AML, Adult RecurrentPhase 1NCT05829226
A Phase 1 Study With LYT-200 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML), or With Relapsed/Refractory, High-risk Myelodysplastic Syndrome (MDS)
A Phase 1 study testing LYT-200, Venetoclax and Azacitidine in people with AML, Adult Recurrent and MDS, run by PureTech at 9 sites in United States.
Trial summary
A Phase 1 Open-label, Multi-center Study of the Safety, Pharmacokinetics (PK), and Anti-tumor Activity of LYT- 200 in Patients with Relapsed/Refractory Acute Myeloid Leukemia (AML), or with Relapsed/refractory, High-risk Myelodysplastic Syndrome (MDS) Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- AML, Adult Recurrent, MDS
- What is being tested?
- LYT-200, Venetoclax and Azacitidine
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 101 participants
- Where
- 9 sitesUnited States
- Study dates
- 12 Dec 2022to 31 Dec 2025
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients ≥ 18 years of age at the time of obtaining informed consent.
- Patients with morphologically documented primary or secondary AML by the World Health Organization(WHO) criteria, whose disease is relapsed/refractory to at least one line of prior therapy, with or without an allogeneic stem cell transplant and for whom no standard therapy that may provide clinical benefit is available or for patients who decline available standard of care.
- Patients with a documented diagnosis of high-risk myelodysplastic syndrome (MDS), whose disease is relapsed/refractory, post at least one line of treatment based on the revised International Prognostic Scoring System (IPSS-R) and for whom no standard therapy that may provide clinical benefit is available
- Patients are able and willing to comply with study procedures as per protocol, including bone marrowbiopsies.
- Patient has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.
- Patient must meet the following criteria as indicated on the clinical laboratory tests:
- oWhite blood cell (WBC) count at the time of the first dose of < 25,000/uL. oAspartate aminotransferase or alanine aminotransferase ≤ 3 × upper limit of normal (ULN; ≤ 5.0× ULN if considered to be due to leukemic involvement). oTotal bilirubin ≤ 2 × ULN (≤ 3 × ULN if considered to be due to leukemic involvement orGilbert's syndrome). oCreatinine clearance of ≥ 60 mL/min.
Who cannot join
- Patient diagnosed with acute promyelocytic leukemia (APL).
- Patient has active malignant tumors other than AML/MDS
- Patient has had HSCT and meets any of the following: has undergone HSCT within the 6- month period prior to the first study dose; has ≥ Grade 2 persistent non-hematological toxicity related to the transplant donor lymphocytes infusion.
- Patient has active graft versus host disease (GVHD) and patients receiving immunosuppressive treatment for GVHD.
- Patient with symptomatic central nervous system (CNS) involvement of leukemia or other CNS diseases related to underlying and secondary effects of malignancy
- Patient has had major surgery within 4 weeks prior to the first study dose.
- Patient has congestive heart failure New York Heart Association (NYHA) class 3 or 4, or patient with a history of congestive heart failure NYHA class 3 or 4 in the past, unless a screening echocardiogram or multigated acquisition (MUGA) scan performed within 3 months prior to study entry results in a left ventricular ejection fraction (LVEF) that is ≥ 45%.
- Patient has any condition which, in the Investigator's opinion, makes the patient unsuitable for study participation.
