Study to Evaluate Imetelstat (GRN163L) in Participants With International Prognostic Scoring System (IPSS) Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)
NCT ID: NCT02598661
Last Updated: 2026-01-05
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
ACTIVE_NOT_RECRUITING
PHASE2/PHASE3
289 participants
INTERVENTIONAL
2016-01-12
2026-10-13
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
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.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Expanded Access for Treatment With Imetelstat
NCT05937568
Pentostatin and Lymphocyte Infusion in Preventing Graft Rejection in Patients Who Have Undergone Donor Stem Cell Transplant
NCT00096161
Adoptive Immunotherapy in Relapsed Hematological Malignancy: Early GVHD Prophylaxis
NCT02593123
Alemtuzumab and Glucocorticoids in Treating Newly Diagnosed Acute Graft-Versus-Host Disease in Patients Who Have Undergone a Donor Stem Cell Transplant
NCT00410657
Selective T-Cell Depletion to Reduce GVHD (Patients) Receiving Stem Cell Tx to Treat Leukemia, Lymphoma or MDS
NCT00025662
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
* Phase 2 is an open-label, single-arm design to assess the efficacy and safety of imetelstat sodium. A total of 57 participants were enrolled in Phase 2, including the expansion cohort.
* Phase 3 is a double-blind, randomized design to compare the efficacy of imetelstat sodium with placebo. In the Phase 3 study, 178 participants were enrolled and randomized in a 2:1 ratio to receive either imetelstat sodium or placebo, respectively.
* In a separate Ventricular Repolarization (VR) Substudy (QTc Substudy), 54 participants were enrolled and randomized 2:1 to receive either imetelstat sodium or placebo. If after a minimum of 2 treatment cycles in the VR substudy, a participant has no significant change to packed red blood cell (pRBC) transfusion burden or evidence of clinical benefit per Investigator, after discussion with the Sponsor the participant may be unblinded. If the participant was on placebo treatment, he/she may be permitted to start treatment with imetelstat sodium.
The Extension Phase was initiated at the end of the Phase 3 study (24 months after the last participant was randomized in the Phase 3) and will continue until participants who entered Phase 3 study participated in the study for up to 5 years from the first dose of imetelstat sodium (including treatment and follow-up), or 3 years of post-treatment follow-up from the last dose of study treatment, whichever occurs later, or until death, withdrawal of consent, study termination, or until a participant is lost to follow-up. Participants ongoing on imetelstat sodium and considered to be benefiting from treatment per Investigator in the Phase 3 Study or Ventricular Repolarization Substudy, have the option to continue receiving imetelstat sodium in the Extension Phase. Participants in the follow-up phase for the Phase 3 study or Ventricular Repolarization Substudy have the option to continue the follow-up in the Extension Phase.
The Phase 2, Phase 3, and VR Substudy all 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 Extension Phase of the study will consist of an extended treatment phase and an extended follow-up phase which will continue until death, lost to follow-up, withdrawal of consent, or the End of the Study (whichever occurs first).
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Phase 2: Imetelstat Sodium
Imetelstat sodium administered intravenously (IV), at a starting dose of 7.5 milligrams per kilogram (mg/kg), every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2.
Imetelstat Sodium
Imetelstat sodium IV infusion.
Phase 3: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Imetelstat Sodium
Imetelstat sodium IV infusion.
Phase 3: Placebo
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Placebo
Imetelstat sodium-matching placebo IV infusion.
QTc Substudy: Imetelstat Sodium
Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first.
Imetelstat Sodium
Imetelstat sodium IV infusion.
QTc Substudy: Placebo
Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. If after a minimum of 2 treatment cycles a participant has no significant change to pRBC transfusion burden or evidence of clinical benefit per Investigator, after discussion with the Sponsor the participant, he/she may be permitted to start treatment with imetelstat sodium.
Placebo
Imetelstat sodium-matching placebo IV infusion.
