Myeloid LeukemiaPhase 1/2NCT06235801
A Phase I/II Study of Gilteritinib and Momelotinib for Patients With Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia
A Phase 1/2 study testing Gilteritinib and Momelotinib in people with Myeloid Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
To learn the recommended dose of momelotinib that can be given in combination with gilteritinib to participants with AML. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myeloid Leukemia
- What is being tested?
- Gilteritinib and Momelotinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteUnited States
- Study dates
- 22 May 2024to 1 Oct 2029
- Phase
- Phase 1/2
- 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
- Diagnosis:
- Adults ≥18 years with relapsed/refractory FLT3-mutated AML. Participants with either FLT3-ITD or FLT3 D835/D836 mutations will be eligible
- Performance status ≤3 (ECOG Scale).
- Adequate liver and renal function as defined by the following criteria:
- Total serum bilirubin ≤ 2 x upper limit of normal (ULN), unless due to Gilbert's syndrome, hemolysis or the underlying leukemia approved by the PI
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≤ 3 x ULN, unless due to the underlying leukemia approved by the PI
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- Creatinine clearance ≥ 30 mL/min
- Willingness to use adequate contraception prior to study entry, for the duration of study participation, and for 4 months after completion of study participation. For women of childbearing potential, adequate methods of contraception include: complete abstinence, hormonal contraception (i.e. birth control pills, injection, implant, transdermal patch, vaginal ring), intrauterine device (IUD), tubal Ligation or hysterectomy, participants/partner post vasectomy, implantable or injectable contraceptives, and condoms plus spermicide
- Ability to understand and the willingness to sign a written informed consent document.
Who cannot join
- Congenital long QT syndrome or QTcF >450 msec. Repeat EKGs after correction of electrolytes or discontinuation of QT prolonging medications are allowed to meet entry criteria. In cases where QTcF >450 msec is considered to be falsely increased due to inaccurate automated reading and not clinically significant (e.g. due to bundle branch block), participants are still eligible if cardiologist reviews and documents that QTcF is ≤ 450 msec when manually measured.
- Active serious infection not controlled by oral or intravenous antibiotics (e.g. persistent fever or lack of improvement despite antimicrobial treatment).
- Active Class III-V cardiac failure as defined by the New York Heart Association Functional Classification.
- Active central nervous system leukemia
- Child-Turcotte-Pugh class C cirrhosis
- Known human immunodeficiency virus (HIV) seropositive.
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- Known hepatitis B surface antigen seropositive or known or suspected active hepatitis C infection Note: Participants who have isolated positive hepatitis B core antibody (ie, in the setting of negative hepatitis B surface antigen and negative hepatitis B surface antibody) must have an undetectable hepatitis B viral load. Participants who have positive hepatitis C antibody may be included if they have an undetectable hepatitis C viral load.
- Participants with a prior or concurrent malignancy whose natural history or treatment is not anticipated to interfere with the safety or efficacy assessment of the investigational regimen may be included only after discussion with the PI
- Treatment with any investigational antileukemic agents or chemotherapy agents in the last 7 days before study entry, unless full recovery from side effects has occurred or participant has rapidly progressive disease judged to be life-threatening by the investigator.
- Prior recent treatment with corticosteroids, hydroxyurea and/or cytarabine (given for cytoreduction) permitted.
- Inability to swallow
- Unable or unwilling to sign informed consent
- Pregnant women will not be eligible; A female participant is eligible to participate if she is not pregnant or breastfeeding, and at least one of the following conditions applies: i. Is a woman of nonchildbearing potential (WONCBP), OR ii. Is a woman of childbearing potential (WOCBP) and using a contraceptive method that is highly effective (with a failure rate of <1% per year), with low user dependency, during the intervention period and for at least 4 month after the last dose of study drug.
- Participants with psychiatric illness/social situations that would limit compliance with study requirements.
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to gilteritinib, momelotinib or other agents used in study.
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 Cancer Center
Houston, Texas, 77030
Nicholas Short, MD, 713-563-4485, nshort@mdanderson.org
Nicholas Short, MD
About this study
To learn the recommended dose of momelotinib that can be given in combination with gilteritinib to participants with AML.
Read the detailed description
Primary Objectives
- Phase I: To establish the minimum safe and biologically-effective dose of momelotinib in combination with gilteritinib in relapsed/refractory FLT3-mutated AML
- Phase II: To determine the modified CRc rate of the regimen, including complete remission (CR), CR with incomplete hematologic recovery (CRi) or morphologic leukemia free state (MLFS)
Secondary Objectives
- To assess other efficacy endpoints (CR rate, measurable residual disease negativity by flow cytometry and FLT3 PCR, relapse-free survival, overall survival)
- To assess proportion of participants proceeding to allogeneic hematopoietic stem cell transplantation
- To determine the safety of the combination regimen
Exploratory Objectives
- To evaluate the impact of baseline genomic alterations on response and survival of the combination regimen
- To determine the impact of baseline FLT3 allelic ratio on response and survival
- To evaluate the pharmacokinetics (e.g. AUC, Cmax, Tmax and half-life) of momelotinib in the combination regimen
- To evaluate changes in circulating cytokine levels in response to treatment
- To evaluate inhibition of FLT3 signaling and inhibition of JAK/STAT signaling pathways
- To evaluate other biomarkers associated with response and resistance to the combination regimen
- Conditions
- Myeloid Leukemia
- Being tested
- Gilteritinib, Momelotinib
- Medical subject headings
- Leukemia, Myeloid
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GilteritinibDrug
Given by PO
MomelotinibDrug
Given by PO
| Study group | Role | What they receive |
|---|---|---|
| Dose Escalation Phase 1/Phase 2 | Experimental | Participants who are enrolled in Part 1, the dose of momelotinib you receive will depend on when you join this study. Participants you are enrolled in Part 2, you will receive momelotinib at the recommended dose that was found in Part 1. All participants will receive the same dose level of gilteritinib. Cycle 1 will be 35 days in length. Momelotinib will be administered once daily by mouth on days 1-35. Gilteritinib will be administered once daily by mouth on days 8-35. Cycles 2 and beyond will be 28 days in length. Momelotinib and gilteritinib will be administered once daily by mouth on days 1-28. Receives: Drug: Gilteritinib; Drug: Momelotinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and adverse events (AEs)
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Measured: Through study completion; an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Feb 2024
- Study start22 May 2024
- Primary completion1 Oct 2027 (estimated)
- Study completion1 Oct 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- GlaxoSmithKline
- Responsible party
- Sponsor
- Investigators
- Nicholas Short, MD, Principal investigator, MD Anderson Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06235801
- Sponsor's protocol ID
- 2023-0438
- Other IDs
- NCI-2024-00669
- Status last verified
- Oct 2026
- First submitted
- 23 Jan 2024
- Last updated
- 3 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Study of Gilteritinib and Momelotinib for Patients With Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT06235801
- Acronym / sponsor ID
- 2023-0438
- Conditions
- Myeloid Leukemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- GlaxoSmithKline
- Study start
- 22 May 2024 (actual)
- Primary completion
- 1 Oct 2027 (estimated)
- Study completion
- 1 Oct 2029 (estimated)
- First posted
- 1 Feb 2024 (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.
