Relapsed/Refractory Acute Myeloid Leukemia (R/R AML)Phase 1NCT06786533
Clinical Study of Anti-FLT3 CAR-T Cells for the Treatment of Relapsed/Refractory AML
A Phase 1 study testing Anti-FLT3 CAR-T cells in people with Relapsed/Refractory Acute Myeloid Leukemia (R/R AML), run by Hemogenyx Pharmaceuticals LLC at 1 site in United States.
Trial summary
This is a phase 1 dose escalation study to determine the safety of anti-FLT3 CAR-T in subjects with R/R AML. The primary objective is to assess safety. Up to 18 evaluable adult and 18 evaluable pediatric subjects will be enrolled. Evaluable subjects are defined as those who have received an infusion of HG-CT-1. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Relapsed/Refractory Acute Myeloid Leukemia (R/R AML)
- What is being tested?
- Anti-FLT3 CAR-T cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- 23 Jan 2025to Jan 2027
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 18 years of age or older at enrollment. Patients ≥12 and <18 12 to 17 years of age weighing ≥ 35 kg at enrollment may be included once safety evaluation at the corresponding adult dose escalation protocol have been completed.
- Subjects with AML unlikely to be cured with currently available therapies. Specifically, the following groups are eligible:
- Refractory AML: i.e., newly diagnosed AML that after two cycles of intensive chemotherapy has not achieved a complete remission or morphologic leukemia free state by ELN criteria.1 Intensive chemotherapy must have included either the combination of cytarabine and an anthracycline (7+3 or similar) or combination of venetoclax with a hypomethylating agent.
- Patients with FLT3 ITD must also have failed treatment with a FLT3 inhibitor and patients with IDH1 or IDH2 mutations must have failed treatment containing ivosidenib or enasidenib respectively (i.e., progression on treatment, or failure to achieve CR after six months of treatment,) OR:
- AML relapsed following allogeneic stem cell transplantation (including MDS evolved to AML post-allogeneic stem cell transplantation). Note: morphologic relapse is not required; persistent/recurrent disease-associated molecular, phenotypic, or cytogenetic abnormalities (measurable residual disease, MRD) at any time after allogeneic HSCT is eligible. OR:
- AML that has relapsed within 12 months after initial induction and consolidation therapy OR:
Show all 20Show fewer
- AML that has relapsed more than 12 months after initial induction but that has failed to achieve CR or morphologic leukemia free state after one reinduction OR:
- AML after second or subsequent relapse.
- FLT3 expression must be detectable in AML blast by flow cytometric analysis.
- Subjects must have a suitable stem cell transplant donor. Donor may be matched or mismatched and must be found to be suitable according to the institution's standard criteria. That donor shall be "cleared" for donation by institutional standards prior to administration of HG-CT-1. Adult donors can be either related or unrelated, HLA-matched or partially matched. Matched or partially matched umbilical cord blood donors are also eligible.
- Subjects with relapsed disease after prior allogeneic transplant must be off systemic immunosuppression for at least 1 month at the time of enrollment without GvHD that requires systemic immunosuppression.
- Satisfactory organ functions:
- Creatinine ≤ 1.6 mg/dl and Creatinine clearance (CrCl) as calculated by the Cockcroft-Gault formula ≥ 60 mL/min.
- ALT/AST must be ≤ 3 x upper limit of normal unless related to disease.
- Direct bilirubin < 2.0mg/dl unless subject has Gilbert's syndrome (in which case it should be ≤3.0 mg/dL).
- Left ventricular ejection fraction ≥ 45% as confirmed by echocardiogram or MUGA.
- DLCO >45% predicted and O2 Saturation > 90% on room air.
- Patients ≥18 must have an ECOG Performance status 0-1. Patients <18 must have a Lansky/Karnofsky score of ≥50.
- Written informed consent is given in patients ≥ 18. In patients <18 or not developmentally appropriate for consent, written consent will be provided to the parent or legal guardian. Patients ≥12 and <18 years of age will be additionally provided with assent documentation.
- Subjects of reproductive potential must agree to use acceptable birth control methods (as described in protocol Section 4.7).
Who cannot join
- Pregnant or lactating (nursing) women.
- Active second malignancy will not be eligible with the following exceptions:
- Carcinoma in situ of the cervix (which may be considered for enrollment),
- Indolent, non-metastatic prostate cancer
- Non melanoma skin cancer
- Other indolent and controlled malignancies not requiring urgent treatment.
Show all 19Show fewer
- Subjects with a history of a prior allogeneic stem cell transplantation are excluded if:
- Subjects are less than 100 days post-transplant OR
- Subjects have evidence of ongoing active GvHD and are taking immunosuppressive agents (>0.5mg/kg/methylprednisolone equivalents or other immunosuppression for GvHD treatment) OR
- Subjects have received DLI within 30 days prior to enrollment.
