Promising new combo aims to boost survival in kids with relapsed leukemia

NCT ID NCT05183035

Disease control Sponsor: PedAL BCU, LLC Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Aug 25, 2026 · Updated 2 times

Summary

This study tests whether adding the drug venetoclax to standard chemotherapy helps children and young adults with relapsed acute myeloid leukemia (AML) live longer. About 130 participants aged 29 days to 21 years will be randomly assigned to receive either chemotherapy alone or chemotherapy plus venetoclax. The goal is to improve overall survival and reduce the chance of the cancer coming back.

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Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 130 people

The number the study aims to enrol. It can still change while the study runs.

Started

Oct 2022

Expected to finish

Apr 2031

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

29 days to 21 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria * Participants must have enrolled on APAL2020SC, NCT Number: NCT04726241 prior to enrollment on ITCC-101/APAL2020D. (This is only applicable for participants in USA/Canada/Australia/New Zealand sites/Blood Cancer United territory). * Participants must be \>28 days of age and \< 22 years of age at enrollment. * Participants must have one of the following: 1. Children, adolescents, and young adults with AML without demonstrated FLT3/internal tandem duplication (ITD) mutation. Ideally, the status of the mutation needs to be proven in the current relapse. Nevertheless, patients with previous FLT3/ITD negative test from prior lines can be included based on local results in order to not delay the start of treatment. 2. And participants must have AML which is either: * Untreated second relapse, in participants who are sufficiently fit to undergo another round of intensive chemotherapy, or * Untreated first relapse, in participants who cannot tolerate additional anthracycline containing chemotherapy per investigator discretion. * Participants must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2 (≥ 50% Lansky or Karnofsky score). * Participants must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to start of protocol treatment: 1. Cytotoxic chemotherapy: Must not have received cytotoxic chemotherapy within 14 days prior to start of protocol treatment, except for corticosteroids, low dose cytarabine or hydroxyurea that can be given up to 24 hours prior to start of protocol treatment. 2. Intrathecal cytotoxic therapy: No wash-out time is required for participants having received any combination of intrathecal cytarabine, methotrexate, and/or hydrocortisone. 3. Antibodies: ≥ 21 days must have elapsed from infusion of last dose of an antibody-drug conjugate before start of protocol treatment. For unmodified antibodies or T cell engaging antibodies, 2 half-lives must have elapsed before start of protocol treatment. Any toxicity related to prior antibody therapy must be recovered to Grade ≤ 1. 4. Interleukins, Interferons and Cytokines (other than Hematopoietic Growth Factors): ≥ 21 days after the completion of interleukins, interferon or cytokines (other than Hematopoietic Growth Factors) before start of protocol treatment. 5. Hematopoietic growth factors: ≥ 14 days after the last dose of a long-acting growth factor (e.g., pegfilgrastim) or ≥7 days for short-acting growth factor before start of protocol treatment. 6. Radiation therapy (RT) (before start of protocol treatment): * ≥ 14 days have elapsed for local palliative RT (small port); * ≥ 84 days must have elapsed if prior craniospinal RT or if ≥ 50% radiation of pelvis; * ≥ 42 days must have elapsed if other substantial bone marrow (BM) radiation. 7. Stem Cell Infusions (before start of protocol treatment): * ≥ 84 days since allogeneic (non-autologous) bone marrow or stem cell transplant (with or without total body irradiation \[TBI\]) or boost infusion (any stem cell product; not including donor lymphocyte infusion \[DLI\]); * No evidence of active graft versus host disease (GVHD). 8. Participants who are receiving cyclosporine, tacrolimus or other agents to treat or prevent either graft-versus-host disease post bone marrow transplant or organ rejection post-transplant are not eligible for this trial. Participants must be off medications to treat or prevent either graft-versus-host disease post bone marrow transplant or organ rejection post-transplant for at least 14 days prior to enrollment. 