Myelodysplastic Syndrome Clinical Trial

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

Summary

The purpose of this study is to evaluate the efficacy and safety of imetelstat in transfusion dependent participants with low or intermediate-1 risk myelodysplastic syndrome (MDS) that is relapsed/refractory to erythropoiesis-stimulating agent (ESA) treatment in Part 1 of the study and to compare the efficacy, in terms of red blood cell (RBC) transfusion independence (TI), of imetelstat to placebo in transfusion dependent participants with low or intermediate-1 risk MDS that is relapsed/refractory to ESA treatment in Part 2 of the study.

View Full Description

Full Description

This is a Phase 2/3, multicenter study of imetelstat that consists of 2 parts and approximately 270 participants may be enrolled.

Part 1 is an open-label, single-arm design to assess the efficacy and safety of imetelstat. Approximately 55 participants were enrolled in Part 1, including the expansion cohort, and be followed-up for safety, hematologic improvement and reduction in transfusion requirement.

Part 2 is a double-blind, randomized design to compare the efficacy of imetelstat with placebo. In the main study in Part 2, 178 participants were enrolled and randomized in a 2:1 ratio to receive either imetelstat or placebo, respectively.

In a separate Ventricular Repolarization substudy of Part 2, approximately 45 participants will be enrolled and randomized 2:1 to receive either imetelstat or placebo. If after a minimum of 2 treatment cycles in the Ventricular Repolarization substudy, a participant has no significant change to 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.

Each part of the study will 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 End of the Study is defined as 2 years after the study entry of the last participant in the main study of Part 2 or anytime the sponsor terminates the study, whichever comes first.

View Eligibility Criteria

Eligibility Criteria

Inclusion Criteria:

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) (Part 1) or randomization [Part 2 (Main Study)]. In Part 2 (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 known allergies, hypersensitivity, or intolerance to imetelstat 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
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)
Part 2 (Main Study): a) Prior treatment with a hypomethylating agent (example [eg], azacitidine, decitabine); b) Prior treatment with lenalidomide

Additional Exclusion Criteria for Part 2 (Ventricular Repolarization Substudy)

Concurrent therapy with medications known to prolong the QT interval and have been associated with Torsade de pointes arrythmia (TdP)

Cardiac function abnormalities on screening ECG as follows:

Resting heart rate outside of 50 to 100 beats per minute
QTcF >470 millisecond (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 bundle branch block or ventricular conduction delay (QRS >119 msec)
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

Study is for people with:

Myelodysplastic Syndrome

Phase:

Phase 2

Estimated Enrollment:

278

Study ID:

NCT02598661

Recruitment Status:

Recruiting

Sponsor:

Geron Corporation

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There are 132 Locations for this study

