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SGI-110 in Adults With Untreated Acute Myeloid Leukemia (AML), Not Considered Candidates for Intensive Remission Induction

Phase 3
Completed
Conditions
Leukemia, Myeloid, Acute
Interventions
Drug: SGI-110 (guadecitabine)
Drug: Treatment Choice
Registration Number
NCT02348489
Lead Sponsor
Astex Pharmaceuticals, Inc.
Brief Summary

To compare efficacy and safety between SGI-110 and Treatment Choice in adults with previously untreated AML who are not considered candidates for intensive remission induction chemotherapy.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
815
Inclusion Criteria

Cytologically or histologically confirmed diagnosis of AML (except M3 acute promyelocytic leukemia) according to World Health Organization (WHO) classification.

Performance status (ECOG) of 0-3. Adults with previously untreated AML except for hydroxyurea or corticosteroids. Prior hydroxyurea or lenalidomide treatment for myelodysplastic syndrome (MDS) is allowed.

Not considered candidates for intensive remission induction chemotherapy at time of enrollment based on EITHER:

  1. ≥75 years of age OR
  2. <75 years of age with at least 1 of the following:

i. Poor performance status (ECOG) score of 2-3.

ii. Clinically significant heart or lung comorbidities, as reflected by at least 1 of:

  1. Left ventricular ejection fraction (LVEF) ≤50%.
  2. Lung diffusing capacity for carbon monoxide (DLCO) ≤65% of expected.
  3. Forced expiratory volume in 1 second (FEV1) ≤65% of expected.
  4. Chronic stable angina or congestive heart failure controlled with medication.

iii. Liver transaminases >3 × upper limit of normal (ULN).

iv. Other contraindication(s) to anthracycline therapy (must be documented).

v. Other comorbidity the investigator judges incompatible with intensive remission induction chemotherapy, which must be documented and approved by the study medical monitor before randomization.

Creatinine clearance as estimated by the Cockcroft-Gault (C-G) or other medically acceptable formulas ≥30 mL/min.

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Exclusion Criteria

Candidate for intensive remission induction chemotherapy at the time of enrollment.

Candidate for best supportive care only, ie, not a candidate for any active therapy with the TC comparators.

Known extramedullary central nervous system (CNS) AML.

Second malignancy currently requiring active therapy except breast or prostate cancer stable on or responding to endocrine therapy.

Prior treatment with decitabine or azacitidine.

Hypersensitivity to decitabine, SGI-110, or SGI-110 excipients.

Known active human immunodeficiency virus (HIV), hepatitis B virus (HBV), or hepatitis C virus (HCV) infection. Inactive hepatitis carrier status or low viral hepatitis titer on antivirals is allowed.

Known significant mental illness or other condition such as active alcohol or other substance abuse or addiction that, in the opinion of the investigator, predisposes the subject to high risk of noncompliance with the protocol.

Refractory congestive heart failure unresponsive to medical treatment; active infection resistant to all antibiotics; or advanced pulmonary disease requiring >2 liters per minute (LPM) oxygen.

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Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
SGI-110 (guadecitabine)SGI-110 (guadecitabine)Guadecitabine 60 mg/m\^2 administered subcutaneously (SC) daily for 5 days (Days 1-5) in 28-day cycles.
Treatment ChoiceTreatment ChoiceOne of the following treatment regimens: 20 mg cytarabine administered subcutaneously (SC) twice daily (BID) on Days 1-10 every 28 days; 20 mg/m\^2 decitabine given as a 1-hour intravenous (IV) infusion daily on Days 1-5 every 28 days; or 75 mg/m\^2 azacitidine given IV or SC daily on Days 1-7 every 28 days.
Primary Outcome Measures
NameTimeMethod
Number of Participants With a Complete Response (CR)Up to 38 months (median follow-up of 25.5 months)

Number of participants with a best response of CR assessed based on International Working Group 2003 acute myeloid leukemia (AML) response criteria by a blinded independent pathologist.

