NCT02598661

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Strong global presence with extensive site network
Enrollment
289

participants targeted

Target at P75+ for phase_2

Timeline
5mo left

Started Jan 2016

Longer than P75 for phase_2

Geographic Reach
17 countries

126 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress96%
Jan 2016Oct 2026

First Submitted

Initial submission to the registry

October 27, 2015

Completed
10 days until next milestone

First Posted

Study publicly available on registry

November 6, 2015

Completed
2 months until next milestone

Study Start

First participant enrolled

January 12, 2016

Completed
7.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 13, 2023

Completed
1.3 years until next milestone

Results Posted

Study results publicly available

February 14, 2025

Completed
1.7 years until next milestone

Study Completion

Last participant's last visit for all outcomes

October 13, 2026

Expected
Last Updated

January 5, 2026

Status Verified

September 1, 2025

Enrollment Period

7.8 years

First QC Date

October 27, 2015

Results QC Date

September 24, 2024

Last Update Submit

December 12, 2025

Conditions

Keywords

Myelodysplastic SyndromesImetelstat SodiumGRN163LRelapsed/refractory to ESAsTransfusion dependentIMerge

Outcome Measures

Primary Outcomes (3)

  • Phase 2: Percentage of Participants Without Any Red Blood Cell (RBC) Transfusion During Any Consecutive 8-Weeks Period (All Participants)

    Percentage of participants without any RBC transfusion during any consecutive 8 weeks (56 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2. The 95% confidence interval (CI) was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.

    Up to 5 years in Phase 2

  • Phase 2: Percentage of Participants Without Any RBC Transfusion During Any Consecutive 8-Weeks Period in Target Population

    Percentage of participants without any RBC transfusion during any consecutive 8 weeks (56 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2. The 95% confidence interval (CI) was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.

    Up to 5 years in Phase 2

  • Phase 3: Percentage of Participants Without Any RBC Transfusion During Any Consecutive 8-Weeks Period

    Percentage of participants without any RBC transfusion during any consecutive 8 weeks (56 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2. The 95% CI was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.

    Up to 3.7 years in Phase 3

Secondary Outcomes (21)

  • Phase 2 and Phase 3: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Deaths

    Adverse events: Up to 5 years in Phase 2 and up to 3.7 years in Phase 3; Deaths: Up to 6.6 years in Phase 2 and up to 4 years in Phase 3

  • Phase 2 and Phase 3: Percentage of Participants Without Any RBC Transfusion During Any Consecutive 24-Weeks Period

    Up to 5 years in Phase 2 and up to 3.7 years in Phase 3

  • Phase 2 and Phase 3: Time to the 8-Weeks RBC Transfusion Independence (TI)

    Up to 5 years in Phase 2 and up to 3.7 years in Phase 3

  • Phase 2 and Phase 3: Time to the 24-Weeks RBC TI

    Up to 5 years in Phase 2 and up to 3.7 years in Phase 3

  • Phase 2 and Phase 3: Duration of RBC TI

    Up to 5 years in Phase 2 and up to 3.7 years in Phase 3

  • +16 more secondary outcomes

Other Outcomes (3)

  • Extension Phase: Number of Participants With AEs

    Up to approximately 3 years in the extension phase

  • Extension Phase: Overall Survival

    Up to approximately 3 years in the extension phase

  • Extension Phase: Progression Free Survival (PFS) Survival

    Up to approximately 3 years in the extension phase

Study Arms (7)

Phase 2: Imetelstat Sodium

EXPERIMENTAL

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.

Drug: Imetelstat Sodium

Phase 3: Imetelstat Sodium

EXPERIMENTAL

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.

Drug: Imetelstat Sodium

Phase 3: Placebo

PLACEBO COMPARATOR

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.

Drug: Placebo

QTc Substudy: Imetelstat Sodium

EXPERIMENTAL

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.

Drug: Imetelstat Sodium

QTc Substudy: Placebo

PLACEBO COMPARATOR

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.

Drug: Placebo

Extension Phase: Imetelstat Sodium

EXPERIMENTAL

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.

Drug: Imetelstat Sodium

Extension Phase: Extended Follow-up

EXPERIMENTAL

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.

Drug: Placebo

Interventions

Imetelstat sodium IV infusion.

Also known as: GRN163L
Extension Phase: Imetelstat SodiumPhase 2: Imetelstat SodiumPhase 3: Imetelstat SodiumQTc Substudy: Imetelstat Sodium

Imetelstat sodium-matching placebo IV infusion.

