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Acute Myeloid Leukemia, in RelapsePhase 1FRIDANCT05546580

Study of Iadademstat and Gilteritinib in Patients With R/R AML With FMS-like Tyrosine Kinase Mutation (FLT3 Mut+)

A Phase 1 study testing Iadademstat and Gilteritinib Oral Tablet in people with Acute Myeloid Leukemia, in Relapse and Acute Myeloid Leukemia Refractory, run by Oryzon Genomics S.A. at 13 sites in United States.

Active, not recruiting

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

Official record
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Trial summary

Iadademstat is being studied as a treatment for subjects with Relapsed or Refractory Acute Myeloid Leukemia (R/R AML) with FMS-like tyrosine kinase mutation (FLT3 mut+). During the trial, iadademstat will be given in combination with gilteritinib, a drug that is already approved to treat patients with FLT3-mutated R/R… Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Iadademstat and Gilteritinib Oral Tablet
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
50 participants
Where
13 sitesUnited States
Study dates
14 Nov 2022to 5 Dec 2026
Phase
Phase 1
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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

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.

Trial locations and contacts

13 sites in 1 country.

13 sites in 1 country

United States13 sites

  • The John Hopkins University School of Medicine

    Baltimore, Maryland, 21287-0013

  • Massachusetts General Hospital (MGH)

    Boston, Massachusetts, 02114

  • Duke University Medical Center

    Durham, North Carolina, 27705

  • Banner MD Anderson Cancer Center

    Gilbert, Arizona, 85234

  • Sylvester Comprehensive Cancer Center

    Miami, Florida, 33136

  • Miami Cancer Institute

    Miami, Florida, 33176

  • Froedtert Hospital & The Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

  • West Virginia University

    Morgantown, West Virginia, 26506

  • Sarah Cannon Research Institute, LLC

    Nashville, Tennessee, 37203

  • Icahn School of Medicine at Mount Sinai and Mount Sinai Hospital

    New York, New York, 10029

  • Rutgers, The State University

    Piscataway, New Jersey, 08854

  • Oregon Health & Science University

    Portland, Oregon, 97239

  • The University of Arizona Cancer Center - North Campus

    Tucson, Arizona, 85724-5024

Other trials in United States

About this study

Official title: 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

Iadademstat is being studied as a treatment for subjects with Relapsed or Refractory Acute Myeloid Leukemia (R/R AML) with FMS-like tyrosine kinase mutation (FLT3 mut+). During the trial, iadademstat will be given in combination with gilteritinib, a drug that is already approved to treat patients with FLT3-mutated R/R AML.

Read the detailed description

This is an escalation/expansion, open label, single arm, study to investigate the safety and the RP2D of the combination of iadademstat with gilteritinib in FLT3-mutated R/R AML.

This study consists of 2 parts. A dose finding part to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD) and emerging activity of iadademstat and gilteritinib combination, and to determine the pharmacologically-active dose (i.e., the minimum safe and biologically active dose) of iadademstat in combination with gilteritinib, and an expansion part at the specific dose/s selected to evaluate the activity of iadademstat in combination with gilteritinib in patients with FLT3-mutated R/R AML.

Being tested
Iadademstat, Gilteritinib Oral Tablet
Keywords
Acute Myeloid Leukemia, FLT3 mut
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.

  • IadademstatDrug

    Also called ORY-1001, RO7051790

    iadademstat oral solution

  • Gilteritinib Oral TabletDrug

    Also called XOSPATA®

    120 mg Gilteritinib

Study groups in NCT05546580
Study groupRoleWhat they receive
Active armExperimentaliadademstat and gilteritinib
Receives: Drug: Iadademstat; Drug: Gilteritinib Oral Tablet

What is being measured

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

  1. Adverse Events (AE)

    Number of participants with Adverse Events (AE) after treatment with iadademstat in combination with gilteritinib in patients with FLT3-mutated R/R AML.

    Measured: Up to 18 months

  2. Laboratory value abnormalities and/or adverse events (AE)

    Number of participants with laboratory value abnormalities and/or Adverse Events (AE) after treatment with iadademstat in combination with gilteritinib in patients with FLT3-mutated R/R AML.

