A multicenter study to assess efficacy, safety, and tolerability of ropeginterferon alfa-2b-njft in patients with essential thrombocythemia in the US and Canada: EXCEED-ET trial

No new drugs have been approved for essential thrombocythemia (ET) treatment since the anagrelide approval in 1997. Ropeginterferon alfa-2b-njft (ropeg) is approved for polycythemia vera, providing a rationale for its use in ET. Its current dosing schema requires dose up-titrations with 50 mcg every 2 weeks and takes approximately 20 weeks to reach a plateau. The goal of this study is to assess the efficacy and safety of ropeg in ET using a higher initial dose and accelerated titration (HDAC) regimen. This is a single-arm, multicenter study in the US and Canada. Patients with ET receive ropeg at 250 mcg on Day 0, 350 mcg at Week 2, and 500 mcg from Week 4 onward with flexibility of dose adjustment. The primary endpoint is: platelets ≤400×10 9 /L, white blood cells <10×10 9 /L, improvement or non-progression of spleen size or major symptoms, and absence of hemorrhagic or thrombotic events, at months 10 and 13. Secondary endpoints include molecular response, safety and tolerability. A total of 91 patients were enrolled with 77 (84.6%) patients in the US and 14 (15.4%) in Canada. The last patient was enrolled on March 28, 2024. JAK2 V617F was found in 52 (57.1%) patients while CALR and MPL mutations in 34 (37.4%) and 5 (5.5%), respectively. As of November 12, 2024, the discontinuation rate was 8.8%. The study results will be available in mid-2025. This study will provide efficacy, tolerability and safety, molecular response and quality of life data that will be critical in assessing ropeg for ET treatment. Clinical trial registration ClinicalTrials.gov , identifier NCT05482971.

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Publication Details

Journal
Frontiers in Medicine
Published
2025-04-17
DOI
https://doi.org/10.3389/fmed.2025.1548590
Citations
2
Primary Topic
Myeloproliferative Neoplasms: Diagnosis and Treatment
Type
article
Field-Weighted Citation Impact
3.25

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article

A multicenter study to assess efficacy, safety, and tolerability of ropeginterferon alfa-2b-njft in patients with essential thrombocythemia in the US and Canada: EXCEED-ET trial

Stephen T. Oh, Fengshuo Lan, Albert Qin, Sunil Babu et al.
2 citations
Frontiers in Medicine
Myeloproliferative Neoplasms: Diagnosis and Treatment
3.25
article

A multicenter study to assess efficacy, safety, and tolerability of ropeginterferon alfa-2b-njft in patients with essential thrombocythemia in the US and Canada: EXCEED-ET trial

Stephen T. Oh, Fengshuo Lan, Albert Qin, Sunil Babu, Prithviraj Bose, Nikolai Alexandrovich Podoltsev, Jennifer Lucas, Joan How, Anthony Michael Hunter, Sonia Cerquozzi, Julia Cunningham, Pankit J. Vachhani, Brandi Reeves, Christopher Hillis, Swati Goel, Oleh Zagrijtschuk, Firas El Chaer, Casey Lee O'Connell, Bruno Fang, Tsewang Tashi, Lynda Foltz, Abdulraheem Yacoub, Ghaith F. Abu-Zeinah, Salman Otoukesh, Lucia Masárová, Dawn Maze, Anna B. Halpern, Zhuoyan Li, Henry Castro, Lindsay Rein, Hana Safah
article en
2 citations

Abstract

No new drugs have been approved for essential thrombocythemia (ET) treatment since the anagrelide approval in 1997. Ropeginterferon alfa-2b-njft (ropeg) is approved for polycythemia vera, providing a rationale for its use in ET. Its current dosing schema requires dose up-titrations with 50 mcg every 2 weeks and takes approximately 20 weeks to reach a plateau. The goal of this study is to assess the efficacy and safety of ropeg in ET using a higher initial dose and accelerated titration (HDAC) regimen. This is a single-arm, multicenter study in the US and Canada. Patients with ET receive ropeg at 250 mcg on Day 0, 350 mcg at Week 2, and 500 mcg from Week 4 onward with flexibility of dose adjustment. The primary endpoint is: platelets ≤400×10 9 /L, white blood cells <10×10 9 /L, improvement or non-progression of spleen size or major symptoms, and absence of hemorrhagic or thrombotic events, at months 10 and 13. Secondary endpoints include molecular response, safety and tolerability. A total of 91 patients were enrolled with 77 (84.6%) patients in the US and 14 (15.4%) in Canada. The last patient was enrolled on March 28, 2024. JAK2 V617F was found in 52 (57.1%) patients while CALR and MPL mutations in 34 (37.4%) and 5 (5.5%), respectively. As of November 12, 2024, the discontinuation rate was 8.8%. The study results will be available in mid-2025. This study will provide efficacy, tolerability and safety, molecular response and quality of life data that will be critical in assessing ropeg for ET treatment. Clinical trial registration ClinicalTrials.gov , identifier NCT05482971.

Frontiers in MedicineVol. 12
University of North Carolina at Chapel Hill (US), University of Southern California (US), Greater Baltimore Medical Center (US), Albert Einstein College of Medicine (US), City Of Hope National Medical Center (US), Our Lady of the Lake Regional Medical Center (US), The University of Texas MD Anderson Cancer Center (US), University of British Columbia (CA), Emory University (US), University of Calgary (CA), Duke University (US), Georgetown University (US), University of Toronto (CA), Washington University in St. Louis (US), Cornell University (US), University of Utah (US), Huntsman Cancer Institute (US), Stony Brook University Hospital (US), University of Washington Medical Center (US), MedStar Georgetown University Hospital (US), Princess Margaret Cancer Centre (CA), Brunswick (United States) (US), University of Alabama at Birmingham (US), Yale University (US), Fort Wayne Medical Institute (US), Dana-Farber Cancer Institute (US), St. Paul's Hospital (CA), Cellular Therapeutics (United Kingdom) (GB), Custom Security Industries (Canada) (CA), Marin Endocrine Care and Research (US), Bridge Pharma (United States) (US), Weill Cornell Medicine (US), University of Virginia (US), McMaster University (CA)
Amgen, Bristol-Myers Squibb, Pfizer, AstraZeneca, Sanofi, Gilead Sciences, Celgene, FibroGen, Astex Pharmaceuticals, MEI Pharma, Arog Pharmaceuticals, Janssen Scientific Affairs, CTI Biopharma, Incyte, Association of Community Cancer Centers, Kiadis Pharma, MorphoSys, Italfarmaco, Genentech
Openalex Percentile: Top 9%
Myeloproliferative Neoplasms: Diagnosis and Treatment
3.25
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