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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.

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.

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)

Mayo Clinic Cancer Center

🇺🇸

Rochester, Minnesota, United States

Scripps Cancer Center

🇺🇸

La Jolla, California, United States

University of Southern California

🇺🇸

Los Angeles, California, United States

University of Chicago

🇺🇸

Chicago, Illinois, United States

University of Kansas Medical Center

🇺🇸

Westwood, Kansas, United States

University of Minnesota Medical Center

🇺🇸

Minneapolis, Minnesota, United States

John Theurer Cancer Center at Hackensack

🇺🇸

Hackensack, New Jersey, United States

University of New Mexico Cancer Center

🇺🇸

Albuquerque, New Mexico, United States

Roswell Park Cancer Institute

🇺🇸

Buffalo, New York, United States

Weill Cornell Medical College

🇺🇸

New York, New York, United States

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Mayo Clinic Cancer Center
🇺🇸Rochester, Minnesota, United States

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