Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at UCLA
Dates
study started
estimated completion

Description

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.

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

Details

This is a Phase 2/3, multicenter 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.

Keywords

Myelodysplastic Syndromes Imetelstat GRN163L Relapsed/refractory to ESAs Transfusion dependent IMerge Preleukemia Syndrome Motesanib diphosphate

Eligibility

You can join if…

Open to people ages 18 years and up

  • Man or woman greater than or equal to (>=) 18 years of age
  • In Part 1, diagnosis of myelodysplastic syndrome (MDS) according to World Health Organization (WHO) criteria
  • International Prognostic Scoring System (IPSS) low Risk or intermediate-1 risk MDS
  • Red blood cell (RBC) transfusion dependent, defined as requiring at least 4 RBC units transfused over an 8-week period during the 16 weeks prior to Study Entry; pre-transfusion hemoglobin (Hb) should be less than or equal to 9.0 gram per deciliter (g/dL) to count towards the 4 units total
  • Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2

You CAN'T join if...

  • Participant has known allergies, hypersensitivity, or intolerance to imetelstat or its excipients
  • Participant has received an investigational drug or used an invasive investigational medical device within 30 days prior to Study Entry or is currently enrolled in an investigational study
  • Prior treatment with imetelstat
  • Have received corticosteroids greater than (>) 30 milligram per day (mg/day) prednisone or equivalent, or growth factor treatment within 4 weeks prior to study entry
  • a) Prior treatment with a hypomethylating agent (example [eg], azacitidine, decitabine); b) Prior treatment with lenalidomide; c) Has received an erythropoiesis-stimulating agent (ESA) or any chemotherapy, immunomodulatory, or immunosuppressive therapy within 4 weeks prior to study entry (8 weeks for long-acting ESAs)

Locations

  • UCLA Ronald Regan Medical Center accepting new patients
    Los Angeles California 90095 United States
  • CBCC Global Research, Inc accepting new patients
    Bakersfield California 93309 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Geron Corporation
ID
NCT02598661
Phase
Phase 2/3
Study Type
Interventional
Last Updated