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
United States9 sites
Mass. General Hospital-Harvard
Boston, Massachusetts, 02114
Karmanos Cancer Institute
Detroit, Michigan, 48201
Cedars-Sinai Medical Center
Los Angeles, California, 90048
Norton Healthcare-Norton Cancer Institute
Louisville, Kentucky, 40207
Baptist Health South Florida-Miami Cancer Institute
Miami, Florida, 02114
Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey, 08901
University of California Irvine Medical Center
Orange, California, 92868
Rhode Island Hospital
Providence, Rhode Island, 02903
Virginia Commonwealth University Medical Center
Richmond, Virginia, 23219
About this study
Official title: A Phase 1 Open-label, Multi-center Study of the Safety, Pharmacokinetics (PK), and Anti-tumor Activity of LYT- 200 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML), or With Relapsed/Refractory, High-risk Myelodysplastic Syndrome (MDS)
A Phase 1 Open-label, Multi-center Study of the Safety, Pharmacokinetics (PK), and Anti-tumor Activity of LYT- 200 in Patients with Relapsed/Refractory Acute Myeloid Leukemia (AML), or with Relapsed/refractory, High-risk Myelodysplastic Syndrome (MDS)
Read the detailed description
This is an open-label, non-randomized, multi-center, Phase 1, dose escalation study in patients with AML relapsed/refractory to at least one line of prior therapy, with or without an allogeneic stem cell transplant, or in patients with a documented diagnosis of relapsed/refractory, high-risk myelodysplastic syndrome (MDS) post at least one line of treatment and for whom no standard therapy that may provide clinical benefit is available. The 4+2 algorithm-based dose-escalation design will be used to help identify the recommended Phase 2 dose (RP2D). Single agent LYT-200 and in combination with venetoclax and/or hypomethylating agents (HMA) safety and tolerability evaluation is the primary study endpoint, Pharmacokinetics (PK), and Anti-tumor Activity of LYT- 200 single agent and in combination with venetoclax and/or HMAs are key secondary study endpoints.
- Conditions
- AML, Adult Recurrent, MDS
- Being tested
- LYT-200, Venetoclax, Azacitidine, Decitabine
- Keywords
- AML Recurrent, AML Relapsed, AML Refractory, Hematological Cancer, Gal-9, Immuno-oncology, MDS, MDS High Risk
- Medical subject headings
- Leukemia, Myeloid, Acute, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LYT-200Drug
monoclonal antibody (mAb), targeting galectin-9 protein
VenetoclaxDrug
Bcl-2 inhibitor
AzacitidineDrug
Hypomethylating agent
DecitabineDrug
Hypomethylating agent
| Study group | Role | What they receive |
|---|---|---|
| Single agent dose escalation | Experimental | LYT-200 in relapsed/refractory AML or relapsed/refractory high-risk MDS, administered via IV infusion over 60 minutes every week. Receives: Drug: LYT-200 |
| Combination agent dose escalation | Experimental | LYT-200 in relapsed/refractory AML or relapsed/refractory high-risk MDS, administered via IV infusion over 60 minutes every week, in combination with oral venetoclax Day 1, 100 mg, Day 2, 200mg, Day 3-28, 400 mg and/or azacitidine, 75 mg/m2 subcutaneously given for 7 days per cycle or decitabine 20 mg/m2 IV for 5 days per cycle. Receives: Drug: LYT-200; Drug: Venetoclax; Drug: Azacitidine; Drug: Decitabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events [Safety and RP2D determination]
Evaluation of safety parameters including treatment emergent adverse events as detected by hematology, chemistry, coagulation safety labs, physical exams, vital signs, ECG, ECHO/MUGA, ECOG status
Measured: approximately 1 year
Incidence of Dose Limiting Toxicities [Tolerability and RP2D determination]
Evaluation of tolerability parameters including dose limiting toxicities as detected by hematology, chemistry, coagulation safety labs, physical exams, vital signs, ECG, ECHO/MUGA, ECOG status
Measured: approximately 1 year
Secondary outcomes (4)
Rate of disease responses, time-to-event endpoints, hematological improvements
Evaluate preliminary efficacy of LYT- 200 as a single agent in AML and MDS
Measured: approximately 1 year
Pharmacokinetic (PK) profile of LYT-200_Area Under the Curve (AUC)
Characterize the PK profile of LYT-200
Measured: approximately 1 year
Pharmacokinetic (PK) profile of LYT-200_Concentration Max (CMax)
Characterize the PK profile of LYT-200
Measured: approximately 1 year
Pharmacokinetic (PK) profile of LYT-200_Time to Reach CMax (TMax)
Characterize the PK profile of LYT-200
Measured: approximately 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Apr 2023
- Study start12 Dec 2022
- Primary completion31 Dec 2025
- Study completion31 Dec 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AML, Adult RecurrentStudies 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
- A Phase 1 open-label, multi-center study. The 4+2 algorithm-based dose-escalation design in single agent LYT-200 and in combination with venetoclax and/or HMAs will be used to help identify the recommended Phase 2 dose (RP2D). Up to 6 patients per single treatment Cohorts 1-5 will receive infusions of LYT-200 every week (QW). Up to 6 patients per combination Cohorts 1-4 will receive infusions of LYT-200 QW and oral venetoclax every day and/or HMAs for 7 days (azacitidine) or 5 days (decitabine) per cycle.
