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.
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
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 130 participants
- Where
- 90 sitesin 20 countries
- Study dates
- 1 Oct 2022to Apr 2031
- Phase
- Phase 3
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
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- 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.
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- 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. Contact the study team or a site near you to ask about taking part.
90 sites in 20 countries
United States45 sites, 45 recruiting
- Recruiting
C.S. Mott Children's Hospital
Ann Arbor, Michigan, 48109-4259
- Recruiting
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
- Recruiting
Children's Hospital Colorado
Aurora, Colorado, 80045
- Recruiting
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
- Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
- Recruiting
Comer Children's Hospital
Chicago, Illinois, 60637
- Recruiting
Prisma Health Richland Hospital
Columbia, South Carolina, 29203
- Recruiting
Nationwide Children's Hospital
Columbus, Ohio, 43205
- Recruiting
Harold C. Simmons Comprehensive Cancer Center
Dallas, Texas, 75235
- Recruiting
Children's Hospital of Michigan
Detroit, Michigan, 48201
- Recruiting
Golisano Children's Hospital of Southwest Florida
Fort Myers, Florida, 33908
- Recruiting
University of Florida Health Shands Children's Hospital
Gainesville, Florida, 32610
- Recruiting
Hackensack University Medical Center, HMH
Hackensack, New Jersey, 07601
- Recruiting
Kapi'olani Medical Center for Women and Children
Honolulu, Hawaii, 96826
- Recruiting
Texas Children's Hospital
Houston, Texas, 77030
- Recruiting
Indiana University School of Medicine
Indianapolis, Indiana, 46202
- Recruiting
University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, 52242
- Recruiting
University of Mississippi Medical Center
Jackson, Mississippi, 39216
- Recruiting
Nemours Children's Specialty Care Jacksonville
Jacksonville, Florida, 32207
- Recruiting
The Children's Mercy Hospital - Adele Hall Campus
Kansas City, Missouri, 64108
- Recruiting
Alliance for Childhood Diseases dba Cure 4 The Kids Foundation
Las Vegas, Nevada, 89135
- Recruiting
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
- Recruiting
MemorialCare Miller Children's and Women's Hospital Long Beach
Long Beach, California, 90806
- Recruiting
Norton Children's Hospital
Louisville, Kentucky, 40202
- Recruiting
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105-3678
- Recruiting
Masonic Cancer Center
Minneapolis, Minnesota, 55455
- Recruiting
Morristown Medical Center
Morristown, New Jersey, 07960
- Recruiting
Monroe Carell Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, 37232
- Recruiting
Yale University
New Haven, Connecticut, 06511
- Recruiting
Columbia University Irving Medical Center
New York, New York, 10032
- Recruiting
Memorial Sloan Kettering Cancer Center - New York
New York, New York, 10065
- Recruiting
Children's Hospital of Orange County Main Campus - Orange
Orange, California, 92868
- Recruiting
Nemours Children's Hospital - Orlando
Orlando, Florida, 32827
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
- Recruiting
Phoenix Children's Hospital
Phoenix, Arizona, 85016
- Recruiting
Doernbecher Children's Hospital
Portland, Oregon, 97239
- Recruiting
Cohen Children's Medical Center
Queens, New York, 11040
- Recruiting
Children's Hospital of Richmond at Virginia Commonwealth University
Richmond, Virginia, 23219
- Recruiting
Primary Children's Hospital
Salt Lake City, Utah, 84113
- Recruiting
Benioff Children's Hospital - Mission Bay
San Francisco, California, 94158
- Recruiting
Seattle Children's Hospital
Seattle, Washington, 98105
- Recruiting
Washington University School of Medicine in St. Louis
St Louis, Missouri, 63110
- Recruiting
Saint Joseph's Hospital - Tampa
Tampa, Florida, 33607
- Recruiting
Children's National - Main Hospital
Washington D.C., District of Columbia, 20010
- Recruiting
Nemours Alfred I. Dupont Hospital for Children
Wilmington, Delaware, 19803
Canada6 sites, 5 recruiting
- Recruiting
Alberta Children's Hospital
Calgary, Alberta, T3B 6A8
- Recruiting
Izaak Walton Killam (IWK) Health Center
Halifax, Nova Scotia, B3K 6R8
- Recruiting
Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1
- Recruiting
SickKids - The Hospital for Sick Children
Toronto, Ontario, M5G 1X8
- Completed
British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3N1
- Recruiting
CancerCare Manitoba
Winnipeg, Manitoba, R3E 0V9
France6 sites, 6 recruiting
- Recruiting
Hôpital Jeanne de Flandre
Loos, Hauts-de-France, 59120
- Recruiting
Institut d'Hématologie et d'Oncologie Pédiatrique
Lyon, Rhône, 69008
- Recruiting
CHU de Nantes - Hôpital Femme-Enfant-Adolescent
Nantes, Loire-Atlantique, 44093
- Recruiting
Hôpital Armand-Trousseau
Paris, Île-de-France Region, 75012
- Recruiting
Hôpital Universitaire Robert-Debré
