A Study of Galunisertib in Participants With Myelodysplastic Syndromes

Lilly

Status and phase: Completed; Phase 3; Phase 2

Conditions: Myelodysplastic Syndromes

Treatments: Drug: Galunisertib; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02008318

2013-003235-30 (EudraCT Number)

15242

H9H-MC-JBAV (Other Identifier)

Details and patient eligibility

About

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.

Enrollment: 43 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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)

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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43 participants in 3 patient groups, including a placebo group

Phase (ph) 2: Galunisertib + BSC

Experimental group

Description:

Ph 2. 150 milligrams Galunisertib given orally twice daily (BID) for 14 days followed by 14 days with no study drug (28 day cycles). Participants will receive best supportive care (BSC) according to institutional guidelines. Treatment is expected to last for 6 cycles. Participants may receive additional cycles if they are deriving clinical benefit.

Treatment:

Drug: Galunisertib

Ph 3: Placebo + BSC

Placebo Comparator group

Description:

Placebo administered orally BID for 14 days followed by 14 days with no study drug (28 day cycles). Participants will receive BSC according to institutional guidelines. Treatment is expected to last for 6 cycles. Participants may receive additional cycles if they are deriving clinical benefit. This arm is contingent on the data from the phase 2 arm.

Treatment:

Drug: Placebo

Ph 3: Galunisertib + BSC

Experimental group

Description:

150 milligrams Galunisertib given orally twice daily (BID) for 14 days followed by 14 days with no study drug (28 day cycles). Participants will receive best supportive care (BSC) according to institutional guidelines. Treatment is expected to last for 6 cycles. Participants may receive additional cycles if they are deriving clinical benefit. This arm is contingent on the data from the phase 2 arm.

Treatment:

Drug: Galunisertib