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

Phase 1

Conditions: Acute Myeloid Leukemia
MedDRA version: 20.0Level: LLTClassification code 10060558Term: Acute myeloid leukemia recurrentSystem Organ Class: 100000012987
Therapeutic area: Diseases [C] - Cancer [C04]

Registration Number: EUCTR2017-003434-87-Outside-EU/EEA

Lead Sponsor: Cincinnati Children's Hospital Medical Center

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: A

Sex: All

Target Recruitment: 27

Inclusion Criteria

- Age 1-21 yrs at the time of study enrollment
- Diagnosis of a hematologic malignancy [acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), or aggressive lymphoma]
- Karnofsky = 50% for patients > 16 years of age and Lansky = 50 for patients =16 years of age
- Patients must have fully recovered from acute toxicities of prior therapy
- Adequate Organ Function requirements, defined as:
o Platelet count = 20 X 109/L (20,000/mcgL) (may receive platelet transfusion)
o Hemoglobin = 8.0 g/dL (may receive RBC transfusion)
o Adequate serum creatinine based on age/gender or a 24 hour creatinine clearance/ radioisotope determined GFR = 70mL/min/1.73 m2
o Direct bilirubin = 1.5 X upper limit of normal (ULN) for age
o SGPT (ALT) , 5.0 X ULN for age and institution (unless elevation is related to leukemia involvement)
o Shortening fraction of =27% by echocardiogram, or Ejection fraction of = 50% by gated radionuclide study or echocardiogram
Are the trial subjects under 18? yes
Number of subjects for this age range: 27
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Patients with Acute Promyelocytic Leukemia (APML), Down Syndrome, Fanconi Anemia, ALL and CNS leukemia (CNS status 3).
- Pregnant or breast-feeding women and males or females of reproductive potential unwilling to use an effective contraceptive method.
- Growth factors that support platelet or white cell number or function must not have been administered within the 7 days prior to enrollment.
- Patients who are currently receiving another investigational drug or anti-cancer agents (with the exception of intrathecal cytarabine and oral hydroxyurea. Hydroxyurea must be discontinued 24 hours prior to initiation of protocol therapy).
- Patients who have an uncontrolled infection, history of Wilson’s disease or other copper metabolism, had major surgery within 4 weeks of enrollment or received prior radiation to the mediastinum.
- Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To determine a safe and tolerable dose of CPX-351 in children and adolescents with relapsed or refractory hematopoietic malignancies and to recommend a dose for future studies.;Secondary Objective: To estimate the overall response rate to a single course of CPX-351 to young patients with recurrent or <br>refractory hematologic malignancies.;Primary end point(s): ? Determine rate of dose limiting toxicities <br>? Number of participants with dose limiting toxicities to determine maximum tolerated dose<br>? Pharmacokinetics: Serum concentration of CPX-351 components and metabolites<br>;Timepoint(s) of evaluation of this end point: ? Day 56<br>? Day 56<br>? Day 10

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): ? Overall response rate after a single course of CPX-351<br>? Electrocardigram, echocardiogram, peripheral blood cardiac troponin-T (cTn-T) and brain natriuretic peptide (BnP);Timepoint(s) of evaluation of this end point: ? Day 28<br>? Day 30