MedTrace Logo

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 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 531

Last updated 2026-05-06

No results posted yet for this study

Summary

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.

Conditions

  • Myelodysplastic Syndrome (MDS)

Interventions

DRUG

Venetoclax

Tablet: Oral

DRUG

Azacitidine

Subcutaneous (SC) or Intravenous (IV) injection

DRUG

Placebo

Tablet; Oral

Sponsors & Collaborators

  • Genentech, Inc.

    collaborator INDUSTRY

  • AbbVie

    lead INDUSTRY

Principal Investigators

  • ABBVIE INC. · AbbVie

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-09-10
Primary Completion
2027-07-31
Completion
2027-07-31
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Austria
  • Belgium
  • Brazil
  • Canada
  • China
  • Czechia
  • France
  • Germany
  • Hungary
  • Israel
  • Italy
  • Japan
  • Netherlands
  • Poland
  • Puerto Rico
  • Russia
  • South Korea
  • Spain
  • Taiwan
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04401748 on ClinicalTrials.gov