- Active hepatitis B (HBV) or active hepatitis C (HCV) or any HIV infection. Note: prior HCV that has been appropriately treated or evidence of past HBV infection do not constitute exclusions.
- Concurrent use of systemic steroids at a prednisone dose of greater than 10 mg, hydrocortisone greater than 10-12.5 mg/m2/day, or equivalent. Recent, or current use of inhaled steroids is not exclusionary.
- Concurrent use of immunosuppressant medications such as calcineurin inhibitors, methotrexate, or alemtuzumab.
- Uncontrolled, symptomatic, intercurrent illness including but not limited to infection, congestive heart failure, unstable angina pectoris, cardiac arrhythmia, psychiatric illness, or social situations that would limit compliance with study requirements or in the opinion of the site Pl would pose an unacceptable risk to the subject.
- Active or uncontrolled viral, bacterial, or fungal infection. May be receiving ongoing therapy for controlled infection.
- Subjects with signs or symptoms indicative of active CNS involvement. A CNS evaluation shall be performed as clinically appropriate to rule out CNS involvement. Subjects with adequately treated CNS leukemia are eligible. History of CNS involvement is not exclusionary if CNS has been cleared with a documented negative lumbar puncture and negative imaging (imaging required only if previously showing evidence of CNS leukemia not otherwise documented by spinal fluid assessment).
- Known history of allergy or hypersensitivity to study product excipients (human serum albumin, DMSO, and Dextran 40).
- Hyperleukocytosis (>50,000 blasts/µL) or rapidly progressive disease that in the estimation of the investigator and sponsor would compromise ability to complete study therapy.
- Patients with Acute Promyelocytic Leukemia are not eligible.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
MD Anderson
Houston, Texas, 77030
Nicholas Short, 1-877-632-6789, nshort@mdanderson.org
About this study
Official title: Phase 1 Study of Anti-FLT3 Chimeric Antigen Receptor-redirected T Cells in Subjects With Relapsed/Refractory Acute Myeloid Leukemia (AML)
This is a phase 1 dose escalation study to determine the safety of anti-FLT3 CAR-T in subjects with R/R AML. The primary objective is to assess safety. Up to 18 evaluable adult and 18 evaluable pediatric subjects will be enrolled. Evaluable subjects are defined as those who have received an infusion of HG-CT-1.
Primary clinical objectives:
Read more
- Determine the safety of HG-CT-1 based on the proportion of subjects infused with HG-CT-1 who experience a dose limiting toxicity (DLT).
Secondary clinical objectives:
- Estimate the efficacy of HG-CT-1 according to standard clinical response criteria for AML. ii. Estimate overall survival of evaluable subjects. iii. Estimate progression-free survival of evaluable subjects. iv. Estimate duration of response in evaluable subjects who achieve a response.
Secondary scientific objectives:
- Describe the persistence and trafficking of HG-CT-1. ii. Describe HG-CT-1 bioactivity and its predictors.
- Being tested
- Anti-FLT3 CAR-T cells
- Medical subject headings
- Recurrence, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Anti-FLT3 CAR-T cellsDrug
Also called HG-CT-1
Anti-FLT3 CAR-T cells is administered by intravenous infusion following standard lymphodepleting chemotherapy consisting of fludarabine and cyclophosphamide.
| Study group | Role | What they receive |
|---|---|---|
| Dose level 1: 7x10^7 Transduced CAR+ HG-CT-1 | Experimental | Not described Receives: Drug: Anti-FLT3 CAR-T cells |
| Dose level 2: 1.4x10^8 Transduced CAR+ HG-CT-1 | Experimental | Not described |
| Dose level 3: 3.5x10^8 Transduced CAR+ HG-CT-1 | Experimental | Not described Receives: Drug: Anti-FLT3 CAR-T cells |
| Dose level -1: 3.5 x 10^7 Transduced CAR+ HG-CT-1 | Experimental | Not described Receives: Drug: Anti-FLT3 CAR-T cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine the safety of HG-CT-1 cells based on the proportion of subjects infused with HG-CT-1 who experience a DLT.
Occurrence of dose-limiting toxicities related to HG-CT-1. First-in-human study with unknown safety of infusion of HG-CT-1.
Measured: From the time of HG-CT-1 infusion until Day 28
Secondary outcomes (4)
Estimate the efficacy of HG-CT-1 cells as defined by clinical response according to established criteria for AML.
Proportion of subjects with response, according to ELN 2022 criteria. {Dohner et al., 2022 #29518}. To demonstrate the anti-AML activity of HG-CT-1.
Measured: Day 14, Day 28, Month 3, Month 6 and Month 12
Estimate the Overall Survival (OS).
Proportion of subjects alive at 6 months and one year. To demonstrate effect on disease biology and prognosis (compared to historical expectations).
