IMerge Imetelstat in Myelodysplastic syndrome (MDS)
Research type
Research Study
Full title
A Study to Evaluate Imetelstat (JNJ-63935937) in Transfusion-Dependent Subjects with IPSS Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) that is Relapsed/Refractory to Erythropoiesis-Stimulating Agent (ESA) Treatment
IRAS ID
199829
Contact name
Andrew Grethlein
Contact email
Sponsor organisation
Geron Corporation
Eudract number
2015-002874-19
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 9 months, 0 days
Research summary
This is a multicentre study of imetelstat in transfusion dependent subjects with low or intermediate-1 risk myelodysplastic syndrome (MDS) that is relapsed/refractory to erythropoiesis stimulating agent (ESA) treatment. The study will consist of two parts. Part 1 is an open label, single arm design to assess the efficacy and safety of imetelstat, and will seek to enroll approximately 30 subjects. Part 2 is a double-blind, randomised, placebo-controlled design to compare the efficacy of imetelstat with placebo. The sponsor will review the data from Part 1, and if this suggests a satisfactory risk/benefit ratio, Part 2 will be initiated and will seek to enroll approximately 170 subjects.
It is expected that imetelstat will improve the rate of red blood cell transfusion independence (RBC TI) as compared to placebo in transfusion dependent subjects with low or intermediate- 1 risk MDS that is relapsed/refractory
to ESA treatment. Such patients currently have limited treatment options, with a median survival of 3.5 years, which is considerably shorter than predicted at time of diagnosis with MDS. Therefore, there remains a highly unmet need for effective treatments, and encouraging early clinical data suggest that imetelstat may provide benefit to this patient population.REC name
London - Brighton & Sussex Research Ethics Committee
REC reference
16/LO/0338
Date of REC Opinion
19 Apr 2016
REC opinion
Further Information Favourable Opinion