NCT01193699: PEGINVERA

An Open-label, Prospective, Multicentre, Phase I/II Dose Escalation Study to Determine the Maximum Tolerated Dose and to Assess the Safety and Efficacy of P1101, PEG-Proline-Interferon Alpha-2b in Patients With Polycythaemia Vera

Completed Phase 1/Phase 2 Last updated 29 January 2018

What this trial tests

Phase 1/Phase 2 trial testing PEG-P-INF alpha-2b (P1101) in Polycythemia Vera in 24 participants. Completed in 25 January 2018.

Timeline

1 August 2010

Primary endpoint
25 January 2018

25 January 2018

Quick facts

Lead sponsor	AOP Orphan Pharmaceuticals AG
Phase	Phase 1/Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	24
Start date	1 August 2010
Primary completion	25 January 2018
Estimated completion	25 January 2018
Sites	6 locations across Austria

Drugs / interventions tested

• PEG-P-INF alpha-2b (P1101) — full drug profile →

Conditions studied

• Polycythemia Vera — all drugs for Polycythemia Vera →

Sponsor

AOP Orphan Pharmaceuticals AG — full company profile →

Who can join

Adults 18 to 90, any sex, with Polycythemia Vera. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Maximum tolerated dose (MTD)
  Time frame: The incidence of dose limiting toxicities (DLTs), which define the MTD are assessed continously until achievement of MTD.
  The definition of MTD is based on a 3+3 dose escalation design. MTD is defined as the next lower dose of that dose which was considered to be untolerated (observed DLT frequency at least 2 out of 3 in one cohort or at least 2 out of six patients in 2 cohorts).

Sponsor's own description

The purpose of this study is the identification of the maximum tolerated dose (MTD) of the investigational medicinal product. Moreover the safety and tolerability will be assessed and an exploratory analysis of efficacy and biomarker modulation will be performed.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Ropeginterferon alfa-2b, a novel IFNα-2b, induces high response rates with low toxicity in patients with polycythemia vera.
   Gisslinger H, Zagrijtschuk O, Buxhofer-Ausch V, Thaler J, et al · · 2015 · cited 139× · PMID 26261238 · DOI 10.1182/blood-2015-04-637280
2. Molecular responses and chromosomal aberrations in patients with polycythemia vera treated with peg-proline-interferon alpha-2b.
   Them NC, Bagienski K, Berg T, Gisslinger B, et al · · 2015 · cited 44× · PMID 25545244 · DOI 10.1002/ajh.23928
3. Interferons in the treatment of myeloproliferative neoplasms.
   Vachhani P, Mascarenhas J, Bose P, Hobbs G, et al · · 2024 · cited 14× · PMID 38380373 · DOI 10.1177/20406207241229588
4. Germline genetic factors influence the outcome of interferon-α therapy in polycythemia vera.
   Jäger R, Gisslinger H, Fuchs E, Bogner E, et al · · 2021 · cited 14× · PMID 32814349 · DOI 10.1182/blood.2020005792
5. Polycythemia Vera Management and Challenges in the Community Health Setting.
   Gerds AT, Dao KH. · · 2017 · cited 7× · PMID 28095380 · DOI 10.1159/000454953
6. SOHO State of the Art Update and Next Questions: Novel Therapies for Polycythemia Vera.
   Masarova L, Chifotides HT. · · 2024 · cited 5× · PMID 38135633 · DOI 10.1016/j.clml.2023.11.004
7. A paradigm shift in the treatment of patients with polycythemia vera. The initial early use of recombinant interferon-alpha.
   Silver RT, Hasselbalch HC. · · 2026 · cited 1× · PMID 41951941 · DOI 10.1038/s41375-026-02882-w
8. Efficacy and safety of ropeginterferon alfa-2b in the treatment of polycythemia vera: a systematic review with single arm meta-analysis.
   Nada EA, Elfagieh MA, Abdelsalam F, Elrashedy AA, et al · · 2025 · PMID 41238945 · DOI 10.1007/s00277-025-06701-7