Extension Phase: Extended Follow-upPhase 3: PlaceboQTc Substudy: Placebo

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

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
  • 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
  • +8 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (126)

UAB Comprehensive Cancer Center

Birmingham, Alabama, 35294, United States

Location

Acrc/Arizona Clinical Research, Inc.

Tucson, Arizona, 85715, United States

Location

CBCC Global Research, Inc.

Bakersfield, California, 93309, United States

Location

UCLA Ronald Regan Medical Center

Los Angeles, California, 90095, United States

Location

Yale-New Haven Hospital (YNHH) - Smilow Cancer Hospital

New Haven, Connecticut, 06510-3220, United States

Location

BRCR Medical Center

Plantation, Florida, 33326, United States

Location

University of South Florida (USF) - H. Lee Moffitt Cancer Center

Tampa, Florida, 33612, United States

Location

Franciscan Health

Indianapolis, Indiana, 46237-8601, United States

Location

St. Agnes Healthcare, Inc

Baltimore, Maryland, 21229, United States

Location

Center for Cancer and Blood Disorders

Bethesda, Maryland, 20817, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

University of New Mexico Cancer Center

Albuquerque, New Mexico, 87131, United States

Location

Icahn School of Medicine at Mount Sinai Program for the Protection of Human Subjects

New York, New York, 10029, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Weill Cornell Medical College-New York Presbyterian Hospital

New York, New York, 10065, United States

Location

Cleveland Clinic Taussig Cancer

Cleveland, Ohio, 44195, United States

Location

The Ohio State Comprehensive Cancer Center

Columbus, Ohio, 43210, United States

Location

Prairie lakes Healthcare system, Inc

Watertown, South Dakota, 57201, United States

Location

Vanderbilt University Medical - Hematology-Oncology

Nashville, Tennessee, 37232-6307, United States

Location

Texas Oncology/Methodist Charlton Cancer Center

Dallas, Texas, 75237, United States

Location

Simmons Comprehensive Cancer Center

Dallas, Texas, 75390, United States

Location

Fred Hutchinson Cancer Research Center (FHCRC)

