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Acute Myeloid LeukemiaPhase 3NCT05183035

Venetoclax in Children With Relapsed Acute Myeloid Leukemia (AML)

A Phase 3 study testing Fludarabine, Cytarabine and Gemtuzumab Ozogamicin in people with Acute Myeloid Leukemia, run by PedAL BCU, LLC at 90 sites in 20 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

A study to evaluate if the randomized addition of venetoclax to a chemotherapy backbone (fludarabine/cytarabine/gemtuzumab ozogamicin [GO]) improves survival of children/adolescents/young adults with acute myeloid leukemia (AML) in 1st relapse who are unable to receive additional anthracyclines, or in 2nd relapse. Read more about this study

Who is it for?
Ages 29 days to 21 years
Conditions the study is for
Acute Myeloid Leukemia
What is being tested?
Fludarabine, Cytarabine and Gemtuzumab Ozogamicin
Could I receive a placebo?
No, everyone receives an active treatment
How many people
120 participants
Where
90 sitesin 20 countries
Study dates
1 Oct 2022to Apr 2031
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 29 days to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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:
  • 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.
  • 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
Show all 37Show fewer
  • 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:
  • 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.
  • Intrathecal cytotoxic therapy: No wash-out time is required for participants having received any combination of intrathecal cytarabine, methotrexate, and/or hydrocortisone.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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).
  • 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.
  • 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.
  • Participants with prior exposure to venetoclax are eligible in this trial.
  • Adequate organ function:
  • 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
  • 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.
  • 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.

Who cannot join

  • 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.
Show all 23Show fewer
  • 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.

Trial locations and contacts

90 sites in 20 countries.

