Safety Study of CPX-351 in Children With Relapsed Leukemia or Lymphoma
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|ClinicalTrials.gov Identifier: NCT01943682|
Recruitment Status : Completed
First Posted : September 17, 2013
Last Update Posted : June 4, 2020
The purpose of this study is to test the safety of a study drug called CPX-351. This drug has been tested in adults but not yet in children and adolescents. This study tests different doses of the drug to see which dose is safer in children and adolescents.
Patients who have blood cancer are being asked to take part in this study . Blood cancers may include leukemia and lymphoma. Patients able to be in this study have already been treated with standard chemotherapy for their disease and the disease is still growing or has come back.
CPX-351 is a drug that is not yet approved by the United States Food and Drug Administration (FDA) and is only used in research studies like this one. CPX-351 is made up of two chemotherapy drugs that patients may have already received called cytarabine and daunorubicin that are now packaged together.
Another purpose of this study is to collect blood samples for special research studies. Researchers want to study how much of the CPX-351 is in the body over time. These studies are call pharmacokinetic studies or PK studies for short. PK studies require the collection of several blood samples before and after participants are given the study drug.
|Condition or disease||Intervention/treatment||Phase|
|Acute Myeloid Leukemia Acute Lymphoblastic Leukemia Burkitt Lymphoma Diffuse Large Cell Lymphoma Gray Zone Lymphoma Lymphoblastic Lymphoma Anaplastic Large Cell Lymphoma Hodgkin Lymphoma||Drug: CPX-351||Phase 1|
Cytarabine in combination with an anthracycline is a frequently used chemotherapy platform for both newly diagnosed and relapsed/refractory acute myeloid leukemia (AML) and other hematologic malignancies. Synergistic antitumor activity has been demonstrated between cytarabine and daunorubicin that is dependent upon the ratio of the drugs with the best therapeutic effect observed with a cytarabine to daunorubicin ratio of 5:1 in in vitro and in vivo models. CPX-351 is a liposomal preparation of cytarabine and daunorubicin that maintains this therapeutic drug ratio 24 hours post infusion. The altered biodistribution from encapsulation may result in a greater therapeutic effect in patients with relapsed hematologic malignancies and demonstrate greater tolerability than non-liposomal cytarabine and daunorubicin.
This is a single institution phase-I pilot study that aims to assess the pharmacokinetics, toxicity and tolerability of CPX-351 in pediatric and young adults with relapsed/refractory hematologic malignancies. Subjects will receive a single course of CPX-351 administered on Days 1, 3, and 5. The study will first open to children in a dose exploration phase, and then be available to an expanded cohort, which will be open to children and young adults once a tolerable dose has been determined.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||27 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase I/Pilot Study of CPX-351 for Children, Adolescents and Young Adults With Recurrent or Refractory Hematologic Malignancies|
|Study Start Date :||September 2013|
|Actual Primary Completion Date :||March 2019|
|Actual Study Completion Date :||April 2019|
CPX-351 is made up of two chemotherapy drugs that patients may have already received called cytarabine and daunorubicin that are now packaged together. Subjects will receive a single course of CPX-351 administered on Days 1, 3, and 5.
Comparison of Different doses of drug
- Determine rate of dose limiting toxicities [ Time Frame: 56 days ]Any Grade 3 or greater adverse event that can be possible/probably/or definitely attributable to CPX-351 that occurs between Day 1 and Day 56.
- Number of participants with dose limiting toxicities to determine maximum tolerated dose. [ Time Frame: 56 Days ]If 2 or more participants have dose limiting toxicities at a given dose level, the maximum tolerated dose will have been exceeded.
- Pharmacokinetics: Serum concentration of CPX-351 components (cytarabine and daunorubicin) and metabolites. [ Time Frame: 10 Days ]Blood samples will be collected on Day 5 (prior to CPX-351 infusion, 45 minutes (mid infusion), 90 minutes (immediately post-infusion), 2 hr, 5 hr, 8 hr, 12 hrs, Day 6, Day 8, and Day 10. Serum will be analyzed for drug and metabolite concentrations.
- Tumor measurement by bone marrow biopsy, blood counts, and/or PET/CT scan [ Time Frame: 28 days ]Tumor measurements will be used to assess disease response per standard response criteria for acute myeloid leukemia, acute lymphoid leukemia and lymphoma.
- Serum levels of biomarkers (troponin-1, troponin-T, and B-type natriuretic) of cardiac injury. [ Time Frame: 30 days ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01943682
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center|
|Cincinnati, Ohio, United States, 45229|
|Principal Investigator:||Michael Absalon, MD, PhD||Children's Hospital Medical Center, Cincinnati|