To learn the recommended dose of momelotinib that can be given in combination with gilteritinib to participants with AML.
Detailed description
Primary Objectives
- Phase I: To establish the minimum safe and biologically-effective dose of momelotinib in combination with gilteritinib in relapsed/refractory FLT3-mutated AML
- Phase II: To determine the modified CRc rate of the regimen, including complete remission (CR), CR with incomplete hematologic recovery (CRi) or morphologic leukemia free state (MLFS)
Secondary Objectives
- To assess other efficacy endpoints (CR rate, measurable residual disease negativity by flow cytometry and FLT3 PCR, relapse-free survival, overall survival)
- To assess proportion of participants proceeding to allogeneic hematopoietic stem cell transplantation
- To determine the safety of the combination regimen
Exploratory Objectives
- To evaluate the impact of baseline genomic alterations on response and survival of the combination regimen
- To determine the impact of baseline FLT3 allelic ratio on response and survival
- To evaluate the pharmacokinetics (e.g. AUC, Cmax, Tmax and half-life) of momelotinib in the combination regimen
- To evaluate changes in circulating cytokine levels in response to treatment
- To evaluate inhibition of FLT3 signaling and inhibition of JAK/STAT signaling pathways
- To evaluate other biomarkers associated with response and resistance to the combination regimen
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dose Escalation Phase 1/Phase 2 | Experimental | Drug: Gilteritinib, Drug: MomelotinibParticipants who are enrolled in Part 1, the dose of momelotinib you receive will depend on when you join this study. Participants you are enrolled in Part 2, you will receive momelotinib at the recommended dose that was found in Part 1. All participants will receive the same dose level of gilteritinib. Cycle 1 will be 35 days in length. Momelotinib will be administered once daily by mouth on days 1-35. Gilteritinib will be administered once daily by mouth on days 8-35. Cycles 2 and beyond will be 28 days in length. Momelotinib and gilteritinib will be administered once daily by mouth on days 1-28. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Diagnosis:
- Performance status ≤3 (ECOG Scale).
- Adequate liver and renal function as defined by the following criteria:
- Willingness to use adequate contraception prior to study entry, for the duration of study participation, and for 4 months after completion of study participation. For women of childbearing potential, adequate methods of contraception include: complete abstinence, hormonal contraception (i.e. birth control pills, injection, implant, transdermal patch, vaginal ring), intrauterine device (IUD), tubal Ligation or hysterectomy, participants/partner post vasectomy, implantable or injectable contraceptives, and condoms plus spermicide
- Ability to understand and the willingness to sign a written informed consent document.
Key exclusion criteria (15 listed)
- Congenital long QT syndrome or QTcF >450 msec. Repeat EKGs after correction of electrolytes or discontinuation of QT prolonging medications are allowed to meet entry criteria. In cases where QTcF >450 msec is considered to be falsely increased due to inaccurate automated reading and not clinically significant (e.g. due to bundle branch block), participants are still eligible if cardiologist reviews and documents that QTcF is ≤ 450 msec when manually measured.
- Active serious infection not controlled by oral or intravenous antibiotics (e.g. persistent fever or lack of improvement despite antimicrobial treatment).
- Active Class III-V cardiac failure as defined by the New York Heart Association Functional Classification.
- Active central nervous system leukemia
- Child-Turcotte-Pugh class C cirrhosis
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 |
|---|---|---|
| GilteritinibGiven by PO | Drug | Dose Escalation Phase 1/Phase 2 |
| MomelotinibGiven by PO | Drug | Dose Escalation Phase 1/Phase 2 |
Primary outcome
Safety and adverse events (AEs)
Time frame: Through study completion; an average of 1 year
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
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 3 Nov 2026.
- MD Anderson Cancer CenterLink provided by the sponsor.
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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.
Nicholas Short, MD principal investigator
MD Anderson Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Gilteritinib and Momelotinib in people with Myeloid Leukemia, run by M.D. Anderson Cancer Center 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 9 inclusion and 15 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 3 Nov 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 combined 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.
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?
Contact Nicholas Short, MD ((713) 563-4485, nshort@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and adverse events (AEs) (time frame: Through study completion; an average of 1 year). 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 3 Nov 2026.