A Study of Galunisertib in Participants With Myelodysplastic Syndromes

Phase 3CompletedNCT02008318

Eli Lilly and Company43 enrolled

Overview

The purpose of this study is to investigate the effect of the study drug known as galunisertib in participants with myelodysplastic syndromes (MDS). Participants with different degrees of disease (very low, low, and intermediate risk) will be studied. The study treatment is expected to last about 6 months for each participant.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Myelodysplastic Syndromes

Interventions

GalunisertibDRUG

Administered orally

PlaceboDRUG

Administered orally

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Confirmed diagnosis of MDS based on the World Health Organization (WHO) criteria * Participants with 5q deletions are allowed only if they have failed or are intolerant of lenalidomide treatment * Participants must have a Revised International Prognostic Scoring System (IPSS-R) category of very low-, low-, or intermediate-risk disease * In the 8 weeks prior to registration, participants in phase 2 should have anemia with Hb ≤10.0 g/dL (based on the average of 2 baseline measurements and untransfused for at least 1 week) with or without red blood cell (RBC) transfusion dependence confirmed for a minimum of 8 weeks before enrollment * For phase 3, participants should have anemia with RBC transfusion dependence confirmed within 8 weeks before enrollment * Performance status ≤2 on the Eastern Cooperative Oncology Group (ECOG) scale Exclusion Criteria: * No history of moderate or severe cardiac disease * No prior history of acute myeloid leukemia (AML)

Locations (14)

Outcomes

Primary Outcomes

Percentage of Participants With Hematological Improvement (HI)

Percentage of participants with hematological improvement (HI) based on International Working Group (IWG) 2006 criteria in participants with very low, low, and intermediate-risk myelodysplastic syndromes treated with Galunisertib plus best supportive care, as assessed by the International Prognostic Scoring System (IPSS-R). To be classified as an HI responder, the HI response must have lasted at least 8 weeks (56 days).

Time frame: Baseline through end of study treatment (24 weeks)

Percentage of Participants Who Are Transfusion-free or Have Hemoglobin (Hb) Increase ≥1.5 Grams/Deciliter Maintained for 8 Weeks During Phase 3

Comparison of the percentage of participants with very low-, low-,and intermediate-risk MDS who were transfusion-free or had an increase ≥1.5 g/dL in hemoglobin (Hb) maintained for at least 8 weeks within the first 24 weeks of treatment with galunisertib plus best supportive care or placebo plus best supportive care and assessed by IPSS-R. The Phase 3 portion of this study was not conducted because efficacy level required in phase 2 to move forward to phase 3 was not achieved.

Time frame: Baseline through end of study treatment (24 weeks)

Secondary Outcomes

Change From Baseline in Brief Fatigue Inventory (BFI)

The Brief Fatigue Inventory (BFI) is a brief participant-reported questionnaire that measures the severity of fatigue based on the worst fatigue experienced during the past 24-hours. The severity of fatigue is assessed using an 11-point numeric scale, with 0 = no fatigue and 10 = fatigue as bad as you can imagine.

Time frame: Baseline, Follow up (final visit up to 24 months)

Change From Baseline in EuroQol 5-Dimension 5 Level Instrument

EuroQol 5-Dimension 5 Level Instrument (EQ-5D-5L) was not conducted, trial terminated prior to Phase 3. No data collected.

Time frame: Phase 3: Baseline, Cycle 2, Cycle 4, Cycle 6 (Cycle = 28 days)

Data from ClinicalTrials.gov

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For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.

Dresden, Germany

Düsseldorf, Germany

Jena, Germany

Lübeck, Germany

Ulm, Germany

Westerstede, Germany

Florence, Italy

Novara, Italy

Rome, Italy

Barcelona, Spain

...and 4 more locations