A Study of Galunisertib in Participants With Myelodysplastic Syndromes

Sponsor
Eli Lilly and Company (Industry)
Overall Status
Completed
CT.gov ID
NCT02008318
Collaborator
(none)
43
14
3
42.1
3.1
0.1

Study Details

Study Description

Brief 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.

Condition or Disease Intervention/Treatment Phase
Phase 2/Phase 3

Study Design

Study Type:
Interventional
Actual Enrollment :
43 participants
Allocation:
Non-Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Phase 2/3 Study of Monotherapy LY2157299 Monohydrate in Very Low-, Low-, and Intermediate-Risk Patients With Myelodysplastic Syndromes
Study Start Date :
Mar 1, 2014
Actual Primary Completion Date :
Mar 1, 2016
Actual Study Completion Date :
Sep 1, 2017

Arms and Interventions

Arm Intervention/Treatment
Experimental: Phase (ph) 2: Galunisertib + BSC

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.

Drug: Galunisertib
Administered orally
Other Names:
  • LY2157299
  • Placebo Comparator: Ph 3: Placebo + BSC

    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.

    Drug: Placebo
    Administered orally

    Experimental: Ph 3: Galunisertib + BSC

    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.

    Drug: Galunisertib
    Administered orally
    Other Names:
  • LY2157299
  • Outcome Measures

    Primary Outcome Measures

    1. Percentage of Participants With Hematological Improvement (HI) [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 [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 Outcome Measures

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

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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)

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 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 01307
    2 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. Düsseldorf Germany 40479
    3 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. Jena Germany 07747
    4 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. Lübeck Germany 23562
    5 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. Ulm Germany 89081
    6 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. Westerstede Germany 26655
    7 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. Firenze Italy 50134
    8 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. Novara Italy 28100
    9 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. Rome Italy 00161
    10 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. Barcelona Spain 08035
    11 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. Madrid Spain 28050
    12 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. Oviedo Spain 33011
    13 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. Salamanca Spain 37007
    14 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. Valencia Spain 46026

    Sponsors and Collaborators

    • Eli Lilly and Company

    Investigators

    • Study Director: Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST), Eli Lilly and Company

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Eli Lilly and Company
    ClinicalTrials.gov Identifier:
    NCT02008318
    Other Study ID Numbers:
    • 15242
    • H9H-MC-JBAV
    • 2013-003235-30
    First Posted:
    Dec 11, 2013
    Last Update Posted:
    Sep 11, 2019
    Last Verified:
    Aug 1, 2019
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    Additional relevant MeSH terms:

    Study Results

    Participant Flow

    Recruitment Details
    Pre-assignment Detail This is a single-arm study (Galunisertib at 150 milligram [mg]); the Galunisertib at 80 mg was considered exploratory and only conducted in parallel with the main study, at one site in Spain.
    Arm/Group Title Galunisertib at 150 mg Galunisertib at 80 mg
    Arm/Group Description Galunisertib at 150 mg given orally twice daily (BID) for 14 days followed by 14 days with no study drug (28 day cycles). Exploratory arm: Galunisertib at 80 mg given orally twice daily (BID) for 14 days followed by 14 days with no study drug (28 day cycles).
    Period Title: Overall Study
    STARTED 41 2
    Received At Least 1 Dose of Study Drug 41 2
    COMPLETED 29 1
    NOT COMPLETED 12 1

