Combining venetoclax with Vyxeos for young patients with relapsed or refractory acute leukemia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase I pilot study evaluates the safety and tolerability of a combination therapy using venetoclax and Vyxeos (CPX-351) in pediatric and young adult patients suffering from relapsed or refractory acute leukemia. Participants will receive a single course of treatment that includes daily oral or crushed venetoclax, with a gradual dose increase, alongside intravenous Vyxeos administered on specific days. The study aims to assess not only the safety and tolerability of this combination but also the overall response rate and pharmacokinetics of venetoclax in this context.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Ages 1 Year to 39 Years
* Diagnosis of one of the following:

  * Acute myeloid leukemia (AML), any subtype except

    * Patients with acute promyelocytic leukemia (APML) are NOT eligible
    * Patients with ML-DS are NOT eligible
  * Myeloid sarcoma
  * Acute leukemia of ambiguous lineage (ALAL)

    * Acute undifferentiated leukemia (AUL)
    * T/myeloid mixed phenotype acute leukemia (MPAL)
    * B/myeloid MPAL
    * MPAL with KMT2A-rearrangement MPAL with t (9;22) are NOT eligible
  * T-cell acute lymphoblastic leukemia (T ALL)
  * Early thymocyte precursor (ETP) ALL
  * KMT2A-rearranged ALL
* Disease Status

  * Relapsed/Refractory AML, MPA, and AUL
  * Untreated therapy related AML
  * Relapsed/Refractory KMT2A-rearranged ALL, T-cell ALL, ETEP ALL
* Karnofsky/Lanksy performance level score of greater than or equal to 50 percent.
* Prior therapy requirements

  * Fully recovered from acute toxicities of Hematopoietic Stem Cell Transplant (HSCT) or Anthracycline Exposure
  * 14 days must have elapsed since the completion of systemic cytotoxic therapy other than hydroxyurea, decitabine or azacitidine
  * 2 weeks must have elapsed for local palliative radiotherapy (RT); 6 months must have elapsed if prior craniospinal RT or if 50% radiation of pelvis, and at least 6 weeks must have elapsed if other substantial bone marrow radiation
* Adequate renal, liver, cardiac, and central nervous system (CNS) function

Exclusion Criteria:

* Diagnosis of one of the following:

  * Myeloid Leukemia associated with Down Syndrome (ML-DS)
  * Acute Promyelocytic Leukemia (APML)
  * Acute leukemia with CNS status 3 involvement
  * Philadelphia chromosome t(9;22) positive leukemia (Ph+ ALL, AML, MPAL, or AUL)
  * Fanconi Anemia, Shwachman-Diamond syndrome, or any other bone marrow failure syndrome or DNA repair disorder
  * Wilson's Disease or other copper-metabolism disorder
* Pregnant or breastfeeding
* Uncontrolled infection
* Received greater than 13.6 Gray (Gy) prior radiation to the mediastinum
* Receipt of growth factors within 7 days prior to enrollment
* Currently receiving another investigational drug
* Currently receiving anti-cancer agents (with the exception of intrathecal (IT) agents or hydroxyurea)
* Unable to comply with the safety monitoring requirements of the study

Where it's running

• Cincinnati Children's Hospital Medical Center
  Cincinnati, Ohio, United States Recruiting

Study contacts

• Principal investigator: John Perentesis, MD — Children's Hospital Medical Center, Cincinnati
• Study coordinator: Site Pulblic Contact
• Email: cancer@cchmc.org
• Phone: 513-636-2799

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.