Clinical trial · Interventional
A Study Evaluating Venetoclax in Combination With Azacitidine in Participants With Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS)
A Phase 1b Dose Escalation Study Evaluating the Safety and Pharmacokinetics of Venetoclax in Combination With Azacitidine in Subjects With Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS)
NCT02942290CI-TRIAL-00121599completedPhase 1ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Myelodysplastic Syndromes (MDS) | Myelodysplastic Syndrome | ALIAS | 0.85 |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Azacitidine | Drug | Azacitidine | ALIAS |
| Venetoclax | Drug | Venetoclax | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Venetoclax + Azacitidine
- interventionNames
- Drug: Azacitidine
- Drug: Venetoclax
Primary outcomes (12)
- measure
- AUCt for Azacitidine
- timeFrame
- Up to 32 days
- description
- Area under the plasma concentration-time curve (AUC) from 0 to the time of the last measurable concentration (AUCt) for azacitidine.
- measure
- Cmax of venetoclax
- timeFrame
- Up to 32 days
- description
- Maximum plasma concentration (Cmax) of venetoclax.
- measure
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
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.
References
Publications (1)
- DERIVEDGarcia JS, Platzbecker U, Odenike O, Fleming S, Fong CY, Borate U, Jacoby MA, Nowak D, Baer MR, Peterlin P, Chyla B, Wang H, Ku G, Hoffman D, Potluri J, Garcia-Manero G. Efficacy and safety of venetoclax plus azacitidine for patients with treatment-naive high-risk myelodysplastic syndromes. Blood. 2025 Mar 13;145(11):1126-1135. doi: 10.1182/blood.2024025464. PMID 39652823