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AMLPhase 2AGORA-1NCT05199051

A Phase 2 Study of Gemtuzumab Ozogamicin (GO)-Gilteritinib Combination in Adults With FLT3-ITD and/or FLT3-TKD Relapse/Refractory (R/R) AML

A Phase 2 study testing Gemtuzumab ozogamicine - Cytarabine - Gilteritinib in people with AML and Leukaemia (Acute Myeloid), run by Centre Antoine Lacassagne at 12 sites in France.

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

This is a national, open-label, single-arm, multicenter phase II trial evaluating the safety and efficacy of adding gilteritinib, a new FLT3 inhibitor to the AGORA platform, consisting of the combination of an intermediate dose of cytarabine and a divided dose of GO in adult patients with R / R AML with an FLT3-ITD… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
AML, Leukaemia (Acute Myeloid)
What is being tested?
Gemtuzumab ozogamicine - Cytarabine - Gilteritinib
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
19 participants
Where
12 sitesFrance
Study dates
3 Jun 2023to 10 Jul 2028
Phase
Phase 2
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

Who may be able to join

  • Patients aged 18 years old or more
  • Confirmed diagnosis of R/R AML positive for CD33 antigen as determined locally by immunophenotyping according to routine practice, defined as:
  • AML refractory to 1 or 2 intensive chemotherapy courses or a treatment by hypomethylating agents (HMAs)
  • Or AML in first hematologic relapse or progression after front-line therapy, including intensive chemotherapy or hypomethylating agents (HMAs).
  • Previous treatments with FLT3 inhibitors (other than gilteritinib) are allowed
  • R/R AML secondary to a prior chemotherapy or radiotherapy for another cancer (tAML) could be included.
Show all 13Show fewer
  • Presence of a FLT3-ITD mutation (allelic ratio ≥0.05 at last evaluation)* or a FLT3 TKD mutation
  • Patient with no contraindication to gemtuzumab ozogamicin (GO), cytarabine and gilteritinib
  • ECOG performance status ≤2
  • AST and ALT ≤ 2.5 x upper the limit of normal (ULN) and/or total and direct serum bilirubin ≤ 1.5 x ULN unless considered due to leukemia
  • Estimated glomerular filtration rate (GFR) ≥ 50 mL/min according to the formula usually used by the investigator
  • Written informed consent obtained prior to any screening procedures
  • Eligible for National Health Insurance in France

