Clinical trial NCT06743035: Ropeginterferon Alfa-2b in Patients With Polycythemia Vera (PV) Without Symptomatic Splenomegaly
Official title: Ropeginterferon Alfa-2b in Patients With Polycythemia Vera (PV) Without Symptomatic Splenomegaly: A Prospective, Longitudinal, Multicenter, Observational Study in Germany
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Polycythemia Vera
Keywords: Ropeginterferon alfa-2b
polycythemia vera
symptom burden
myeloproliferative neoplasm
Lead sponsor: iOMEDICO AG
Sponsor class: INDUSTRY
Collaborators: name: AOP Orphan Pharmaceuticals Germany GmbH
First posted: 2024-12-19
Enrollment: 200
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study 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).

Brief summary: 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: Inclusion Criteria:

* 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.)
* Dated signature of informed consent form
* Participation in Patient-Reported Outcome (PRO) assessment in German language and completion of questionnaire at time of study enrollment
* Other criteria according to current Summary of Product Characteristics

Exclusion Criteria:

* Participation in an interventional clinical trial (except follow-up)
* Other contraindications according to current Summary of Product Characteristics

Primary outcomes:
measure: Symptom Burden
timeFrame: From Time of enrollment until month 36.
Secondary outcomes:
measure: Effectiveness: Complete hematologic response (CHR) rate
timeFrame: From time of treatment start until end of study (max. 54 months after FPI)
measure: Effectiveness: Event-free survival (EFS)
timeFrame: From time of treatment start until end of study (max. 54 months after FPI)
measure: Effectiveness: Proportion of patients with platelet count ≤400 ×109/L
timeFrame: From time of treatment start until end of study (max. 54 months after FPI)
measure: Effectiveness: Proportion of patients with WBC count <10 ×109/L
timeFrame: From time of treatment start until end of study (max. 54 months after FPI)
measure: Effectiveness: Proportion of patients with HCT value <45%
timeFrame: From time of treatment start until end of study (max. 54 months after FPI)
measure: Effectiveness: Proportion of patients without phlebotomy during course of study
timeFrame: From time of treatment start until end of study (max. 54 months after FPI)
measure: Drug safety
timeFrame: From time of treatment start until end of study (max. 54 months after FPI)
measure: Dosing
timeFrame: From start until end of treatment (max. 54 months after FPI)
measure: Treatment discontinuation
timeFrame: From start until end of treatment (max. 54 months after FPI)
measure: (S)ADRs leading to permanent treatment discontinuation
timeFrame: From start until end of treatment (max. 54 months after FPI)
measure: Symptom burden
timeFrame: From time of enrollment until month 36 after treatment start (max. 54 months after FPI)
measure: Treatment reality: previous cytoreductive therapies
timeFrame: From time of treatment start until end of study (max. 54 months after FPI)
measure: Treatment reality: Switch to ropeginterferon alfa-2b
timeFrame: From time of treatment start until end of study (max. 54 months after FPI)
measure: Treatment reality: parallel cytoreductive therapies
timeFrame: From time of treatment start until end of study (max. 54 months after FPI)
measure: Treatment reality: subsequent cytoreductive therapies
timeFrame: From time of treatment start until end of study (max. 54 months after FPI)
Other outcomes:
Locations:
city: Hannover
country: Germany
facility: Onkologisches Studienzentrum Dr. med. Ingo Zander & Dr. med. Eyck von der Heyde
state: Niedersachsen
Study officials:
affiliation: Onkologische Schwerpunktpraxis
name: Eyck von der Heyde, Dr.
role: PRINCIPAL_INVESTIGATOR
References: