Double-Blind study comparing venetoclax and azacitidine versus placebo and azacitidine in patients that are newly diagnosed with Higher-Risk Myelodysplastic Syndrome
- Conditions
- Higher-Risk Myelodysplastic SyndromeMedDRA version: 21.1Level: PTClassification code: 10028533Term: Myelodysplastic syndrome Class: 100000004864Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]
- Registration Number
- CTIS2023-507153-16-00
- Lead Sponsor
- AbbVie Deutschland GmbH & Co. KG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 526
Subjects must be = 18 years of age with a diagnosis of MDS according to the 2016 World Health Organization (WHO) classification with presence of < 20% bone marrow blasts per marrow biopsy/aspirate at screening., Subjects must have no prior therapy for MDS with any hypomethylating agent, chemotherapy, or allogeneic stem cell transplantation, and must meet the following disease activity criteria: ? Overall Revised International Prognostic Scoring System (IPSS-R) score > 3 (intermediate, high, or very high); ? Eastern Cooperative Oncology Group (ECOG) performance status of = 2; ? Hematopoietic stem cell transplant (HSCT) eligible with no pre-arranged HSCT at the time of Study Day 1 (not to exceed approximately 19% of subjects enrolled on study), or HSCT ineligible without plan for HSCT at the time of Study Day 1.
For further information on the exclusion criteria, please refer to section 5.1 Eligibility Criteria of the Study Protocol
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 azacitidine compared to placebo with azacitidine in treatment-naïve, higher-risk MDS population.<br>The hypothesis corresponding to the primary objective is that venetoclax will improve the overall survival (OS) when added to standard of care azacitidine treatment in patients newly diagnosed with higher-risk MDS.;Secondary Objective: To evaluate various efficacy measures of venetoclax in combination with azacitidine compared to placebo with azacitidine in treatment-naïve, higher-risk MDS population., To evaluate the safety of venetoclax in combination with azacitidine compared to placebo with azacitidine in treatment-naïve, higher-risk MDS population., To evaluate patient-reported outcomes (PROs) for subjects on venetoclax in combination with azacitidine compared to placebo with azacitidine in treatment-naïve, higher-risk MDS population.;Primary end point(s): The primary endpoint of this study is Overall Survival.
- Secondary Outcome Measures
Name Time Method Secondary end point(s):Modified overall response (mOR) defined as complete remission (CR) + partial remission (PR) + marrow CR (mCR);Secondary end point(s):Overall hematological improvement (HI) (platelet, neutrophil, or erythroid);Secondary end point(s):Complete remission;Secondary end point(s):Red blood cell (RBC) and platelet transfusion independence for subjects who are transfusion dependent on RBC and/or platelet at baseline;Secondary end point(s):Time to deterioration in physical functioning, as measured by the physical functioning domain of EORTC QLQ-C30;Secondary end point(s):Change from baseline in fatigue, as measured by the Patient-Reported Outcomes Measurement Information System (PROMIS)-Fatigue SF 7a;Secondary end point(s):Overall response (OR) defined as CR + PR