Seattle, Washington, 98109-1024, United States

Location

ZAS Middelheim

Antwerp, Antwerpen, 2020, Belgium

Location

ZAS Cadix

Antwerp, Antwerpen, 2030, Belgium

Location

GZA Ziekenhuizen - Campus Sint

Wilrijk, Antwerpen, 2610, Belgium

Location

AZ Sint-Jan Burgge-Oostende

Bruges, West-Vlaanderen, 8000, Belgium

Location

Az Groeninge

Kortrijk, West-Vlaanderen, 8500, Belgium

Location

AZ Klina

Brasschaat, 2930, Belgium

Location

Universitair Ziekenhuis Gent

Ghent, 9000, Belgium

Location

UZ Leuven - Campus Gasthuisberg

Leuven, 3000, Belgium

Location

Tom Baker Cancer Centre

Calgary, Alberta, T2N 4N2, Canada

Location

University of Alberta Hospital - Hematology Research

Edmonton, Alberta, T6G 2R3, Canada

Location

The Ottawa Hospital

Ottawa, Ontario, K1G 8L6, Canada

Location

Sunnybrook Health Sciences Centre

Toronto, Ontario, M4N 3M5, Canada

Location

Princess Margaret Hospital

Toronto, Ontario, M5G 2L7, Canada

Location

Jewish General Hospital

Montreal, Quebec, H3T 1E2, Canada

Location

Fakultni nemocnice Brno

Brno, Brno-město, 625 00, Czechia

Location

FN Hradec Kralove

Hradec Králové, Hradec Králové, 500 05, Czechia

Location

FN Kralovske Vinohrady

Prague, 100 34, Czechia

Location

Vseobecna fakultni nemocnice v Praze

Prague, 128 08, Czechia

Location

Hopital de l'Archet

Nice, Alpes-Maritimes, 6202, France

Location

CHU Tours

Tours, Centre-Val de Loire, 37044, France

Location

CHU de Limoges, Hopital Dupuytren

Limoges, Haute-Vienne, 87042, France

Location

CHU de Grenoble - Hôpital Albe

La Tronche, Isère, 38700, France

Location

CHRU Nancy Brabois

Vandœuvre-lès-Nancy, Meurthe-et-Moselle, 54511, France

Location

CH Le Mans - HAEMATOLOGY

Le Mans, Sarthe, 72037, France

Location

CHU de Poitiers

Poitiers, Vienne, 86021, France

Location

Centre Hospitalier Universitai

Angers, 49100, France

Location

CHRU de Lille - Hopital Claude Huriez - Maladies du Sang

Lille, 59037, France

Location

CHU - Hôpital Saint Louis - H

Paris, Île-de-France Region, 75010, France

Location

University Hospital Freiburg

Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

Location

Fachärztliche Gemeinschaftspraxis mit Schwerpunkt

Dresden, Saxony, 1307, Germany

Location

University Hospital Leipzig

Leipzig, Saxony, 4107, Germany

Location

Studienzentrum für Hämatologie, Onkologie,Diabetologie, Endoskopie und Fußambulanz

Aschaffenburg, 63739, Germany

Location

University Hospital Bonn

Bonn, 53127, Germany

Location

Universitatsklinikum Carl Gustav Carcus Dresden

Dresden, 01307, Germany

Location

Universitätsklinikum Düsseldorf

Düsseldorf, 40225, Germany

Location

Johannes Gutenberg Universität

Mainz, 55131, Germany

Location

The Edith Wolfson Medical Center

H̱olon, Central District, 58100, Israel

Location

Meir Medical Center

Kfar Saba, Central District, 44281, Israel

Location

Kaplan Medical Center

Rehovot, Hagalil Saint, 7610001, Israel

Location

Hadassah Medical Organization

Jerusalem, Jerusalem, 9112001, Israel

Location

Ha'Emek Medical Center

Afula, Northern District, 1834111, Israel

Location

Tel Aviv Sourasky Medical Center

Tel Aviv, Tel Aviv, 49372, Israel

Location

The Chaim Sheba Medical Center

Tel Litwinsky, Tel Aviv, 5265601, Israel

Location

Carmel MC

Haifa, 3436212, Israel

Location

Rabin Medical Center, Beilinson Hospital

Petah Tikva, 4941492, Israel

Location

A.O. Ospedale Niguarda Ca' Granda

Milan, Lombardy, 20162, Italy

Location

Istituto Clinico Humanitas Rozzano, IRCCS

Rozzano, Milano, 20089, Italy

Location

Irccs Crob

Rionero in Vulture, Potenza, 85028, Italy

Location

A.O. Universitaria Policlinico Tor Vergata

Roma, Roma, 00133, Italy

Location

AOU Ospedali Riuniti Umberto I G.M. Lancisi G. Salesi

Ancona, 60020, Italy

Location

AOU di Bologna Policlinico S. Orsola Malpighi

Bologna, 40138, Italy

Location

Azienda Ospedaliera Universitaria Careggi di Firenze

Florence, 50134, Italy

Location

Grande Ospedale Metropolitano 'Bianchi-Melacrino-Morelli' Reggio Calabria

Reggio Calabria, 89124, Italy

Location

AO S. Andrea, Università degli Studi di Roma La Sapienza

Roma, 189, Italy

Location

Ospedale di Circolo, PO Varese

Varese, 21100, Italy

Location

Radboud Umcn

Nijmegen, Gelderland, 6525 GA, Netherlands

Location

Meander Medisch Centrum

Amersfoort, 3813 TZ, Netherlands

Location

VU Medisch Centrum

Amsterdam, 1081 HV, Netherlands

Location

Universitair Medisch Centrum Groningen

Groningen, 9713 GZ, Netherlands

Location

Uniwersytecki Szpital Kliniczny im. J. Mikulicza-Radeckiego

Wroclaw, Lower Silesian Voivodeship, 50-367, Poland

Location

Wojewódzki Szpital Specjalistyczny sp.z o.o.

Słupsk, Pomeranian Voivodeship, 76-200, Poland

Location

PRATIA Poznań

Poznan, Ul. Gryfińska 1, 60-192, Poland

Location

SPZOZ MSWiA z Warminsko - Mazurskim Centrum Onkologii

Olsztyn, Warmian-Masurian Voivodeship, 10-228, Poland

Location

Ars Medical sp. z o.o.

