Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02008318 | A Study of Galunisertib in Participants With Myelodysplastic Syndromes | PHASE2 | COMPLETED | 43 | — | — | Mar 1, 2014 | Sep 1, 2017 | Sep 11, 2019 | 14 | Germany, Italy +1 |
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).
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.
| Arm | Type | Description |
|---|---|---|
| Phase (ph) 2: Galunisertib + BSC | EXPERIMENTAL | 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. |
| Ph 3: Placebo + BSC | 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. |
| Ph 3: Galunisertib + BSC | EXPERIMENTAL | 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. |
| Name | Type | Description |
|---|---|---|
| Galunisertib | DRUG | Administered orally |
| Placebo | DRUG | Administered orally |
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...