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

Running, not enrolling · Phase 2/Phase 3 · Has a placebo group

Conditions studied: Myelodysplastic Syndromes

In brief

The purpose of this study is to evaluate the efficacy and safety of imetelstat sodium in transfusion-dependent participants with low or intermediate-1 risk myelodysplastic syndrome (MDS) that is relapsed/refractory to erythropoiesis-stimulating agent (ESA) treatment in Phase 2 study and to compare the efficacy, in terms of red blood cell (RBC) transfusion independence (TI), of imetelstat sodium to placebo in transfusion-dependent participants with low or intermediate-1 risk MDS that is relapsed/refractory to ESA treatment in Phase 3 study. A separate Ventricular Repolarization Substudy (QTc Substudy) will evaluate the effect of imetelstat sodium on ventricular repolarization. An Extension Phase has been included to allow continued treatment for those participants who are benefitting from imetelstat sodium and to continue to evaluate the long-term safety, overall survival (OS), and disease progression, including progression to acute myeloid leukemia (AML) in transfusion-dependent participants with low or immediate-1 risk MDS that is relapsed/refractory to ESA treatment.

Key facts

Study ID

NCT02598661

Run by

Geron Corporation

People needed

289

Starts

2016-01-12

Expected to finish

2026-10-13

Last updated by the study team

2026-07-17

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Man or woman greater than or equal to (≥) 18 years of age
• Diagnosis of myelodysplastic syndrome (MDS) according to World Health Organization (WHO) criteria confirmed by bone marrow aspirate and biopsy within 12 weeks prior to Cycle 1 Day 1 (C1D1) (Phase 2) or randomization (Phase 3). In Ventricular Repolarization Substudy, diagnosis of MDS or myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T) according to WHO criteria confirmed by bone marrow aspirate and biopsy within 12 weeks prior to C1D1
• 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 may not qualify if…

• Participant has known allergies, hypersensitivity, or intolerance to imetelstat sodium 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 sodium
• 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
• 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)
• Phase 3: a) Prior treatment with a hypomethylating agent (example [eg], azacitidine, decitabine); b) Prior treatment with lenalidomide
• Additional Exclusion Criteria for the Ventricular Repolarization Substudy:
• Concurrent therapy with medications known to prolong the QT interval and have been associated with Torsade de pointes arrhythmia (TdP)
• Cardiac function abnormalities on screening ECG as follows:
• Resting heart rate outside of 50 to 100 beats per minute
• QT interval by Fridericia's correction method (QTcF) >470 millisecond (msec) (or QTcF >490 msec in the presence of a right bundle branch block or ventricular conduction delay [QRS >119 msec]), determined by central assessment based on the average value of a triplicate set of ECGs
• Diagnosed or suspected congenital long QT syndrome
• Family history of sudden unexpected death from cardiac-related causes if indicative of a pathogenic mutation of cardiac ion channels
• Family history of congenital long QT syndrome
• History of Mobitz II second degree or third degree heart block
• Implantable pacemaker or automatic implantable cardioverter defibrillator
• Complete left bundle branch block
• Chronic or persistent atrial arrhythmia including atrial fibrillation and atrial flutter
• History or presence of clinically relevant heart rhythm disturbances including atrial, junctional, re-entry, and ventricular tachycardia
• Unusual T-wave morphology (i.e., bifid T-wave) likely to interfere with QT measurements
• History or evidence for any of the following: severe or unstable angina, myocardial infarction, symptomatic congestive heart failure, arterial or venous thromboembolic events (example, pulmonary embolism, cerebrovascular accident including transient ischemic attacks) within 12 months prior to Cycle 1 Day 1, New York Heart Association (NYHA) Class II to IV heart disease
• Presence of uncontrolled hypertension (persistent systolic blood pressure [BP] ≥160 mmHg or diastolic BP ≥100 mmHg). Participants with a history of hypertension are permitted, provided that BP is controlled to within these limits by anti-hypertensive treatment
• Any skin condition likely to interfere with electrocardiographic electrode placement or adhesion
• History of thoracic surgery likely to cause abnormality of the electrical conduction through thoracic tissues

Where it is running

• Acrc/Arizona Clinical Research, Inc. — Tucson, Arizona, United States
• CBCC Global Research, Inc. — Bakersfield, California, United States
• UCLA Ronald Regan Medical Center — Los Angeles, California, United States
• Yale-New Haven Hospital (YNHH) - Smilow Cancer Hospital — New Haven, Connecticut, United States
• BRCR Medical Center — Plantation, Florida, United States
• University of South Florida (USF) - H. Lee Moffitt Cancer Center — Tampa, Florida, United States
• Franciscan Health — Indianapolis, Indiana, United States
• St. Agnes Healthcare, Inc — Baltimore, Maryland, United States
• Center for Cancer and Blood Disorders — Bethesda, Maryland, United States
• Washington University School of Medicine — St Louis, Missouri, United States
• University of New Mexico Cancer Center — Albuquerque, New Mexico, United States
• Icahn School of Medicine at Mount Sinai Program for the Protection of Human Subjects — New York, New York, United States
• Columbia University Medical Center — New York, New York, United States
• Weill Cornell Medical College-New York Presbyterian Hospital — New York, New York, United States
• Cleveland Clinic Taussig Cancer — Cleveland, Ohio, United States
• The Ohio State Comprehensive Cancer Center — Columbus, Ohio, United States
• Prairie lakes Healthcare system, Inc — Watertown, South Dakota, United States
• Vanderbilt University Medical - Hematology-Oncology — Nashville, Tennessee, United States
• Texas Oncology/Methodist Charlton Cancer Center — Dallas, Texas, United States
• Simmons Comprehensive Cancer Center — Dallas, Texas, United States
• Fred Hutchinson Cancer Research Center (FHCRC) — Seattle, Washington, United States
• ZAS Middelheim — Antwerp, Antwerpen, Belgium
• ZAS Cadix — Antwerp, Antwerpen, Belgium
• GZA Ziekenhuizen - Campus Sint — Wilrijk, Antwerpen, Belgium
• UAB Comprehensive Cancer Center — Birmingham, Alabama, United States

Full record on ClinicalTrials.gov

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