    Baseline Characteristics

    Arm/Group Title Galunisertib at 150 mg Galunisertib at 80 mg Total
    Arm/Group Description Galunisertib at 150 mg 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. Exploratory arm: Galunisertib at 80 mg 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. Total of all reporting groups
    Overall Participants 41 2 43
    Age (years) [Mean (Standard Deviation) ]
    Mean (Standard Deviation) [years]
    70.49
    (7.65)
    62.50
    (10.61)
    70.12
    (7.83)
    Sex: Female, Male (Count of Participants)
    Female
    15
    36.6%
    1
    50%
    16
    37.2%
    Male
    26
    63.4%
    1
    50%
    27
    62.8%
    Ethnicity (NIH/OMB) (Count of Participants)
    Hispanic or Latino
    5
    12.2%
    0
    0%
    5
    11.6%
    Not Hispanic or Latino
    32
    78%
    2
    100%
    34
    79.1%
    Unknown or Not Reported
    4
    9.8%
    0
    0%
    4
    9.3%
    Race (NIH/OMB) (Count of Participants)
    American Indian or Alaska Native
    0
    0%
    0
    0%
    0
    0%
    Asian
    0
    0%
    0
    0%
    0
    0%
    Native Hawaiian or Other Pacific Islander
    0
    0%
    0
    0%
    0
    0%
    Black or African American
    0
    0%
    0
    0%
    0
    0%
    White
    41
    100%
    2
    100%
    43
    100%
    More than one race
    0
    0%
    0
    0%
    0
    0%
    Unknown or Not Reported
    0
    0%
    0
    0%
    0
    0%
    Region of Enrollment (Count of Participants)
    Italy
    8
    19.5%
    0
    0%
    8
    18.6%
    Germany
    23
    56.1%
    0
    0%
    23
    53.5%
    Spain
    10
    24.4%
    2
    100%
    12
    27.9%
    IPSS-R Prognostic Risk Score (Count of Participants)
    Very Low= (≤1.5)
    2
    4.9%
    0
    0%
    2
    4.7%
    Low= (>1.5 - 3)
    30
    73.2%
    1
    50%
    31
    72.1%
    Intermediate= (>3 - 4.5)
    9
    22%
    1
    50%
    10
    23.3%

    Outcome Measures

    1. Primary Outcome
    Title Percentage of Participants With Hematological Improvement (HI)
    Description 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)

    Outcome Measure Data

    Analysis Population Description
    Participants who received at least one dose of study drug.
    Arm/Group Title Galunisertib at 150 mg Arm: Galunisertib at 80 mg
    Arm/Group Description Galunisertib at 150 mg 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. Exploratory arm: Galunisertib at 80 mg given orally twice daily (BID) for 14 days followed by 14... more days with no study drug (28 day cycles). Completed participants completed at least 6 cycles.
    Measure Participants 41 2
    Number (95% Confidence Interval) [percentage of participants]
    31.7
    77.3%
    0.0
    0%
    2. Primary Outcome
    Title Percentage of Participants Who Are Transfusion-free or Have Hemoglobin (Hb) Increase ≥1.5 Grams/Deciliter Maintained for 8 Weeks During Phase 3
    Description 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)

    Outcome Measure Data

    Analysis Population Description
    Participants who received at least one dose of study drug during Phase 3.
    Arm/Group Title Galunisertib at 150 mg Galunisertib at 80 mg
    Arm/Group Description Galunisertib at 150 mg 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. Exploratory arm: Galunisertib at 80 mg given orally twice daily (BID) for 14 days followed by 14... more days with no study drug (28 day cycles). Completed participants completed at least 6 cycles.
    Measure Participants 0 0
    3. Secondary Outcome
    Title Change From Baseline in Brief Fatigue Inventory (BFI)
    Description 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)

    Outcome Measure Data

    Analysis Population Description
    Participants who received at least one dose of study drug.
    Arm/Group Title Galunisertib at 150 mg Galunisertib at 80 mg
    Arm/Group Description Galunisertib at 150 mg 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. Exploratory arm: Galunisertib at 80 mg 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.
    Measure Participants 41 2
    Current Fatigue at Follow-up
    0.818
    (0.42)
    -1.005
    (2.08)
    Usual Fatigue at Follow-up
    -0.017
    (0.37)
    -0.157
    (1.87)
    Worst Fatigue at Follow-up
    -0.191
    (0.38)
    -0.063
    (1.93)
    4. Secondary Outcome
    Title Change From Baseline in EuroQol 5-Dimension 5 Level Instrument
    Description 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)