See Locations Near You

UAB Comprehensive Cancer Center
Birmingham Alabama, 35294, United States
Acrc/Arizona Clinical Research, Inc.
Tucson Arizona, 85715, United States
CBCC Global Research, Inc.
Bakersfield California, 93309, United States
UCLA Ronald Regan Medical Center
Los Angeles California, 90095, United States
Yale-New Haven Hospital (YNHH) - Smilow Cancer Hospital
New Haven Connecticut, 06510, United States
BRCR Medical Center
Plantation Florida, 33326, United States
University of South Florida (USF) - H. Lee Moffitt Cancer Center
Tampa Florida, 33612, United States
University of Chicago
Chicago Illinois, 60637, United States
Franciscan Health
Indianapolis Indiana, 46237, United States
St. Agnes Healthcare, Inc
Baltimore Maryland, 21229, United States
Center for Cancer and Blood Disorders
Bethesda Maryland, 20817, United States
Mayo Clinic Rochester
Rochester Minnesota, 55902, United States
Washington University School of Medicine
Saint Louis Missouri, 63110, United States
University of New Mexico Cancer Center
Albuquerque New Mexico, 87131, United States
Icahn School of Medicine at Mount Sinai Program for the Protection of Human Subjects
New York New York, 10029, United States
Columbia Presbyterian
New York New York, 10032, United States
Columbia University Medical Center
New York New York, 10032, United States
Weill Cornell Medical College-New York Presbyterian Hospital
New York New York, 10065, United States
Cleveland Clinic Taussig Cancer
Cleveland Ohio, 44195, United States
The Ohio State Comprehensive Cancer Center
Columbus Ohio, 43210, United States
Prairie lakes Healthcare system, Inc
Watertown South Dakota, 57201, United States
Tennessee Oncology at Centennial Medical Center
Nashville Tennessee, 37203, United States
Vanderbilt University Medical - Hematology-Oncology
Nashville Tennessee, 37232, United States
Texas Oncology/Methodist Charlton Cancer Center
Dallas Texas, 75237, United States
Simmons Comprehensive Cancer Center
Dallas Texas, 75390, United States
Fred Hutchinson Cancer Research Center (FHCRC)
Seattle Washington, 98109, United States
GZA Ziekenhuizen - Campus Sint
Wilrijk Antwerpen, 2610, Belgium
UZ Gent
Ghent Oost-Vlaanderen, 9000, Belgium
AZ Sint-Jan Burgge-Oostende
Brugge West-Vlaanderen, 8000, Belgium
Az Groeninge
Kortrijk West-Vlaanderen, 8500, Belgium
ZNA Middelheim
Antwerpen , 2020, Belgium
ZNA Stuyvenberg Antwerpen
Antwerpen , 2610, Belgium
AZ Klina
Brasschaat , 2930, Belgium
Algemeen Ziekenhuis Sint-Jan
Brugge , 8000, Belgium
Universitair Ziekenhuis Gent
Gent , 9000, Belgium
UZ Leuven - Campus Gasthuisberg
Leuven , 3000, Belgium
Tom Baker Cancer Centre
Calgary Alberta, T2N 4, Canada
University of Alberta Hospital - Hematology Research
Edmonton Alberta, T6G 2, Canada
The Ottawa Hospital
Ottawa Ontario, K1G 8, Canada
Sunnybrook Health Sciences Centre
Toronto Ontario, M4N 3, Canada
Princess Margaret Hospital
Toronto Ontario, M5G 2, Canada
Jewish General Hospital
Montréal Quebec, H3T 1, Canada
Fakultni nemocnice Brno
Brno Brno-město, 625 0, Czechia
FN Hradec Kralove
Hradec Králové , 50005, Czechia
FN Kralovske Vinohrady
Praha 10 , 100 3, Czechia
Vseobecna fakultni nemocnice v Praze
Praha 2 , 128 0, Czechia
Hopital de l'Archet
Nice Alpes-Maritimes, 6202, France
CHU Tours
Tours Centre, 37044, France
CHU de Limoges, Hopital Dupuytren
Limoges Haute-Vienne, 87042, France
CHU de Grenoble - Hôpital Albe
La Tronche Isère, 38700, France
CHRU Nancy Brabois
Vandœuvre-lès-Nancy Meurthe-et-Moselle, 54511, France
CH Le Mans - HAEMATOLOGY
Le Mans Sarthe, 72037, France
Hopital Paul Brousse - Aphp Hôpitaux Universitaires Paris Su
Villejuif Val-de-Marne, 94804, France
CHU de Poitiers
Poitiers Vienne, 86021, France
Centre Hospitalier Universitai
Angers , 49100, France
CHRU de Lille - Hopital Claude Huriez - Maladies du Sang
Lille , 59037, France
CHU - Hôpital Saint Louis - H
Paris Île-de-France, 75010, France
University Hospital Freiburg
Freiburg Baden-Württemberg, 79106, Germany
Fachärztliche Gemeinschaftspraxis mit Schwerpunkt
Dresden Sachsen, 1307, Germany
University Hospital Leipzig
Leipzig Sachsen, 4107, Germany
Studienzentrum für Hämatologie, Onkologie,Diabetologie, Endoskopie und Fußambulanz
Aschaffenburg , 63739, Germany
University Hospital Bonn
Bonn , 53127, Germany
Universitatsklinikum Carl Gustav Carcus Dresden
Dresden , 01307, Germany
Universitätsklinikum Düsseldorf
Duesseldorf , 40225, Germany
Johannes Gutenberg Universität
Mainz , 55131, Germany
Kaplan Medical Center
Reẖovot Hagalil Saint, 76100, Israel
The Edith Wolfson Medical Center
H̱olon HaMerkaz, 58100, Israel
Meir Medical Center
Kfar Saba HaMerkaz, 44281, Israel
Ha'Emek Medical Center
Afula HaZafon, 18341, Israel
Tel Aviv Sourasky Medical Center
Tel Aviv Tel-Aviv, 49372, Israel
The Chaim Sheba Medical Center
Tel HaShomer Tel-Aviv, 52656, Israel
Carmel MC
Haifa , 34362, Israel