    Measured: Up to 18 months

  3. Vital sign abnormalities and/or adverse events (AEs)

    Number of participants with vital signs abnormalities and/or Adverse Events (AE) after treatment with iadademstat in combination with gilteritinib in patients with FLT3-mutated R/R AML.

    Measured: Up to 18 months

  4. Routine 12-lead electrocardiogram (ECG) abnormalities and/or Adverse Events (AEs)

    Number of participants with Routine 12-lead electrocardiogram (ECG )abnormalities and/or Adverse Events (AE) after treatment with iadademstat in combination with gilteritinib in patients with FLT3-mutated R/R AML.

    Measured: Up to 18 months

  5. Recommend Phase 2 dose (RP2D)

    Determine the recommended Phase 2 dose (RP2D) of iadademstat in combination with gilteritinib in patients with FLT3-mutated R/R

    Measured: Up to 18 months

  6. iadademstat tmax

    Measurement of the time it takes for iadademstat to reach the maximum concentration (Cmax) in blood.

    Measured: Up to 26 days

  7. Iadademstat Cmax

    Measurement of the highest concentration of iadademstat in the blood after a dose is given.

    Measured: Up to 26 days

  8. iadademstat Cmin

    Measurement of the lowest concentration of iadademstat in the blood, after a dose is given.

    Measured: Up to 26 days

  9. iadademstat AUC

    Measurement of how much iadadmestat reaches a person's bloodstream in a given period of time after a dose is given.

    Measured: Up to 26 days

  10. iadademstat Target Engagement (TE)

    Percent of drug covalently bound to LSD1 molecule

    Measured: Up to 26 days

  11. OR rate

    Proportion of patients achieving complete remission (CR), CR with incomplete hematologic recovery (CRi), and partial remission (PR).

    Measured: Up to 18 months

Secondary outcomes (7)

  1. Overall Survival (OS)

    Time from start of treatment to the time of death from any cause.

    Measured: Up to 24 months

  2. Event-Free-Survival (EFS)

    Time from start of treatment to the date of failure to achieve CR or CRi, relapse from CR/CRi, or death from any cause, whichever occurs first.

    Measured: Up to 18 months

  3. Overall response rate

    Percentage of patients with complete remission (CR), CR with incomplete blood count recovery (CRi), or PR.

    Measured: Up to 6 months

  4. Time to Response (TTR)

    Time from the date of initial dosing at RP2D/expansion dose to first documentation of either a type of CR or Partial Response (PR).

    Measured: Up to 6 months

  5. Duration of Remission (DoR)

    Time from the date of first documentation of any type of remission to the date of first documentation of progression of remission for remitters

    Measured: Up to 18 months

  6. Transfusion independence rate

    A patient is defined as red blood cell (RBC) and/or platelet-transfusion independent if he/she receives no RBC and/or platelet transfusions for a period of at least 8 weeks. Rate of transfusion independence is the percentage of patients who become RBC and/or platelet transfusion independent (from the number of patients transfusion dependent at baseline).

    Measured: Up to 18 months

  7. Transplantation Rate Time Frame

    Percentage of patients undergoing Hematopoietic Stem Cell Transplantation (HSCT) during the study period.

    Measured: Up to 18 months

Timeline

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

  1. First posted21 Sep 2022
  2. Study start14 Nov 2022
  3. Primary completion30 Jan 2026
  4. Study completion5 Dec 2026 (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 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Model description
Escalation/extension open label study
Masking
None (open label)
Enrolment
50 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05546580
Sponsor's protocol ID
CL04-ORY-1001
Acronym
FRIDA
Status last verified
Aug 2026
First submitted
15 Sep 2022
Last updated
24 Aug 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Iadademstat and Gilteritinib Oral Tablet in people with Acute Myeloid Leukemia, in Relapse and Acute Myeloid Leukemia Refractory, run by Oryzon Genomics S.A. at 13 sites in United States.

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

Who may be eligible to take part?

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 24 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 13 sites in United States. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

Adverse Events (AE) (time frame: Up to 18 months). The study lists 11 primary outcomes in all. 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 NCT05546580.

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 24 Aug 2026.

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