    Outcome Measure Data

    Analysis Population Description
    Participants who received at least one dose of study drug during Phase 3.
    Arm/Group Title Galunisertib at 150 mg Galunisertib at 80 mg
    Arm/Group Description Galunisertib at 150 mg 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. Exploratory arm: Galunisertib at 80 mg 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.
    Measure Participants 0 0
    5. Secondary Outcome
    Title Percentage of Participants With Cytogenetic Response
    Description 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.
    Time Frame Baseline through end of study treatment (24 weeks)

    Outcome Measure Data

    Analysis Population Description
    Participants who received at least one dose of study drug.
    Arm/Group Title Galunisertib at 150 mg Galunisertib at 80 mg
    Arm/Group Description Galunisertib at 150 mg 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. Exploratory arm: Galunisertib at 80 mg given orally twice daily (BID) for 14 days followed by 14... more days with no study drug (28 day cycles). Completed participants completed at least 6 cycles.
    Measure Participants 41 2
    Number (95% Confidence Interval) [percentage of participants]
    2.4
    5.9%
    0
    0%
    6. Secondary Outcome
    Title Percentage of Participants Who Are Hospitalized (Resource Utilization)
    Description 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.
    Time Frame Baseline through end of study treatment (24 weeks)

    Outcome Measure Data

    Analysis Population Description
    Participants who received at least one dose of study drug.
    Arm/Group Title Galunisertib at 150 mg Galunisertib at 80 mg
    Arm/Group Description Galunisertib at 150 mg 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. Exploratory arm: Galunisertib at 80 mg given orally twice daily (BID) for 14 days followed by 14... more days with no study drug (28 day cycles). Completed participants completed at least 6 cycles.
    Measure Participants 41 2
    Number [percentage of participants]
    24.3
    59.3%
    0
    0%
    7. Secondary Outcome
    Title Population Pharmacokinetics (PK): Mean Population Clearance of Galunisertib
    Description Population mean (between-participant coefficient variation [CV%]) apparent clearance.
    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

    Outcome Measure Data

    Analysis Population Description
    All participants who received at least one dose of study drug, regardless of dose, with evaluable PK data.
    Arm/Group Title Galunisertib
    Arm/Group Description Galunisertib given orally twice daily (BID) for 14 days followed by 14 days with no study drug (28 day cycles).
    Measure Participants 43
    Geometric Mean (Geometric Coefficient of Variation) [Liter per hour (L/h)]
    32
    (52)
    8. Secondary Outcome
    Title Overall Survival (OS)
    Description Overall survival is defined as the time from the date of first dose to the date of death from any cause.
    Time Frame Baseline to date of death from any cause (Up to 2 years)

    Outcome Measure Data

    Analysis Population Description
    Participants who received at least one dose of study drug excluding the exploratory participants.
    Arm/Group Title Galunisertib at 150 mg
    Arm/Group Description Galunisertib at 150 mg 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.
    Measure Participants 41
    Median (Full Range) [days]
    679
    9. Secondary Outcome
    Title Number of Participants With a Change in Bone Marrow Fibrosis Grading
    Description 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).
    Time Frame Baseline, Cycle 6 (Cycle = 28 days)

    Outcome Measure Data

    Analysis Population Description
    Participants who received at least one dose of study drug and had both a baseline and postbaseline assessment excluding the exploratory participants.
    Arm/Group Title Galunisertib at 150 mg
    Arm/Group Description Galunisertib at 150 mg 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.
    Measure Participants 30
    Number [participants]
    11
    26.8%

