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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
117
Ropeginterferon alfa-2b 250 micrograms/0.5 mL or 500 micrograms/0.5 ml solution for injection in pre-filled pen.
University Hospital Graz, Department of Internal Medicine, Clinical Department of Hematology
Graz, Styria, Austria
Medical University Innsbruck, Department of Internal Medicine V (Hematology and Oncology)
Innsbruck, Tyrol, Austria
Ordensklinikum Linz GmbH Elisabethinen Hospital, Department of Internal Medicine I - Hemato-Oncology
Linz, Upper Austria, Austria
Medical University Vienna, Department of Internal Medicine I, Clinical Department of Hematology and Hemostaseology
Vienna, Austria
University Hospital Brno, Clinic of Internal Medicine - Hematology and Oncology
Brno, Czechia
Durable (for at least 3 months) peripheral blood count remission
PLTs ≤400 x 109/L AND WBC \<10 x 109/L
Time frame: At month 12
Absence of haemorrhagic or thrombotic events and absence of disease progression*
\*Progression is defined as conversion from asymptomatic to symptomatic splenomegaly or clinically relevant progression of spleen size at the Investigator's discretion, respectively.
Time frame: At month 12
Absence or Non-progression* in disease-related signs
\*Progression is defined as conversion from asymptomatic to symptomatic splenomegaly or clinically relevant progression of spleen size at the Investigator's discretion, respectively.
Time frame: At month 12
Durable (for at least 3 months) large symptoms improvement or maintenance of non-progression based on the Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)#
\# Large symptoms improvement for ET patients is defined as follows: * Baseline TSS ≥20: 10-points reduction in TSS * Baseline TSS 15-19, inclusive: 5-points reduction in TSS * Baseline TSS 10-14, inclusive: TSS decreases to ≤10 * Baseline TSS \<10: TSS stays \<10
Time frame: At month 12
Response at month 9, 18, 24, 30 and 36
Time frame: Month 9, 18, 24, 30 and 36
Longitudinal changes in the ELN response rates over 12 months
Time frame: up to 36 months
Time to first response (as defined by ELN criteria)
Time frame: up to 36 months
Duration of first response (as defined by ELN criteria)
Time frame: up to 36 months
Duration of first durable response (as defined by ELN criteria)
Time frame: up to 36 months
Time to first peripheral blood count remission response
Time frame: up to 36 months
Duration of first durable peripheral blood count remission response
Time frame: up to 36 months
Occurrence of thromboembolic and bleeding events response
Time frame: up to 36 months
Occurrence of disease progression response
Time frame: up to 36 months
Symptomatic improvement assessed by EQ-5D-5L questionnaire response
Time frame: up to 36 months
Symptomatic improvement assessed by the 10-item MPN-SAF TSS
Time frame: up to 36 months
Change in inflammation markers over time
Time frame: up to 36 months
Change in CALR, MPL, or JAK2 allelic burden over time
Time frame: up to 36 months
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University Hospital Kralovske Vinohrady, Clinic of Internal Hematology
Prague, Czechia
Centre Hospitalier Universitaire De Poitiers
Poitiers, Poitiers, France
Saint-Louis Hospital, Department of Adult Hematology
Paris, France
Bordeaux University Hospital, Haut-Leveque Hospital
Pessac, France
University Hospital Aachen, Clinic of Oncology, Hematology and Stem Cell Transplantation (Medical Clinic IV)
Aachen, Germany
...and 26 more locations