NCT05317403

A Phase I Study of Venetoclax to Augment Epigenetic Modification and Chemotherapy in Pediatric and Young Adult Patients With Relapsed and Refractory Acute Myeloid Leukemia

Recruiting now Phase 1 Last updated 9 January 2026

What this trial tests

Phase 1 trial testing Venetoclax in Acute Myeloid Leukemia, in Relapse in 40 participants. Currently enrolling.

Timeline

31 March 2023

Primary endpoint
1 January 2027

1 January 2027

Quick facts

Lead sponsor	Medical College of Wisconsin
Phase	Phase 1
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	40
Start date	31 March 2023
Primary completion	1 January 2027
Estimated completion	1 January 2027
Sites	1 location across United States

Drugs / interventions tested

• Venetoclax (venetoclax) — full drug profile →
• Azacitadine
• Vorinostat (VORINOSTAT) — full drug profile →
• Cytarabine — full drug profile →
• Fludarabine (FLUDARABINE) — full drug profile →
• Filgrastim

Conditions studied

• Acute Myeloid Leukemia, in Relapse — all drugs for Acute Myeloid Leukemia, in Relapse →
• Acute Myeloid Leukemia Refractory — all drugs for Acute Myeloid Leukemia Refractory →

Sponsor

Medical College of Wisconsin

Who can join

Adults 1 to 25, any sex, with Acute Myeloid Leukemia, in Relapse or Acute Myeloid Leukemia Refractory. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Venetoclax Dose-Limiting Toxicity
  Time frame: 42 months
  The primary endpoint, for dose escalation of venetoclax, is the occurrence of a dose-limiting toxicity (DLT) observed during the first course of therapy.

Sponsor's own description

The investigator is testing the addition of venetoclax to 5-azacitidine and vorinostat followed by standard chemotherapy to enhance treatment response in AML patients.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Epigenetic regulation in hematopoiesis and its implications in the targeted therapy of hematologic malignancies.
   Zhao A, Zhou H, Yang J, Li M, et al · · 2023 · cited 81× · PMID 36797244 · DOI 10.1038/s41392-023-01342-6
2. Mechanisms of venetoclax resistance and solutions.
   Liu J, Chen Y, Yu L, Yang L. · · 2022 · cited 44× · PMID 36313732 · DOI 10.3389/fonc.2022.1005659
3. Histone deacetylase inhibitors for leukemia treatment: current status and future directions.
   Hosseini MS, Sanaat Z, Akbarzadeh MA, Vaez-Gharamaleki Y, et al · · 2024 · cited 18× · PMID 39456044 · DOI 10.1186/s40001-024-02108-8
4. The evolution of targeted therapy in pediatric AML: gemtuzumab ozogamicin, FLT3/IDH/BCL2 inhibitors, and other therapies.
   Pommert L, Tarlock K. · · 2022 · cited 15× · PMID 36485125 · DOI 10.1182/hematology.2022000358
5. AML under the Scope: Current Strategies and Treatment Involving FLT3 Inhibitors and Venetoclax-Based Regimens.
   Milnerowicz S, Maszewska J, Skowera P, Stelmach M, et al · · 2023 · cited 10× · PMID 37958832 · DOI 10.3390/ijms242115849
6. Emerging and Future Targeted Therapies for Pediatric Acute Myeloid Leukemia: Targeting the Leukemia Stem Cells.
   Murphy LA, Winters AC. · · 2023 · cited 6× · PMID 38137469 · DOI 10.3390/biomedicines11123248
7. Epigenetic targets to enhance antitumor immune response through the induction of tertiary lymphoid structures.
   Omotesho QA, Escamilla A, Pérez-Ruiz E, Frecha CA, et al · · 2024 · cited 4× · PMID 38333212 · DOI 10.3389/fimmu.2024.1348156
8. Role of epigenetics in paediatric cancer pathogenesis & drug resistance.
   Leung JY, Chiu HY, Taneja R. · · 2025 · cited 1× · PMID 40055485 · DOI 10.1038/s41416-025-02961-2