- Masking
- None (open label)
- Enrolment
- 101 participants (actual)
Sponsor and investigators
- Lead sponsor
- PureTech
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Aleksandra Filipovic, MD, PhD., Study director, PureTech Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05829226
- Sponsor's protocol ID
- LYT-200-2022-02
- Status last verified
- May 2025
- First submitted
- 30 Mar 2023
- Last updated
- 6 May 2026
- 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 6 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Open-label, Multi-center Study of the Safety, Pharmacokinetics (PK), and Anti-tumor Activity of LYT- 200 in Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML), or With Relapsed/Refractory, High-risk Myelodysplastic Syndrome (MDS)
- ClinicalTrials.gov ID
- NCT05829226
- Acronym / sponsor ID
- LYT-200-2022-02
- Conditions
- AML, Adult Recurrent, MDS
- Keywords
- AML Recurrent, AML Relapsed, AML Refractory, Hematological Cancer, Gal-9, Immuno-oncology, MDS, MDS High Risk
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 101 participants (actual)
- Lead sponsor
- PureTechIndustry (pharma, biotech, devices)
- Study start
- 12 Dec 2022 (actual)
- Primary completion
- 31 Dec 2025 (actual)
- Study completion
- 31 Dec 2025 (actual)
- First posted
- 25 Apr 2023 (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.
A Phase 1 Open-label, Multi-center Study of the Safety, Pharmacokinetics (PK), and Anti-tumor Activity of LYT- 200 in Patients with Relapsed/Refractory Acute Myeloid Leukemia (AML), or with Relapsed/refractory, High-risk Myelodysplastic Syndrome (MDS)
Detailed description
This is an open-label, non-randomized, multi-center, Phase 1, dose escalation study in patients with AML relapsed/refractory to at least one line of prior therapy, with or without an allogeneic stem cell transplant, or in patients with a documented diagnosis of relapsed/refractory, high-risk myelodysplastic syndrome (MDS) post at least one line of treatment and for whom no standard therapy that may provide clinical benefit is available. The 4+2 algorithm-based dose-escalation design will be used to help identify the recommended Phase 2 dose (RP2D). Single agent LYT-200 and in combination with venetoclax and/or hypomethylating agents (HMA) safety and tolerability evaluation is the primary study endpoint, Pharmacokinetics (PK), and Anti-tumor Activity of LYT- 200 single agent and in combination with venetoclax and/or HMAs are key secondary study endpoints.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- A Phase 1 open-label, multi-center study. The 4+2 algorithm-based dose-escalation design in single agent LYT-200 and in combination with venetoclax and/or HMAs will be used to help identify the recommended Phase 2 dose (RP2D). Up to 6 patients per single treatment Cohorts 1-5 will receive infusions of LYT-200 every week (QW). Up to 6 patients per combination Cohorts 1-4 will receive infusions of LYT-200 QW and oral venetoclax every day and/or HMAs for 7 days (azacitidine) or 5 days (decitabine) per cycle.
