Ropeginterferon Alfa-2b in Patients With Polycythemia Vera (PV) Without Symptomatic Splenomegaly (ROPE)

Ropeginterferon Alfa-2b in Patients With Polycythemia Vera (PV) Without Symptomatic Splenomegaly: A Prospective, Longitudinal, Multicenter, Observational Study in Germany

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.

Study Overview

• Status: Recruiting
• Conditions: Polycythemia Vera

• Study Type: Observational
• Enrollment (Estimated): 200

Contacts and Locations

• Study Contact: Name: Laura Serrer; Phone Number: +49 761 15242-0; Email: rope@iomedico.com

Study Locations

• Germany
  • Lower Saxony
    • Hanover, Lower Saxony, Germany, 30161
      • Recruiting
      • Onkologisches Studienzentrum Dr. med. Ingo Zander & Dr. med. Eyck von der Heyde
      • Contact: Eyck von der Heyde, Dr.; Phone Number: +49511311660; Email: vdheyde@onkologie-am-raschplatz.de

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• 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).

Description

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

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	From Time of enrollment until month 36.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	From time of treatment start until end of study (max. 54 months after FPI)
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.	From time of treatment start until end of study (max. 54 months after FPI)
Effectiveness: Proportion of patients with platelet count ≤400 ×109/L	Platelet count (descriptive statistics and frequencies ≤400 vs. > 400 ×109/L)	From time of treatment start until end of study (max. 54 months after FPI)
Effectiveness: Proportion of patients with WBC count <10 ×109/L	WBC count (descriptive statistics and frequencies <10 vs. ≥10 ×109/L)	From time of treatment start until end of study (max. 54 months after FPI)
Effectiveness: Proportion of patients with HCT value <45%	HCT value (descriptive statistics and frequencies <45% vs. ≥45%)	From time of treatment start until end of study (max. 54 months after FPI)
Effectiveness: Proportion of patients without phlebotomy during course of study	Proportion of patients without phlebotomy from treatment start until respective time point	From time of treatment start until end of study (max. 54 months after FPI)
Drug safety	Incidence of serious adverse events (SAEs), adverse drug reactions (ADRs) and serious adverse drug reactions (SADRs) related to ropeginterferon alfa-2b as characterized by severity, and seriousness	From time of treatment start until end of study (max. 54 months after FPI)
Dosing	Dose intensity (average dose in µg/4 weeks) and dose over time per patient and overall	From start until end of treatment (max. 54 months after FPI)
Treatment discontinuation	Frequency of treatment discontinuation and reasons thereof	From start until end of treatment (max. 54 months after FPI)
(S)ADRs leading to permanent treatment discontinuation	Frequency of patients with (S)ADRs leading to permanent treatment discontinuation	From start until end of treatment (max. 54 months after FPI)
Symptom burden	Absolute values of single items regarding symptom burden during course of study until month 36.	From time of enrollment until month 36 after treatment start (max. 54 months after FPI)
Treatment reality: previous cytoreductive therapies	Frequency of distinct previous cytoreductive therapies (if applicable)	From time of treatment start until end of study (max. 54 months after FPI)
Treatment reality: Switch to ropeginterferon alfa-2b	Reason for switch to ropeginterferon alfa-2b (if applicable) (i.e., frequencies of answers per reasons in the physician´s questionnaire)	From time of treatment start until end of study (max. 54 months after FPI)
Treatment reality: parallel cytoreductive therapies	Frequency of parallel cytoreductive therapies (combinations)	From time of treatment start until end of study (max. 54 months after FPI)
Treatment reality: subsequent cytoreductive therapies	Frequency of subsequent cytoreductive therapies (switch to another therapeutic agent)	From time of treatment start until end of study (max. 54 months after FPI)

Collaborators and Investigators

• Sponsor: iOMEDICO AG
• Collaborators: AOP Orphan Pharmaceuticals Germany GmbH
• Investigators: Principal Investigator: Eyck von der Heyde, Dr., Onkologische Schwerpunktpraxis

Study record dates

Study Major Dates

• Study Start (Actual): December 3, 2024
• Primary Completion (Estimated): July 1, 2029
• Study Completion (Estimated): July 1, 2029

Study Registration Dates

• First Submitted: December 16, 2024
• First Submitted That Met QC Criteria: December 16, 2024
• First Posted (Actual): December 19, 2024

Study Record Updates

• Last Update Posted (Actual): January 9, 2026
• Last Update Submitted That Met QC Criteria: January 8, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Keywords: polycythemia vera; myeloproliferative neoplasm; symptom burden; Ropeginterferon alfa-2b
• Additional Relevant MeSH Terms: Neoplasms by Site; Neoplasms; Hematologic Diseases; Bone Marrow Diseases; Bone Marrow Neoplasms; Hematologic Neoplasms; Hemic and Lymphatic Diseases; Myeloproliferative Disorders; Polycythemia Vera
• Other Study ID Numbers: IOM-060513

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No