Who cannot join

  • Acute promyelocytic leukemia or AML with BCR-ABL1 gene fusion
  • Secondary AML (sAML) defined by a history of prior myelodysplastic syndrome (MDS) or myeloproliferative syndrome (MPN) including chronic myelomonocytic leukemia (CMML)
  • Patient with contraindications to the administration of gemtuzumab ozogamicin (GO), cytarabine and gilteritinib. Refer to the SPCs of the molecules mentioned concerning the contraindications, special warnings, precautions for use, dose modifications in the event of toxicity, contraception and monitoring of patients and drugs prohibited or to be used with caution.
  • Proven central nervous system leukemic involvement
  • Prior allogeneic HSCT within the last 6 months and/or history of acute GVHD of grade >1
  • Prior treatment with gemtuzumab ozogamicin within the last 3 months preceding the initiation of the treatment in the present clinical trial
Show all 35Show fewer
  • Uncontrolled or active malignant disease within prior 12 months (excluding cutaneous basal cell carcinoma, "in-situ" carcinoma of the cervix or breast, or other local malignancy excised)
  • Uncontrolled or significant cardiovascular history or symptoms including:
  • Prior anthracycline exposure equivalent to more than 550 mg/m2 of daunorubicin
  • History of clinically relevant ventricular arrhythmias (e.g. ventricular tachycardia, ventricular fibrillation or torsade de pointes)
  • History of 2° (Mobitz II) or 3° heart block (subjects with pacemakers are allowed if they have no history of clinically relevant arrhythmias with the pacemaker)
  • History of uncontrolled angina pectoris or MI within 6 months
  • History of NYHA Class 3 or 4 heart failure
  • Left ventricular ejection fraction ≤ 50% or less than the institutional lower limit of normal
  • History of complete left bundle branch block
  • Unstable angina, New York Heart Association (NYHA) class 3 or 4 congestive heart failure
  • QTcF > or equal to 450 msec, long QT syndrome (including family history)
  • Bradycardia < 50 bpm (unless subject has a pacemaker)
  • Systolic BP ≥ 180 mmHg or diastolic BP ≥ 110 mmHg
  • Uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate treatment)
  • Active known HBV or HCV hepatitis or positive HIV serology
  • Concurrent therapy with any other investigational agent or cytotoxic drug, within 28 days before starting treatment. Only hydroxyurea ± dexamethasone is permitted for the control of blood counts
  • Current use or anticipated requirement for drugs that are known strong inducers of CYP3 A4/5
  • Current use or anticipated requirement for drugs that are known as strong inhibitors or inducers of P glycoprotein (P-gp), as mentioned in the appendix 14 of the protocol, with the exception of drugs that are considered absolutely essential for the care of the subject
  • Current use or anticipated treatment with concomitant drugs that target 5HT1R or 5HT2BR receptors or sigma non-specific receptor, as mentioned in the appendix 15 of the protocol, with exception of drugs that are considered absolutely essential for the care of the subject
  • Known malabsorption syndrome or other condition that may significantly impair absorption of oral study medications
  • Any of concurrent severe and/or uncontrolled medical condition, which could compromise participation in the study.
  • Patient currently receiving one or more inadvisable or prohibited treatments described in section 6.4.2 of the protocol.
  • Females who are pregnant or breastfeeding.
  • Women of childbearing potential or partners of women of childbearing potential should use a highly effective method of contraception during treatment and change the duration of contraception after the last dose of the medicines of the study (GO, cytarabine and gilteritinib).
  • For women of childbearing potential (WOCBP): agreement to remain abstinent (refrain from heterosexual intercourse) or use contraception with a failure rate of < 1% per year during the treatment period and after the final dose of study treatment for at least: - 7 months for GO, - 6 months for cytarabine, - 6 months for gilteritinib. Women must refrain from donating eggs during this same period. A woman is considered to be of childbearing potential if she is postmenarcheal, has not reached a postmenopausal state (≥ 12 continuous months of amenorrhea with no identified cause other than menopause), and has not undergone surgical sterilization is permanently infertile due to surgery (i.e. removal of ovaries, fallopian tubes, and/or uterus) or another cause as determined by the investigator (e.g., Müllerian agenesis). The definition of childbearing potential may be adapted for alignment with local guidelines or regulations. Examples of contraceptive methods with a failure rate of < 1% per year include bilateral tubal ligation, male sterilization, hormonal contraceptives that inhibit ovulation, hormone-releasing intrauterine devices, and copper intrauterine devices. Hormonal contraceptive methods must be supplemented by a barrier method. The reliability of sexual abstinence should be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, or postovulation methods) and withdrawal are not acceptable methods of contraception. If required per local guidelines or regulations, locally recognized acceptable methods of contraception and information about the reliability of abstinence will be described in the local Informed Consent Form. Women of childbearing potential must have a negative serum pregnancy test result within 7 days prior to first dose.
  • For male patients: acceptance that most of the study treatments require specific reliable and effective contraception measures, as well as measures related to sperm donation Agreement to remain abstinent (refrain from heterosexual intercourse) or use contraception, and agreement to refrain from donating sperm, as defined below With a female partner of childbearing potential who is not pregnant, men who are not surgically sterile must remain abstinent or use a condom plus an additional contraceptive method that together result in a failure rate of < 1% per year during the treatment period and after the final dose of study treatment for at least: - 4 months for GO, - 6 months for cytarabine, - 4 months for gilteritinib.
  • Men must refrain from donating sperm during this same period With pregnant female partners, men must remain abstinent or use a condom to avoid exposing the embryo during the treatment period and for at least: - 4 months for GO, - 6 months for cytarabine, - 4 months for gilteritinib. The reliability of sexual abstinence should be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, or postovulation methods) and withdrawal are not acceptable methods of contraception. If required per local guidelines or regulations, locally recognized acceptable methods of contraception and information about the reliability of abstinence will be described in the local Informed Consent Form.
  • Adults subject to a legal protection order or unable to give their consent
  • Persons deprived of their freedom by judicial or administrative decision, persons hospitalized without their consent by virtue of articles L. 3212-1 and L. 3213-1 and who are not subject to the provisions of article L. 1121-8.

