Combining venetoclax with Vyxeos for young patients with relapsed or refractory acute leukemia
A Phase I Study of Venetoclax Combined With Vyxeos (CPX-351) for Children, Adolescents and Young Adults With Relapsed or Refractory Acute Leukemia
This study is testing a new treatment combining venetoclax and Vyxeos to see if it helps young patients with relapsed or hard-to-treat acute leukemia feel better.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 21 (estimated) |
| Ages | 1 Year to 39 Years |
| Sex | All |
| Sponsor | Children's Hospital Medical Center, Cincinnati Academic / other |
| Drugs / interventions | radiation |
| Locations | 1 site (Cincinnati, Ohio) |
| Trial ID | NCT03826992 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are pediatric and young adult patients aged 1 to 39 years with relapsed or refractory acute leukemia, excluding certain subtypes.
Not a fit: Patients with acute promyelocytic leukemia or specific subtypes of acute leukemia that are not eligible will not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for young patients with difficult-to-treat forms of acute leukemia.
How similar studies have performed: While this approach is novel in this specific combination, other studies have explored similar therapies with varying degrees of success.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Cincinnati, Ohio
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.