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)

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) - VERONA

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON54378

Enrollment

Registered

2020-09-23

Start date

2020-11-16

Completion date

Unknown

Last updated

2024-09-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Myelodisplastic Syndrome (MDS) myelodysplasia

Interventions

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 rece

Eligibility

Age

18 Years to 99 Years

Inclusion criteria

Inclusion criteria: Adult male or female, at least 18 years old. Diagnosis of MDS according to the 2016 WHO classification with presence of 3 (intermediate, high, or very high; Appendix E); * Eastern Cooperative Oncology Group (ECOG) performance status

Exclusion criteria

Exclusion criteria: 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. Lenalidomide, anti-thymocyte globulin (ATG), and cyclosporin are also excluded as these are considered disease-modifying agents. * No known active SARS-CoV-2 infection. If a subject has signs/symptoms of SARS-CoV-2 infection, they should undergo molecular (e.g., PCR) testing to rule out SARS-CoV-2 infection.

Design outcomes

Primary

Measure	Time frame
Overall Survival (OS)	—

Secondary

Measure	Time frame
- Complete remission (CR) - Overall hematological improvement (HI) (HI-platelet, HI-neutrophil, or HI-erythroid) - Red blood cell (RBC) and platelet transfusion independence for subjects who are transfusion dependent on RBC and/or platelet at baseline - Change from baseline in fatigue, as measured by the Patient-Reported Outcomes Measurement Information System (PROMIS)-Fatigue SF 7a - Time to deterioration in physical functioning, as measured by the physical functioning domain of EORTC QLQ-C30 - Overall response (OR) defined as CR + partial response (PR) - Modified overall response (mOR) defined as CR + PR + marrow CR (mCR).	—

Measure

Time frame

- Complete remission (CR) - Overall hematological improvement (HI) (HI-platelet, HI-neutrophil, or HI-erythroid) - Red blood cell (RBC) and platelet transfusion independence for subjects who are transfusion dependent on RBC and/or platelet at baseline - Change from baseline in fatigue, as measured by the Patient-Reported Outcomes Measurement Information System (PROMIS)-Fatigue SF 7a - Time to deterioration in physical functioning, as measured by the physical functioning domain of EORTC QLQ-C30 - Overall response (OR) defined as CR + partial response (PR) - Modified overall response (mOR) defined as CR + PR + marrow CR (mCR).

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Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)