent with the established profile for eltrombopag and no new safety concerns were observed. The most common adverse events (AEs) occurring most frequently in the eltrombopag arm included nasopharyngitis, rhinitis, cough and respiratory tract infection. Grade 3/4 AEs occurred in 12.7 percent of patients treated with eltrombopag and 10.3 percent of patients in the placebo group. Serious AEs were reported in 8 percent of eltrombopag-treated patients vs. 14 percent in the placebo arm.
Additional results from an early phase study of eltrombopag in previously treated paediatric patients with cITP were also presented at this meeting.
Immune (idiopathic) thrombocytopenic purpura (ITP) - characterised by a low platelet count - affects as many as 5 in 100,000 children each year.1 While many children with ITP do not require treatment and/or their disease resolves, up to 30 percent of patients experience persistent disease at 12 months and are diagnosed with cITP.2,3,4 Patients with paediatric cITP are at a high risk of severe bleeding.
PETIT2 (TRA115450) was 2-part, double-blind, randomised placebo-controlled and open-label study to investigate the efficacy, safety and tolerability of eltrombopag in paediatric patients with previously treated cITP. The multi-centre study enrolled 93 subjects at 38 centres in 14 countries. The primary objective of the study was to assess the efficacy of eltrombopag, relative to placebo, in achieving platelet counts of ≥50 Gi/L among paediatric patients with previously treated cITP for at least 12 months. The initial phase of the study compared eltrombopag to placebo for 13 weeks. All study participants were then treated with eltrombopag in the second phase of the study (through to week 24).
About Eltrombopag (PromactaTM/RevoladeTM)
Eltrombopag is not approved or licensed anywhere in the world for use in chronic immune (idiopathic) thrombocytopenic purpura in the paediatric setting.
For full US Prescribing Information for Promacta® (eltrombopag), including Boxed Warning, please visit: https://www.gsksource.com/gskprm/htdocs/documents/PROMACTA-PI-MG-COMBINED.PDF. For the European Union (EU) Summary of Product Characteristics (SPC) for Revolade® (eltrombopag) in approved indications, please visit http://health.gsk.com/.