Extension Phase: Imetelstat Sodium
Participants randomized to the imetelstat sodium arm in the Phase 3 and the VR QTc Substudy, based on the response will continue to receive imetelstat sodium IV, at the dose they were receiving in the Phase 3 or VR QTc Substudy, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, study termination, or up to 3 years whichever occurs first.
Imetelstat Sodium
Imetelstat sodium IV infusion.
Extension Phase: Extended Follow-up
Participants randomized to the placebo arm in the Phase 3 study will enter the Extended Follow-up part of the Extension Phase and continue in follow up until death, lost to follow-up, withdrawal of consent, study termination, or whichever occurs first up to approximately 3 years.
Placebo
Imetelstat sodium-matching placebo IV infusion.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Imetelstat Sodium
Imetelstat sodium IV infusion.
Placebo
Imetelstat sodium-matching placebo IV infusion.
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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
Exclusion Criteria
* 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
* 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
18 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Geron Corporation
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Tymara Berry, MD
Role: STUDY_DIRECTOR
Geron Corporation
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
UAB Comprehensive Cancer Center
Birmingham, Alabama, United States
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
AZ Sint-Jan Burgge-Oostende
Bruges, West-Vlaanderen, Belgium
Az Groeninge
Kortrijk, West-Vlaanderen, Belgium
AZ Klina
Brasschaat, , Belgium
Universitair Ziekenhuis Gent
Ghent, , Belgium
UZ Leuven - Campus Gasthuisberg
Leuven, , Belgium
Tom Baker Cancer Centre
Calgary, Alberta, Canada
University of Alberta Hospital - Hematology Research
Edmonton, Alberta, Canada
The Ottawa Hospital
Ottawa, Ontario, Canada
Sunnybrook Health Sciences Centre
Toronto, Ontario, Canada
Princess Margaret Hospital
Toronto, Ontario, Canada
Jewish General Hospital
Montreal, Quebec, Canada
Fakultni nemocnice Brno
Brno, Brno-město, Czechia
FN Hradec Kralove
Hradec Králové, Hradec Králové, Czechia
FN Kralovske Vinohrady
Prague, , Czechia
Vseobecna fakultni nemocnice v Praze
Prague, , Czechia
Hopital de l'Archet
Nice, Alpes-Maritimes, France
CHU Tours
Tours, Centre-Val de Loire, France
CHU de Limoges, Hopital Dupuytren
Limoges, Haute-Vienne, France
CHU de Grenoble - Hôpital Albe
La Tronche, Isère, France
CHRU Nancy Brabois
Vandœuvre-lès-Nancy, Meurthe-et-Moselle, France
CH Le Mans - HAEMATOLOGY
Le Mans, Sarthe, France
CHU de Poitiers
Poitiers, Vienne, France
Centre Hospitalier Universitai
Angers, , France
CHRU de Lille - Hopital Claude Huriez - Maladies du Sang
Lille, , France
CHU - Hôpital Saint Louis - H
Paris, Île-de-France Region, France
University Hospital Freiburg
Freiburg im Breisgau, Baden-Wurttemberg, Germany
Fachärztliche Gemeinschaftspraxis mit Schwerpunkt
Dresden, Saxony, Germany
University Hospital Leipzig
Leipzig, Saxony, Germany
Studienzentrum für Hämatologie, Onkologie,Diabetologie, Endoskopie und Fußambulanz
Aschaffenburg, , Germany
University Hospital Bonn
Bonn, , Germany