Measured: At 6 months and 1 year
Estimate the progression-free survival (PFS).
Proportion of subjects alive without progression at 6 months and one year. To demonstrate durability of anti-AML effect.
Measured: At 6 months and 1 year
Estimate the Duration of response (DOR).
Proportion of subjects with response who remain alive and without progression at 6 months and at one year. To demonstrate durability of anti-AML effect.
Measured: At 6 months and 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jan 2025
- Study start23 Jan 2025
- Primary completionJan 2027 (estimated)
- Study completionJan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Relapsed/Refractory Acute Myeloid Leukemia (R/R AML)Studies 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Dose and route of administration HG-CT-1 cells will be administered by intravenous infusion following standard lymphodepleting chemotherapy consisting of fludarabine and cyclophosphamide. Statistical Design The trial will use a modified toxicity probability interval (mTPI-2) design while matching the cohort size with the traditional 3 + 3 design. Dose Levels Dose level 1: 7x10\^7 Transduced CAR+ HG-CT-1 Dose level 2: 1.4x10\^8 Transduced CAR+ HG-CT-1 Dose level 3: 3.5x10\^8 Transduced CAR+ HG-CT-1 If Dose level 1 is found not to be safe, dose will be de-escalated to 3.5 x 10\^7 Transduced CAR+ HG-CT-1. (Dose level -1) Pediatric Dosing: Enrollment on any pediatric (≥12 and \<18 years of age) cohort shall begin after the corresponding adult cohort has been safely completed.
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hemogenyx Pharmaceuticals LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- M.D. Anderson Cancer Center
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06786533
- Sponsor's protocol ID
- HEM-001-AML-1101
- Other IDs
- IND 029573
- Status last verified
- Sep 2026
- First submitted
- 10 Jan 2025
- Last updated
- 17 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1 Study of Anti-FLT3 Chimeric Antigen Receptor-redirected T Cells in Subjects With Relapsed/Refractory Acute Myeloid Leukemia (AML)
- ClinicalTrials.gov ID
- NCT06786533
- Acronym / sponsor ID
- HEM-001-AML-1101
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- Hemogenyx Pharmaceuticals LLCIndustry (pharma, biotech, devices)
- Collaborators
- M.D. Anderson Cancer Center
- Study start
- 23 Jan 2025 (actual)
- Primary completion
- Jan 2027 (estimated)
- Study completion
- Jan 2027 (estimated)
- First posted
- 22 Jan 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase 1 dose escalation study to determine the safety of anti-FLT3 CAR-T in subjects with R/R AML. The primary objective is to assess safety. Up to 18 evaluable adult and 18 evaluable pediatric subjects will be enrolled. Evaluable subjects are defined as those who have received an infusion of HG-CT-1.
Primary clinical objectives:
- Determine the safety of HG-CT-1 based on the proportion of subjects infused with HG-CT-1 who experience a dose limiting toxicity (DLT).
Secondary clinical objectives:
- Estimate the efficacy of HG-CT-1 according to standard clinical response criteria for AML. ii. Estimate overall survival of evaluable subjects. iii. Estimate progression-free survival of evaluable subjects. iv. Estimate duration of response in evaluable subjects who achieve a response.
Secondary scientific objectives:
- Describe the persistence and trafficking of HG-CT-1. ii. Describe HG-CT-1 bioactivity and its predictors.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
- Model description
- Dose and route of administration HG-CT-1 cells will be administered by intravenous infusion following standard lymphodepleting chemotherapy consisting of fludarabine and cyclophosphamide. Statistical Design The trial will use a modified toxicity probability interval (mTPI-2) design while matching the cohort size with the traditional 3 + 3 design. Dose Levels Dose level 1: 7x10^7 Transduced CAR+ HG-CT-1 Dose level 2: 1.4x10^8 Transduced CAR+ HG-CT-1 Dose level 3: 3.5x10^8 Transduced CAR+ HG-CT-1 If Dose level 1 is found not to be safe, dose will be de-escalated to 3.5 x 10^7 Transduced CAR+ HG-CT-1. (Dose level -1) Pediatric Dosing: Enrollment on any pediatric (≥12 and <18 years of age) cohort shall begin after the corresponding adult cohort has been safely completed.
| Arm | Type | Receives |
|---|---|---|
| Dose level 1: 7x10^7 Transduced CAR+ HG-CT-1 | Experimental | Drug: Anti-FLT3 CAR-T cells |
| Dose level 2: 1.4x10^8 Transduced CAR+ HG-CT-1 | Experimental | – |
| Dose level 3: 3.5x10^8 Transduced CAR+ HG-CT-1 | Experimental | Drug: Anti-FLT3 CAR-T cells |
| Dose level -1: 3.5 x 10^7 Transduced CAR+ HG-CT-1 | Experimental | Drug: Anti-FLT3 CAR-T cells |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (20 listed)
- 18 years of age or older at enrollment. Patients ≥12 and <18 12 to 17 years of age weighing ≥ 35 kg at enrollment may be included once safety evaluation at the corresponding adult dose escalation protocol have been completed.
