Treatment Study for Children and Adolescents With Acute Promyelocytic Leukemia

NCT04793919 | Recruiting Phase 2 DMC

• 2 other identifiers: ICC APL STUDY 022017-002383-40
• Study Type: interventional
• Target: 89
• Locations: 12 countries
• Sites: 31

Brief Summary

The trial is open to all patients with a diagnosis of acute promyelocytic leukemia (APL) who are PCR-positive for the PML-RARα transcript and less than 18 years of age.

Conditions

Acute Promyelocytic Leukemia

Outcome Measures

Primary Outcomes (1)

• Event Free Survival (EFS) probability

  SR patients: To evaluate the efficacy in terms of event-free survival of a treatment combining arsenic trioxide (ATO) and all-trans retinoic acid (ATRA) in newly diagnosed APL standard-risk children and adolescents HR patients: To evaluate the efficacy in terms of event-free survival of a treatment combining arsenic trioxide (ATO), all-trans retinoic acid (ATRA) and gemtuzumab ozogamicin (GO) in newly diagnosed APL high-risk children and adolescents

  3 years

Secondary Outcomes (10)

• Rate of hematological CR/CRi after induction

  5 years

• Rate of molecular CR/CRi after induction

  5 years

• Rate of early death during induction

  5 years

• Probability of overall survival (OS) at 3 years

  3 years

• Cumulative incidence of relapse (CIR) at 3 years

  3 years

Study Arms (2)

Standard Risk (SR)

ACTIVE COMPARATOR

Patient with APL and WBC less than 10x10e9/L at presentation before start treatment

Drug: Arsenic Trioxide; Drug: All-trans retinoic acid

High Risk (HR)

EXPERIMENTAL

Patient with APL, with the highest pre-treatment WBC count equal to or greater than 10x10e9/L at presentation

Drug: Mylotarg; Drug: Arsenic Trioxide; Drug: All-trans retinoic acid

Interventions

Mylotarg DRUG

See the protocol

Also known as: GO

High Risk (HR)

Arsenic Trioxide DRUG

See the protocol

Also known as: ATO

High Risk (HR); Standard Risk (SR)

All-trans retinoic acid DRUG

See the protocol

Also known as: ATRA

High Risk (HR); Standard Risk (SR)

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

• Patients with a clinical diagnosis of APL but subsequently found to lack PML/RARα rearrangement should be withdrawn from the study and treated on an alternative protocol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

Sponsors & Collaborators

• Associazione Italiana Ematologia Oncologia Pediatrica: lead

Study Sites (31)

Hôpital Universitaire des Enfants Reine Fabiola (Huderf)

Brussels, 1020, Belgium

NOT YET RECRUITING

University Hospital Motol

Prague, 15006, Czechia

NOT YET RECRUITING

Pediatrics and Adolescent Medicine Aarhus University Hospital

Aarhus N, 8200, Denmark

NOT YET RECRUITING

CHU de Bordeaux - Hôpital des Enfants

Bordeaux, 33076, France

RECRUITING

Universitätsklinikum Essen (AöR) Zentrum für Kinder-und Jugendmedizin Klinik für Kinderheilkunde III

Essen, 45147, Germany

NOT YET RECRUITING

Our Lady's Children's Hospital Crumlin

Dublin, 12, Ireland

NOT YET RECRUITING

Rappaport Children'S Hospital, Rambam Health Care Campus

Haifa, Israel

NOT YET RECRUITING

Ospedale "Casa Sollievo della Sofferenza" - UO Oncoematologia Pediatrica

San Giovanni Rotondo, Foggia, 71013, Italy

RECRUITING

AOU Policlinico Dipartimento di Pediatria

Bari, 70124, Italy

NOT YET RECRUITING

Ospedale Papa Giovanni XXIII - USS Oncoematologia Pediatrica

Bergamo, 24100, Italy

NOT YET RECRUITING

AOU Policlinico Sant'Orsola-Malpighi - Oncologia ed Ematologia Pediatrica

Bologna, 40138, Italy

RECRUITING

Ospedale Pediatrico Microcitemico "A.Cau", Az.Ospedaliera Brotzu - SC Oncoematologia Ped. e Patologia della coagulazione

