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

Titre officiel

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

Sommaire:

L’objectif de cette étude est d’évaluer l’innocuité et la sécurité de l’imetelstat chez les participants ayant une dépendance transfusionnelle et présentant un syndrome myélodysplasique (SMD) à risque faible ou intermédiaire 1, qui est récidivant ou réfractaire au traitement par un agent stimulant l’érythropoïèse (ASE).

Description de l'essai

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.

Voir cet essai sur ClinicalTrials.gov

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