Overall SurvivalAt 676 death events (up to 38 months)

Survival time was defined as the number of days from the day the participant was randomly assigned to study treatment to the date of death, regardless of cause.

Secondary Outcome Measures
NameTimeMethod
Number of Red Blood Cell or Platelet TransfusionsMonth 6

The total number of red blood cells (RBCs) transfused or, separately, the total number of platelets transfused up to the 6-month time point for each participant was counted from the date of randomization to Day 180, the date of last contact, or date of death, whichever occurred earlier. One RBC or platelet transfusion was defined as one unit, and a single bag of RBCs or platelets was considered one unit.

Change in Health-related Quality of Life (QOL) Scores From Baseline: EQ-5D-5LBaseline to Month 6

EuroQol 5-level 5-dimension (EQ-5D-5L) descriptive scores were collected for each participant for a minimum of 6 months, unless the participant died or withdrew consent. Scores within each dimension for EQ-5D (mobility, self-care, usual activity, pain/discomfort, anxiety/depression) were calculated using counts and proportions. Mean change in scores from baseline are summarized in which an index score of 0 represents the worst health state and 1 represents the best health state.

Change in Health-related Quality of Life (QOL) Scores From Baseline: EQ-VASBaseline to Month 6

EuroQol-Visual Analogue Scale (EQ-VAS) descriptive scores were collected for each participant for a minimum of 6 months, unless the participant died or withdrew consent. A vertical 20-cm scale for EQ-VAS was used where the lowest value of 0 was labeled "the worst health you can imagine" and the top value of 100 was labeled "the best health you can imagine." Mean change in scores from baseline are summarized.

Number of Participants With Composite CR (CRc)Up to 38 months (median follow-up of 25.5 months)

CRc is reported as the number of participants with a best response of CR, complete response with incomplete platelet recovery (CRp), or complete response with incomplete blood count recovery (CRi).

Number of Days Alive and Out of the HospitalMonth 6

The date of each hospital admission and discharge was collected for each participant for up to 6 months, unless the participant died or withdrew consent prior to that time. Duration of each hospital stay in days was calculated as date of discharge minus date of admission. The Number of Days Alive and Out of the Hospital (NDAOH) was calculated as: NDAOH=180 - total duration of all hospital stays within 180 days from the first treatment - number of death days before Day 180. For subjects who were lost to follow-up within 6 months, the NDAOH was calculated conservatively assuming that the subject would have died the day after the last contact day.

Progression-free Survival (PFS)Up to 38 months (median follow-up of 25.5 months)

Progression-free survival was defined as the number of days from randomization to the earliest date of investigator's assessment of disease progression, participant receiving an alternative anti-leukemia therapy (including hematopoietic cell transplant), or relapse by peripheral blood (PB) assessment or blinded bone marrow (BM) assessment, whichever occurred first, or death, regardless of cause.

Duration of CRUp to 38 months (median follow-up of 25.5 months)

Duration of CR (in number of days) was calculated from the first time a CR was observed to the time of relapse, defined as the earliest time point whereby BM assessment or PB assessment indicated relapse/disease progression due to reappearance of leukemic blasts in PB or ≥ 5% leukemic blasts in BM.

Trial Locations

Locations (127)

