Double-Blind study comparing venetoclax and azacitidine versus placebo and azacitidine in patients that are newly diagnosed with Higher-Risk Myelodysplastic Syndrome
- Conditions
- Myelodysplastic SyndromeTherapeutic area: Diseases [C] - Cancer [C04]
- Registration Number
- EUCTR2020-000744-55-CZ
- Lead Sponsor
- AbbVie
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 500
Adult male or female, at least 18 years old. Diagnosis of MDS according to the 2016 WHO classification with presence of < 20% bone marrow blasts per bone marrow biopsy/aspirate at screening. Subject meets the following disease activity criteria:
•Overall IPSS-R score > 3 (intermediate, high, or very high; Appendix E);
•Eastern Cooperative Oncology Group (ECOG) performance status = 2;
•HSCT eligible with no pre-arranged HSCT at the time of Study Day 1, or HSCT ineligible without plan for HSCT at the time of Study Day 1. No prior therapy for MDS with any hypomethylating agent (for example, azacitidine, decitabine), chemotherapy or allogeneic stem cell transplantation.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 300
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 200
No previous diagnosis of:
•Therapy-related MDS (t-MDS)
•MDS evolving from a pre-existing myeloproliferative neoplasm (MPN)
•MDS/MPN including chronic myelomonocytic leukemia (CMML), atypical chronic myeloid leukemia (aCML), juvenile myelomonocytic leukemia (JMML) and unclassifiable MDS/MPN
•No prior therapy for MDS with any hypomethylating agent (for example, azacitidine, decitabine), chemotherapy or allogeneic stem cell transplantation.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To assess the efficacy of venetoclax in combination with AZA compared to placebo with AZA in treatment-naive higher-risk MDS.<br>The hypotheses corresponding to the primary objective is that venetoclax will improve the complete remission (CR) rate and overall survival (OS) when added to standard of care AZA treatment in patients newly diagnosed with higher-risk MDS.;Secondary Objective: 1.To evaluate the safety of venetoclax in combination with AZA compared to placebo with AZA in higher-risk MDS populations<br>2.To evaluate patient-reported outcomes (PROs) for subjects on venetoclax in combination with AZA compared to placebo with AZA.;Primary end point(s): - Complete Remission and Overall Survival;Timepoint(s) of evaluation of this end point: - Interim complete remission analysis will occur afer randomization of 422 subjects and followed up for 12 months. Interim analysis of overall survival after approximately 267 death events
- Secondary Outcome Measures
Name Time Method Secondary end point(s): - Modified overall response (mOR) defined as CR + partial response [PR] + marrow CR (mCR)<br>- Red blood cell (RBC) transfusion independence (TI) rate for subjects who are transfusion dependent at baseline <br>- Change from baseline in fatigue as measured by the Patient-Reported Outcomes Measurement Information System (PROMIS)-Fatigue SF 7a<br>- Change from baseline in physical functioning, as measured by the EORTC QLQ-C30 physical functioning domain<br>- Platelet Transfusion independence (TI) rate for subjects who are transfusion dependent at baseline<br>- Overall response based on CR+PR<br>- Time to deterioration in physical functioning, as measured by the EORTC QLQ-C30 physical functioning domain;Timepoint(s) of evaluation of this end point: Up to 5 years