Universitatsklinikum Carl Gustav Carcus Dresden
Dresden, , Germany
Universitätsklinikum Düsseldorf
Düsseldorf, , Germany
Johannes Gutenberg Universität
Mainz, , Germany
The Edith Wolfson Medical Center
H̱olon, Central District, Israel
Meir Medical Center
Kfar Saba, Central District, Israel
Kaplan Medical Center
Rehovot, Hagalil Saint, Israel
Hadassah Medical Organization
Jerusalem, Jerusalem, Israel
Ha'Emek Medical Center
Afula, Northern District, Israel
Tel Aviv Sourasky Medical Center
Tel Aviv, Tel Aviv, Israel
The Chaim Sheba Medical Center
Tel Litwinsky, Tel Aviv, Israel
Carmel MC
Haifa, , Israel
Rabin Medical Center, Beilinson Hospital
Petah Tikva, , Israel
A.O. Ospedale Niguarda Ca' Granda
Milan, Lombardy, Italy
Istituto Clinico Humanitas Rozzano, IRCCS
Rozzano, Milano, Italy
Irccs Crob
Rionero in Vulture, Potenza, Italy
A.O. Universitaria Policlinico Tor Vergata
Roma, Roma, Italy
AOU Ospedali Riuniti Umberto I G.M. Lancisi G. Salesi
Ancona, , Italy
AOU di Bologna Policlinico S. Orsola Malpighi
Bologna, , Italy
Azienda Ospedaliera Universitaria Careggi di Firenze
Florence, , Italy
Grande Ospedale Metropolitano 'Bianchi-Melacrino-Morelli' Reggio Calabria
Reggio Calabria, , Italy
AO S. Andrea, Università degli Studi di Roma La Sapienza
Roma, , Italy
Ospedale di Circolo, PO Varese
Varese, , Italy
Radboud Umcn
Nijmegen, Gelderland, Netherlands
Meander Medisch Centrum
Amersfoort, , Netherlands
VU Medisch Centrum
Amsterdam, , Netherlands
Universitair Medisch Centrum Groningen
Groningen, , Netherlands
Uniwersytecki Szpital Kliniczny im. J. Mikulicza-Radeckiego
Wroclaw, Lower Silesian Voivodeship, Poland
Wojewódzki Szpital Specjalistyczny sp.z o.o.
Słupsk, Pomeranian Voivodeship, Poland
PRATIA Poznań
Poznan, Ul. Gryfińska 1, Poland
SPZOZ MSWiA z Warminsko - Mazurskim Centrum Onkologii
Olsztyn, Warmian-Masurian Voivodeship, Poland
Ars Medical sp. z o.o.
Piła, Wielkopolskie Województwo, Poland
Clinics of Samarskiy GMU
Samara, Volga, Russia
Emergency Hospital of Dzerzhinsk
Dzerzhinsk, , Russia
City Clinical Hospital
Moscow, , Russia
Nizhniy Novgorod Region Clinical Hospital
Nizhny Novgorod, , Russia
Ryazan Regional Clinical Hospital
Ryazan, , Russia
FGU-Russian Research Institut
Saint Petersburg, , Russia
Oncologic Dispensary No.2
Sochi, , Russia
Pusan National University Hospital - Hematology and Oncology
Seogu, Incheon, South Korea
Gachon University Gil Medical Center - oncology
Incheon, Incheon Gwang'Yeogsi, South Korea
Chonnam National University Hwasun Hospital
Hwasun, Jeollanam-do, South Korea
Seoul National University Hospital
Seoul, Seoul Teugbyeolsi, South Korea
Asan Medical Center
Seoul, , South Korea
Samsung Medical Center
Seoul, , South Korea
The Catholic University of Korea Seoul St. Mary's Hospital
Seoul, , South Korea
Severance Hospital, Yonsei Uni
Seoul, , South Korea
H.U.Pta.del Mar
Cadiz, Cádiz, Spain
Hospital Universitario Puerta de Hierro Majadahonda
Majadahonda, Madrid, Spain
Hospital Universitario Nuestra Señora de Valme
Seville, Sevilla, Spain
Hospital Universitario Doctor
Valencia, Valencia, Spain
Hospital de Cruces
Barakaldo, Vizcaya, Spain
Hosp. Univ. Germans Trias I Pujol
Badalona, , Spain
Hosp. Univ. Vall D Hebron
Barcelona, , Spain
Hosp. Gral. Univ. Gregorio Maranon
Madrid, , Spain
