The purpose of the study is to identify the recommended Part 2 dose (R2PD) of imetelstat sodium in combination with ruxolitinib in participants with myelofibrosis (MF) in Part 1, and to evaluate the safety and preliminary clinical activity of the R2PD of imetelstat sodium in combination with ruxolitinib in participants with MF in Part 2.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
36
Imetelstat sodium will be administered as intravenous (IV) every 28 days.
Ruxolitinib will be administered, orally (PO), twice daily (BID) in cohort B as the standard of care per local prescribing guidelines.
City of Hope
Duarte, California, United States
RECRUITINGCity of Hope
Irvine, California, United States
RECRUITINGUniversity of Miami
Coral Gables, Florida, United States
Part 1: Incidence, Type, and Severity of Adverse Events, Including Dose-limiting Toxicity (DLT) During the DLT Observation Period and/or Study Treatment
Time frame: 28 days after first dose
Part 2: Number of Participants With Treatment-emergent Adverse Event (AE)
Safety will be assessed based on incidence and severity (according to Common Terminology Criteria for Adverse Events) of treatment emergent adverse events from the first dose of study treatment until 30 days after completion of treatment.
Time frame: First dose of study treatment until 30 days after the last dose of study treatment (up to approximately 5 years)
Part 2: Symptom Response Rate at Week 24
Symptom response rate is defined as percentage of participants with \>=50% reduction in the Total Symptom Score (TSS) measured by the Myelofibrosis Symptom Assessment Form (MFSAF) v4.0 e-diary at 24 week compared to baseline.
Time frame: Week 24
Part 1: Pharmacokinetic Profile of Ruxolitinib (Maximum Observed Plasma Concentration [Cmax]
Time frame: From first dose of Ruxolitinib treatment up to approximately 5 years
Part 1: Pharmacokinetic Profile of Ruxolitinib Time to Reach Maximum Plasma Concentration [Tmax])
Time frame: From first dose of imetelstat treatment up to approximately 5 years
Part 1 and Part 2: Pharmacokinetic Profile of Imetelstat Sodium Maximum Observed Plasma Concentration [Cmax]
Time frame: From first dose of imetelstat treatment up to approximately 5 years
Part 1 and Part 2: Pharmacokinetic Profile of Imetelstat Time to Reach Maximum Plasma Concentration [Tmax])
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H. Lee Moffitt Cancer Center and Research Institute, Inc.
Tampa, Florida, United States
RECRUITINGIcahn School of Medicine at Mount Sinai
New York, New York, United States
RECRUITINGTexas Oncology
Denison, Texas, United States
WITHDRAWNTexas Oncology
Tyler, Texas, United States
WITHDRAWNFred Hutchinson Cancer Center
Seattle, Washington, United States
RECRUITINGTime frame: From first dose of imetelstat treatment up to approximately 5 years
Part 1 and Part 2: Percentage of Participants with Anti-imetelstat Antibodies
Time frame: From first dose of imetelstat treatment up to approximately 5 years
Part 1: Symptom Response at Week 24
Symptom response rate is defined as percentage of participants with \>50% reduction in the TSS measured by the MFSAF v4.0 e-diary at 24 week compared to baseline.
Time frame: Baseline, Week 24
Part 1 and Part 2: Absolute Change From Baseline in TSS at Week 24
Absolute change is defined as change in total symptom score from baseline to week 24 as measured by the MFSAF v4.0 e-diary.
Time frame: Baseline, Week 24
Part 1 and Part 2: Average Absolute Change in TSS Over 24 weeks
Average absolute change in TSS is defined as change in the average of absolute change in total symptom score from week 1(baseline) to week 24 as measured by the MFSAF v4.0 e-diary.
Time frame: Baseline, Week 24
Part 1 and Part 2: Spleen Response at Week 24
Spleen response is the proportion of participants who achieve a reduction in spleen volume of ≥35% from baseline confirmed by magnetic resonance imaging (MRI) or computed tomography (CT).
Time frame: Week 24
Part 1 and Part 2: Progression Free Survival (PFS)
The time interval from start of study treatment date to the first date of disease progression or death from any cause, whichever occurs first.
Time frame: From start of study treatment date to the disease progression or death (up to approximately 5 years)
Part 1 and Part 2: Percentage of Participants With Complete Remission (CR), Partial Remission (PR), Clinical Improvement (CI) Per the Modified 2013 International Working Group - Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) Criteria.
Time frame: From first dose to end of the treatment (up to approximately 5 years)
Part 1 and Part 2: Time to Response
Time to response was defined as the duration from first dose of study treatment to the earliest date that a response is first documented. The response CI/CR/PR was assessed by IWG-MRT criteria.
Time frame: From first dose of study treatment to the earliest date that a response was first documented (Up to approximately 5 years)
Part 1 and Part 2: Duration of Response (DOR) Per IWG-MRT Criteria
DOR measured from time of initial response (CR/PR/CI) until documented PD or death whichever occurs first.
Time frame: From time of initial response to PD or death whichever occurs first (up to approximately 5 years)
Part 1 and Part 2: Reduction of Bone Marrow Fibrosis
Reduction of bone marrow fibrosis is defined as the percentage of participants with a post-baseline bone marrow fibrosis degree smaller than the baseline fibrosis degree prior to start of subsequent anticancer therapy.
Time frame: From first dose to end of the treatment (up to approximately 5 years)
Part 1 and Part 2: Time to Progression to Acute Myeloid Leukemia (AML)
Time to progression to AML, defined as the time interval from the start of study treatment date to the first date of documented progression to AML or death from any cause, whichever occurs first.
Time frame: From first dose to end of the treatment (up to approximately 5 years)