90 sites in 20 countries

United States45 sites

  • C.S. Mott Children's Hospital

    Ann Arbor, Michigan, 48109-4259

  • Children's Healthcare of Atlanta

    Atlanta, Georgia, 30322

  • Children's Hospital Colorado

    Aurora, Colorado, 80045

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611

  • Comer Children's Hospital

    Chicago, Illinois, 60637

  • Prisma Health Richland Hospital

    Columbia, South Carolina, 29203

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205

  • Harold C. Simmons Comprehensive Cancer Center

    Dallas, Texas, 75235

  • Children's Hospital of Michigan

    Detroit, Michigan, 48201

  • Golisano Children's Hospital of Southwest Florida

    Fort Myers, Florida, 33908

  • University of Florida Health Shands Children's Hospital

    Gainesville, Florida, 32610

  • Hackensack University Medical Center, HMH

    Hackensack, New Jersey, 07601

  • Kapi'olani Medical Center for Women and Children

    Honolulu, Hawaii, 96826

  • Texas Children's Hospital

    Houston, Texas, 77030

  • Indiana University School of Medicine

    Indianapolis, Indiana, 46202

  • University of Iowa Stead Family Children's Hospital

    Iowa City, Iowa, 52242

  • University of Mississippi Medical Center

    Jackson, Mississippi, 39216

  • Nemours Children's Specialty Care Jacksonville

    Jacksonville, Florida, 32207

  • The Children's Mercy Hospital - Adele Hall Campus

    Kansas City, Missouri, 64108

  • Alliance for Childhood Diseases dba Cure 4 The Kids Foundation

    Las Vegas, Nevada, 89135

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202

  • MemorialCare Miller Children's and Women's Hospital Long Beach

    Long Beach, California, 90806

  • Norton Children's Hospital

    Louisville, Kentucky, 40202

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105-3678

  • Masonic Cancer Center

    Minneapolis, Minnesota, 55455

  • Morristown Medical Center

    Morristown, New Jersey, 07960

  • Monroe Carell Jr. Children's Hospital at Vanderbilt

    Nashville, Tennessee, 37232

  • Yale University

    New Haven, Connecticut, 06511

  • Columbia University Irving Medical Center

    New York, New York, 10032

  • Memorial Sloan Kettering Cancer Center - New York

    New York, New York, 10065

  • Children's Hospital of Orange County Main Campus - Orange

    Orange, California, 92868

  • Nemours Children's Hospital - Orlando

    Orlando, Florida, 32827

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016

  • Doernbecher Children's Hospital

    Portland, Oregon, 97239

  • Cohen Children's Medical Center

    Queens, New York, 11040

  • Children's Hospital of Richmond at Virginia Commonwealth University

    Richmond, Virginia, 23219

  • Primary Children's Hospital

    Salt Lake City, Utah, 84113

  • Benioff Children's Hospital - Mission Bay

    San Francisco, California, 94158

  • Seattle Children's Hospital

    Seattle, Washington, 98105

  • Washington University School of Medicine in St. Louis

    St Louis, Missouri, 63110

  • Saint Joseph's Hospital - Tampa

    Tampa, Florida, 33607

  • Children's National - Main Hospital

    Washington D.C., District of Columbia, 20010

  • Nemours Alfred I. Dupont Hospital for Children

    Wilmington, Delaware, 19803

Other trials in United States

Canada6 sites

  • Alberta Children's Hospital

    Calgary, Alberta, T3B 6A8

  • Izaak Walton Killam (IWK) Health Center

    Halifax, Nova Scotia, B3K 6R8

  • Children's Hospital of Eastern Ontario

    Ottawa, Ontario, K1H 8L1

  • SickKids - The Hospital for Sick Children

    Toronto, Ontario, M5G 1X8

  • British Columbia Children's Hospital

    Vancouver, British Columbia, V6H 3N1

  • CancerCare Manitoba

    Winnipeg, Manitoba, R3E 0V9

Other trials in Canada

France6 sites

  • Hôpital Jeanne de Flandre

    Loos, Hauts-de-France, 59120

  • Institut d'Hématologie et d'Oncologie Pédiatrique

    Lyon, Rhône, 69008

  • CHU de Nantes - Hôpital Femme-Enfant-Adolescent

    Nantes, Loire-Atlantique, 44093

  • Hôpital Armand-Trousseau

    Paris, Île-de-France Region, 75012

  • Hôpital Universitaire Robert-Debré

    Paris, Île-de-France Region, 75019

  • CHU de Toulouse - Hôpital des Enfants

    Toulouse, Haute-Garonne, 31059

Other trials in France

Germany5 sites

  • Universitätsklinikum Augsburg

    Augsburg, 86156

  • Charité - Universitätsmedizin Berlin

    Berlin, 13353

  • Universitätsklinikum Frankfurt

    Frankfurt, 60590

  • Padiatrische Hamatologie und Onkologie

    Münster, 48149

  • Universitätsklinikum Münster

    Münster, 49149

Other trials in Germany

Japan5 sites

  • Hyogo Prefectural Kobe Children's Hospital

    Kobe, Hyōgo, 650-0047

  • Japanese Red Cross Aichi Medical Center Nagoya Daiichi Hospital

    Nagoya, Aiti, 453-8511

  • Osaka City General Hospital

    Osaka, 534-0021

  • Saitama Prefectural Children's Medical Center

    Saitama-Shi, Saitama, 330-8777

  • National Center for Child Health and Development

    Setagaya-Ku, Tokyo, 157-8535

Other trials in Japan

Italy4 sites

  • Istituto Giannina Gaslini

    Genova, Genoa, 16147

  • Fondazione IRCCS San Gerardo dei Tintori

    Monza, Monza and Brianza, 20900

  • Ospedale Pediatrico Bambino Gesù

    Roma, Rome, 00165

  • Ospedale Infantile Regina Margherita

    Torino, Turin, 10126

Other trials in Italy

Spain4 sites

  • Hospital Universitari Vall d'Hebrón

    Barcelona, 08035

  • Hospital Sant Joan de Déu Barcelona

    Barcelona, 08950

  • Hospital Infantil Universitario Niño Jesús

    Madrid, 28009

  • Hospital Universitario La Fe

    Valencia, 46026

Other trials in Spain

Australia3 sites

  • Perth Children's Hospital

