Study to Evaluate Imetelstat (GRN163L) in Participants With International Prognostic Scoring System (IPSS) Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)
Condition(s) studied
Investigational drug(s) / intervention(s)
Imetelstat Sodium: Imetelstat sodium IV infusion.
Placebo: Imetelstat sodium-matching placebo IV infusion.
Study summary
The purpose of this study is to evaluate the efficacy and safety of imetelstat sodium in transfusion-dependent participants with low or intermediate-1 risk myelodysplastic syndrome (MDS) that is relapsed/refractory to erythropoiesis-stimulating agent (ESA) treatment in Phase 2 study and to compare the efficacy, in terms of red blood cell (RBC) transfusion independence (TI), of imetelstat sodium to placebo in transfusion-dependent participants with low or intermediate-1 risk MDS that is relapsed/refractory to ESA treatment in Phase 3 study.
A separate Ventricular Repolarization Substudy (QTc Substudy) will evaluate the effect of imetelstat sodium on ventricular repolarization.
An Extension Phase has been included to allow continued treatment for those participants who are benefitting from imetelstat sodium and to continue to evaluate the long-term safety, overall survival (OS), and disease progression, including progression to acute myeloid leukemia (AML) in transfusion-dependent participants with low or immediate-1 risk MDS that is relapsed/refractory to ESA treatment.
Eligibility
Primary outcome measure(s)
- Phase 2: Percentage of Participants Without Any Red Blood Cell (RBC) Transfusion During Any Consecutive 8-Weeks Period (All Participants) — Up to 5 years in Phase 2
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. - Phase 2: Percentage of Participants Without Any RBC Transfusion During Any Consecutive 8-Weeks Period in Target Population — Up to 5 years in Phase 2
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. - Phase 3: Percentage of Participants Without Any RBC Transfusion During Any Consecutive 8-Weeks Period — Up to 3.7 years in Phase 3
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.
Trial sites (126)
| Facility | City | Region | Status |
|---|---|---|---|
| UAB Comprehensive Cancer Center | Birmingham | Alabama | |
| Acrc/Arizona Clinical Research, Inc. | Tucson | Arizona | |
| CBCC Global Research, Inc. | Bakersfield | California | |
| UCLA Ronald Regan Medical Center | Los Angeles | California | |
| Yale-New Haven Hospital (YNHH) - Smilow Cancer Hospital | New Haven | Connecticut | |
| BRCR Medical Center | Plantation | Florida | |
| University of South Florida (USF) - H. Lee Moffitt Cancer Center | Tampa | Florida | |
| Franciscan Health | Indianapolis | Indiana | |
| St. Agnes Healthcare, Inc | Baltimore | Maryland | |
| Center for Cancer and Blood Disorders | Bethesda | Maryland | |
| Washington University School of Medicine | St Louis | Missouri | |
| University of New Mexico Cancer Center | Albuquerque | New Mexico | |
| Icahn School of Medicine at Mount Sinai Program for the Protection of Human Subjects | New York | New York | |
| Columbia University Medical Center | New York | New York | |
| Weill Cornell Medical College-New York Presbyterian Hospital | New York | New York | |
| Cleveland Clinic Taussig Cancer | Cleveland | Ohio | |
| The Ohio State Comprehensive Cancer Center | Columbus | Ohio | |
| Prairie lakes Healthcare system, Inc | Watertown | South Dakota | |
| Vanderbilt University Medical - Hematology-Oncology | Nashville | Tennessee | |
| Texas Oncology/Methodist Charlton Cancer Center | Dallas | Texas | |
| Simmons Comprehensive Cancer Center | Dallas | Texas | |
| Fred Hutchinson Cancer Research Center (FHCRC) | Seattle | Washington | |
| ZAS Middelheim | Antwerp | Antwerpen | |
| ZAS Cadix | Antwerp | Antwerpen | |
| GZA Ziekenhuizen - Campus Sint | Wilrijk | Antwerpen | |
| AZ Sint-Jan Burgge-Oostende | Bruges | West-Vlaanderen | |
| Az Groeninge | Kortrijk | West-Vlaanderen | |
| AZ Klina | Brasschaat | Belgium | |
| Universitair Ziekenhuis Gent | Ghent | Belgium | |
| UZ Leuven - Campus Gasthuisberg | Leuven | Belgium | |
| Tom Baker Cancer Centre | Calgary | Alberta | |
| University of Alberta Hospital - Hematology Research | Edmonton | Alberta | |
| The Ottawa Hospital | Ottawa | Ontario | |
| Sunnybrook Health Sciences Centre | Toronto | Ontario | |
| Princess Margaret Hospital | Toronto | Ontario | |
| Jewish General Hospital | Montreal | Quebec | |
| Fakultni nemocnice Brno | Brno | Brno-město | |
| FN Hradec Kralove | Hradec Králové | Hradec Králové | |
| FN Kralovske Vinohrady | Prague | Czechia | |
| Vseobecna fakultni nemocnice v Praze | Prague | Czechia |
+ 86 more sites — see the full list on the official registry below.
More Geron Corporation trials in the UK
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT02598661 on ClinicalTrials.gov ↗ ← All trials in the UK