Treatment study for children and adolescents with Acute Promyelocytic Leukemia

Phase 2

Conditions: acute promyelocytic leukemia
blood cancer
10024324

Registration Number: NL-OMON54336

Lead Sponsor: AIEOP- Associazione Italiana Ematologia Oncologia Pediatrica

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Pending

Sex: Not specified

Target Recruitment: 5

Inclusion Criteria

- Newly diagnosed APL confirmed by the presence of PML/RARa fusion gene
- Age <18 years
- Written informed consent by parents or legal guardians
- If applicable, female participants must have pregnancy test by beta-HCG
dosing and be negative.
- Patients of child-bearing or child-fathering potential must be willing to
practice the most appropriate approach for birth control from the time of
enrollment in this study and for 3 months after receiving the latest infusion.

Exclusion Criteria

- Patients with a clinical diagnosis of APL but subsequently found to lack
PML/RARa rearrangement should be withdrawn from the study and treated with an
alternative protocol
- Significant liver dysfunction (bilirubin serum levels >3 mg/dL, ALT/AST serum
levels >5x the normal values)
- Creatinine serum levels >2 times the normal value for age
- Significant arrhythmias, ECG abnormalities, other cardiac contraindications
(L-FEV <= 50% or LV-FS <=28%)
- Neuropathy grade 2 or greater
- Concurrent active malignancy
- Uncontrolled life-threatening infections
- Pregnant or lactating females
- Patients who had received alternative therapy (APL not initially suspected;
ATRA and/or ATO not available)

Study & Design

Study Type: Observational non invasive

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>The primary endpoint of the study is event-free survival (EFS). This cumulative<br /><br>endpoint includes the following events:<br /><br>• no achievement of hematological complete remission after induction therapy;<br /><br>• no achievement of molecular remission after three consolidation courses<br /><br>(molecular resistance);<br /><br>• relapse (hematological/molecular);<br /><br>• death, including early death, at 2 years from diagnosis.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>• Rate of hematological CR after induction<br /><br>• Rate of early and aplastic death during induction<br /><br>• Overall survival (OS)<br /><br>• Cumulative incidence of either hematological and molecular relapse (CIR)<br /><br>• Incidence of hematological and non-hematological toxicity<br /><br>• Kinetics of MRD clearance<br /><br>• Rate of molecular remission after 3 consolidation cycles<br /><br>• Assessment of PML/RARa transcript level reduction during treatment<br /><br>• Toxicity - hematological and non-hematological<br /><br>• Supportive care requirements<br /><br>• Total hospitalization days during therapy and health economic impact</p><br>