Categories:
Diseases Treated:
Eligibility Overview:
- 1–22 years old
- Treatment-related or secondary MDS/AML
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It is rare for pediatric patients treated for primary cancers or hematologic diseases to develop secondary myeloid malignancies such as myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). Those who do have second malignancies often have poor prognoses, with survival rates below 30%.
Traditionally, patients with secondary myeloid neoplasms (SMNs) are treated with intensive chemotherapy followed by hematopoietic cell transplantation. However, most of these patients have already received significant amounts of chemotherapy or were chronically ill. Early mortality and morbidity are high.
This phase 2 clinical trial will study the treatment of secondary AML and MDS with a new FDA-approved drug, CPX-351 (VYXEOS). This lipid-based vesicle contains 2 commonly used induction chemotherapy drugs, daunorubicin and cytarabine. The lipid delivery method minimizes cardiotoxicity until the drug reaches cancer cells in the bone marrow, spleen, and liver. This drug has already shown promise in some patients with SMNs.
Primary Objectives
This study will:
- Examine the efficacy of CPX-351 for treatment of secondary MDS and AML
- Examine side effects (toxicity) of this drug
- Study how the body reacts to CPX-351
- Study the biology and genetics of patients with SMNs
- Estimate the 3-year survival of patients receiving 1 or 2 courses of CPX-351 followed by hematopoietic stem cell transplantation
Eligibility Criteria
Inclusion criteria include:
- 1–22 years old
- Treatment-related or secondary MDS/AML
- Performance status for an Eastern Cooperative Oncology Group (ECOG) score of 0, 1 or 2 Karnowski for patients older than 16 and Lansky for patients younger than 16
- Adequate renal, liver, cardiac, and CNS function
- Recovered from acute toxic effects of prior chemotherapy, immunotherapy, HSCT, or radiotherapy
Exclusion criteria include:
- Down syndrome
- Dyskeratosis congenita (telomeropathy)
- Copper-related metabolic disorders
- Philadelphia chromosome-positive myeloid neoplasms
- Active uncontrolled infection
- Receiving medications for left ventricular systolic dysfunction
- Active HBV and HCV infections
- Prior treatment with drugs that cause heart damage
- Allergy to daunorubicin and/or cytarabine
- Currently receiving another investigational drug
- Pregnant or breastfeeding
Study Sites
St. Jude Children’s Research Hospital, Memphis, Tennessee
Other collaborating sites -
About this study
Some patients treated for cancer or blood diseases may get cancer again. This second cancer may come from the disease or its treatment.
We want to study the effects of a new drug, CPX-351, to treat 2 kinds of secondary cancers:
- Myelodysplastic syndrome (MDS)
- Acute myeloid leukemia (AML)
We aim to:
- Study how well CPX-351 treats MDS and AML
- Learn more about the side effects of CPX-351
- Learn more about how the body reacts to CPX-351
- Learn more about the biology and genetics of patients with MDS and AML
In this study patients will be treated with:
- 2 rounds of CPX-351
A stem cell transplant or other therapies may follow this treatment. We will study DNA changes in your bone marrow and blood.
Purpose of this clinical trial
This study will help us understand the effects of CPX-351 treatment in patients with MDS and AML.
Eligibility
To take part in this study, patients must:
- Be 1–22 years old
- Have 1 of the following:
- MDS or AML as a second cancer
- MDS or AML that was caused by earlier treatments
- Pass tests for kidney, liver, heart, brain, and spinal cord function
- Have recovered from earlier treatments
Patients are not eligible for this study if they are:
- Taking another new drug as part of a research study
- Taking medicine for a heart problem known as left ventricular systolic dysfunction
- Pregnant or breastfeeding
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CPXSMN Quick View Sponsor St. Jude Children's Research Hospital ClinicalTrials.gov Identifier NCT05656248 Trial start date December 2022 Estimated enrollment 10 St. Jude patients, 15 patients from other institutions
Study type Prospective Phase 2 Conditions Secondary myeloid neoplasms (SMNs), acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS)
Ages 1–22 years old Site Principal Investigator Raul Ribeiro, MD
Study site St. Jude Children’s Research Hospital For a consultation or to discuss St. Jude Physician/Patient Referral Office
1-888-226-4343
referralinfo@stjude.org
Contact
St. Jude Children’s Research Hospital
262 Danny Thomas Place
Memphis, TN 38105 USA
Voice: 1-888-226-4343 or 901-595-4055
24-Hour Emergency Access Pager: 1-800-349-4334
Email: referralinfo@stjude.org
The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.