9. Cellular Therapy: ≥ 42 days after the completion of donor lymphocyte infusion (DLI) or any type of cellular therapy (e.g., modified T cells, natural killer \[NK\] cells, dendritic cells, etc.) before start of protocol treatment. 10. Participants with prior exposure to venetoclax are eligible in this trial. * Adequate organ function: 1. Adequate Renal Function defined as: * Creatinine clearance or radioisotope glomerular filtration rate (GFR) ≥ 60ml/min/1.73 m\^2, or * Normal serum creatinine based on age/sex 2. Adequate Liver Function defined as: * Direct bilirubin \< 1.5 x upper limit of normal (ULN), and * Alkaline phosphatase ≤ 2.5 x ULN, and * Serum glutamic pyruvic transaminase (SGPT) alanine aminotransferase (ALT) ≤ 2.5 x ULN. If higher transaminases outside these ranges (up to 5x ULN) are due to a radiographically identifiable leukemia infiltrate, the participant will remain eligible. Transaminase elevation up to 5x ULN is also allowed in case of steatosis on echography. 3. Cardiac performance: Minimum cardiac function defined as: * No history of congestive heart failure in need of medical treatment * No pre-treatment diminished left ventricular function on echocardiography (shortening fraction \[SF\] \< 25% or ejection fraction \[EF\] \< 40%) * No signs of congestive heart failure at presentation of relapse. * Participant, parent or guardian must sign and date informed consent and pediatric assent (when required), prior to the initiation of screening or study specific procedures, according to local law and legislation. Exclusion Criteria * Participants who in the opinion of the investigator may not be able to comply with the study requirements of the study, are not eligible. * Participants with Down syndrome. * Participants with Acute promyelocytic leukemia (APL) or Juvenile myelomonocytic leukemia (JMML). * Participants with isolated CNS3 disease or symptomatic CNS3 disease. * Participants with malabsorption syndrome or any other condition that precludes enteral administration of venetoclax. * Participants who are currently receiving an investigational drug other than those specified for this study. * Participants with Fanconi anemia, Kostmann syndrome, Shwachman syndrome or any other known congenital bone marrow failure syndrome. * Participants with known prior allergy to any of the medications used in protocol therapy. * Participants with documented active, uncontrolled infection at the time of study entry. * Known hepatitis C virus (HCV), hepatitis B virus (HBV) (known positive hepatitis B virus (HBV) surface antigen (HBsAg) results), or human immunodeficiency virus (HIV) infection. * Concomitant Medications * Participants who have received strong and moderate CYP3A inducers such as rifampin, carbamazepine, phenytoin, and St. John's wort within 7 days of the start of study treatment. * Participants who have consumed grapefruit, grapefruit products, Seville oranges (including marmalade containing Seville oranges) or starfruit within 3 days of the start of study treatment. * Participants who have hypersensitivity to the active substance or to any of the excipients listed in summary of product characteristics (SPC). * Pregnancy or Breast-Feeding: * Participants who are pregnant or breast-feeding. * Participants of reproductive potential may not participate unless they have agreed to use a highly effective contraceptive method per Clinical Trial Facilitation Group (CTFG) guidelines for the duration of study therapy and at least 30 days after last dose of venetoclax, or 7 months after gemtuzumab ozogamicin treatment, or for 6 months after the completion of all study therapy, whichever is longer. * Male participants must use a condom during intercourse and agree not to father a child or donate sperm during therapy and for the duration of study therapy and at least 30 days after last dose of venetoclax or 4 months after last dose of gemtuzumab ozogamicin, 6 months from the last dose of cytarabine, or 90-days after last exposure to any other chemotherapy, whichever is longer. Additional criteria to receive a gemtuzumab ozogamicin infusion: Gemtuzumab ozogamicin should not be given: * to participants with history of veno-occlusive disease (VOD)/Sinusoidal obstruction syndrome (SOS) grade 3 or 4 * to participants with CD33 negative leukemic blasts (determined at local lab) Note that these participants are eligible for the study but will not be treated with gemtuzumab ozogamicin.

