Safety And Efficacy Study Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Complete Remission and Overall Survival In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic Syndrome (Verona)
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|ClinicalTrials.gov Identifier: NCT04401748|
Recruitment Status : Recruiting
First Posted : May 26, 2020
Last Update Posted : September 10, 2020
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 200 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.
|Condition or disease||Intervention/treatment||Phase|
|Myelodysplastic Syndrome (MDS)||Drug: Venetoclax Drug: Azacitidine Drug: Placebo||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||500 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||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)|
|Actual Study Start Date :||August 16, 2020|
|Estimated Primary Completion Date :||February 14, 2025|
|Estimated Study Completion Date :||February 14, 2025|
Experimental: Arm 1: Venetoclax + Azacitidine (AZA)
Participants will receive venetoclax once daily (QD) (Days 1-14) in combination with AZA QD (Days 1-7) of each 28 day cycle.
Subcutaneous (SC) or Intravenous (IV) injection
Other Name: AZA
Active Comparator: Arm 2: Placebo + Azacitidine
Participants will receive placebo once daily (QD) (Days 1-14) in combination with AZA QD (Days 1-7) of each 28 day cycle.
Subcutaneous (SC) or Intravenous (IV) injection
Other Name: AZA
- Complete Remission (CR) [ Time Frame: Up To 36 Months ]CR is defined as achieving a complete remission at any time point during the study per the modified International Working Group (IWG) 2006 criteria for myelodysplastic syndrome (MDS).
- Overall survival (OS) [ Time Frame: Up To 5 Years ]OS is defined as the number of days from the date of randomization to the date of death of any cause, or last known date to be alive.
- Percentage of Participants who Achieve Red Blood Cell (RBC) transfusion independence (TI) [ Time Frame: Up To 5 Years ]RBC TI is when the participants who were transfusion dependent at baseline achieve transfusion independence post baseline. RBC TI is a period of at least 56 days with no transfusion after the date of the first dose of study drug to the last dose of study drug + 30 days, the initiation of post-treatment therapy, or death, whichever is earliest.
- Percentage of Participants who Achieve Platelet Transfusion Independence (TI) [ Time Frame: Up To 5 Years ]Platelet TI is when participants who were transfusion dependent at baseline achieve transfusion independence post baseline. Platelet TI is a period of at least 56 days with no transfusion after the date of the first dose of study drug to the last dose of study drug + 30 days, the initiation of post-treatment therapy, or death, whichever is earliest.
- Change in Patient-Reported Outcomes Measurement Information System (PROMIS)-Fatigue Short Form (SF) 7a Scale Score. [ Time Frame: Up To 5 Years ]Fatigue will be assessed using the PROMIS Fatigue SF 7a Global Fatigue Score. PROMIS Fatigue SF 7a is a 7-item questionnaire that assesses the impact and experience of fatigue over the past 7 days. Participants rate items on a 5-point scale, with 1 as "never" an 5 as "always".
- Time to Deterioration in Physical Functioning as Measured by EORTC QLQ-C30 Scale [ Time Frame: Up To 5 Years ]Time to deterioration in physical functioning, as measured by the EORTC QLQ-C30 physical functioning score is defined as the time from the date of randomization to the date of death of any cause, or the first time worsening of score from baseline >= a pre-specified threshold. Participants rate items on a 4-point scale, with 1 as "not at all" and 4 as "very much."
- Overall Response (OR) [ Time Frame: Up To 5 Years ]OR [complete remission (CR) + partial response (PR)] is defined as achieving a CR or PR at any time point during the study per the modified IWG 2006 criteria for MDS.
- Modified Overall Response (mOR) [ Time Frame: Up To 5 Years ]mOR [complete remission (CR) + marrow complete remission (mCR) + partial response (PR)] is defined as achieving a CR, mCR, or PR at any time point during the study per the modified IWG 2006 criteria for MDS.
- Change in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC-QLQ) Physical Functioning Domain Score [ Time Frame: Up To 5 Years ]The EORTC-QLQ-C30 is a 30-item subject self-report questionnaire composed of both multi-item and single scales, including global health status/quality of life, functional Scales (physical, role, emotional, cognitive, and social) , symptom scales (fatigue, nausea and vomiting, and pain), and 6 single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, and financial difficulties). Participants rate items on a 4-point scale, with 1 as "not at all" and 4 as "very much."
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04401748
|Contact: ABBVIE CALL CENTERfirstname.lastname@example.org|
|Study Director:||AbbVie Inc.||AbbVie|