Concord Repatriation General Hospital

🇦🇺

Concord, New South Wales, Australia

University of Chicago

🇺🇸

Chicago, Illinois, United States

Sunnybrook Health Sciences Centre

🇨🇦

Toronto, Ontario, Canada

CHRU de Limoges - Hôpital Dupuytren

🇫🇷

Limoges Cedex, Limousin, Lorraine, France

Universitaetsklinikum Freiburg

🇩🇪

Freiburg, Baden-wuerttemberg, Germany

MD Anderson Cancer Center

🇺🇸

Houston, Texas, United States

Rigshospitalet-Copenhagen University Hospital

🇩🇰

Copenhagen, Denmark

Hospital Universitari Germans Trias i Pujol

🇪🇸

Badalona, Barcelona, Spain

University of Minnesota Medical Center

🇺🇸

Minneapolis, Minnesota, United States

Duke Cancer Center

🇺🇸

Durham, North Carolina, United States

Skånes Universitetssjukhus i Lund

🇸🇪

Lund, Skane, Sweden

Taipei Veterans General Hospital

🇨🇳

Taipei, Taiwan

Medway Maritime Hospital

🇬🇧

Gillingham, Kent, United Kingdom

Vanderbilt University Medical Center

🇺🇸

Nashville, Tennessee, United States

Somogy Megyei Kaposi Mór Oktató Kórház

🇭🇺

Kaposvár, Hungary

Algemeen Ziekenhuis Sint-Jan

🇧🇪

Brugge, West-vlaanderen, Belgium

Debreceni Egyetem Klinikai Kozpont

🇭🇺

Debrecen, Hungary

GHR Mulhouse Sud-Alsace

🇫🇷

Mulhouse Cedex, Alsace, France

Universitair Medisch Centrum Utrecht

🇳🇱

Utrecht, Netherlands

Spitalul Clinic Judetean de Urgenta Tirgu-Mures

🇷🇴

Targu-Mures, Mures, Romania

China Medical University Hospital

🇨🇳

Taichung, Taiwan

Semmelweis Egyetem

🇭🇺

Budapest, Hungary

Mackay Memorial Hospital

🇨🇳

Taipei, Taiwan

UZ Gent

🇧🇪

Ghent, Oost-vlaanderen, Belgium

Wojewodzki Szpital Specjalistyczny im. M. Kopernika

🇵🇱

Lódz, Lodzkie, Poland

Institutul Regional de Oncologie Iasi

🇷🇴

Iasi, Romania

Sverdlovsk Regional Clinical Hospital #1

🇷🇺

Ekaterinburg, Russian Federation

Tom Baker Cancer Center

🇨🇦

Calgary, Alberta, Canada

Medizinische Universität Graz

🇦🇹

Graz, Styria, Austria

Austin Health

🇦🇺

Heidelberg, Victoria, Australia

National Taiwan University Hospital

🇨🇳

Taipei, Taiwan

Bacs-Kiskun Megyei Korhaz

🇭🇺

Kecskemét, Bacs-kiskun, Hungary

Instytut Hematologii i Transfuzjologii

🇵🇱

Warszawa, Mazowieckie, Poland

Samodzielny Publiczny Zaklad Opieki Zdrowotnej Zespól Szpitali Miejskich

🇵🇱

Chorzów, Slaskie, Poland

Ryazan Regional Clinical Hospital

🇷🇺

Ryazan, Russian Federation

Clinical Centre of Vojvodina

🇷🇸

Novi Sad, Serbia

Vancouver General Hospital

🇨🇦

Vancouver, British Columbia, Canada

King's College Hospital

🇬🇧

London, England, United Kingdom

IRCCS Centro di Riferimento Oncologico di Basilicata di Rionero in Vulture

🇮🇹

Rionero in Vulture, Potenza, Italy

Helsinki University Central Hospital

🇫🇮

Helsinki, Finland

Samodzielny Publiczny Szpital Kliniczny nr 1 w Lublinie

🇵🇱

Lublin, Lubelskie, Poland

Clinical Center of Serbia

🇷🇸

Belgrade, Serbia

Hospital Universitario Central de Asturias

🇪🇸

Oviedo, Asturias, Spain

Monash Medical Centre

🇦🇺

Clayton, Victoria, Australia

Hanusch Krankenhaus Wiener Gebietskrankenkasse

🇦🇹

Wien, Vienna, Austria

