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Combination treatment for relapsed or refractory acute myeloid leukemia with FLT3 mutation

An Escalation/Expansion, Open Label, Multicenter Study of Iadademstat and Gilteritinib in Patients With Relapsed or Refractory Acute Myeloid Leukemia (R/R AML) With FMS-like Tyrosine Kinase Mutation (FLT3 Mut+): The FRIDA Study

Phase 1 Interventional Oryzon Genomics S.A. · NCT05546580

This study is testing a new combination of two medications to see if they can help people with relapsed or refractory acute myeloid leukemia that has a specific genetic mutation.

Quick facts

PhasePhase 1
Study typeInterventional
Enrollment50 (estimated)
Ages18 Years and up
SexAll
SponsorOryzon Genomics S.A. Industry-sponsored
Drugs / interventionsquizartinib, gilteritinib, chemotherapy, radiation
Locations13 sites (Gilbert, Arizona and 12 other locations)
Trial IDNCT05546580 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the safety and effectiveness of iadademstat combined with gilteritinib in patients suffering from relapsed or refractory acute myeloid leukemia (AML) with FLT3 mutations. The study is divided into two parts: a dose-finding phase to determine the optimal dosage and assess safety, and an expansion phase to evaluate the treatment's activity at the selected dose. Participants will receive both medications orally, and the trial aims to establish a pharmacologically active dose of iadademstat. The study is open-label and single-arm, focusing on patients with specific genetic markers associated with their AML.

Who should consider this trial

Good fit: Ideal candidates include adults diagnosed with relapsed or refractory AML who have specific FLT3 mutations and meet other eligibility criteria.

Not a fit: Patients without FLT3 mutations or those with other types of AML may not benefit from this treatment.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with difficult-to-treat FLT3-mutated AML.

How similar studies have performed: Previous studies have shown promise in targeting FLT3 mutations in AML, suggesting potential for success with this combination approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Main Inclusion Criteria:

* Diagnosis of primary AML or AML with myelodysplasia-related changes (AML-MRC)
* Patient is in first or second relapse or has refractory disease. Patients must have had histologic verification of AML at the original diagnosis.
* Patient must be positive for the following FLT3 mutations in bone marrow or PB: FLT3 internal tandem duplication (ITD), FLT3 tyrosine kinase domain (TKD) D835 or I836 or FLT3-ITD and specified FLT3-TKD.
* ECOG performance status 0-2
* Life expectancy of at least 3 months in the opinion of the investigator.
* Normal hepatic and renal function.
* Patient is able to swallow oral medications.
* Female patients are postmenopausal, documented as surgically sterile, use two methods of contraception or practice true abstinence and have a negative urine pregnancy test at screening.
* Male patients even if surgically sterilized agree to practice true abstinence or use highly effective barrier contraception.

Main Exclusion Criteria:

* Diagnosis of acute promyelocytic leukemia.
* Known BCR-ABL-positive leukemia.
* AML secondary to prior chemotherapy for other neoplasms (except for MDS).
* AML that has relapsed after or is refractory to more than 2 lines of therapy.
* Clinically active central nervous system leukemia or prior history of NCI CTCAE Grade ≥ 3 drug-related CNS toxicity.
* Major surgery or radiation therapy within 4 weeks prior to the first study dose.
* Prior treatment with iadademstat is not allowed. Treatment with any other agents with KDM1A/LSD1 inhibitory activity is only allowed if treatment finalized at least 3 weeks prior to first dose on study. Previous treatment with FLT3 inhibitors is allowed in the following cases: midostaurin and sorafenib are allowed when used in first-line therapy regimen as part of induction, consolidation and/or maintenance: quizartinib and gilteritinib are allowed when used in first-line therapy regimen, as part of induction, consolidation and/or maintenance, ONLY if patients were not refractory to the drugs or if responding, relapse did not occur while on these drugs.
* Patients not eligible to receive gilteritinib per label.
* Prior treatment with 3 or more lines of AML therapy.
* Treatment with any investigational products within 3 weeks prior to first dose of study treatment.
* Uncontrolled hypertension or poorly controlled diabetes.
* Evidence of active uncontrolled viral, bacterial, or systemic fungal infection.
* Pregnant or lactating women.

Where this trial is running

Gilbert, Arizona and 12 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Acute Myeloid Leukemia, in RelapseAcute Myeloid Leukemia RefractoryAcute Myeloid Leukemia, FLT3 mut
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.