Trial locations and contacts

12 sites in 1 country.

12 sites in 1 country

France12 sites

  • CHU d'Amiens

    Amiens

  • CHU d'Angers

    Angers

  • CHU Bordeaux

    Bordeaux

  • CHU de Caen

    Caen

  • CHMS de Chambery

    Chambéry

  • HIA Percy

    Clamart

  • CHU Limoges

    Limoges

  • CHRU Nancy

    Nancy

  • Centre Hospitalier Universitaire de Nice

    Nice

  • Hôpital Saint Louis

    Paris

  • Centre Henri Becquerel

    Rouen

  • Centre Hospitalier de Versailles

    Versailles

Other trials in France

About this study

Official title: AGORA-1 /ALFA 2100 Study : A Phase 2 Study of Gemtuzumab Ozogamicin (GO)-Gilteritinib Combination in Adults With FLT3-ITD and/or FLT3-TKD Relapse/Refractory (R/R) AML

This is a national, open-label, single-arm, multicenter phase II trial evaluating the safety and efficacy of adding gilteritinib, a new FLT3 inhibitor to the AGORA platform, consisting of the combination of an intermediate dose of cytarabine and a divided dose of GO in adult patients with R / R AML with an FLT3-ITD and/or FLT3-TKD mutation.

Being tested
Gemtuzumab ozogamicine - Cytarabine - Gilteritinib
Keywords
AML, Gemtuzumab Ozogamicin Gilteritinib Combination, relapse, refractory, Leukaemia
Medical subject headings
Leukemia, Myeloid, Acute, Recurrence, Leukemia

What participants receive

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

  • Gemtuzumab ozogamicine - Cytarabine - GilteritinibCombination product

    Also called Mylotarg - Aracytine - Xospata

    See Arms description paragraph

Study groups in NCT05199051
Study groupRoleWhat they receive
Gemtuzumab ozogamicine - Cytarabine - GilteritinibExperimentalFor Gemtuzumab ozogamicine administrated during the induction phase at D1, D4 and D7, 3mg/m2/day (5mg max), IV, 2h of infusion. For Cytarabine during induction and consolidation phase at D1 to D5, 1000 mg/m2, IV, 2h of infusion. For Gilteritinib during induction phase from D10 for 14 consecutive days, per os, two doses level study with dose level 1 (80mg/d) in part 1 or dose level 2 (80 or 120mg/d depending of the result of part 1) in part 2. During consolidation (2 cycles max) from D8 for 14 consecutive days, per os, 120mg/d or reduced dose of 80 mg/kg is planned to be used in patients receiving concomitantly CYP3A4 inhibitors. During the maintenance (24 months max) dose level 2 (120mg/d), per os.
Receives: Combination Product: Gemtuzumab ozogamicine - Cytarabine - Gilteritinib

What is being measured

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

  1. Stage 1 - Safety of the addition of gilteritinib to the AGORA treatment platform

    The primary objective of the first stage is to evaluate the safety of combining gilteritinib with the Gemtuzumab Ozogamicin (GO) -cytarabine AGORA platform in patients with FLT3-ITD and/or FLT3-TKD mutated Relapse/Refractory (R/R) Acute Myeloïd Leukemia (AML), through occurrence of dose-limiting toxicity (DLT).

    Measured: 18 months

  2. Stage 2 - Event-free survival (EFS)

    The primary objective of the second extension stage is to evaluate the efficacy of combining gilteritinib with the Gemtuzumab Ozogamicin (GO)-cytarabine AGORA platform in patients with FLT3-ITD and/or FLT3-TKD mutated Relapse/Refractory (R/R) Acute Myeloïd Leukemia (AML) through event-free survival (EFS).