Szpital Wojewódzki w Opolu - Samodzielny Publiczny Zaklad Opieki Zdrowotnej

🇵🇱

Opole, Opolskie, Poland

Saratov State Medical University

🇷🇺

Saratov, Russian Federation

Chelsea and Westminster Hospital NHS Foundation Trust

🇬🇧

London, England, United Kingdom

Aarhus University Hospital

🇩🇰

Aarhus, Denmark

Azienda Ospedaliera Ospedali Riuniti Marche Nord

🇮🇹

Pesaro, Pesaro E Urbino, Italy

Azienda Ospedaliero-Universitaria di Bologna - Policlinico S.Orsola-Malpighi

🇮🇹

Bologna, Italy

Azienda Ospedaliero Universitaria S. Maria della Misericordia di Udine

🇮🇹

Udine, Italy

Mayo Clinic Cancer Center

🇺🇸

Rochester, Minnesota, United States

Medical College of Wisconsin

🇺🇸

Milwaukee, Wisconsin, United States

Weill Cornell Medical College

🇺🇸

New York, New York, United States

Scripps Cancer Center

🇺🇸

La Jolla, California, United States

University of Southern California

🇺🇸

Los Angeles, California, United States

John Theurer Cancer Center at Hackensack

🇺🇸

Hackensack, New Jersey, United States

Roswell Park Cancer Institute

🇺🇸

Buffalo, New York, United States

Columbia University Medical Center

🇺🇸

New York, New York, United States

Stony Brook University Medical Center

🇺🇸

Stony Brook, New York, United States

University Hospitals of Cleveland

🇺🇸

Cleveland, Ohio, United States

University Hospitals Monarch Medical Center

🇺🇸

Cleveland, Ohio, United States

Western Pennsylvania Hospital

🇺🇸

Pittsburgh, Pennsylvania, United States

Penn State Milton S. Hershey Medical Center

🇺🇸

Hershey, Pennsylvania, United States

Temple University

🇺🇸

Philadelphia, Pennsylvania, United States

Royal Adelaide Hospital

🇦🇺

Adelaide, South Australia, Australia

Grand Hôpital de Charleroi

🇧🇪

Charleroi, Hainaut, Belgium

UMHAT 'Sveti Georgi' EAD

🇧🇬

Plovdiv, Bulgaria

Multiprofile Hospital for Active Treatment "Sveta Marina'' EAD

🇧🇬

Varna, Bulgaria

The Ottawa Hospital

🇨🇦

Ottawa, Ontario, Canada

Princess Margaret Hospital

🇨🇦

Toronto, Ontario, Canada

Fakultní nemocnice Královské Vinohrady

🇨🇿

Praha 10, Czechia

Fakultní nemocnice Brno

🇨🇿

Brno, Jihormoravsky KRAJ, Czechia

Všeobecná fakultní nemocnice v Praze

🇨🇿

Praha 2, Praha, Czechia

Odense University Hospital

🇩🇰

Odense, Denmark

Tampere University Hospital

🇫🇮

Tampere, Southern Finland, Finland

Hôpital Hôtel-Dieu

🇫🇷

Nantes cedex 1, PAYS DE LA Loire, France

Centre Henri-Becquerel

🇫🇷

Rouen Cedex 1, Haute-normandie, France

Hôpital Saint Louis

🇫🇷

Paris Cedex 10, Ile-de-france, France

Institut Paoli Calmettes

🇫🇷

Marseille Cedex 9, Provence Alpes COTE D'azur, France

Centre Hospitalier Universitaire de Toulouse

🇫🇷

Toulouse cedex 9, Midi-pyrenees, France

Centre Hospitalier Universitaire Grenoble

🇫🇷

La Tronche, Rhone-alpes, France

Centre Hospitalier Lyon Sud

🇫🇷

Pierre Bénite Cedex, Rhone-alpes, France

Centre Antoine Lacassagne

🇫🇷

Nice, Provence Alpes COTE D'azur, France

Centre Léon Bérard

🇫🇷

Lyon Cedex 08, Rhone-alpes, France

Universitätsklinikum Ulm

