Overview

This trial is active, not recruiting.

Condition myelodysplastic syndromes
Treatments ly2157299, placebo
Phase phase 2/phase 3
Target TGFBR1
Sponsor Eli Lilly and Company
Start date March 2014
End date March 2016
Trial size 140 participants
Trial identifier NCT02008318, 15242, 2013-003235-30, H9H-MC-JBAV

Summary

The purpose of this study is to investigate the effect of the study drug known as LY2157299 on red blood cells 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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, caregiver, investigator)
Primary purpose treatment
Arm
(Experimental)
Phase (ph) 2. 150 milligrams LY2157299 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.
ly2157299
Administered orally
(Placebo Comparator)
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.
placebo
Administered orally
(Experimental)
150 milligrams LY2157299 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.
ly2157299
Administered orally

Primary Outcomes

Measure
Percentage of Participants with Hematological Improvement (HI) Based on International Working Group (IWG) 2006 Criteria
time frame: Baseline through HI (24 weeks)
Percentage of Participants who are Transfusion-free or have Hemoglobin (Hb) Increase ≥1.5 Grams/Deciliter (g/dL) Maintained for 8 Weeks
time frame: Baseline through first 24 weeks of study treatment

Secondary Outcomes

Measure
Change from Baseline in Brief Fatigue Inventory (BFI)
time frame: Baseline, Cycle 2, Cycle 4, Cycle 6 (Cycle = 28 days)
Change from Baseline in EuroQol 5-Dimension 5 Level Instrument (EQ-5D-5L)
time frame: Phase 3: Baseline, Cycle 2, Cycle 4, Cycle 6 (Cycle = 28 days)
Percentage of Participants with Cytogenetic Response
time frame: Baseline, Cycle 3, Cycle 6 (Cycle = 28 days)
Resource Utilization: Percentage of Participants who are Hospitalized
time frame: Baseline through end of study treatment (24 weeks)
Population Pharmacokinetics: Median Population Clearance of LY2157299
time frame: Cycle 1, Predose Cycle 2, 4, 6 (Cycle = 28 days)
Overall Survival
time frame: Baseline to date of death from any cause (estimated up to 2 years)
Change from Baseline in Bone Marrow Fibrosis
time frame: Baseline, Cycle 3, Cycle 6 (Cycle = 28 days)

Eligibility Criteria

Male or female participants at least 18 years old.

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)

Additional Information

Official title Phase 2/3 Study of Monotherapy LY2157299 Monohydrate in Very Low-, Low-, and Intermediate-Risk Patients With Myelodysplastic Syndromes
Trial information was received from ClinicalTrials.gov and was last updated in August 2016.
Information provided to ClinicalTrials.gov by Eli Lilly and Company.