- Subjects with AML unlikely to be cured with currently available therapies. Specifically, the following groups are eligible:
- Patients with FLT3 ITD must also have failed treatment with a FLT3 inhibitor and patients with IDH1 or IDH2 mutations must have failed treatment containing ivosidenib or enasidenib respectively (i.e., progression on treatment, or failure to achieve CR after six months of treatment,) OR:
- FLT3 expression must be detectable in AML blast by flow cytometric analysis.
- Subjects must have a suitable stem cell transplant donor. Donor may be matched or mismatched and must be found to be suitable according to the institution's standard criteria. That donor shall be "cleared" for donation by institutional standards prior to administration of HG-CT-1. Adult donors can be either related or unrelated, HLA-matched or partially matched. Matched or partially matched umbilical cord blood donors are also eligible.
Key exclusion criteria (19 listed)
- Pregnant or lactating (nursing) women.
- Active second malignancy will not be eligible with the following exceptions:
- Subjects with a history of a prior allogeneic stem cell transplantation are excluded if:
- Active hepatitis B (HBV) or active hepatitis C (HCV) or any HIV infection. Note: prior HCV that has been appropriately treated or evidence of past HBV infection do not constitute exclusions.
- Concurrent use of systemic steroids at a prednisone dose of greater than 10 mg, hydrocortisone greater than 10-12.5 mg/m2/day, or equivalent. Recent, or current use of inhaled steroids is not exclusionary.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Anti-FLT3 CAR-T cellsAlso: HG-CT-1Anti-FLT3 CAR-T cells is administered by intravenous infusion following standard lymphodepleting chemotherapy consisting of fludarabine and cyclophosphamide. | Drug | Dose level -1: 3.5 x 10^7 Transduced CAR+ HG-CT-1, Dose level 1: 7x10^7 Transduced CAR+ HG-CT-1, Dose level 3: 3.5x10^8 Transduced CAR+ HG-CT-1 |
Primary outcome
Determine the safety of HG-CT-1 cells based on the proportion of subjects infused with HG-CT-1 who experience a DLT.
Time frame: From the time of HG-CT-1 infusion until Day 28
Occurrence of dose-limiting toxicities related to HG-CT-1. First-in-human study with unknown safety of infusion of HG-CT-1.
4 secondary outcomes
Estimate the efficacy of HG-CT-1 cells as defined by clinical response according to established criteria for AML.
Time frame: Day 14, Day 28, Month 3, Month 6 and Month 12
Estimate the Overall Survival (OS).
Time frame: At 6 months and 1 year
Estimate the progression-free survival (PFS).
Time frame: At 6 months and 1 year
Estimate the Duration of response (DOR).
Time frame: At 6 months and 1 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2026.
- Individual participant dataSharing planned: Undecided
Related trials for Relapsed/Refractory Acute Myeloid Leukemia (R/R AML)
Not yet recruitingPhase 1
A Study to Assess the Pharmacokinetics and Safety of Revumenib in Participants With Advanced Cancer and Hepatic or Renal Impairment
Relapsed/Refractory (R/R) Hematologic Malignancies, Acute Leukemia
RecruitingPhase 1
First-in-human (FIH) Trial of GEN3018 in Relapsed or Refractory (R/R) Acute Myeloid Leukemia (AML) or Higher-risk Myelodysplastic Syndrome (HR-MDS)
R/R AML, R/R HR-MDS
Not yet recruitingPhase 1/2
A Study of Sonrotoclax (BGB-11417) in Children With Relapsed or Refractory Acute Myeloid Leukemia and B-cell Acute Lymphoblastic Leukemia
Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
Active, not recruitingPhase 1
BXCL701 Phase 1 R/R Acute Myeloid Leukemia or Myelodysplastic Syndrome
Acute Myeloid Leukemia, Myelodysplastic Syndrome With Excess Blasts-2
RecruitingPhase 1
Phase 1 Study of AUTX-703 in Relapsed/Refractory AML and MDS
Relapsed Acute Myeloid Leukemia (AML), Refractory Acute Myeloid Leukemia (AML)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Anti-FLT3 CAR-T cells in people with Relapsed/Refractory Acute Myeloid Leukemia (R/R AML), run by Hemogenyx Pharmaceuticals LLC at 1 site 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?
The sponsor lists 20 inclusion and 19 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: looking for participants now, as of the sponsor's last update on 17 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Determine the safety of HG-CT-1 cells based on the proportion of subjects infused with HG-CT-1 who experience a DLT. (time frame: From the time of HG-CT-1 infusion until Day 28). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 17 Sep 2026.