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

Phase 3

Recruiting

Conditions: myelodysplasia
Myelodysplastic Syndrome (MDS)
10018865

Registration Number: NL-OMON54841

Lead Sponsor: Geron Corporation

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: Not specified

Target Recruitment: 2

Inclusion Criteria

# Diagnosis of MDS according to WHO criteria confirmed by bone marrow aspirate
and biopsy within 12 weeks prior to Randomization
# IPSS low or intermediate-1 risk MDS
# RBC transfusion dependent
# Relapsed/refractory to ESA treatment
# ECOG performance status 0, 1 or 2

Exclusion Criteria

# Prior treatment with imetelstat, or known allergies, hypersensitivity or
intolerance to imetelstat or its excipients
# Prior treatment with a hypomethylating agent (eg, azacitidine, decitabine)
# Prior treatment with lenalidomide
# Any ESA, chemotherapy, immunomodulatory or immunosuppressive therapy,
corticosteroids (defined dosage, refer to protocol) or growth factor treatment
within 4 weeks prior to C1D1 (part 1) or Randomization (Part 2) (8 weeks for
long acting ESA)
# Prior history of hematopoietic stem cell transplant
# Anemia attributed to factors other than MDS
# Active systemic hepatitis infection requiring treatment
# Previously assessed as having IPSS intermediate-2 or high risk
# Del(5q) karyotype
# MDS/myeloproliferative neoplasm overlap syndrome

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The (primary) hypothesis is that imetelstat will improve the rate of RBC TI<br /><br>(transfusion independence) as compared to placebo in transfusion dependent<br /><br>subjects with low or intermediate-1 risk MDS that is relapsed/refractory to ESA<br /><br>treatment.<br /><br>The primary efficacy endpoint is the rate of RBC TI lasting at least 8 weeks.<br /><br>The 8-week RBC TI rate is defined as the proportion of subjects without any RBC<br /><br>transfusion during any consecutive 8 weeks starting from Study Day 1.</p><br>

Secondary Outcome Measures