Phase 3
N=43
A Study of Galunisertib in Participants With Myelodysplastic Syndromes
Myelodysplastic Syndromes
Bottom Line
View on ClinicalTrials.gov: NCT02008318 ↗Enrolled (actual)
43
Serious AEs
18.6%
Results posted
Mar 2019
Primary outcome: Primary: Percentage of Participants With Hematological Improvement (HI) — 31.7; 0.0 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Galunisertib (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Eli Lilly and Company
- Primary completion
- Mar 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Hematological Improvement (HI) |
31.7; 0.0 | — |
| PRIMARY Percentage of Participants Who Are Transfusion-free or Have Hemoglobin (Hb) Increase ≥1.5 Grams/Deciliter Maintained for 8 Weeks During Phase 3 |
— | — |
| SECONDARY Change From Baseline in Brief Fatigue Inventory (BFI) |
0.818; -1.005; -0.017; -0.157; -0.191; -0.063 | — |
| SECONDARY Change From Baseline in EuroQol 5-Dimension 5 Level Instrument |
— | — |
| SECONDARY Percentage of Participants With Cytogenetic Response |
2.4; 0 | — |
| SECONDARY Percentage of Participants Who Are Hospitalized (Resource Utilization) |
24.3; 0 | — |
| SECONDARY Population Pharmacokinetics (PK): Mean Population Clearance of Galunisertib |
32 | — |
| SECONDARY Overall Survival (OS) |
679 | — |
| SECONDARY Number of Participants With a Change in Bone Marrow Fibrosis Grading |
11 | — |
Summary
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.
Eligibility Criteria
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)
Data sourced from ClinicalTrials.gov (NCT02008318). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.