    Nedlands, Western Australia, 6009

  • The Royal Children's Hospital - Children's Cancer Centre

    Parkville, Victoria, 3052

  • Children's Health Queensland Hospital and Health Service

    South Brisbane, Queensland, 4101

Other trials in Australia

Austria1 site

  • Sankt Anna-Kinderspital

    Vienna, 1090

Other trials in Austria

Belgium1 site

  • Universitair Ziekenhuis Gent

    Ghent, Oost-Vlaanderen, 9000

Other trials in Belgium

Czechia1 site

  • Fakultni nemocnice v Motole

    Prague, Prague, 150 06

Other trials in Czechia

Denmark1 site

  • Rigshospitalet

    Copenhagen, Capital Region, 2100

Other trials in Denmark

Finland1 site

  • Uusi Lastensairaala

    Helsinki, Etelä-Suomen Lääni, FI-00290

Other trials in Finland

Israel1 site

  • Schneider Children's Medical Center of Israel

    Petach Tikvah, Central District, 4920235

Other trials in Israel

Netherlands1 site

  • Prinses Maxima Centrum Kinderoncologie

    Utrecht, 3584 CS

Other trials in Netherlands

New Zealand1 site

  • Starship Children's Hospital

    Grafton, Auckland, 1023

Other trials in New Zealand

Norway1 site

  • Oslo Universitetssykehus

    Oslo, 0372

Other trials in Norway

Portugal1 site

  • Instituto Portugues De Oncologia De Lisboa Francisco Gentil

    Lisbon, Lisbon District, 1099-023

Other trials in Portugal

Sweden1 site

  • Karolinska Universitetssjukhuset Solna

    Stockholm, Stockholm County, 171 76

Other trials in Sweden

Switzerland1 site

  • Universitaets - Kinderspital Zürich

    Zurich, 8032

Other trials in Switzerland

About this study

Official title: A Randomized Phase 3 Trial of Fludarabine/Cytarabine/Gemtuzumab Ozogamicin With or Without Venetoclax in Children With Relapsed AML

A study to evaluate if the randomized addition of venetoclax to a chemotherapy backbone (fludarabine/cytarabine/gemtuzumab ozogamicin [GO]) improves survival of children/adolescents/young adults with acute myeloid leukemia (AML) in 1st relapse who are unable to receive additional anthracyclines, or in 2nd relapse.

Read the detailed description

Relapse of AML is driven by chemotherapy resistant stem cells. One mechanism of chemotherapeutic resistance in AML is the overexpression of the protein B-cell lymphoma 2 (BCL-2), an anti-apoptotic protein which sequesters intracellular activators of apoptosis. Venetoclax is a selective, potent, orally bioavailable, small molecule inhibitor of BCL-2 that restores programmed cell death in cancer cells.

This is a trial for children, adolescents and young adults with 2nd relapsed AML or 1st relapsed AML unable to receive additional anthracycline.

This is randomized trial of venetoclax in combination with intensive chemotherapy (fludarabine/cytarabine/gemtuzumab ozogamicin) for the first two cycles (42-day-cycles) that would inform and evaluate if this agent is an effective option for this population to improve its poor prognosis. Participants can receive up to two cycles of induction chemotherapy before hematopoietic stem cell transplantation (HSCT). If participants who have perceived clinical benefit cannot be transplanted after the 2 cycles, maintenance treatment may be given at the discretion of the investigator. In Arm B (experimental arm), participants can continue venetoclax if they have perceived clinical benefit, and maintenance therapy will combine venetoclax with azacitidine for a maximum of 24 cycles. In Arm A (control arm), participants will receive azacitidine in monotherapy. Maintenance is continued until clinical progression or unacceptable toxicity with a maximum of 24 cycles.

Being tested
Fludarabine, Cytarabine, Gemtuzumab Ozogamicin, Azacitidine, Venetoclax
Keywords
Venetoclax, Gemtuzumab Ozogamicin, Fludarabine, Cytarabine, Relapsed refractory, Azacitidine
Medical subject headings
Leukemia, Myeloid, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • FludarabineDrug

    Intravenous (IV) infusion

  • CytarabineDrug

    Intravenous (IV) infusion

  • Gemtuzumab OzogamicinDrug

    Intravenous (IV) infusion

  • AzacitidineDrug

    Intravenous (IV) infusion or subcutaneous injection

  • VenetoclaxDrug

    Orally via tablet or powder suspension

Study groups in NCT05183035
Study groupRoleWhat they receive
Arm A: Control Arm without VenetoclaxActive comparatorDuring Cycle 1 (42-day-cycles), participants will receive 30 mg/m^2 of fludarabine followed by 2 g/m^2 of cytarabine on Days 1-5. Gemtuzumab 3 mg/m^2 will be given on Day 6 (only for participants with CD33 expression on leukemia blasts). During Cycle 2 participants will receive 30 mg/m^2 of fludarabine followed by 2 g/m^2 of cytarabine on Days 1-5. After Cycle 2 participants are assessed for HSCT or azacitidine maintenance therapy.
Receives: Drug: Fludarabine; Drug: Cytarabine; Drug: Gemtuzumab Ozogamicin; Drug: Azacitidine
Arm B: Experimental Arm with VenetoclaxExperimentalDuring Cycle 1 (42-day-cycles), participants will receive 300 mg adult dose equivalent of venetoclax once on Day 1 followed by 600 mg adult dose equivalent of venetoclax on Days 2-21. Participants will also receive 30 mg/m^2 of fludarabine followed by 2 g/m^2 of cytarabine on Days 8-12. Gemtuzumab 3 mg/m^2 will be given on Day 13 (only for participants with CD33 expression on leukemia blasts). During Cycle 2, participants will receive 600 mg adult dose equivalent of venetoclax on Days 1-21. Participants will receive 30 mg/m^2 of fludarabine followed by 2 g/m^2 of cytarabine on Days 1-5. After Cycle 2 participants are assessed for HSCT or azacitidine maintenance therapy in combination with venetoclax.
Receives: Drug: Fludarabine; Drug: Cytarabine; Drug: Gemtuzumab Ozogamicin; Drug: Azacitidine; Drug: Venetoclax

