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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05583552 | Study to Evaluate Imetelstat in Patients With High-Risk MDS or AML Failing HMA-based Therapy | PHASE2 | ACTIVE NOT_RECRUITING | 46 | — | — | Jun 5, 2023 | Jun 1, 2026 | May 12, 2026 | 11 | Australia, France +1 |
| NCT02598661 | Study to Evaluate Imetelstat (GRN163L) in Participants With International Prognostic Scoring System (IPSS) Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) | PHASE2 | ACTIVE NOT_RECRUITING | 289 | — | — | Jan 12, 2016 | Oct 13, 2026 | Jan 5, 2026 | 126 | United States, Belgium +15 |
The combined response assessment criteria for MDS and AML based on IWG (International Working Group) 2018 criteria (MDS) and the criteria of the European LeukemiaNet (AML) will be used to define responders. The response rate is calculated as number of responders divided by the number of all participants of the analysis set.
Percentage of participants without any RBC transfusion during any consecutive 8 weeks (56 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2. The 95% confidence interval (CI) was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
Percentage of participants without any RBC transfusion during any consecutive 8 weeks (56 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2. The 95% confidence interval (CI) was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
Percentage of participants without any RBC transfusion during any consecutive 8 weeks (56 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2. The 95% CI was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.
| Arm | Type | Description |
|---|---|---|
| Single-arm imetelstat | EXPERIMENTAL | - |
| Phase 2: Imetelstat Sodium | EXPERIMENTAL | Imetelstat sodium administered intravenously (IV), at a starting dose of 7.5 milligrams per kilogram (mg/kg), every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. Dose escalation to 9.4 mg/kg was allowed before Protocol Amendment 2. |
| Phase 3: Imetelstat Sodium | EXPERIMENTAL | Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. |
| Phase 3: Placebo | PLACEBO_COMPARATOR | Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. |
| QTc Substudy: Imetelstat Sodium | EXPERIMENTAL | Imetelstat sodium administered IV, at a starting dose of 7.5 mg/kg, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. |
| QTc Substudy: Placebo | PLACEBO_COMPARATOR | Imetelstat sodium-matching placebo administered IV, every 4 weeks (on a 28-day cycle), until death, lost to follow-up, withdrawal of consent, or study termination whichever occurs first. If after a minimum of 2 treatment cycles a participant has no significant change to pRBC transfusion burden or evidence of clinical benefit per Investigator, after discussion with the Sponsor the participant, he/she may be permitted to start treatment with imetelstat sodium. |
| Extension Phase: Imetelstat Sodium | EXPERIMENTAL | Participants randomized to the imetelstat sodium arm in the Phase 3 and the VR QTc Substudy, based on the response will continue to receive imetelstat sodium IV, at the dose they were receiving in the Phase 3 or VR QTc Substudy, every 4 weeks (on a 28-day cycle) until death, lost to follow-up, withdrawal of consent, study termination, or up to 3 years whichever occurs first. |
| Extension Phase: Extended Follow-up | EXPERIMENTAL | Participants randomized to the placebo arm in the Phase 3 study will enter the Extended Follow-up part of the Extension Phase and continue in follow up until death, lost to follow-up, withdrawal of consent, study termination, or whichever occurs first up to approximately 3 years. |
| Name | Type | Description |
|---|---|---|
| Imetelstat sodium | DRUG | Intravenous injection |
| Placebo | DRUG | Imetelstat sodium-matching placebo IV infusion. |
Inclusion Criteria: * Signed written informed consent * Male and female ≥ 18 years at the first screening * Must be able to adhere to the study visit schedule and other protocol requirements * Initial diagnosis of AML or MDS according to WHO (World Health Organization) 2016 classification * At leas...