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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Acute myeloid leukemia

How to take part

Only the study team decides who joins. These are the ways to reach them.

The places running it

89 sites in 20 countries. The list below names each one and where it is.
The official record

ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

Open the record ↗
A doctor treating you

A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

Alberta Children's Hospital
RECRUITING

Calgary, Alberta, T3B 6A8, Canada
Alliance for Childhood Diseases dba Cure 4 The Kids Foundation
RECRUITING

Las Vegas, Nevada, 89135, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITING

Chicago, Illinois, 60611, United States
Arkansas Children's Hospital
RECRUITING

Little Rock, Arkansas, 72202, United States
Benioff Children's Hospital - Mission Bay
RECRUITING

San Francisco, California, 94158, United States
British Columbia Children's Hospital
COMPLETED

Vancouver, British Columbia, V6H 3N1, Canada
C.S. Mott Children's Hospital
RECRUITING

Ann Arbor, Michigan, 48109-4259, United States
CHU de Nantes - Hôpital Femme-Enfant-Adolescent
RECRUITING

Nantes, Loire-Atlantique, 44093, France
CHU de Toulouse - Hôpital des Enfants
RECRUITING

Toulouse, Haute-Garonne, 31059, France
CancerCare Manitoba
RECRUITING

Winnipeg, Manitoba, R3E 0V9, Canada
Charité - Universitätsmedizin Berlin
RECRUITING

Berlin, 13353, Germany
Children's Health Queensland Hospital and Health Service
RECRUITING

South Brisbane, Queensland, 4101, Australia
Children's Healthcare of Atlanta
RECRUITING

Atlanta, Georgia, 30322, United States
Children's Hospital Colorado
RECRUITING

Aurora, Colorado, 80045, United States
Children's Hospital of Eastern Ontario
RECRUITING

Ottawa, Ontario, K1H 8L1, Canada
Children's Hospital of Michigan
RECRUITING

Detroit, Michigan, 48201, United States
Children's Hospital of Orange County Main Campus - Orange
RECRUITING

Orange, California, 92868, United States
Children's Hospital of Philadelphia
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Richmond at Virginia Commonwealth University
RECRUITING

Richmond, Virginia, 23219, United States
Children's National - Main Hospital
RECRUITING

Washington D.C., District of Columbia, 20010, United States
Cohen Children's Medical Center
RECRUITING

Queens, New York, 11040, United States
Columbia University Irving Medical Center
RECRUITING

New York, New York, 10032, United States
Comer Children's Hospital
RECRUITING

Chicago, Illinois, 60637, United States
Dana-Farber Cancer Institute
RECRUITING

Boston, Massachusetts, 02215, United States
Doernbecher Children's Hospital
RECRUITING

Portland, Oregon, 97239, United States
Fakultni nemocnice v Motole
RECRUITING

Prague, Prague, 150 06, Czechia
Fondazione IRCCS San Gerardo dei Tintori
RECRUITING

Monza, Monza and Brianza, 20900, Italy
Golisano Children's Hospital of Southwest Florida
RECRUITING

Fort Myers, Florida, 33908, United States
Hackensack University Medical Center, HMH
RECRUITING

Hackensack, New Jersey, 07601, United States
Harold C. Simmons Comprehensive Cancer Center
RECRUITING

Dallas, Texas, 75235, United States
Hospital Infantil Universitario Niño Jesús
RECRUITING

Madrid, 28009, Spain
Hospital Sant Joan de Déu Barcelona
RECRUITING

Barcelona, 08950, Spain
Hospital Universitari Vall d'Hebrón
RECRUITING

Barcelona, 08035, Spain
Hospital Universitario La Fe
RECRUITING

Valencia, 46026, Spain
Hyogo Prefectural Kobe Children's Hospital
RECRUITING

Kobe, Hyōgo, 650-0047, Japan
Hôpital Armand-Trousseau
RECRUITING

Paris, Île-de-France Region, 75012, France
Hôpital Jeanne de Flandre
RECRUITING

Loos, Hauts-de-France, 59120, France
Hôpital Universitaire Robert-Debré
RECRUITING

Paris, Île-de-France Region, 75019, France
Indiana University School of Medicine
RECRUITING

Indianapolis, Indiana, 46202, United States
Institut d'Hématologie et d'Oncologie Pédiatrique
RECRUITING

Lyon, Rhône, 69008, France
Instituto Portugues De Oncologia De Lisboa Francisco Gentil
RECRUITING

Lisbon, Lisbon District, 1099-023, Portugal
Istituto Giannina Gaslini
RECRUITING

Genova, Genoa, 16147, Italy
Izaak Walton Killam (IWK) Health Center
RECRUITING

Halifax, Nova Scotia, B3K 6R8, Canada
Japanese Red Cross Aichi Medical Center Nagoya Daiichi Hospital
RECRUITING

Nagoya, Aiti, 453-8511, Japan
Kapi'olani Medical Center for Women and Children
RECRUITING

Honolulu, Hawaii, 96826, United States
Karolinska Universitetssjukhuset Solna
RECRUITING

Stockholm, Stockholm County, 171 76, Sweden
Masonic Cancer Center
RECRUITING

Minneapolis, Minnesota, 55455, United States
Memorial Sloan Kettering Cancer Center - New York
RECRUITING