| Arm | Type | Receives |
|---|---|---|
| Single agent dose escalation | Experimental | Drug: LYT-200LYT-200 in relapsed/refractory AML or relapsed/refractory high-risk MDS, administered via IV infusion over 60 minutes every week. |
| Combination agent dose escalation | Experimental | Drug: LYT-200, Drug: Venetoclax, Drug: Azacitidine, Drug: DecitabineLYT-200 in relapsed/refractory AML or relapsed/refractory high-risk MDS, administered via IV infusion over 60 minutes every week, in combination with oral venetoclax Day 1, 100 mg, Day 2, 200mg, Day 3-28, 400 mg and/or azacitidine, 75 mg/m2 subcutaneously given for 7 days per cycle or decitabine 20 mg/m2 IV for 5 days per cycle. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Patients ≥ 18 years of age at the time of obtaining informed consent.
- Patients with morphologically documented primary or secondary AML by the World Health Organization(WHO) criteria, whose disease is relapsed/refractory to at least one line of prior therapy, with or without an allogeneic stem cell transplant and for whom no standard therapy that may provide clinical benefit is available or for patients who decline available standard of care.
- Patients with a documented diagnosis of high-risk myelodysplastic syndrome (MDS), whose disease is relapsed/refractory, post at least one line of treatment based on the revised International Prognostic Scoring System (IPSS-R) and for whom no standard therapy that may provide clinical benefit is available
- Patients are able and willing to comply with study procedures as per protocol, including bone marrowbiopsies.
- Patient has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.
Key exclusion criteria (8 listed)
- Patient diagnosed with acute promyelocytic leukemia (APL).
- Patient has active malignant tumors other than AML/MDS
- Patient has had HSCT and meets any of the following: has undergone HSCT within the 6- month period prior to the first study dose; has ≥ Grade 2 persistent non-hematological toxicity related to the transplant donor lymphocytes infusion.
- Patient has active graft versus host disease (GVHD) and patients receiving immunosuppressive treatment for GVHD.
- Patient with symptomatic central nervous system (CNS) involvement of leukemia or other CNS diseases related to underlying and secondary effects of malignancy
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 |
|---|---|---|
| LYT-200monoclonal antibody (mAb), targeting galectin-9 protein | Drug | Combination agent dose escalation, Single agent dose escalation |
| VenetoclaxBcl-2 inhibitor | Drug | Combination agent dose escalation |
| AzacitidineHypomethylating agent | Drug | Combination agent dose escalation |
| DecitabineHypomethylating agent | Drug | Combination agent dose escalation |
Primary outcome
Incidence of Treatment-Emergent Adverse Events [Safety and RP2D determination]
Time frame: approximately 1 year
Evaluation of safety parameters including treatment emergent adverse events as detected by hematology, chemistry, coagulation safety labs, physical exams, vital signs, ECG, ECHO/MUGA, ECOG status
Incidence of Dose Limiting Toxicities [Tolerability and RP2D determination]
Time frame: approximately 1 year
Evaluation of tolerability parameters including dose limiting toxicities as detected by hematology, chemistry, coagulation safety labs, physical exams, vital signs, ECG, ECHO/MUGA, ECOG status
4 secondary outcomes
Rate of disease responses, time-to-event endpoints, hematological improvements
Time frame: approximately 1 year
Pharmacokinetic (PK) profile of LYT-200_Area Under the Curve (AUC)
Time frame: approximately 1 year
Pharmacokinetic (PK) profile of LYT-200_Concentration Max (CMax)
Time frame: approximately 1 year
Pharmacokinetic (PK) profile of LYT-200_Time to Reach CMax (TMax)
Time frame: approximately 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 May 2026.
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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.
Aleksandra Filipovic, MD, PhD. study director
PureTech Health
9 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 LYT-200, Venetoclax and Azacitidine in people with AML, Adult Recurrent and MDS, run by PureTech at 9 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 7 inclusion and 8 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: finished, as of the sponsor's last update on 6 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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 of Treatment-Emergent Adverse Events [Safety and RP2D determination] (time frame: approximately 1 year). 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?
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 6 May 2026.