Hosp. Univ. La Paz
Madrid, , Spain
Hosp. Clinico Univ. de Salamanca
Salamanca, , Spain
Hospital Universitari i Politecnic La Fe
Valencia, , Spain
University Hospital in Basel
Basel, Basel-Stadt (de), Switzerland
Inselspital - Universitätsspital Bern
Bern, Canton of Bern, Switzerland
Kantonsspital St. Gallen - Onkologie/Hämatologie
Sankt Gallen, Canton of St. Gallen, Switzerland
Universitaetsspital Zuerich
Zurich, Canton of Zurich, Switzerland
Ankara University Medical Faculty - Hematology
Ankara, Anatolia, Turkey (Türkiye)
Ege Universitesi Tip Fakultesi - Hematology
Izmir, İzmir, Turkey (Türkiye)
Cukurova University Medical Faculty
Adana, , Turkey (Türkiye)
KNP "Cherkaskyi oblasnyi onkolohichnyi dyspanser Cherkaskoi
Cherkasy, Cherkasy Oblast, Ukraine
KZ "Miska bahatoprofilna klinichna likarnia No4", hematolohi
Dnipro, Dnipropetrovsk Oblast, Ukraine
Instytut patolohii krovi ta transfusiynoi medytsyny NAMN Ukr
Lviv, Lviv Oblast, Ukraine
The Leeds Teaching Hospitals NHS Trust
Leeds, Leeds, United Kingdom
Nottingham City Hospital - Clinical Haematology
Nottingham, Nottinghamshire, United Kingdom
Southampton University Hospital
Southampton, Southampton, United Kingdom
Aberdeen Royal Infirmary
Aberdeen, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
Kim N, Pulte ED, Ehrlich LA, Ionan AC, Haupert S, Vallejo J, Green F, Zheng N, Wang Y, Liu J, Blanco JG, Dorff SE, Booth B, Choe M, Gehrke B, Bhatnagar V, Theoret M, Pazdur R, De Claro RA, Norsworthy KJ. US Food and Drug Administration Approval Summary: Imetelstat for Selected Patients With Low- to Intermediate-1 Risk Myelodysplastic Syndromes With Transfusion-Dependent Anemia. J Clin Oncol. 2025 Dec 10;43(35):3760-3768. doi: 10.1200/JCO-25-01369. Epub 2025 Oct 24.
Platzbecker U, Santini V, Fenaux P, Sekeres MA, Savona MR, Madanat YF, Diez-Campelo M, Valcarcel D, Illmer T, Jonasova A, Belohlavkova P, Sherman LJ, Berry T, Dougherty S, Shah S, Xia Q, Sun L, Wan Y, Huang F, Ikin A, Navada S, Feller F, Komrokji RS, Zeidan AM. Imetelstat in patients with lower-risk myelodysplastic syndromes who have relapsed or are refractory to erythropoiesis-stimulating agents (IMerge): a multinational, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2024 Jan 20;403(10423):249-260. doi: 10.1016/S0140-6736(23)01724-5. Epub 2023 Dec 1.
Steensma DP, Fenaux P, Van Eygen K, Raza A, Santini V, Germing U, Font P, Diez-Campelo M, Thepot S, Vellenga E, Patnaik MM, Jang JH, Varsos H, Bussolari J, Rose E, Sherman L, Sun L, Wan Y, Dougherty S, Huang F, Feller F, Rizo A, Platzbecker U. Imetelstat Achieves Meaningful and Durable Transfusion Independence in High Transfusion-Burden Patients With Lower-Risk Myelodysplastic Syndromes in a Phase II Study. J Clin Oncol. 2021 Jan 1;39(1):48-56. doi: 10.1200/JCO.20.01895. Epub 2020 Oct 27.
Provided Documents
Download supplemental materials such as informed consent forms, study protocols, or participant manuals.
Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
63935937MDS3001
Identifier Type: OTHER
Identifier Source: secondary_id
EU CTIS number
Identifier Type: OTHER
Identifier Source: secondary_id
2015-002874-19
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
CR107947
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.