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall Survival (OS)

    Measured: Up to 5 years

Secondary outcomes (15)

  1. Morphology Event Free Survival (EFS)

    Measured: Up to 5 years

  2. Flow-based Event Free Survival (EFS)

    Measured: Up to 5 years

  3. Flow-based Overall Response Rate (ORR)

    Measured: Up to Day 84

  4. Morphological Overall Response Rate (ORR)

    Measured: Up to Day 84

  5. Duration of Response (DOR)

    Measured: Up to 5 years

  6. Cumulative Incidence of Relapse (CIR)

    Measured: Up to 5 years

  7. Disease-related Mortality

    Measured: Up to 5 years

  8. Non-disease-related Mortality

    Measured: Up to 5 years

  9. Hematopoietic Stem Cell Transplantation (HSCT) Rate

    Measured: Up to 5 years

  10. Number of Participants with Adverse Events (AEs)

    Measured: Up to 5 years

  11. Maximum Observed Plasma Concentration (Cmax) of Venetoclax

    Measured: Pre-dose, 2, 4, 6, 8, and 24 hours post-dose on Cycle 1 Day 8 and Day 13 (cycle is 42 days); once on follow-up visits of Cycle 2 between Day 5 and Day 21

  12. Time to Maximum Observed Plasma Concentration (Tmax) of Venetoclax

    Measured: Pre-dose, 2, 4, 6, 8, and 24 hours post-dose on Cycle 1 Day 8 and Day 13 (cycle is 42 days); once on follow-up visits of Cycle 2 between Day 5 and Day 21

  13. Area Under the Plasma Concentration-time Curve Over a 24-hour Dose Interval (AUC0-24)

    Measured: Pre-dose, 2, 4, 6, 8, and 24 hours post-dose on Cycle 1 Day 8 and Day 13 (cycle is 42 days); once on follow-up visits of Cycle 2 between Day 5 and Day 21

  14. Number of Participants That Are Pediatric Minimal Residual Disease (Ped-MRD) Negative with Complete Remission (CR), Partial Complete Remission (CRp), or Complete Remission with Incomplete Hematologic Recovery (CRi)

    Measured: Up to 5 years

  15. Number of Participants with International Working Group Complete Response (IWG-CR)

    Measured: Up to 5 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 Jan 2022
  2. Study start1 Oct 2022
  3. Primary completionFeb 2027 (estimated)
  4. Study completionApr 2031 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
120 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05183035
Sponsor's protocol ID
ITCC-101/APAL2020D
Other IDs
2021-003212-11, 2023-510160-12-00, 2023
Status last verified
Nov 2026
First submitted
21 Dec 2021
Last updated
6 Nov 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Daniels KP, Pounds SB, Wu H, Wang L, Alexander TB, Budhraja A, Wolf J, Westover T, Mead PE, Mehr CM, Gupta SD, Lacayo NJ, Inaba H, Rees M, Pui CH, Opferman JT, Klco JM, Rubnitz JE, Karol SE. Venetoclax in combination with cytarabine with or without idarubicin or azacitidine in children, adolescents, and young adults with relapsed or refractory acute myeloid leukaemia (VENAML): a multicentre, phase 1 expansion study. Lancet Haematol. 2026 Aug;13(8):e545-e554. doi: 10.1016/S2352-3026(26)00136-5. Epub 2026 Jul 8.

    PubMed 42419338

  • Badawi M, Gopalakrishnan S, Engelhardt B, Palenski T, Karol SE, Rubnitz JE, Menon R, Salem AH. Dosing of Venetoclax in Pediatric Patients with Relapsed Acute Myeloid Leukemia: Analysis of Developmental Pharmacokinetics and Exposure-Response Relationships. Clin Ther. 2024 Oct;46(10):759-767. doi: 10.1016/j.clinthera.2024.09.008. Epub 2024 Oct 5.

    PubMed 39368878

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Fludarabine, Cytarabine and Gemtuzumab Ozogamicin in people with Acute Myeloid Leukemia, run by PedAL BCU, LLC at 90 sites in 20 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 37 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 6 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 90 sites in United States, Canada, France, Germany, Japan and 15 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall Survival (OS) (time frame: Up to 5 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05183035, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Nov 2026.

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