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Completed

NCT Number: NCT02008318

A Study of Galunisertib in Participants With Myelodysplastic Syndromes

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.

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Key information

Conditions

Myelodysplastic Syndromes Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

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

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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)

Treatment and study plan

Galunisertib

Drug

Administered orally

Other names: LY2157299

Placebo

Drug

Administered orally

Primary outcomes

  1. Percentage of Participants With Hematological Improvement (HI)

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

    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).

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

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

    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.

Secondary outcomes

  1. Change From Baseline in Brief Fatigue Inventory (BFI)

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

    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.

  2. Change From Baseline in EuroQol 5-Dimension 5 Level Instrument

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

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

  3. Percentage of Participants With Cytogenetic Response

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

    Percentage of Participants with Cytogenetic Response with either complete or partial response. Complete cytogenetic response is the disappearance of the chromosomal abnormality without appearance of new ones. Partial cytogenetic response is at least 50% reduction of the chromosomal abnormality.

  4. Percentage of Participants Who Are Hospitalized (Resource Utilization)

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

    Percentage of any participant with a hospitalization admission and discharge date on the same day are counted as a half-day in the duration of hospitalization.

  5. Population Pharmacokinetics (PK): Mean Population Clearance of Galunisertib

    Time frame: Day 1 pre-dose & between 0.5 to 2 hours post dose; Day 14 pre-dose, between 0.5 to 2 & between 3 to 5 hours post dose; Days 15 & 16 (if logistically possible) between 0.5 to 2 hours post dose

    Population mean (between-participant coefficient variation [CV%]) apparent clearance.

  6. Overall Survival (OS)

    Time frame: Baseline to date of death from any cause (Up to 2 years)

    Overall survival is defined as the time from the date of first dose to the date of death from any cause.

  7. Number of Participants With a Change in Bone Marrow Fibrosis Grading

    Time frame: Baseline, Cycle 6 (Cycle = 28 days)

    Change from baseline in bone marrow fibrosis measured the number of participants with a change in bone marrow fibrosis grading (negative, mild, moderate, and severe).

Sponsors and collaborators

Lead sponsor

Eli Lilly and Company

Industry

Registry information

Official study title

Phase 2/3 Study of Monotherapy LY2157299 Monohydrate in Very Low-, Low-, and Intermediate-Risk Patients With Myelodysplastic Syndromes

Important dates

Study start
2014
Primary completion
2016
Study completion
2017
First posted
Dec 11, 2013
Registry last updated
Sep 11, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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