A Study Evaluating Venetoclax in Combination With Azacitidine in Participants With Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS)

AbbVie

Status and phase: Active, not recruiting; Phase 1

Conditions: Myelodysplastic Syndromes (MDS)

Treatments: Drug: Azacitidine; Drug: Venetoclax

Study type: Interventional

Funder types: Industry

Identifiers

NCT02942290

2016-001657-41 (EudraCT Number)

M15-531

2023-507154-32-00 (Other Identifier)

Details and patient eligibility

About

This is a Phase 1b, open-label, non-randomized, multicenter, dose-finding study evaluating venetoclax in combination with azacitidine in participants with treatment-naïve higher-risk MDS comprising a dose-escalation portion and a safety expansion portion.

Enrollment: 129 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Participant must have documented diagnosis of untreated de novo MDS with:

  • International Prognostic Scoring System (IPSS) risk categories Int-2 or High (minimum IPSS overall score of 1.5) OR Revised IPSS (IPSS-R) categories intermediate, high or very high (score of > 3) and
  • Presence of less than 20% bone marrow blasts per bone marrow biopsy/aspirate.

• Eastern Cooperative Oncology Group (ECOG) performance score of less than or equal to 2.

Exclusion criteria

• Participant has received prior therapy for MDS. (Prior supportive care in form of transfusions or growth factors, etc., is not considered prior therapy).

• Participant has received prior therapy with a BCL-2 Homology 3 (BH3) mimetic.

• Participant has a diagnosis other than previously untreated de novo MDS (as defined in the protocol) including:

  • MDS with IPSS risk categories Low or Int-1 (overall IPSS score < 1.5)
  • 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 (CML), juvenile myelomonocytic leukemia (JMML) and unclassifiable MDS/MPN.

• Participant has received allogeneic Hematopoietic Stem Cell Transplantation (HSCT) or solid organ transplantation.

• Participant has received a live attenuated vaccine within 4 weeks prior to the first dose of study drug.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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129 participants in 1 patient group

Venetoclax + Azacitidine

Experimental group

Treatment:

Drug: Venetoclax

Drug: Azacitidine