Paris, Île-de-France Region, 75019
- Recruiting
CHU de Toulouse - Hôpital des Enfants
Toulouse, Haute-Garonne, 31059
Germany5 sites, 5 recruiting
- Recruiting
Universitätsklinikum Augsburg
Augsburg, 86156
- Recruiting
Charité - Universitätsmedizin Berlin
Berlin, 13353
- Recruiting
Universitätsklinikum Frankfurt
Frankfurt, 60590
- Recruiting
Padiatrische Hamatologie und Onkologie
Münster, 48149
- Recruiting
Universitätsklinikum Münster
Münster, 49149
Japan5 sites, 5 recruiting
- Recruiting
Hyogo Prefectural Kobe Children's Hospital
Kobe, Hyōgo, 650-0047
- Recruiting
Japanese Red Cross Aichi Medical Center Nagoya Daiichi Hospital
Nagoya, Aiti, 453-8511
- Recruiting
Osaka City General Hospital
Osaka, 534-0021
- Recruiting
Saitama Prefectural Children's Medical Center
Saitama-Shi, Saitama, 330-8777
- Recruiting
National Center for Child Health and Development
Setagaya-Ku, Tokyo, 157-8535
Italy4 sites, 4 recruiting
- Recruiting
Istituto Giannina Gaslini
Genova, Genoa, 16147
- Recruiting
Fondazione IRCCS San Gerardo dei Tintori
Monza, Monza and Brianza, 20900
- Recruiting
Ospedale Pediatrico Bambino Gesù
Roma, Rome, 00165
- Recruiting
Ospedale Infantile Regina Margherita
Torino, Turin, 10126
Spain4 sites, 4 recruiting
- Recruiting
Hospital Universitari Vall d'Hebrón
Barcelona, 08035
- Recruiting
Hospital Sant Joan de Déu Barcelona
Barcelona, 08950
- Recruiting
Hospital Infantil Universitario Niño Jesús
Madrid, 28009
- Recruiting
Hospital Universitario La Fe
Valencia, 46026
Australia3 sites, 3 recruiting
- Recruiting
Perth Children's Hospital
Nedlands, Western Australia, 6009
- Recruiting
The Royal Children's Hospital - Children's Cancer Centre
Parkville, Victoria, 3052
- Recruiting
Children's Health Queensland Hospital and Health Service
South Brisbane, Queensland, 4101
Belgium1 site, 1 recruiting
- Recruiting
Universitair Ziekenhuis Gent
Ghent, Oost-Vlaanderen, 9000
Czechia1 site, 1 recruiting
- Recruiting
Fakultni nemocnice v Motole
Prague, Prague, 150 06
Denmark1 site, 1 recruiting
- Recruiting
Rigshospitalet
Copenhagen, Capital Region, 2100
Finland1 site, 1 recruiting
- Recruiting
Uusi Lastensairaala
Helsinki, Etelä-Suomen Lääni, FI-00290
Israel1 site, 1 recruiting
- Recruiting
Schneider Children's Medical Center of Israel
Petach Tikvah, Central District, 4920235
Netherlands1 site, 1 recruiting
- Recruiting
Prinses Maxima Centrum Kinderoncologie
Utrecht, 3584 CS
New Zealand1 site, 1 recruiting
- Recruiting
Starship Children's Hospital
Grafton, Auckland, 1023
Portugal1 site, 1 recruiting
- Recruiting
Instituto Portugues De Oncologia De Lisboa Francisco Gentil
Lisbon, Lisbon District, 1099-023
Sweden1 site, 1 recruiting
- Recruiting
Karolinska Universitetssjukhuset Solna
Stockholm, Stockholm County, 171 76
Switzerland1 site, 1 recruiting
- Recruiting
Universitaets - Kinderspital Zürich
Zurich, 8032
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.
- Conditions
- Acute Myeloid Leukemia
- 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 group | Role | What they receive |
|---|---|---|
| Arm A: Control Arm without Venetoclax | Active comparator | During 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 Venetoclax | Experimental | During 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.
Overall Survival (OS)
Measured: Up to 5 years
Secondary outcomes (15)
Morphology Event Free Survival (EFS)
Measured: Up to 5 years
Flow-based Event Free Survival (EFS)
Measured: Up to 5 years
Flow-based Overall Response Rate (ORR)
Measured: Up to Day 84
Morphological Overall Response Rate (ORR)
Measured: Up to Day 84
Duration of Response (DOR)
Measured: Up to 5 years
Cumulative Incidence of Relapse (CIR)
Measured: Up to 5 years
Disease-related Mortality
Measured: Up to 5 years
Non-disease-related Mortality
Measured: Up to 5 years
Hematopoietic Stem Cell Transplantation (HSCT) Rate
Measured: Up to 5 years
Number of Participants with Adverse Events (AEs)
Measured: Up to 5 years
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
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
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
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
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.
- First posted10 Jan 2022
- Study start1 Oct 2022
- Primary completionFeb 2027 (estimated)
- Study completionApr 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- 130 participants (estimated)
Sponsor and investigators
- Lead sponsor
- PedAL BCU, LLC
- Sponsor type
- University, hospital or other
- Collaborators
- Princess Maxima Center for Pediatric Oncology (European Sponsor), AbbVie, Roche-Genentech, EuPAL
- Responsible party
- Sponsor
- Investigators
- Seth Karol, MD, Principal investigator, St. Jude Children's Research Hospital
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
- Aug 2026
- First submitted
- 21 Dec 2021
- Last updated
- 24 Aug 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 42419338Badawi 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