NCT01259817

Single Arm Salvage Therapy With Pegylated Interferon Alfa-2a for Patients With High Risk Polycythemia Vera or High Risk Essential Thrombocythemia Who Are Either Hydroxyurea Resistant or Intolerant or Have Had Abdominal Vein Thrombosis

Completed Phase 2 Last updated 10 January 2017

What this trial tests

Phase 2 trial testing PEGASYS in High Risk Polycythemia Vera in 135 participants. Completed in 1 December 2016.

Timeline

1 September 2011

Primary endpoint
1 December 2016

1 December 2016

Quick facts

Lead sponsor	Ronald Hoffman
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	135
Start date	1 September 2011
Primary completion	1 December 2016
Estimated completion	1 December 2016
Sites	21 locations across United States, Italy

Drugs / interventions tested

• PEGASYS — full drug profile →
• Aspirin — full drug profile →

Conditions studied

• High Risk Polycythemia Vera — all drugs for High Risk Polycythemia Vera →
• High Risk Essential Thrombocythemia — all drugs for High Risk Essential Thrombocythemia →

Sponsor

Ronald Hoffman — full company profile →

Who can join

18 and older, any sex, with High Risk Polycythemia Vera or High Risk Essential Thrombocythemia. 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.

• Evaluate the ability of Pegylated Interferon Alfa-2a to achieve Complete Response or Partial Response in patients with (1) high risk polycythemia vera or (2) high risk essential thrombocythemia or (3) splanchnic vein thrombosis
  Time frame: 4 years

Sponsor's own description

The aim of this research is to look at two conditions, Essential Thrombocythemia (ET) and Polycythemia Vera (PV). ET causes people to produce too many blood cells called platelets and PV causes too many platelets and red blood cells to be made. Platelets are particles which circulate in the blood stream and normally prevent bleeding and bruising. Having too many platelets in the blood increases the risk of developing blood clots, which can result in life threatening events like heart attacks and strokes. When the number of red blood cells is increased in PV this will slow the speed of blood flow in the body and increases the risk of developing blood clots.

It is important for patients with ET or PV who are at risk of blood clots to receive drugs which will minimize the risks of developing these blood clots but at the moment the investigators are not sure which drugs will best control the disorder.

The purpose of this study is to look at the effectiveness of giving patients who have been diagnosed with ET and PV a study drug regimen using Aspirin and PEGASYS (also known as Pegylated interferon alfa-2a, instead of the standard treatment drug called Hydroxyurea (or hydroxycarbamide or Hydroxyurea), for whom this drug may not be suitable. The drug may not be suitable either because it is not adequately controlling the number of blood cells or some specific side effects occur.

Publications & conference data

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

1. Pegylated interferon alfa-2a for polycythemia vera or essential thrombocythemia resistant or intolerant to hydroxyurea.
   Yacoub A, Mascarenhas J, Kosiorek H, Prchal JT, et al · · 2019 · cited 125× · PMID 31515250 · DOI 10.1182/blood.2019000428
2. Polycythemia Vera: An Appraisal of the Biology and Management 10 Years After the Discovery of JAK2 V617F.
   Stein BL, Oh ST, Berenzon D, Hobbs GS, et al · · 2015 · cited 52× · PMID 26324368 · DOI 10.1200/jco.2015.61.6474
3. Mutations in MPNs: prognostic implications, window to biology, and impact on treatment decisions.
   Shammo JM, Stein BL. · · 2016 · cited 29× · PMID 27913528 · DOI 10.1182/asheducation-2016.1.552
4. Symptom burden and quality of life in patients with high-risk essential thrombocythaemia and polycythaemia vera receiving hydroxyurea or pegylated interferon alfa-2a: a post-hoc analysis of the MPN-RC 111 and 112 trials.
   Mazza GL, Mead-Harvey C, Mascarenhas J, Yacoub A, et al · · 2022 · cited 20× · PMID 34971581 · DOI 10.1016/s2352-3026(21)00343-4
5. Historical views, conventional approaches, and evolving management strategies for myeloproliferative neoplasms.
   Stein BL, Gotlib J, Arcasoy M, Nguyen MH, et al · · 2015 · cited 19× · PMID 25870379 · DOI 10.6004/jnccn.2015.0058
6. Discovery of a signaling feedback circuit that defines interferon responses in myeloproliferative neoplasms.
   Saleiro D, Wen JQ, Kosciuczuk EM, Eckerdt F, et al · · 2022 · cited 18× · PMID 35365653 · DOI 10.1038/s41467-022-29381-7
7. Changes in peripheral blood lymphocytes in polycythemia vera and essential thrombocythemia patients treated with pegylated-interferon alpha and correlation with <i>JAK2</i>^V617F allelic burden.
   Kovacsovics-Bankowski M, Kelley TW, Efimova O, Kim SJ, et al · · 2015 · cited 16× · PMID 27708986 · DOI 10.1186/s40164-016-0057-y
8. 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