Actelion has announced the launch of a clinical trial to evaluate the effectiveness of a pediatric formulation of Opsumit in delaying disease progression in children with pulmonary arterial hypertension.
Opsumit is an orally available endothelin receptor antagonist approved both in Europe and the U.S. for the treatment of PAH in adults.
The TOMORROW (pediaTric use Of Macitentan tO delay disease pProgRessiOn in PAH Worldwide) trial is a multicenter, controlled, and open-label assessment of the efficacy, safety and pharmacokinetics of Opsumit versus standard of care in children with PAH. If successful, it could lead to the first approved treatment for young PAH patients.
“Clinical studies in children come with big challenges, such as the influence of growth stage and body weight on the dosage scheme and on potential side effects. PAH is a rare disease in adults and even more so in children, so pediatric PAH physicians have to mostly rely on research data collected in adults when weighing up treatment options for their younger patients,” said Maurice Beghetti, head of Pediatric Cardiology at the University Hospital of Geneva, in a press release. “There is a significant medical need for showing the benefit of adequately adapted formulations and doses of PAH-specific medications in order to provide children with PAH with the most appropriate treatment. TOMORROW as a robust study assessing long-term treatment with the appropriate pediatric formulation of macitentan will deliver the sought-after data on all fronts.”
The primary efficacy endpoint of the TOMORROW trial is time to the first Clinical Event Committee-confirmed outcome event, including clinical worsening of PAH, hospitalization due to PAH, or atrial septostomy or Pott’s anastomosis.