Acute Myeloid LeukemiaPhase 1NCT05223699
Study of MGTA-117 in Patients With Adult Acute Myeloid Leukemia (AML) and Myelodysplasia-Excess Blasts (MDS-EB)
A Phase 1 study testing MGTA-117 in people with Acute Myeloid Leukemia and Myelodysplasia, run by Magenta Therapeutics, Inc. at 8 sites in United States.
Trial summary
This research study is designed to selectively deplete CD117-positive cells from participants with AML and MDS-EB. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Acute Myeloid Leukemia, Myelodysplasia
- What is being tested?
- MGTA-117
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 8 sitesUnited States
- Study dates
- 14 Feb 2022to 2 Feb 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participant must have a World Health Organization (WHO)-defined diagnosis of R/R AML and meet one of the following criteria:
- The participant has experienced primary AML induction failure or R/R AML
- OR
- The participant has a WHO-defined diagnosis of MDS-EB and has failed/is refractory to HMA
- OR
- Presence of MRD in morphologic CR
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- CD117+ based on IHC or flow cytometry
- Participant must have an identified HSC donor (related donor or unrelated donor), haplo-identical transplant donor, or umbilical blood donor.
- Participant's Eastern Cooperative Oncology Group (ECOG) performance status must be ≤2.
- Participant must have adequate baseline hepatic function. Alanine aminotransferase (ALT), aspartate aminotransferase (AST), and alkaline phosphatase (ALP) ≤2 x upper limit of normal (ULN), and serum bilirubin ≤1.5 x ULN.
- Estimated creatinine clearance ≥60 mL/min
- Adequate cardiac function as demonstrated by cardiac left ventricular ejection fraction ≥40% or perform New York Heart Association (NYHA) classification I and II
Who cannot join
- Acute promyelocytic leukemia (APL).
- Known active central nervous system (CNS) leukemia or chloroma (granulocyte sarcoma).
- Received HSCT within 6 months prior to dosing
- Received chimeric antigen-receptor cell therapies within 6 months prior to dosing
- Has active graft-versus-host disease (GVHD).
- Active hepatitis B (Hep-B) or hepatitis C (Hep-C) infection or history of human immunodeficiency virus (HIV).
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- Participant with a QTc value >470 msec
- Participant has received another investigational drug or device within 14 days or 5 half-lives of dosing, whichever is longer.
- Participant has any clinically significant medical condition, which in the opinion of the Investigator may place the participant at an unacceptable risk.
- Active uncontrolled systemic bacterial, fungal, or viral infection
- Participant has a history of serious allergic reactions, which in the opinion of the Investigator may pose an increased risk of serious infusion reactions.
- Participant has had any systemic antileukemia treatment within 14 days except hydroxyurea, which is permitted until 24 hours prior to MGTA-117 dosing.
- Participant has received prior anti-CD117 antibody treatment.
- Participant has received gemtuzumab ozogamicin (Mylotarg) within the last 3 months prior to dosing.
- Participant has received recent monoclonal antibody as anti-leukemic therapy within the last 30 days or 5 half-lives, whichever is longer.
- Participant has received recent vaccination within the last 14 days prior to dosing.
- Participant has Grade 2 or higher electrolyte abnormality at screening
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
Roswell Park Comprehensive Cancer Center
Buffalo, New York, 14203
Sarah Cannon Research Institute at HealthONE
Denver, Colorado, 80218
City of Hope
Duarte, California, 91010
MD Anderson Cancer Center
Houston, Texas, 77030
University of Minnesota
Minneapolis, Minnesota, 55455
Washington University School of Medicine
St Louis, Missouri, 63110
Moffitt Cancer Center
Tampa, Florida, 33612
The University of Kansas Cancer Center
Westwood, Kansas, 66205
About this study
Official title: A Phase I/II, Dose-Escalation Study of MGTA-117 in Patients With Adult Acute Myeloid Leukemia (AML) and Myelodysplasia-Excess Blasts (MDS-EB)
This research study is designed to selectively deplete CD117-positive cells from participants with AML and MDS-EB.
Read the detailed description
This is a multicenter, open-label, dose-escalation study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and potential anti-leukemia activity and to establish the minimum safe and biologically-effective dose of a single dose of MGTA-117 in relapsed/refractory (R/R) CD117+ AML participants and participants with MDS-EB. The study consists of escalating single-dose cohorts using a standard 3+3 design.