New York, New York, 10065, United States
MemorialCare Miller Children's and Women's Hospital Long Beach
RECRUITING

Long Beach, California, 90806, United States
Monroe Carell Jr. Children's Hospital at Vanderbilt
RECRUITING

Nashville, Tennessee, 37232, United States
Morristown Medical Center
RECRUITING

Morristown, New Jersey, 07960, United States
National Center for Child Health and Development
RECRUITING

Setagaya-Ku, Tokyo, 157-8535, Japan
Nationwide Children's Hospital
RECRUITING

Columbus, Ohio, 43205, United States
Nemours Alfred I. Dupont Hospital for Children
RECRUITING

Wilmington, Delaware, 19803, United States
Nemours Children's Hospital - Orlando
RECRUITING

Orlando, Florida, 32827, United States
Nemours Children's Specialty Care Jacksonville
RECRUITING

Jacksonville, Florida, 32207, United States
Norton Children's Hospital
RECRUITING

Louisville, Kentucky, 40202, United States
Osaka City General Hospital
RECRUITING

Osaka, 534-0021, Japan
Oslo Universitetssykehus
RECRUITING

Oslo, 0372, Norway
Ospedale Infantile Regina Margherita
RECRUITING

Torino, Turin, 10126, Italy
Ospedale Pediatrico Bambino Gesù
RECRUITING

Roma, Rome, 00165, Italy
Padiatrische Hamatologie und Onkologie
RECRUITING

Münster, 48149, Germany
Perth Children's Hospital
RECRUITING

Nedlands, Western Australia, 6009, Australia
Phoenix Children's Hospital
RECRUITING

Phoenix, Arizona, 85016, United States
Primary Children's Hospital
RECRUITING

Salt Lake City, Utah, 84113, United States
Prinses Maxima Centrum Kinderoncologie
RECRUITING

Utrecht, 3584 CS, Netherlands
Prisma Health Richland Hospital
RECRUITING

Columbia, South Carolina, 29203, United States
Rigshospitalet
RECRUITING

Copenhagen, Capital Region, 2100, Denmark
Saint Joseph's Hospital - Tampa
RECRUITING

Tampa, Florida, 33607, United States
Saitama Prefectural Children's Medical Center
RECRUITING

Saitama-Shi, Saitama, 330-8777, Japan
Sankt Anna-Kinderspital
RECRUITING

Vienna, 1090, Austria
Schneider Children's Medical Center of Israel
RECRUITING

Petach Tikvah, Central District, 4920235, Israel
Seattle Children's Hospital
RECRUITING

Seattle, Washington, 98105, United States
SickKids - The Hospital for Sick Children
RECRUITING

Toronto, Ontario, M5G 1X8, Canada
St. Jude Children's Research Hospital
RECRUITING

Memphis, Tennessee, 38105-3678, United States
Starship Children's Hospital
RECRUITING

Grafton, Auckland, 1023, New Zealand
Texas Children's Hospital
RECRUITING

Houston, Texas, 77030, United States
The Children's Mercy Hospital - Adele Hall Campus
RECRUITING

Kansas City, Missouri, 64108, United States
The Royal Children's Hospital - Children's Cancer Centre
RECRUITING

Parkville, Victoria, 3052, Australia
Universitaets - Kinderspital Zürich
RECRUITING

Zurich, 8032, Switzerland
Universitair Ziekenhuis Gent
RECRUITING

Ghent, Oost-Vlaanderen, 9000, Belgium
University of Florida Health Shands Children's Hospital
RECRUITING

Gainesville, Florida, 32610, United States
University of Iowa Stead Family Children's Hospital
RECRUITING

Iowa City, Iowa, 52242, United States
University of Mississippi Medical Center
RECRUITING

Jackson, Mississippi, 39216, United States
Universitätsklinikum Augsburg
RECRUITING

Augsburg, 86156, Germany
Universitätsklinikum Frankfurt
RECRUITING

Frankfurt, 60590, Germany
Universitätsklinikum Münster
RECRUITING

Münster, 49149, Germany
Uusi Lastensairaala
RECRUITING

Helsinki, Etelä-Suomen Lääni, FI-00290, Finland
Washington University School of Medicine in St. Louis
RECRUITING

St Louis, Missouri, 63110, United States
Yale University
RECRUITING

New Haven, Connecticut, 06511, United States

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