Study Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Disease Activity In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic Syndrome (NCT04401748)
A Randomized, Double-Blind, Phase 3 Study Evaluating the Safety and Efficacy of Venetoclax in Combination With Azacitidine in Patients Newly Diagnosed With Higher-Risk Myelodysplastic Syndrome (Higher-Risk MDS)
🩺 Plain-English Summary (8th-Grade Level)
Myelodysplastic Syndrome (MDS) is a group of disorders that gradually affect the ability of a person's bone marrow (semi-liquid tissue present in many bones like backbones) to produce normal blood cells. Some people with MDS have a risk of the disease progressing to acute myeloid leukemia (AML), and a risk of death from the disease itself. Symptoms of MDS include fatigue, shortness of breath, unusual paleness due to anemia (low red blood cell count), easy or unusual bruising, and red spots just beneath the skin caused by bleeding. The purpose of this study is to see how safe and effective venetoclax and azacitidine (AZA) combination are when compared to AZA and a placebo (contains no medicine), in participants with newly diagnosed higher-risk MDS. Venetoclax is an investigational drug being developed for the treatment of MDS. The study consists of two treatment arms - In one arm, participants will receive venetoclax and AZA. In another arm, participants will receive AZA and placebo. Adult participants with newly diagnosed higher-risk MDS will be enrolled. Around 500 participants will be enrolled in approximately 220 sites worldwide. Participants in one arm will receive oral doses of venetoclax tablet and intravenous (infusion in the vein) or subcutaneous (given under the skin) AZA solution. Participants in another arm will receive oral doses of placebo tablet and intravenous or subcutaneous AZA solution. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the course of the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood and bone marrow tests, checking for side effects, and completing questionnaires.
Who can join: Review the eligibility criteria below.
Location: Duplicate_Providence Medical Foundation /ID# 222633 — Fullerton, California
Age range: 18 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (220 Sites)
Showing the first 25 of 220 sites.
- Duplicate_Providence Medical Foundation /ID# 222633 — Fullerton, California
- University of California, Los Angeles /ID# 221760 — Los Angeles, California
- Torrance Memorial Physician Network Cancer Care /ID# 222702 — Torrance, California
- PIH Health Whittier Hospital /ID# 222647 — Whittier, California
- Rocky Mountain Cancer Centers - Boulder /ID# 223723 — Boulder, Colorado
- Yale University School of Medicine /ID# 222764 — New Haven, Connecticut
- Helen F. Graham Cancer Center & Research Institute /ID# 223731 — Newark, Delaware
- Florida Cancer Specialists - Fort Myers /ID# 221319 — Fort Myers, Florida
- Memorial Healthcare System /ID# 222703 — Hollywood, Florida
- Florida Cancer Specialists - North /ID# 221318 — St. Petersburg, Florida
- Florida Cancer Specialists - Panhandle /ID# 221315 — Tallahassee, Florida
- Florida Cancer Specialists - East /ID# 221317 — West Palm Beach, Florida
- St. Luke's Mountain State Tumor Institute /ID# 220838 — Boise, Idaho
- Northwestern University Feinberg School of Medicine /ID# 220843 — Chicago, Illinois
- Duplicate_Rush University Medical Center /ID# 221007 — Chicago, Illinois
- Ingalls Memorial Hosp /ID# 220844 — Harvey, Illinois
- Illinois Cancer Care, PC /ID# 220840 — Peoria, Illinois
- Duplicate_Parkview Cancer Institute /ID# 223620 — Fort Wayne, Indiana
- Cancer Center of Kansas /ID# 222648 — Wichita, Kansas
- Hematology/Oncology Clinic /ID# 224257 — Baton Rouge, Louisiana
- Pontchartrain Cancer Center - Covington /ID# 221005 — Covington, Louisiana
- Maryland Oncology Hematology /ID# 223776 — Columbia, Maryland
- Massachusetts General Hospital /ID# 225068 — Boston, Massachusetts
- Beth Israel Deaconess Medical Center /ID# 225069 — Boston, Massachusetts
- Dana-Farber Cancer Institute /ID# 219162 — Boston, Massachusetts
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
Explore by condition: All Myelodysplastic Syndrome (MDS) clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.