- Conditions
- Acute Myeloid Leukemia, Myelodysplasia
- Being tested
- MGTA-117
- Keywords
- Acute Myeloid Leukemia, Myelodysplasia-Excess Blasts, MGTA-117, Hematopoietic stem cell transplant
- Medical subject headings
- Leukemia, Myeloid, Acute, Anemia, Refractory, with Excess of Blasts
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MGTA-117Biological
MGTA-117 will be administered as an IV infusion
| Study group | Role | What they receive |
|---|---|---|
| Single dose MGTA-117 | Experimental | Dosing of MGTA-117 prepared and administered by IV infusion. Receives: Biological: MGTA-117 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence rate of treatment emergent adverse events (TEAEs) leading to study drug discontinuation
Measured: 21 days
Incidence rate of treatment emergent >= Grade 3 clinical laboratory abnormalities as assessed by CTCAE v5.0
Measured: 21 days
Assess the clinically significant changes from baseline in vital signs, ECGs and laboratory parameters
Measured: 21 days
Pharmacokinetics profile of MGTA-117
Investigate area under the curve (AUC)
Measured: 21 days
Pharmacokinetics profile of MGTA-117
Investigate maximum plasma concentration (Cmax)
Measured: 21 days
Pharmacokinetics profile of MGTA-117
Investigate time of maximum concentration (Tmax)
Measured: 21 days
Pharmacokinetics profile of MGTA-117
Investigate the half-life (t1/2)
Measured: 21 days
Pharmacokinetics profile of MGTA-117
Investigate the plasma concentration
Measured: 21 days
To establish a minimum safe and biologically effective dose
Assess the CD117 receptor occupancy in circulating leukemic blasts
Measured: 7 days
To establish a minimum safe and biologically effective dose
The incidence of qualifying protocol-defined dose-limiting toxicities
Measured: 21 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Feb 2022
- Study start14 Feb 2022
- Primary completion2 Feb 2023
- Study completion2 Feb 2023
Why it stopped: Sponsor Decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies 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
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Magenta Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05223699
- Sponsor's protocol ID
- 117-HEM-101
- Status last verified
- Feb 2023
- First submitted
- 14 Jan 2022
- Last updated
- 9 Feb 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II, Dose-Escalation Study of MGTA-117 in Patients With Adult Acute Myeloid Leukemia (AML) and Myelodysplasia-Excess Blasts (MDS-EB)
- ClinicalTrials.gov ID
- NCT05223699
- Acronym / sponsor ID
- 117-HEM-101
- Conditions
- Acute Myeloid Leukemia, Myelodysplasia
- Keywords
- Acute Myeloid Leukemia, Myelodysplasia-Excess Blasts, MGTA-117, Hematopoietic stem cell transplant
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor Decision
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Magenta Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 14 Feb 2022 (actual)
- Primary completion
- 2 Feb 2023 (actual)
- Study completion
- 2 Feb 2023 (actual)
- First posted
- 4 Feb 2022 (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 research study is designed to selectively deplete CD117-positive cells from participants with AML and MDS-EB.
Detailed description
This is a multicenter, open-label, dose-escalation study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and potential anti-leukemia activity and to establish the minimum safe and biologically-effective dose of a single dose of MGTA-117 in relapsed/refractory (R/R) CD117+ AML participants and participants with MDS-EB. The study consists of escalating single-dose cohorts using a standard 3+3 design.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single dose MGTA-117 | Experimental | Biological: MGTA-117Dosing of MGTA-117 prepared and administered by IV infusion. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Participant must have a World Health Organization (WHO)-defined diagnosis of R/R AML and meet one of the following criteria:
- OR
- OR
- CD117+ based on IHC or flow cytometry
- Participant must have an identified HSC donor (related donor or unrelated donor), haplo-identical transplant donor, or umbilical blood donor.
Key exclusion criteria (17 listed)
- Acute promyelocytic leukemia (APL).
- Known active central nervous system (CNS) leukemia or chloroma (granulocyte sarcoma).
- Received HSCT within 6 months prior to dosing
- Received chimeric antigen-receptor cell therapies within 6 months prior to dosing
- Has active graft-versus-host disease (GVHD).
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 |
|---|---|---|
| MGTA-117MGTA-117 will be administered as an IV infusion | Biological | Single dose MGTA-117 |
Primary outcome
Incidence rate of treatment emergent adverse events (TEAEs) leading to study drug discontinuation
Time frame: 21 days
Incidence rate of treatment emergent >= Grade 3 clinical laboratory abnormalities as assessed by CTCAE v5.0
Time frame: 21 days
Assess the clinically significant changes from baseline in vital signs, ECGs and laboratory parameters
Time frame: 21 days
Pharmacokinetics profile of MGTA-117
Time frame: 21 days
Investigate area under the curve (AUC)
Pharmacokinetics profile of MGTA-117
Time frame: 21 days
Investigate maximum plasma concentration (Cmax)
Pharmacokinetics profile of MGTA-117
Time frame: 21 days
Investigate time of maximum concentration (Tmax)
Pharmacokinetics profile of MGTA-117
Time frame: 21 days
Investigate the half-life (t1/2)
Pharmacokinetics profile of MGTA-117
Time frame: 21 days
Investigate the plasma concentration
To establish a minimum safe and biologically effective dose
Time frame: 7 days
Assess the CD117 receptor occupancy in circulating leukemic blasts
To establish a minimum safe and biologically effective dose
Time frame: 21 days
The incidence of qualifying protocol-defined dose-limiting toxicities
Results and safety
The study was stopped early (reason given: Sponsor Decision). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Feb 2023.
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing MGTA-117 in people with Acute Myeloid Leukemia and Myelodysplasia, run by Magenta Therapeutics, Inc. at 8 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?
The sponsor lists 12 inclusion and 17 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: stopped early, as of the sponsor's last update on 9 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 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?
The study was stopped early (reason given: Sponsor Decision). Results are not available in the source record.
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?
Incidence rate of treatment emergent adverse events (TEAEs) leading to study drug discontinuation (time frame: 21 days). The study lists 10 primary outcomes in all. 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 9 Feb 2023.