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

Conditions

Essential Thrombocythaemia

Brief summary

Rate of disease response at month 12 according to modified ELN Criteria: 1. Durable (≥3 months) peripheral blood count remission (PLTs ≤400 x 10^9/L AND WBC <10 x 10^9/L), AND 2. Absence of haemorrhagic or thrombotic events and disease progression, AND 3. Absence or Non-progression in disease-related signs, AND 4. Durable (for at least 3 months) large symptoms improvement or maintenance of non-progression based on the MPN-SAF TSS

Detailed description

Response at month 9, 18, 24, 30 and 36, Longitudinal changes in the ELN response rates over 12 months, Time to first response (as defined by ELN criteria), Duration of first response (as defined by ELN criteria), Duration of first durable response (as defined by ELN criteria), Time to first peripheral blood count remission response, Duration of first durable peripheral blood count remission response, Occurrence of thromboembolic and bleeding events, Occurrence of disease progression, Symptomatic improvement assessed by EQ-5D-5L questionnaire, Symptomatic improvement assessed by the 10-item MPN-SAF TSS, Change in inflammation markers over time, Change in CALR, MPL, or JAK2 allelic burden over time

Jean‐Jacques Kiladjian, Francisca Ferrer‐Marín, Haifa Kathrin Al‐Ali, Alberto Álvarez‐Larrán, Eloise Beggiato et al. (41 authors)

Blood2024-11-05

10.1182/blood-2024-199028

ROP-ET: a prospective phase III trial investigating the efficacy and safety of ropeginterferon alfa-2b in essential thrombocythemia patients with limited treatment options.

Kiladjian JJ, Marin FF, Al-Ali HK, Alvarez-Larrán A, Beggiato E, Bieniaszewska M, Breccia M, Buxhofer-Ausch V, Cerna O, Crisan AM, Danaila CD, De Stefano V, Döhner K, Empson V, Gora-Tybor J, Griesshammer M, Grosicki S, Guglielmelli P, García-Gutierrez V, Heidel FH, Illés A, Tomuleasa C, James C, Koschmieder S, Krauth MT, Krejcy K, Lazaroiu MC, Mayer J, Nagy ZG, Nicolini FE, Palandri F, Pappa V, Reiter AJ, Sacha T, Schlager S, Schmidt S, Terpos E, Unger M, Wölfler A, Cirici BX, Klade C.

2024-03-0411 citations

10.1007/s00277-024-05665-4

Interventions

DRUGBesremi 500 micrograms/0.5 mL solution for injection in pre-filled pen

DRUGBesremi 250 micrograms/0.5 mL solution for injection in pre-filled pen

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Rate of disease response at month 12 according to modified ELN Criteria: 1. Durable (≥3 months) peripheral blood count remission (PLTs ≤400 x 10^9/L AND WBC <10 x 10^9/L), AND 2. Absence of haemorrhagic or thrombotic events and disease progression, AND 3. Absence or Non-progression in disease-related signs, AND 4. Durable (for at least 3 months) large symptoms improvement or maintenance of non-progression based on the MPN-SAF TSS	—

Measure

Time frame

Rate of disease response at month 12 according to modified ELN Criteria: 1. Durable (≥3 months) peripheral blood count remission (PLTs ≤400 x 10^9/L AND WBC <10 x 10^9/L), AND 2. Absence of haemorrhagic or thrombotic events and disease progression, AND 3. Absence or Non-progression in disease-related signs, AND 4. Durable (for at least 3 months) large symptoms improvement or maintenance of non-progression based on the MPN-SAF TSS

—

Secondary

Measure	Time frame
Response at month 9, 18, 24, 30 and 36, Longitudinal changes in the ELN response rates over 12 months, Time to first response (as defined by ELN criteria), Duration of first response (as defined by ELN criteria), Duration of first durable response (as defined by ELN criteria), Time to first peripheral blood count remission response, Duration of first durable peripheral blood count remission response, Occurrence of thromboembolic and bleeding events, Occurrence of disease progression, Symptomatic improvement assessed by EQ-5D-5L questionnaire, Symptomatic improvement assessed by the 10-item MPN-SAF TSS, Change in inflammation markers over time, Change in CALR, MPL, or JAK2 allelic burden over time	—

Measure

Time frame

Response at month 9, 18, 24, 30 and 36, Longitudinal changes in the ELN response rates over 12 months, Time to first response (as defined by ELN criteria), Duration of first response (as defined by ELN criteria), Duration of first durable response (as defined by ELN criteria), Time to first peripheral blood count remission response, Duration of first durable peripheral blood count remission response, Occurrence of thromboembolic and bleeding events, Occurrence of disease progression, Symptomatic improvement assessed by EQ-5D-5L questionnaire, Symptomatic improvement assessed by the 10-item MPN-SAF TSS, Change in inflammation markers over time, Change in CALR, MPL, or JAK2 allelic burden over time

—

Countries

Austria, Czechia, France, Germany, Greece, Hungary, Italy, Poland, Romania, Spain

Outcome results

None listed