A Study to Evaluate Imetelstat (JNJ-63935937) in Transfusion-Dependent Subjects with IPSS Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) that isRelapsed/Refractory to Erythropoiesis-Stimulating Agent(ESA) Treatment

Protocol: 
AAAQ4700
Phase: 
II/III

A Study to Evaluate Imetelstat (JNJ-63935937) in Transfusion-Dependent Subjects with IPSS Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) that isRelapsed/Refractory to Erythropoiesis-Stimulating Agent(ESA) Treatment

The purpose of the study is to evaluate the efficacy and safety of imetelstat for the treatment of patients with myelodysplastic syndrome.All patients participating in Part 1 of this study will get 7.5 mg/kg of imetelstat as a 2-hour intravenous infusion once every 4 weeks. The dose of imetelstat may be increased, held or decreased depending on how well imetelstat works to treat your MDS and whether or not you have certain side effects to treatment. Before receiving imetelstat, you will receive 25-50mg of diphenhydramine (an antihistamine) and 100-200 mg of hydrocortisone (a corticosteroid), or equivalent.

Are you Eligible? (Inclusion Criteria)

1) IPSS low Risk or intermediate-1 risk MDS
2) RBC transfusion dependent, defined as requiring 4 units RBC over 8 weeks during the 12 weeks prior to Study Entry (defined in Section 3.1); pre-transfusion Hb should be <=9.0 g/dL to count towards the 4 units total
3) Has MDS that is relapsed/refractory to ESA treatment; as defined by meeting any of the criteria
below:
- Received at least 8 weeks of treatment with a minimum weekly dose of epoetin alfa 40,000 U,
epoetin beta 30,000 U or darbepoetin alfa 150 mcg (or equivalent
agent/dose), without having achieved a Hb rise >=1.5 g/dL or decreased RBC
transfusion requirement by at least 4 units over 8 weeks;
- Transfusion dependence or reduction in Hb by >=1.5 g/dL after hematologic
improvement, in the absence of another explanation;
- Serum EPO level >500 mU/mL

Specialty Area(s)

Trial Location

Herbert Irving Comprehensive Cancer Center
161 Fort Washington Ave.
Herbert Irving Pavilion
New York, NY 10032
United States