    Measured: 60 months

Secondary outcomes (14)

  1. Response rates to the study treatment

    Response rate, including Complete Response (CR), Incomplete Hematologis Recovery (CRi) and partial hematologic recovery (CRh)*; the overall response rate (ORR) being defined as CR/CRi/CRh rates

    *: CRi, CR with incomplete hematologic recovery, meaning CR with platelet count <100,000/µL or absolute neutrophil count <1000/µL; CRh, CR with partial hematologic recovery, meaning CR not fulfilling CR or CRi criteria but with platelet count >50,000/µL AND absolute neutrophil count >500/µL.

    Measured: 60 months

  2. Early mortality rates, at day-30

    Mortality rates between day 1 and day 30

    Measured: 30 months

  3. Incidence of subsequent allogeneic Hematopoietic Stem Cell Transplantation (HSCT) overall.

    Incidence of subsequent allogeneic HSCT of all patients of the study.

    Measured: 60 months

  4. Incidence of subsequent allogeneic Hematopoietic Stem Cell Transplantation in responding patients specifically.

    Incidence of subsequent allogeneic HSCT in responding patients specifically.

    Measured: 60 months

  5. Evaluation of Duration of response (DOR) of treatment

    Duration of response (DOR).

    Measured: 60 months

  6. Evaluation of relapse-free survival (RFS)

    Duration of relapse-free survival (RFS) of patient.

    Measured: 60 months

  7. Evaluation of overall survival (OS)

    Duration of overall survival (OS) defined as the time between the date of the inclusion and the date of death or the date of last news for those still alive at the end of the follow-up.

    Measured: 60 months

  8. Subgroup analyses defined by patient

    Subgroups defined by a patient related factor: age (<65 vs ≥65y)

    Measured: 60 months

  9. Subgroup analyses defined by disease

    Subgroups defined by disease related factors: cytogenetics, mutation profiles (including NPM1 and FLT3-ITD), ELN-risk classification 2017.

    Measured: 60 months

  10. Subgroup analyses defined by allograft

    Subgroups defined by treatment related factor by the performance of a subsequent allogeneic HSCT.

    Measured: 60 months

  11. Evaluation of the Safety : incidence of SAE (SAEs)

    Safety through Serious Adverse event (SAEs)

    Measured: 60 months

  12. Evaluation of the Safety : incidence of Adverses Events (AEs)

    Safety through Adverse event (AEs), Serious Adverse event (SAEs), Treatment Emergent Adverse Event (TEAEs) and incidence of sinusoidal obstruction syndrome (SOS))

    Measured: 60 months

  13. Evaluation of the Safety : incidence of Treatment Emergent Adverse Event (TEAEs)

    Safety through Treatment Emergent Adverse Event (TEAEs)

    Measured: 60 months

  14. Evaluation of the Safety : incidence of sinusoidal obstruction syndrome (SOS)

    Safety through incidence of sinusoidal obstruction syndrome (SOS)

    Measured: 60 months

Timeline

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

  1. First posted20 Jan 2022
  2. Study start3 Jun 2023
  3. Primary completion10 Jul 2028 (estimated)
  4. Study completion10 Jul 2028 (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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
19 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05199051
Sponsor's protocol ID
2020/65
Other IDs
2023-504176-25-00, 2021-001813-35, 2023
Acronym
AGORA-1
Status last verified
Dec 2025
First submitted
12 Nov 2021
Last updated
5 Jan 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Gemtuzumab ozogamicine - Cytarabine - Gilteritinib in people with AML and Leukaemia (Acute Myeloid), run by Centre Antoine Lacassagne at 12 sites in France.

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 13 inclusion and 35 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 5 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 12 sites in France. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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?

Stage 1 - Safety of the addition of gilteritinib to the AGORA treatment platform (time frame: 18 months). The study lists 2 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 NCT05199051.

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 5 Jan 2026.

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