    Adverse Events

    Time Frame Baseline through end of study treatment or death from any cause (Up to 2 years)
    Adverse Event Reporting Description
    Arm/Group Title Galunisertib at 150 mg Galunisertib at 80 mg
    Arm/Group Description Galunisertib at 150 mg given orally twice daily (BID) for 14 days followed by 14 days with no study drug (28 day cycles). Exploratory arm: Galunisertib at 80 mg given orally twice daily (BID) for 14 days followed by 14 days with no study drug (28 day cycles).
    All Cause Mortality
    Galunisertib at 150 mg Galunisertib at 80 mg
    Affected / at Risk (%) # Events Affected / at Risk (%) # Events
    Total / (NaN) / (NaN)
    Serious Adverse Events
    Galunisertib at 150 mg Galunisertib at 80 mg
    Affected / at Risk (%) # Events Affected / at Risk (%) # Events
    Total 8/41 (19.5%) 0/2 (0%)
    Blood and lymphatic system disorders
    Febrile neutropenia 1/41 (2.4%) 1 0/2 (0%) 0
    Lymphadenopathy 1/41 (2.4%) 1 0/2 (0%) 0
    Cardiac disorders
    Cardiac failure 2/41 (4.9%) 2 0/2 (0%) 0
    Gastrointestinal disorders
    Crohn's disease 1/41 (2.4%) 1 0/2 (0%) 0
    Retroperitoneal haemorrhage 1/41 (2.4%) 1 0/2 (0%) 0
    Infections and infestations
    Pneumonia 1/41 (2.4%) 2 0/2 (0%) 0
    Respiratory syncytial virus infection 1/41 (2.4%) 1 0/2 (0%) 0
    Tongue abscess 1/41 (2.4%) 1 0/2 (0%) 0
    Respiratory, thoracic and mediastinal disorders
    Respiratory failure 2/41 (4.9%) 2 0/2 (0%) 0
    Other (Not Including Serious) Adverse Events
    Galunisertib at 150 mg Galunisertib at 80 mg
    Affected / at Risk (%) # Events Affected / at Risk (%) # Events
    Total 33/41 (80.5%) 2/2 (100%)
    Blood and lymphatic system disorders
    Anaemia 3/41 (7.3%) 18 0/2 (0%) 0
    Gastrointestinal disorders
    Diarrhoea 7/41 (17.1%) 8 0/2 (0%) 0
    Nausea 3/41 (7.3%) 4 0/2 (0%) 0
    Vomiting 5/41 (12.2%) 6 0/2 (0%) 0
    General disorders
    Asthenia 5/41 (12.2%) 13 1/2 (50%) 1
    Fatigue 4/41 (9.8%) 4 0/2 (0%) 0
    Oedema peripheral 3/41 (7.3%) 3 0/2 (0%) 0
    Pyrexia 5/41 (12.2%) 8 0/2 (0%) 0
    Infections and infestations
    Nasopharyngitis 4/41 (9.8%) 4 0/2 (0%) 0
    Musculoskeletal and connective tissue disorders
    Back pain 3/41 (7.3%) 3 0/2 (0%) 0
    Nervous system disorders
    Dizziness 3/41 (7.3%) 3 0/2 (0%) 0
    Respiratory, thoracic and mediastinal disorders
    Cough 0/41 (0%) 0 1/2 (50%) 1
    Rhinorrhoea 0/41 (0%) 0 1/2 (50%) 1

    Limitations/Caveats

    The response rate at interim analysis did not meet the predefined rate. The Phase 3 portion was not initiated and the sponsor decided on early discontinuation of the study at the conclusion of Phase 2.

    More Information

    Certain Agreements

    Principal Investigators are NOT employed by the organization sponsoring the study.

    The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.

    Results Point of Contact

    Name/Title Chief Medical Officer
    Organization Eli Lilly and Company
    Phone 800-545-5979
    Email ClinicalTrials.gov@lilly.com
    Responsible Party:
    Eli Lilly and Company
    ClinicalTrials.gov Identifier:
    NCT02008318
    Other Study ID Numbers:
    • 15242
    • H9H-MC-JBAV
    • 2013-003235-30
    First Posted:
    Dec 11, 2013
    Last Update Posted:
    Sep 11, 2019
    Last Verified:
    Aug 1, 2019