92 Participants Needed

Venetoclax Combination Therapy for Blood Cancers

Recruiting at 4 trial locations
JA
Andrew Place, MD, PhD profile photo
Overseen ByAndrew Place, MD, PhD
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial is evaluating the safety and tolerability of venetoclax with chemotherapy in pediatric and young adult patients with hematologic malignancies, including myelodysplastic syndrome (MDS), acute myeloid leukemia derived from myelodysplastic syndrome (MDS/AML), and acute lymphoblastic leukemia (ALL)/lymphoblastic lymphoma (LBL).The names of the study drugs involved in this study are below. Please note this is a list for the study as a whole, participants will receive drugs according to disease cohort.* Venetoclax* Azacitidine* Cytarabine* Methotrexate* Hydrocortisone* Leucovorin* Dexamethasone* Vincristine* Doxorubicin* Dexrazoxane* Calaspargase pegol* Hydrocortisone

Will I have to stop taking my current medications?

The trial does not require stopping all current medications. Some medications like dexamethasone, prednisone, vincristine, and low-dose methotrexate can be continued without a break. However, you must stop using strong or moderate CYP3A inhibitors/inducers at least 3 days before starting the trial.

Is Venetoclax combination therapy safe for treating blood cancers?

Venetoclax combined with azacitidine has been shown to be generally safe for patients with acute myeloid leukemia who are not suitable for standard chemotherapy. Common side effects include low levels of white blood cells (neutropenia), low platelet counts (thrombocytopenia), and low red blood cell counts (anemia), but these were considered tolerable.12345

What makes Venetoclax combination therapy unique for blood cancers?

Venetoclax combination therapy is unique because it targets the B-cell leukemia/lymphoma-2 protein, which helps cancer cells survive, and is used in combination with other drugs like azacitidine to treat acute myeloid leukemia (AML). This combination can be a bridge to a potentially curative transplant for some patients and offers a less aggressive treatment option for older patients who cannot undergo intensive therapies.24678

What data supports the effectiveness of the drug combination therapy for blood cancers?

Research shows that venetoclax combined with azacitidine improves remission rates and survival in older patients with acute myeloid leukemia (AML) compared to azacitidine alone. This suggests that the combination therapy could be effective for treating certain blood cancers.126910

Who Is on the Research Team?

Andrew E. Place, MD, PhD - Dana-Farber ...

Andrew Place, MD, PhD

Principal Investigator

Dana-Farber Cancer Institute

Are You a Good Fit for This Trial?

This trial is for pediatric and young adult patients up to 40 years old with high-risk blood cancers like MDS, AML from MDS, or ALL/LBL. They should have recovered from previous treatments, not have severe organ damage or active infections, and can't be pregnant or breastfeeding. Participants must agree to use effective contraception.

Inclusion Criteria

I can do most activities but need help with some.
I am between 1 and 21 years old.
My leukemia has not responded to treatment or has come back.
See 7 more

Exclusion Criteria

Cohort B: Same exclusion criteria as Cohort A
Pregnant or nursing women
I do not have active GVHD, hepatitis, systemic infections, HIV, or other significant diseases.
See 2 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive venetoclax in combination with chemotherapy. Treatment cycles vary by cohort: Cohorts A and B have cycles up to 35 days, and Cohort C has a single cycle of 32 days.

Up to 4 cycles of 35 days each for Cohorts A and B; 32 days for Cohort C

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessment of treatment-related toxicities and overall response.

Up to 30 days after last dose of study treatment

Long-term Follow-up

Participants are monitored for overall survival and event-free survival up to 2 years post-treatment.

Up to 2 years

What Are the Treatments Tested in This Trial?

Interventions

  • Azacitidine
  • Calaspargase Pegol
  • Cytarabine
  • Dexamethasone
  • Dexrazoxane
  • Doxorubicin
  • Leucovorin
  • Methotrexate
  • Venetoclax
  • Vincristine
Trial Overview The trial tests the safety of venetoclax combined with chemotherapy drugs in treating various aggressive blood cancers. It aims to see how well patients tolerate this combination therapy and involves a range of medications tailored to specific disease cohorts.
How Is the Trial Designed?
3Treatment groups
Experimental Treatment
Group I: Cohort CExperimental Treatment11 Interventions
Group II: Cohort BExperimental Treatment6 Interventions
Group III: Cohort AExperimental Treatment6 Interventions

Azacitidine is already approved in European Union, United States, Canada, Japan for the following indications:

