Treatment for Children and Adolescents with Acute Promyelocytic Leukemia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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* Patients with a clinical diagnosis of APL but subsequently found to lack PML/RARα rearrangement should be withdrawn from the study and treated on an alternative protocol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 it's running

• CHU de Bordeaux - Hôpital des Enfants
  Bordeaux, France Recruiting
• Ospedale "Casa Sollievo della Sofferenza" - UO Oncoematologia Pediatrica
  San Giovanni Rotondo, Foggia, Italy Recruiting
• AOU Policlinico Sant'Orsola-Malpighi - Oncologia ed Ematologia Pediatrica
  Bologna, Italy Recruiting
• Ospedale Pediatrico Microcitemico "A.Cau", Az.Ospedaliera Brotzu - SC Oncoematologia Ped. e Patologia della coagulazione
  Cagliari, Italy Recruiting
• AOU Policlinico Vittorio Emanuele - UOC Ematologia ed Oncologia Pediatrica con TNO
  Catania, Italy Recruiting
• IRCCS Istituto Gannina Gaslini - Dipartimento di Oncoematologia
  Genova, Italy Recruiting

Study contacts

• Principal investigator: Fanco Locatelli, Prof — Dept. of Pediatric Hematology Oncology - Bambino Gesù Children's Hospital Rome
• Study coordinator: Aieop
• Email: studiclinici@aieop.org
• Phone: 0039 051 2144667

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• QTX-2101 (oral arsenic) plus ATRA for adults with low- or intermediate-risk APL

  This Phase 3 trial tests whether an oral arsenic capsule (QTX-2101) combined with ATRA works well and is safe for adults aged 18–70 with newly diagnosed low- or intermediate-risk acute promyelocytic leukemia.

  Recruiting at 36 sites Phase 3 Quetzal Therapeutics · Birmingham, Alabama and 35 other locations · NCT07504458

• Using advanced imaging to detect extramedullary myeloid leukemia in newly diagnosed acute myeloid leukemia patients

  This study is testing new imaging methods to see if they can help find extramedullary myeloid leukemia in people who have just been diagnosed with acute myeloid leukemia.

  Recruiting Phase 1 M.D. Anderson Cancer Center · Houston, Texas · NCT02390635

• Combining tretinoin and arsenic trioxide with or without gemtuzumab ozogamicin for treating acute promyelocytic leukemia

  This study is testing a new combination of treatments for people with acute promyelocytic leukemia to see if it helps them stay healthy longer and improves their chances of recovery without causing more side effects.

  Recruiting at 5 sites Phase 2 M.D. Anderson Cancer Center · Houston, Texas and 4 other locations · NCT01409161

• Combining oral arsenic and ATRA for treating newly diagnosed acute promyelocytic leukemia

  This study is testing if a new treatment combining oral arsenic and ATRA can help newly diagnosed patients with acute promyelocytic leukemia live longer and feel better, especially those at high risk.

  Recruiting Phase 2 Instituto do Cancer do Estado de São Paulo · São Paulo, São Paulo · NCT06982274

• Testing TURALIO(R) for treating hard-to-treat cancers in young people

  This study is testing if TURALIO(R) can safely help children and young adults with tough-to-treat cancers by seeing how well it works to shrink tumors or stop them from growing.

  Recruiting Phase 1 National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT02390752

• Evaluating first line treatment for adult acute promyelocytic leukemia with ATO and ATRA

  This study is testing how well a combination of arsenic trioxide and all-trans retinoic acid works as the first treatment for adults with acute promyelocytic leukemia in Argentina.

  Recruiting at 6 sites Grupo Argentino de Tratamiento de la Leucemia Aguda · La Plata, Buenos Aires and 5 other locations · NCT04897490

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.