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Treatment for Children and Adolescents with Acute Promyelocytic Leukemia

Treatment Study for Children and Adolescents With Acute Promyelocytic Leukemia

Phase 2 Interventional Associazione Italiana Ematologia Oncologia Pediatrica · NCT04793919

This study is testing a new treatment for children and teenagers with acute promyelocytic leukemia that uses a combination of medications instead of traditional chemotherapy to see if it works better and is safer.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment89 (estimated)
AgesN/A to 18 Years
SexAll
SponsorAssociazione Italiana Ematologia Oncologia Pediatrica Academic / other
Drugs / interventionsgemtuzumab, chemotherapy
Locations31 sites (Brussels and 30 other locations)
Trial IDNCT04793919 on ClinicalTrials.gov

What this trial studies

This trial aims to evaluate a non-chemotherapy treatment approach for children and adolescents diagnosed with acute promyelocytic leukemia (APL). It involves administering a combination of arsenic trioxide (ATO) and all-trans retinoic acid (ATRA) for standard-risk patients, and adding gemtuzumab ozogamicin (GO) for high-risk patients. The study seeks to validate the efficacy and safety of this treatment compared to traditional chemotherapy-based protocols. Participants will undergo an induction course followed by consolidation blocks of ATO and ATRA. This is the first pediatric trial focusing on a treatment strategy that avoids cytostatic agents.

Who should consider this trial

Good fit: Ideal candidates are children and adolescents under 18 years old with newly diagnosed APL confirmed by the presence of the PML/RARα fusion gene.

Not a fit: Patients with significant liver dysfunction, concurrent active malignancies, or those who have received alternative therapies may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a safer, chemotherapy-free option for children with APL, potentially improving their quality of life during treatment.

How similar studies have performed: Other studies in adults have shown promising results with similar non-chemotherapy approaches, indicating potential for success in this pediatric population.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Newly diagnosed APL confirmed by the presence of PML/RARα fusion gene
* Age \<18 years
* Written informed consent by parents or legal guardians

Exclusion Criteria:

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

Where this trial is running

Brussels and 30 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Acute Promyelocytic Leukemia
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.