A Phase I Study of Venetoclax in Combination With Cytotoxic Chemotherapy, Including Calaspargase Pegol, for Children, Adolescents and Young Adults With High-Risk Hematologic Malignancies
Andrew E. Place, MD
Summary
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
Description
This is an investigator-initiated open-label multi-institutional phase I study of venetoclax combination therapy in both myeloid and lymphoid hematologic malignancies. This study is designed as a basket trial with three separate cohorts. All cohorts include a dose finding portion (Part I) followed by a dose expansion at the Recommended Phase II dose (RP2D, Part II). This research study is looking to learn more about how Venetoclax works and aims to determine the safest, highest possible dose that can be combined with standard of care chemotherapies. Because of this, not everyone who participa…
Eligibility
- Age range
- 1–40 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria Cohort A Inclusion Criteria: * MDS, AML arising from MDS (MDS/AML), therapy related myeloid neoplasm (tMDS/AML) meeting at least one of the following criteria: * MDS with excess blasts (\>10%) * MDS with blasts \<10% with high-risk features * MDS refractory to initial treatment * Relapsed MDS * MDS/AML: May be newly diagnosed or relapsed/refractory disease. * Therapy related myeloid neoplasm (tMDS/AML): May be initial or relapsed/refractory disease. * Note: MDS or MDS/AML may be derived from a germline predisposition to myeloid malignancy as long as that…
Interventions
- DrugVenetoclax
Tablet taken orally
- DrugAzacitidine
Taken intravenously
- DrugCytarabine
Lumbar Puncture
- DrugMethotrexate
Lumbar Puncture
- DrugHydrocortisone
Lumbar Puncture
- DrugLeucovorin
Taken Orally or intravenously
- DrugDexamethasone
Taken Orally or intravenously
Locations (5)
- University of California San Francisco-Benioff Children's HospitalSan Francisco, California
- Children's Hospital ColoradoAurora, Colorado
- Children's Healthcare of Atlanta at Arthur M. Blank HospitalAtlanta, Georgia
- Ann & Robert H Lurie Children's Hospital of ChicagoChicago, Illinois
- Dana-Farber Cancer InstituteBoston, Massachusetts