Hadassah Medical Organization
Jerusalem , 91120, Israel
Rabin Medical Center, Beilinson Hospital
Petah Tikva , 49414, Israel
A.O. Ospedale Niguarda Ca' Granda
Milano Lombardia, 20162, Italy
Istituto Clinico Humanitas Rozzano, IRCCS
Rozzano Milano, 20089, Italy
Irccs Crob
Rionero In Vulture Potenza, 85028, Italy
AOU Ospedali Riuniti Umberto I G.M. Lancisi G. Salesi
Ancona , 60020, Italy
Policlinico Sant'Orsola Malpighi
Bologna , 40138, Italy
Azienda Ospedaliera Universitaria Careggi di Firenze
Firenze , 50134, Italy
IRCCS Policlinico San Matteo
Pavia , 27100, Italy
A.O. Ospedali Riuniti Marche Nord Presidio Ospedaliero S. Salvatore di Pesaro-Stabilim. di Muraglia
Pesaro , 61122, Italy
Grande Ospedale Metropolitano 'Bianchi-Melacrino-Morelli' Reggio Calabria
Reggio Calabria , 89124, Italy
A.O. Universitaria Policlinico Tor Vergata
Roma , 00133, Italy
AO S. Andrea, Università degli Studi di Roma La Sapienza
Roma , 189, Italy
Ospedale di Circolo, PO Varese
Varese , 21100, Italy
Gachon University Gil Medical Center - oncology
Incheon Incheon Gwang'yeogsi, 21565, Korea, Republic of
Pusan National University Hospital - Hematology and Oncology
Seogu Incheon, 42941, Korea, Republic of
Seoul National University Hospital
Seoul Seoul Teugbyeolsi, 03080, Korea, Republic of
Chonnam National University Hwasun Hospital
Hwasun South Jeolla, 58128, Korea, Republic of
Asan Medical Center
Seoul , 05505, Korea, Republic of
Samsung Medical Center
Seoul , 06351, Korea, Republic of
The Catholic University of Korea Seoul St. Mary's Hospital
Seoul , 06591, Korea, Republic of
Severance Hospital, Yonsei Uni
Seoul , 3722, Korea, Republic of
Radboud Umcn
Nijmegen Gelderland, 6525 , Netherlands
Meander Medisch Centrum
Amersfoort , 3813 , Netherlands
VU Medisch Centrum
Amsterdam , 1081 , Netherlands
Universitair Medisch Centrum Groningen
Groningen , 9713 , Netherlands
Uniwersytecki Szpital Kliniczny im. J. Mikulicza-Radeckiego
Wrocław Dolnoslaskie, 50-36, Poland
Centrum Medyczne Pratia Poznan
Skorzewo Kościerzyna, 60-18, Poland
Wojewódzki Szpital Specjalistyczny sp.z o.o.
Słupsk Pomorskie, 76-20, Poland
SPZOZ MSWiA z Warminsko - Mazurskim Centrum Onkologii
Olsztyn Warminsko-mazurskie, 10-22, Poland
Ars Medical sp. z o.o.
Piła Wielkopolskie Województwo, 64-92, Poland
Clinics of Samarskiy GMU
Samara Volga, 44307, Russian Federation
Emergency Hospital of Dzerzhinsk
Dzerzhinsk , 60601, Russian Federation
City Clinical Hospital
Moscow , 12930, Russian Federation
Nizhniy Novgorod Region Clinical Hospital
Nizhny Novgorod , 60312, Russian Federation
Ryazan Regional Clinical Hospital
Ryazan , 39003, Russian Federation
FGU-Russian Research Institut
Saint Petersburg , 19102, Russian Federation
Oncologic Dispensary No.2
Sochi , 35405, Russian Federation
H.U.Pta.del Mar
Cadiz Cádiz, 11009, Spain
Hospital Universitario Puerta de Hierro Majadahonda
Majadahonda Madrid, 28222, Spain
Hospital de Cruces
Baracaldo Vizcaya, 48903, Spain
Hosp. Univ. Germans Trias I Pujol
Badalona , 08916, Spain
Hosp. Univ. Vall D Hebron
Barcelona , 08035, Spain
Hosp. Clinic I Provincial de Barcelona
Barcelona , 08036, Spain
Hosp. Gral. Univ. Gregorio Maranon
Madrid , 28007, Spain
Hosp. Univ. La Paz
Madrid , 28046, Spain
Hosp. Clinico Univ. de Salamanca
Salamanca , 37007, Spain
Hospital Universitario Nuestra Señora de Valme
Sevilla , 41014, Spain
Hospital Universitario Doctor
Valencia , 46017, Spain
Hospital Universitari i Politecnic La Fe
Valencia , 46026, Spain
University Hospital in Basel
Basel Basel-Stadt (de), 4031, Switzerland
Kantonsspital St. Gallen - Onkologie/Hämatologie
Saint Gallen Sankt Gallen, 9007, Switzerland
Inselspital - Universitätsspital Bern
Bern , 3010, Switzerland
Universitaetsspital Zuerich
Zuerich , 8091, Switzerland
Ankara University Medical Faculty - Hematology
Ankara Anatolia, 6590, Turkey
Cukurova University Medical Faculty
Adana , 1330, Turkey
Ege Universitesi Tip Fakultesi - Hematology
İzmir , 35040, Turkey
KZ "Miska bahatoprofilna klinichna likarnia No4", hematolohi
Dnipropetrovs'k Dnipropetrovs'ka Oblast', 49102, Ukraine
Instytut patolohii krovi ta transfusiynoi medytsyny NAMN Ukr
Lviv L'vivs'ka Oblast', 79044, Ukraine
KNP "Cherkaskyi oblasnyi onkolohichnyi dyspanser Cherkaskoi
Cherkasy , 18009, Ukraine
Nottingham City Hospital - Clinical Haematology
Nottingham Nottinghamshire, NG5 1, United Kingdom
Aberdeen Royal Infirmary
Aberdeen , AB252, United Kingdom
The Leeds Teaching Hospitals NHS Trust
Leeds , LS9 7, United Kingdom
Southampton University Hospital
Southampton , SO16 , United Kingdom

How clear is this clinincal trial information?

Study is for people with:

Myelodysplastic Syndrome

Phase:

Phase 2

Estimated Enrollment:

278

Study ID:

NCT02598661

Recruitment Status:

Recruiting

Sponsor:


Geron Corporation

How clear is this clinincal trial information?

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