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Approved in European Union as Vidaza for:
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Approved in United States as Vidaza for:
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Approved in Canada as Vidaza for:
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Approved in Japan as Vidaza for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

Andrew E. Place

Lead Sponsor

Trials
1
Recruited
90+

Andrew E. Place, MD

Lead Sponsor

Trials
1
Recruited
90+

Children's Cancer Research Fund

Collaborator

AbbVie

Industry Sponsor

Trials
1,079
Recruited
535,000+
Founded
2013
Headquarters
North Chicago, USA
Known For
Immunology treatments
Top Products
Humira (adalimumab), Skyrizi (risankizumab), Rinvoq (upadacitinib)

Dr. Roopal Thakkar

AbbVie

Chief Medical Officer since 2023

MD from Wayne State University School of Medicine

Robert A. Michael profile image

Robert A. Michael

AbbVie

Chief Executive Officer

Bachelor's degree in Finance from the University of Illinois

Servier

Industry Sponsor

Trials
55
Recruited
45,600+

Boston Children's Hospital

Collaborator

Trials
801
Recruited
5,584,000+

University of Colorado, Denver

Collaborator

Trials
1,842
Recruited
3,028,000+

Children's Cancer Research Fund

Collaborator

Trials
3
Recruited
150+

Gateway for Cancer Research

Collaborator

Trials
47
Recruited
2,500+

Published Research Related to This Trial

In a phase II study involving 60 older or unfit patients with newly diagnosed acute myeloid leukemia (AML), the combination of venetoclax with cladribine and low-dose cytarabine alternating with venetoclax and 5-azacitidine resulted in a high composite complete response rate of 93%.
The treatment showed promising overall survival and disease-free survival rates, with only one death occurring within 4 weeks, indicating that this regimen is effective and has a favorable safety profile for this patient population.
Phase II Study of Venetoclax Added to Cladribine Plus Low-Dose Cytarabine Alternating With 5-Azacitidine in Older Patients With Newly Diagnosed Acute Myeloid Leukemia.Kadia, TM., Reville, PK., Wang, X., et al.[2023]
In a study of 13 patients with acute leukemia treated with venetoclax and azacitidine, 46% developed COVID-19, highlighting a significant risk associated with this treatment during the pandemic.
The study found that 33% of those who contracted COVID-19 died from the virus, indicating that COVID-19 can lead to severe outcomes in patients receiving AZA-VEN therapy.
SARS-CoV-2 Infection in Patients Treated with Azacitidine and Venetoclax for Acute Leukemia: A Report of a Case Series Treated in a Single Institution.Drozd-Sokołowska, J., Mądry, K., Barankiewicz, J., et al.[2023]
In patients with newly diagnosed unfit acute myeloid leukemia (AML), the combination of azacitidine and venetoclax is a standard first-line treatment.
However, patients with TP53-mutated AML and poor-risk cytogenetics do not benefit from adding venetoclax to azacitidine, suggesting that alternative treatment regimens should be considered for these individuals.
TP53 or Not TP53: That Is the Question.Green, SD., Zeidner, JF.[2023]

Citations

Phase II Study of Venetoclax Added to Cladribine Plus Low-Dose Cytarabine Alternating With 5-Azacitidine in Older Patients With Newly Diagnosed Acute Myeloid Leukemia. [2023]
SARS-CoV-2 Infection in Patients Treated with Azacitidine and Venetoclax for Acute Leukemia: A Report of a Case Series Treated in a Single Institution. [2023]
TP53 or Not TP53: That Is the Question. [2023]
Not BCL2 mutation but dominant mutation conversation contributed to acquired venetoclax resistance in acute myeloid leukemia. [2022]
Venetoclax, bendamustine, and rituximab in patients with relapsed or refractory NHL: a phase Ib dose-finding study. [2021]
[Efficacy of venetoclax combined azacitidine in newly diagnosed acute myeloid leukemia unfit for standard chemotherapy: a single center experience]. [2023]
Effect of Azithromycin on Venetoclax Pharmacokinetics in Healthy Volunteers: Implications for Dosing Venetoclax with P-gp Inhibitors. [2019]
[Short-term efficacy of venetoclax combined with azacitidine in acute myeloid leukemia: a single-institution experience]. [2022]
BCL2 Inhibition by Venetoclax: Targeting the Achilles' Heel of the Acute Myeloid Leukemia Stem Cell? [2021]
Outpatient initiation of venetoclax in patients with acute myeloid leukemia. [2023]
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