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

Sponsor
AOP Orphan Pharmaceuticals AG
Study ID
NCT06514807
Phase
PHASE3
Status
Active Not Recruiting

Conditions

  • Essential Thrombocythaemia

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

Study Details

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.

Key Dates

Start date
Dec 4, 2023
Status verified
Dec 2024
Primary completion
Mar 15, 2028
Completion
Mar 15, 2028

Study Design

Enrollment
117 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Ropeginterferon alfa-2b (tradename BESREMi®)
    Ropeginterferon alfa-2b (tradename BESREMi®) 250 micrograms/0.5 mL or 500 micrograms/0.5 ml solution for injection in pre-filled pen. Ropeginterferon alfa-2b will be administered subcutaneously every 2 weeks at a dose of 125 µg / 250 µg / 500 µg per injection (depends on the optimal disease response) for up to 36 months of treatment.

Primary Outcome Measure

Durable (for at least 3 months) peripheral blood count remission [ Time Frame: At month 12 ]