NCT06514807

A Study to Evaluate the Efficacy and Safety of Ropeginterferon Alfa-2b in Essential Thrombocythaemia Patients

A Phase III, Single Arm, Multicentre Study to Evaluate the Efficacy and Safety of Ropeginterferon Alfa-2b in Essential Thrombocythaemia Patients Who Are Intolerant or Refractory to or Not Eligible for Other Cytoreductive Treatments

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 117 (estimated)
• Sponsor: AOP Orphan Pharmaceuticals AG · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this study is to assess the efficacy of ropeginterferon alfa-2b in patients with ET who need cytoreductive treatment but are intolerant or refractory to, and/or ineligible for cytoreductive treatments approved and available for the treatment of ET (i.e., HU, ANA, BUS, and PB, when they are available and approved for ET treatment). Ropeginterferon alfa-2b is currently the only interferon authorised as a cytoreductive treatment of a myeloproliferative neoplasm (MPN), and the long-term treatment data from its comprehensive clinical development program show its efficacy in the induction of haematologic remission, resolution of disease-associated symptoms, disease-modifying effect, as well as its favourable safety profile (Gisslinger et al., 2020; Kiladjian et al. 2022). Available clinical data and experience show that ropeginterferon alfa-2b normalises various haematological parameters, including platelets. In addition, suppression of the malignant clone causing ET may be achieved, at least after long-term treatment, which is expected to possibly defer the onset of, or avoid long-term sequelae of ET.

Conditions

• Essential Thrombocythaemia

Interventions

Type	Name	Description
DRUG	Ropeginterferon alfa-2b (BESREMi®)	Ropeginterferon alfa-2b 250 micrograms/0.5 mL or 500 micrograms/0.5 ml solution for injection in pre-filled pen.

Timeline

• Start date: 2023-12-04
• Primary completion: 2028-03-15
• Completion: 2028-03-15
• First posted: 2024-07-23
• Last updated: 2024-12-19

Locations

36 sites across 10 countries: Austria, Czechia, France, Germany, Greece, Hungary, Italy, Poland, Romania, Spain