Randomized, Single-Blind, Multicenter Phase II Study of Two Doses of Imetelstat in Relapsed or Refractory Myelofibrosis.
John O MascarenhasRami S KomrokjiFrancesca PalandriBruno MartinoDietger NiederwieserAndreas ReiterBart L ScottMaria R BaerRonald HoffmanOlatoyosi OdenikeAlessandro M VannucchiJacqueline BussolariEugene ZhuEsther RoseLaurie ShermanSouria DoughertyLibo SunFei HuangYing WanFaye M FellerAleksandra RizoJean-Jacques KiladjianPublished in: Journal of clinical oncology : official journal of the American Society of Clinical Oncology (2021)
In this phase II study of two imetelstat doses, 9.4 mg/kg once every 3 weeks demonstrated clinical benefits in symptom response rate, with an acceptable safety profile for this poor-risk JAKi R/R population. Biomarker and bone marrow fibrosis assessments suggested selective effects on the malignant clone. A confirmatory phase III study is currently underway.
Keyphrases
- phase ii study
- open label
- phase iii
- placebo controlled
- double blind
- phase ii
- bone marrow
- clinical trial
- study protocol
- locally advanced
- acute lymphoblastic leukemia
- acute myeloid leukemia
- mesenchymal stem cells
- randomized controlled trial
- cross sectional
- radiation therapy
- gestational age
- liver fibrosis
- patient reported