Safety Study of CPX-351 in Children With Relapsed Leukemia or Lymphoma
A Phase I/Pilot Study of CPX-351 for Children, Adolescents and Young Adults With Recurrent or Refractory Hematologic Malignancies
2 other identifiers
interventional
27
1 country
1
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_1
Started Sep 2013
Longer than P75 for phase_1
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
September 1, 2013
CompletedFirst Submitted
Initial submission to the registry
September 12, 2013
CompletedFirst Posted
Study publicly available on registry
September 17, 2013
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
April 1, 2019
CompletedNovember 9, 2020
November 1, 2020
5.5 years
September 12, 2013
November 6, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (3)
Determine rate of dose limiting toxicities
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.
56 days
Number of participants with dose limiting toxicities to determine maximum tolerated dose.
If 2 or more participants have dose limiting toxicities at a given dose level, the maximum tolerated dose will have been exceeded.
56 Days
Pharmacokinetics: Serum concentration of CPX-351 components (cytarabine and daunorubicin) and metabolites.
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.
10 Days
Secondary Outcomes (2)
Tumor measurement by bone marrow biopsy, blood counts, and/or PET/CT scan
28 days
Serum levels of biomarkers (troponin-1, troponin-T, and B-type natriuretic) of cardiac injury.
30 days
Study Arms (1)
CPX-351
EXPERIMENTALCPX-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.
Interventions
Eligibility Criteria
You may qualify if:
- Age
- months to 21 years at time of enrollment into dose exploration phase
- months to 30 years at time of enrollment into expanded phase
- Diagnosis: Patients must have a diagnosis of a hematologic malignancy (acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), or aggressive lymphoma.
- Disease Status
- Acute myeloid leukemia - patients with non-therapy related AML must be in first or greater relapse or have refractory disease to at least two courses of induction therapy.
- Acute lymphoid leukemia - patients with ALL must be in second or greater relapse or have relapsed disease refractory to re-induction therapy.
- Aggressive Lymphoma - patients must have relapsed or refractory disease for which there is no known curative therapy available. Patients must have measurable disease by CT scan.
- Performance status: Karnofsky \> or = to 50% or Lansky \> or = to 50.
- Prior therapy: Patients must have fully recovered from acute toxicities of prior therapy.
- Hematopoetic Stem cell transplant (HSCT): Patients who relapsed after HSCT, are eligible provided they have no evidence of active graft versus host disease (GVHD) and are at least 2 months post-transplant.
- Anthracycline exposure: Patients who have not previously had TBI (total body irradiation) must have a total previous cumulative anthracycline exposure ≤ 450 mg/m2 daunorubicin equivalents. Patients who have had prior TBI or radiation to the mediastinum must have a previous cumulative anthracycline exposure e ≤ 300 mg/m2 daunorubicin equivalents.
- Cytotoxic therapy:
- AML and Lymphoma: at least 14 days must have elapsed since the completion of systemic cytotoxic therapy, with the exception of hydroxyurea.
- ALL: patients who relapsed while receiving standard maintenance therapy do not have a waiting period. At least 14 days must have elapsed since receiving systemic cytarabine or an anthracycline/anthracenedione.
- +7 more criteria
You may not qualify if:
- Patients with the following diagnosis are not eligible: acute promyelocytic leukemia (APML), Down Syndrome, Fanconi Anemia, acute lymphoblastic leukemia with central nervous system leukemia (CNS status 3), Wilson's disease
- Pregnant or breast-feeding women. Males and females of reproductive potential may not participate unless they have agreed to use an effective method of contraception.
- Concomitant medications
- Growth factors- growth factors that support platelet or white cell number or function must not be administered within 7 days prior to enrollment.
- Investigational drugs - patients currently receiving another investigational drug are not eligible.
- Anti-cancer agents- patients who are currently receiving other anti-cancer agents are not eligible with the exception of intrathecal cytarabine and oral hydroxyurea. Hydroxyurea must be discontinued 24 hours prior to initiation of protocol therapy.
- Infection: Patients who have an uncontrolled infection are not eligible.
- Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible.
- History of Wilson's disease or other copper-metabolism disorder
- Major surgery within 4 weeks of enrollment.
- Greater than 13.6 Gy prior radiation to the mediastinum
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
John Perentesis, MD
Children's Hospital Medical Center, Cincinnati
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 12, 2013
First Posted
September 17, 2013
Study Start
September 1, 2013
Primary Completion
March 1, 2019
Study Completion
April 1, 2019
Last Updated
November 9, 2020
Record last verified: 2020-11