A Pilot Open-Label Study of the Efficacy and Safety of Imetelstat (GRN163L) in Patients With DIPSS_plus Intermediate-2 or High Risk Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (Post-PV MF), or Post-Essential Thrombocythemia Myelofibrosis (Post-ET-MF).
I. To evaluate overall response rate.
I. To evaluate the safety and tolerability of imetelstat (imetelstat sodium) in
myelofibrosis (MF) (per common terminology criteria for adverse events, version 4.03).
II. To evaluate the efficacy of imetelstat in the reduction of spleen size, as measured by
physical examination (palpable distance from the left costal margin).
III. To evaluate the efficacy of imetelstat in inducing red blood cell
transfusion-independence in previously transfusion-dependent patients (per International
Working Group for Myelofibrosis Research and Treatment [IWG-MRT] criteria).
I. To evaluate the effect of imetelstat on bone marrow histology and karyotype. II. To
evaluate the effect of imetelstat on leukocytosis and thrombocytosis.
OUTLINE: Patients receive imetelstat sodium intravenously (IV) over 2 hours on day 1.
Treatment repeats every 21 days for up to 52 courses in the absence of disease progression
or unacceptable toxicity.
After completion of study treatment, patients are followed up every 6 months for 5 years.
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Overall response rate defined as a clinical improvement (CI), partial remission (PR), or complete remission (CR) according to the IWG-MRT consensus criteria
Will be estimated by the number of successes divided by the total number of evaluable patients. Ninety-five percent Duffy Santner confidence intervals for the true success proportion will be calculated.
Up to 27 weeks
Ayalew Tefferi, M.D.
United States: Food and Drug Administration
|Mayo Clinic||Rochester, Minnesota 55905|