Study to Evaluate Imetelstat (GRN163L) in Subjects With International Prognostic Scoring System (IPSS) Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

Official Title

A Study to Evaluate Imetelstat (GRN163L) in Transfusion-Dependent Subjects With IPSS Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) That is Relapsed/Refractory to Erythropoiesis-Stimulating Agent (ESA) Treatment

Summary:

The purpose of this study is to evaluate the efficacy and safety of imetelstat in transfusion dependent participants with low or intermediate-1 risk myelodysplastic syndrome (MDS) that is relapsed/refractory to erythropoiesis-stimulating agent (ESA) treatment.

Trial Description

Primary Outcome:

• Percentage of participants without any red blood cell (RBC) transfusion during any consecutive 8 week period

Secondary Outcome:

• Number of Participants with Adverse Events (AEs)
• Percentage of participants without any red blood cell (RBC) transfusion during any consecutive 24 week period
• Time to the 8-week RBC transfusion independence (TI)
• Duration of RBC TI
• Percentage of Participants with hematologic improvement
• Percentage of Participants with Complete remission (CR) or Partial remission (PR) as Per International Working Group (IWG) Response Criteria 2006
• Overall survival
• Progression Free Survival (PFS)
• Time to Progression to Acute Myeloid Leukemia
• Percentage of Participants with Transfusion
• Amount of Transfusions
• Percentage of Participants receiving any myeloid growth factors
• Maximum Observed Plasma Concentration (Cmax)
• Area under the drug concentration-plasma time curve from time zero to last measurable concentration (AUC0-t)
• Percentage of Participants with antibodies to imetelstat
• Medical Resource Utilization Data in Part 2
• Assessment of Functional Assessment of Cancer Therapy- Anemia-Related Effects (FACT-An) in Part 2
• Assessment of EuroQol 5 Dimension Questionnaire (EQ-5D-5L) in Part 2
• Assessment of Quality of Life in Myelodysplasia Scale (QUALMS) in Part 2
• Assessment of Participant Global Impression of Change (PGIC) in Part 2
• Change From Baseline in QTc Interval at Day 1 of Part 2

This is a Phase 2/3, multicentre study of imetelstat that consists of 2 parts. Part 1 is an open-label, single-arm design to assess the efficacy and safety of imetelstat. Approximately 55 participants will be enrolled in Part 1, including the expansion cohort, and be followed-up for safety, hematologic improvement and reduction in transfusion requirement. Part 2 of the study will be initiated if data from Part 1 are supportive of a satisfactory benefit/risk profile. Part 2 is a double-blind, randomized design to compare the efficacy of imetelstat with placebo. Approximately 170 eligible participants will be randomized in a 2:1 ratio to receive either imetelstat or placebo, respectively. Each part of the study will consist of 3 phases: a Screening phase (up to 28 days); a treatment phase; and a post-treatment follow-up phase which will continue until death, lost to follow-up, withdrawal of consent, or the End of the Study (whichever occurs first). The End of the Study is defined as 2 years after the study entry of the last participant or anytime the sponsor terminates the study, whichever comes first.

View this trial on ClinicalTrials.gov