NCT05183035

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

Recruiting now Phase 3 Last updated 18 May 2026

What this trial tests

Phase 3 trial testing Fludarabine in Acute Myeloid Leukemia in 130 participants. Currently enrolling.

Timeline

1 October 2022

Primary endpoint
1 February 2027

1 April 2031

Quick facts

Lead sponsor	PedAL BCU, LLC
Phase	Phase 3
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	130
Start date	1 October 2022
Primary completion	1 February 2027
Estimated completion	1 April 2031
Sites	90 locations across United States, Australia, Austria, Belgium, Canada, Czechia, Denmark, Finland

Drugs / interventions tested

• Fludarabine (FLUDARABINE) — full drug profile →
• Cytarabine — full drug profile →
• Gemtuzumab Ozogamicin (Gemtuzumab Ozogamicin) — full drug profile →
• Azacitidine (azacitidine) — full drug profile →
• Venetoclax (venetoclax) — full drug profile →

Conditions studied

• Acute Myeloid Leukemia — all drugs for Acute Myeloid Leukemia →

Sponsor

PedAL BCU, LLC

Who can join

Adults 29 Days to 21, any sex, with Acute Myeloid Leukemia. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Overall Survival (OS)
  Time frame: Up to 5 years

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Relapsed pediatric acute myeloid leukaemia: state-of-the-art in 2023.
   Egan G, Tasian SK. · · 2023 · cited 28× · PMID 36861399 · DOI 10.3324/haematol.2022.281106
2. Risk-Stratified Therapy for Pediatric Acute Myeloid Leukemia.
   Tomizawa D, Tsujimoto SI. · · 2023 · cited 22× · PMID 37627199 · DOI 10.3390/cancers15164171
3. Children's Oncology Group's 2023 blueprint for research: Myeloid neoplasms.
   Cooper TM, Alonzo TA, Tasian SK, Kutny MA, et al · · 2023 · cited 22× · PMID 37480164 · DOI 10.1002/pbc.30584
4. AML under the Scope: Current Strategies and Treatment Involving FLT3 Inhibitors and Venetoclax-Based Regimens.
   Milnerowicz S, Maszewska J, Skowera P, Stelmach M, et al · · 2023 · cited 10× · PMID 37958832 · DOI 10.3390/ijms242115849
5. Emerging and Future Targeted Therapies for Pediatric Acute Myeloid Leukemia: Targeting the Leukemia Stem Cells.
   Murphy LA, Winters AC. · · 2023 · cited 6× · PMID 38137469 · DOI 10.3390/biomedicines11123248
6. Venetoclax Combination Treatment of Acute Myeloid Leukemia in Adolescents and Young Adult Patients.
   Chatzikalil E, Roka K, Diamantopoulos PT, Rigatou E, et al · · 2024 · cited 5× · PMID 38610812 · DOI 10.3390/jcm13072046
7. Dosing of Venetoclax in Pediatric Patients with Relapsed Acute Myeloid Leukemia: Analysis of Developmental Pharmacokinetics and Exposure-Response Relationships.
   Badawi M, Gopalakrishnan S, Engelhardt B, Palenski T, et al · · 2024 · cited 4× · PMID 39368878 · DOI 10.1016/j.clinthera.2024.09.008
8. The PedAL/EuPAL Project: A Global Initiative to Address the Unmet Medical Needs of Pediatric Patients with Relapsed or Refractory Acute Myeloid Leukemia.
   Ceolin V, Ishimaru S, Karol SE, Bautista F, et al · · 2023 · cited 3× · PMID 38201506 · DOI 10.3390/cancers16010078