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Acute Myeloid LeukemiaPhase 1NCT01943682

Safety Study of CPX-351 in Children With Relapsed Leukemia or Lymphoma

A Phase 1 study testing CPX-351 in people with Acute Myeloid Leukemia and Acute Lymphoblastic Leukemia, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 12 months to 30 years
What is being tested?
CPX-351
Could I receive a placebo?
No, everyone receives an active treatment
How many people
27 participants
Where
1 siteUnited States
Study dates
Sep 2013to Apr 2019
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 months to 30 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Age
  • 12 months to 21 years at time of enrollment into dose exploration phase
  • 12 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.
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  • 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.
  • Intrathecal cytotoxic therapy: no waiting period is required for patients receiving intrathecal cytarabine, methotrexate and/or hydrocortisone. At least 14 days must have elapsed since receiving liposomal cytarabine in intrathecal injection.
  • Organ function requirements
  • Adequate bone marrow function - platelet count >/= 20,000/uL (may receive platelet transfusions; Hemoglobin >/= 8 g/dL (may receive red blood cell transfusions)
  • Adequate Renal function - a maximum serum creatinine is based on age/gender. Subjects that do not meet eligibility criteria based upon serum creatinine may meet eligibility criteria based upon a 24 hour creatinine clearance or radioisotope determined GFR >/= 70 mL/min/1.73 m2.
  • Adequate liver function - Direct bilirubin </= 1.5 x upper limit of normal (ULN) for age and SGPT (ALT) < 5.0 x upper limit of normal (ULN) for age and institution (unless elevation is related to leukemia involvement).
  • Adequate cardiac function - Shortening fraction of >/= 27% by echocardiogram, or Ejection fraction of >/= 50% by gated radionuclide study or echocardiogram.
  • Central Nervous system function - patients with seizure disorder may be enrolled if on anticonvulsants and well controlled and CNS toxicity </= Grade 2.

Who cannot join

  • 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.
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  • 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

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229

Other trials in United States

About this study

Official title: A Phase I/Pilot Study of CPX-351 for Children, Adolescents and Young Adults With Recurrent or Refractory Hematologic Malignancies

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.

Read more

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.

Read the detailed description

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.

Being tested
CPX-351
Keywords
Relapsed acute myeloid leukemia, non-therapy related acute myeloid leukemia, Refractory acute myeloid leukemia, Untreated acute myeloid leukemia, Lymphoma, Leukemia
Medical subject headings
Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Burkitt Lymphoma, Lymphoma, Large B-Cell, Diffuse, Lymphoma, Large-Cell, Anaplastic, Hodgkin Disease, Lymphoma, Leukemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CPX-351Drug

    Comparison of Different doses of drug

Study groups in NCT01943682
Study groupRoleWhat they receive
CPX-351ExperimentalCPX-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.
Receives: Drug: CPX-351

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: 56 days

  2. 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.

    Measured: 56 Days

  3. 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.

    Measured: 10 Days

Secondary outcomes (2)

  1. Tumor measurement by bone marrow biopsy, blood counts, and/or PET/CT scan

    Tumor measurements will be used to assess disease response per standard response criteria for acute myeloid leukemia, acute lymphoid leukemia and lymphoma.

    Measured: 28 days

  2. Serum levels of biomarkers (troponin-1, troponin-T, and B-type natriuretic) of cardiac injury.

    Measured: 30 days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Sep 2013 (estimated)
  2. Study startSep 2013
  3. Primary completionMar 2019
  4. Study completionApr 2019

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
27 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01943682
Sponsor's protocol ID
CPX-MA-1201
Other IDs
2013-4305
Status last verified
Nov 2020
First submitted
12 Sep 2013
Last updated
9 Nov 2020
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing CPX-351 in people with Acute Myeloid Leukemia and Acute Lymphoblastic Leukemia, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 22 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 9 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Determine rate of dose limiting toxicities (time frame: 56 days). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01943682.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Nov 2020.

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