Imetelstat in intermediate-2 or high-risk myelofibrosis refractory to JAK inhibitor: IMpactMF phase III study design.
John O MascarenhasClaire N HarrisonJean-Jacques KiladjianRami S KomrokjiSteffen KoschmiederAlessandro M VannucchiTymara BerryDenise ReddingLaurie ShermanSouria DoughertyLixian PengLibo SunFei HuangYing WanFaye M FellerAleksandra RizoSrdan VerstovsekPublished in: Future oncology (London, England) (2022)
Imetelstat, a first-in-class telomerase inhibitor, demonstrated meaningful clinical benefit including a robust symptom response rate and potential overall survival benefit in IMbark, a phase II study in intermediate-2 or high-risk myelofibrosis (MF) patients who have relapsed after or are refractory to JAK inhibitors. We describe the rationale and design for the phase III trial, IMpactMF (NCT04576156), an open-label evaluation of imetelstat versus best available therapy, excluding JAK inhibitors, in MF patients refractory to JAK inhibitor. Imetelstat 9.4 mg/kg is administered as an intravenous infusion every 21 days. Primary objective is to assess overall survival. Secondary objectives include symptom and spleen responses, progression-free survival, clinical response assessment, bone marrow fibrosis reduction, safety and pharmacokinetics. Biomarker, cytogenetics and mutation analyses will be performed.
Keyphrases
- phase iii
- open label
- free survival
- end stage renal disease
- clinical trial
- phase ii study
- bone marrow
- ejection fraction
- newly diagnosed
- chronic kidney disease
- phase ii
- mesenchymal stem cells
- prognostic factors
- double blind
- acute lymphoblastic leukemia
- acute myeloid leukemia
- squamous cell carcinoma
- placebo controlled
- low dose
- high dose
- risk assessment
- radiation therapy
- smoking cessation
- cell therapy