Ropeginterferon Alfa-2b in Patients With Polycythemia Vera (PV) Without Symptomatic Splenomegaly
ConditionPolycythemia Vera
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsoriOMEDICO AG
About this trial
The primary objective of this non interventional study is to evaluate symptom burden in adult patients with PV without symptomatic splenomegaly during treatment with ropeginterferon alfa-2b in a real-world setting. Further patient-relevant endpoints include effectiveness including complete hematologic response (CHR), event-free survival (EFS), safety and tolerability, treatment reality including dosing details as well as factors affecting treatment decision making.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age ≥18 years
Confirmed diagnosis of PV without symptomatic splenomegaly
Indication and decision for treatment with ropeginterferon alfa-2b in accordance with current SmPC
No prior treatment with ropeginterferon alfa-2b (Patients are allowed to be enrolled up to 6 weeks after their first dose of ropeginterferon alfa-2b but must still be on treatment at the time of enrollment.)
Disqualifiers
Participation in an interventional clinical trial (except follow-up)
Other contraindications according to current Summary of Product Characteristics
Trial population
Adult patients with polycythemia vera (PV) without symptomatic splenomegaly with the decision for treatment with ropeginterferon alfa 2b according to Summary of Product Characteristics (SmPC).
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
No trial groups listed
Trial outcomes
Primary outcomes
1
Symptom Burden
Absolute values of the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) total symptom score (TSS) at time of study enrollment, during course of study until month 36.
Time frame
From Time of enrollment until month 36.Secondary outcomes
1
Effectiveness: Complete hematologic response (CHR) rate
CHR rate is defined as the proportion of patients with * Hematocrit (HCT) \<45% and * Phlebotomy-free (absence of phlebotomy during the previous 3 months) and * Platelet count ≤400 x 10\^9/L and * white blood cell count ≤10 x 10\^9/L
Time frame
From time of treatment start until end of study (max. 54 months after FPI)2
Effectiveness: Event-free survival (EFS)
EFS is defined as the time from the first administration of ropeginterferon alfa-2b until the first occurrence of a thromboembolic event, disease progression to post-PV myelofibrosis, acute myeloid leukemia (AML) transformation, or death, whichever comes first. Patients without any event at the time point of analysis will be censored with their respective date of last contact.
Time frame
From time of treatment start until end of study (max. 54 months after FPI)3
Effectiveness: Proportion of patients with platelet count ≤400 ×109/L
Platelet count (descriptive statistics and frequencies ≤400 vs. \> 400 ×109/L)
Time frame
From time of treatment start until end of study (max. 54 months after FPI)4
Effectiveness: Proportion of patients with WBC count <10 ×109/L
WBC count (descriptive statistics and frequencies \<10 vs. ≥10 ×109/L)
Time frame
From time of treatment start until end of study (max. 54 months after FPI)Sponsors and contacts
Click on the lead sponsor to view all of their trials.
iOMEDICO AG
Lead sponsor
AOP Orphan Pharmaceuticals Germany GmbH
Collaborator
Source ClinicalTrials.gov