Piła, Wielkopolskie Województwo, 64-920, Poland

Location

Clinics of Samarskiy GMU

Samara, Volga, 443079, Russia

Location

Emergency Hospital of Dzerzhinsk

Dzerzhinsk, 606019, Russia

Location

City Clinical Hospital

Moscow, 129301, Russia

Location

Nizhniy Novgorod Region Clinical Hospital

Nizhny Novgorod, 603126, Russia

Location

Ryazan Regional Clinical Hospital

Ryazan, 390039, Russia

Location

FGU-Russian Research Institut

Saint Petersburg, 191024, Russia

Location

Oncologic Dispensary No.2

Sochi, 354057, Russia

Location

Pusan National University Hospital - Hematology and Oncology

Seogu, Incheon, 42941, South Korea

Location

Gachon University Gil Medical Center - oncology

Incheon, Incheon Gwang'Yeogsi, 21565, South Korea

Location

Chonnam National University Hwasun Hospital

Hwasun, Jeollanam-do, 58128, South Korea

Location

Seoul National University Hospital

Seoul, Seoul Teugbyeolsi, 03080, South Korea

Location

Asan Medical Center

Seoul, 05505, South Korea

Location

Samsung Medical Center

Seoul, 06351, South Korea

Location

The Catholic University of Korea Seoul St. Mary's Hospital

Seoul, 06591, South Korea

Location

Severance Hospital, Yonsei Uni

Seoul, 3722, South Korea

Location

H.U.Pta.del Mar

Cadiz, Cádiz, 11009, Spain

Location

Hospital Universitario Puerta de Hierro Majadahonda

Majadahonda, Madrid, 28222, Spain

Location

Hospital Universitario Nuestra Señora de Valme

Seville, Sevilla, 41014, Spain

Location

Hospital Universitario Doctor

Valencia, Valencia, 46017, Spain

Location

Hospital de Cruces

Barakaldo, Vizcaya, 48903, Spain

Location

Hosp. Univ. Germans Trias I Pujol

Badalona, 08916, Spain

Location

Hosp. Univ. Vall D Hebron

Barcelona, 08035, Spain

Location

Hosp. Gral. Univ. Gregorio Maranon

Madrid, 28007, Spain

Location

Hosp. Univ. La Paz

Madrid, 28046, Spain

Location

Hosp. Clinico Univ. de Salamanca

Salamanca, 37007, Spain

Location

Hospital Universitari i Politecnic La Fe

Valencia, 46026, Spain

Location

University Hospital in Basel

Basel, Basel-Stadt (de), 4031, Switzerland

Location

Inselspital - Universitätsspital Bern

Bern, Canton of Bern, 3010, Switzerland

Location

Kantonsspital St. Gallen - Onkologie/Hämatologie

Sankt Gallen, Canton of St. Gallen, 9007, Switzerland

Location

Universitaetsspital Zuerich

Zurich, Canton of Zurich, 8091, Switzerland

Location

Ankara University Medical Faculty - Hematology

Ankara, Anatolia, 6590, Turkey (Türkiye)

Location

Ege Universitesi Tip Fakultesi - Hematology

Izmir, İzmir, 35040, Turkey (Türkiye)

Location

Cukurova University Medical Faculty

Adana, 1330, Turkey (Türkiye)

Location

KNP "Cherkaskyi oblasnyi onkolohichnyi dyspanser Cherkaskoi

Cherkasy, Cherkasy Oblast, 18009, Ukraine

Location

KZ "Miska bahatoprofilna klinichna likarnia No4", hematolohi

Dnipro, Dnipropetrovsk Oblast, 49102, Ukraine

Location

Instytut patolohii krovi ta transfusiynoi medytsyny NAMN Ukr

Lviv, Lviv Oblast, 79044, Ukraine

Location

The Leeds Teaching Hospitals NHS Trust

Leeds, Leeds, LS9 7TF, United Kingdom

Location

Nottingham City Hospital - Clinical Haematology

Nottingham, Nottinghamshire, NG5 1PB, United Kingdom

Location

Southampton University Hospital

Southampton, Southampton, SO16 6YD, United Kingdom

Location

Aberdeen Royal Infirmary

Aberdeen, AB252ZL, United Kingdom

Location

Related Publications (3)

  • 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.

MeSH Terms

Conditions

Myelodysplastic SyndromesRecurrence

Interventions

imetelstatGRN163L peptide

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Results Point of Contact

Title
Study Director
Organization
Geron Corp.

Study Officials

  • Tymara Berry, MD

    Geron Corporation

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 27, 2015

First Posted

November 6, 2015

Study Start

January 12, 2016

Primary Completion

October 13, 2023

Study Completion (Estimated)

October 13, 2026

Last Updated

January 5, 2026

Results First Posted

February 14, 2025

Record last verified: 2025-09

Data Sharing

IPD Sharing
Will not share

Locations