Cagliari, 09121, Italy

RECRUITING

AOU Policlinico Vittorio Emanuele - UOC Ematologia ed Oncologia Pediatrica con TNO

Catania, 95123, Italy

RECRUITING

A.O. Universitaria Meyer - DAI Oncoematologia Pediatrica

Florence, 50139, Italy

NOT YET RECRUITING

IRCCS Istituto Gannina Gaslini - Dipartimento di Oncoematologia

Genova, 16147, Italy

RECRUITING

Fondazione Monza e Brianza per il Bambino e la sua Mamma (MBBM) - Ospedale San Gerardo

Monza, 20900, Italy

RECRUITING

AORN Santobono-Pausilipon

Napoli, 80123, Italy

RECRUITING

Univerità degli Studi della Campania- Luigi Vanvitelli - Sevizio di Oncologia Pediatrica

Napoli, 80138, Italy

RECRUITING

Azienda Ospedaliera di Padova - Oncoematologia Pediatrica

Padua, 35128, Italy

NOT YET RECRUITING

ARNAS Civico di Cristina e Benfratelli - UOC Oncoematologia Pediatrica

Palermo, 90127, Italy

NOT YET RECRUITING

Fondazione IRCCS Policlinico San Matteo - Oncoematologia Pediatrica

Pavia, 27100, Italy

RECRUITING

Ospedale santa Chiara - AOU Pisana, UO Oncoematologia Pediatrica

Pisa, 56126, Italy

NOT YET RECRUITING

Policlinico Umberto I Università "LA Sapienza" - Dip. Biotecnologie cellulari ed ematologia UOS Ematologia Pediatrica

Roma, 00161, Italy

NOT YET RECRUITING

Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica - Ospedale Pediatrico "Bambino Gesù"

Roma, 00165, Italy

RECRUITING

AOU Città della Salute e della Scienza di Torino - Presidio Infantile Regina Margherita

Torino, 10126, Italy

RECRUITING

VU medisch centrum

Amsterdam, Netherlands

NOT YET RECRUITING

Centro Hospitalar Universitário de Coimbra - Hospital Pediátrico de Coimbra

Coimbra, 3000-602, Portugal

NOT YET RECRUITING

Instituto Português de Oncologia de Lisboa Francisco Gentil, EPE

Lisbon, 1099-023, Portugal

NOT YET RECRUITING

Instituto Português de Oncologia do Porto Francisco Gentil, E. P. E.

Porto, 4200-072, Portugal

NOT YET RECRUITING

Valencia University Medical School University Hospital La Fe

Valencia, 46026, Spain

NOT YET RECRUITING

Childrens hematology and oncology Uppsala University

Uppsala, .O. Box 256, SE-751 05, Sweden

NOT YET RECRUITING

MeSH Terms

Conditions

Leukemia, Promyelocytic, Acute

Interventions

Gemtuzumab; Arsenic Trioxide; Tretinoin

Condition Hierarchy (Ancestors)

Leukemia, Myeloid, Acute; Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Calicheamicins; Aminoglycosides; Glycosides; Carbohydrates; Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Arsenicals; Inorganic Chemicals; Oxides; Oxygen Compounds; Vitamin A; Retinoids; Carotenoids; Polyenes; Alkenes; Hydrocarbons, Acyclic; Hydrocarbons; Organic Chemicals; Cyclohexenes; Cyclohexanes; Cycloparaffins; Hydrocarbons, Alicyclic; Hydrocarbons, Cyclic; Terpenes; Diterpenes; Pigments, Biological; Biological Factors

Study Officials

• Fanco Locatelli, Prof

  Dept. of Pediatric Hematology Oncology - Bambino Gesù Children's Hospital Rome

  PRINCIPAL INVESTIGATOR

Central Study Contacts

AIEOP

CONTACT

0039 051 2144667; studiclinici@aieop.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 13, 2019
• First Posted: March 11, 2021
• Study Start: October 9, 2019
• Primary Completion: October 9, 2025
• Study Completion (Estimated): October 10, 2027
• Last Updated: April 15, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will not share

Locations

CZ (1); NL (1); ES (1); IE (1); IT (18); SE (1); PT (3); FR (1); DK (1); BE (1); IL (1); DE (1)