🇩🇪

Ulm, Baden-wuerttemberg, Germany

Schwarzwald-Baar Klinikum Villingen-Schwenningen GmbH

🇩🇪

Villingen-Schwenningen, Baden-wuerttemberg, Germany

Universitätsklinikum Frankfurt Goethe Universität

🇩🇪

Frankfurt am Main, Hessen, Germany

Städtisches Klinikum Braunschweig gGmbH

🇩🇪

Braunschweig, Niedersachsen, Germany

Marien Hospital Düsseldorf GmbH

🇩🇪

Düsseldorf, Nordrhein-westfalen, Germany

Azienda Ospedaliero-Univesitaria San Luigi Gonzaga

🇮🇹

Orbassano, Torino, Italy

Azienda Ospedaliera SS. Antonio E. Biagio E. Cesare Arrigo di Alessandria

🇮🇹

Alessandria, Italy

Azienda Ospedaliera Ospedale di Busto Arsizio

🇮🇹

Busto Arsizio, Italy

Azienda Ospedaliera-Universitaria Vittorio Emanuele-Ferrarotto-Santo Bambino

🇮🇹

Catania, Italy

IRCCS Azienda Ospedaliera Universitaria San Martino - IST

🇮🇹

Genova, Italy

Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

🇮🇹

Milano, Italy

Azienda Ospedaliero-Universitaria Policlinico di Modena

🇮🇹

Modena, Italy

AORN A. Cardarelli

🇮🇹

Napoli, Italy

Azienda Policlinico Umberto I di Roma

🇮🇹

Roma, Italy

Seoul National University Bundang Hospital

🇰🇷

Seongnam-si, Gyeonggi-do, Korea, Republic of

Seoul National University Hospital

🇰🇷

Jongno Gu, Seoul, Korea, Republic of

Chonnam National University Hwasun Hospital

🇰🇷

Hwasun, Jeollanam-do, Korea, Republic of

Inje University Busan Paik Hospital

🇰🇷

Busan, Korea, Republic of

Asan Medical Center

🇰🇷

Seoul, Korea, Republic of

Seoul Saint Mary's Hospital

🇰🇷

Seoul, Korea, Republic of

Samsung Medical Center

🇰🇷

Seoul, Korea, Republic of

Kyungpook National University Hospital

🇰🇷

Daegu, Korea, Republic of

Severance Hospital, Yonsei University Health System

🇰🇷

Seoul, Korea, Republic of

Samodzielny Publiczny Szpital Kliniczny Nr 1 we Wroclawiu

🇵🇱

Wroclaw, Dolnoslaskie, Poland

Ulsan University Hospital

🇰🇷

Ulsan, Korea, Republic of

Samodzielny Publiczny Centralny Szpital Kliniczny

🇵🇱

Warszawa, Mazowieckie, Poland

Hospital General Virgen de las Nieves

🇪🇸

Granada, Spain

Hospital Vall d´Hebrón

🇪🇸

Barcelona, Spain

Hospital San Pedro de Alcantara

🇪🇸

Caceres, Spain

Hospital Clínico Universitario de Salamanca

🇪🇸

Salamanca, Spain

Hospital General Universitario Gregorio Marañon

🇪🇸

Madrid, Spain

Hospital Universitario 12 de Octubre

🇪🇸

Madrid, Spain

Hospital Universitario Virgen del Rocio

🇪🇸

Sevilla, Spain

Chang Gung Medical Foundation-LinKou Branch

🇨🇳

Tao-Yuan, Taoyuan, Taiwan

Hospital Universitari i Politecnic La Fe de Valencia

🇪🇸

Valencia, Spain

Karolinska University Hospital Huddinge

🇸🇪

Stockholm, Sweden

University of New Mexico Cancer Center

🇺🇸

Albuquerque, New Mexico, United States

University of Wisconsin

🇺🇸

Madison, Wisconsin, United States

University of Alberta Hospital

🇨🇦

Edmonton, Alberta, Canada

Universitätsklinikum Schleswig-Holstein

🇩🇪

Kiel, Schleswig-holstein, Germany

University of Kansas Medical Center

🇺🇸

Westwood, Kansas, United States

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