Acute Myeloid LeukemiaPhase 1NCT07107126
Safety and Proof-of-Concept Study of RPT1G in Adults With Acute Myeloid Leukemia and High-Risk Myelodysplastic Syndromes
A Phase 1 study testing RPT1G in people with Acute Myeloid Leukemia and Myelodysplastic Syndrome, run by Remedy Plan, Inc. at 2 sites in United States.
Trial summary
The main goals of this study are to learn if RPT1G is safe and tolerable and to determine the best dose and schedule of RPT1G for patients with relapsed or refractory acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia, Myelodysplastic Syndrome
- What is being tested?
- RPT1G
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 2 sitesUnited States
- Study dates
- 13 Feb 2026to 1 Dec 2028
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Eastern Cooperative Oncology Group (ECOG) performance status ≤2
- Histological confirmation of AML (ELN 2022 criteria) that relapsed and/or refractory AML or high-risk MDS as defined by International Consortium for MDS (icMDS) 2023 criteria that have received appropriate standard of care therapy, in the opinion of the investigator or declined receipt of these
- Organ function/reserve as per the following laboratory criteria:
- Hepatic: Aspartate aminotransferase (AST) ≤2.5 x upper limit of normal (ULN), alanine aminotransferase (ALT) ≤2.5 x ULN, and total bilirubin <2 x ULN (except for study patients with known Gilbert's where 1.5 x UL of the subject's baseline or in the case of suspected Gilbert's syndrome where a maximum total bilirubin level of 4.0 mg/dL is acceptable) for the local laboratory. If due to disease, higher values may be approved after discussion with medical monitor.
- Renal: Adequate renal function as defined by calculated creatinine clearance >50 mL/min for the CLIA certified local laboratory. Creatinine clearance must be calculated by Cockcroft-Gault equation.
Who cannot join
- Patients without evidence of blood or marrow involvement
- Acute promyelocytic leukemia
- Symptomatic central nervous system involvement by AML
- Clinical signs/symptoms of leukostasis requiring urgent therapy
- Active infections
- Radiotherapy <14 days prior to the first day of RPT1G administration
Show all 9Show fewer
- Ongoing complications from prior therapy
- Prior or concurrent malignancy
- Any other condition, therapy, treatment, or comorbidity that leads the investigator to determine that the study is not in the best interest of the patient
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Eunice Vukosavljevic, CP, Director of Clinical Operations contact
United States2 sites, 2 recruiting
- Recruiting
MD Anderson Cancer Center
Houston, Texas, 77030
- Recruiting
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
About this study
Official title: A Phase 1 Multi-Center, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Anti-Leukemia Activity of RPT1G in Patients With Acute Myeloid Leukemia (AML) and High-Risk Myelodysplastic Syndromes (MDS)
The main goals of this study are to learn if RPT1G is safe and tolerable and to determine the best dose and schedule of RPT1G for patients with relapsed or refractory acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS).
- Conditions
- Acute Myeloid Leukemia, Myelodysplastic Syndrome
- Being tested
- RPT1G
- Medical subject headings
- Leukemia, Myeloid, Acute, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RPT1GDrug
RPT1G is a selective hyperbolic NAMPT inhibitor. RPT1G is administered orally.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Starting Dose of RPT1G Receives: Drug: RPT1G |
| Cohort 2 | Experimental | 2nd ascending dose of RPT1G Receives: Drug: RPT1G |
| Cohort 3 | Experimental | 3rd ascending dose of RPT1G Receives: Drug: RPT1G |
| Cohort 4 | Experimental | 4th ascending dose of RPT1G Receives: Drug: RPT1G |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and nature of dose-limiting toxicity(ies) (DLTs)
Measured: 3 years
Incidence of adverse events, serious adverse events, and treatment-emergent adverse events
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Aug 2025
- Study start13 Feb 2026
- Primary completion1 Dec 2028 (estimated)
- Study completion1 Dec 2028 (estimated)
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
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Remedy Plan, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07107126
- Sponsor's protocol ID
- RPT1G-1002
- Status last verified
- Mar 2026
- First submitted
- 30 Jul 2025
- Last updated
- 25 Mar 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Multi-Center, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Anti-Leukemia Activity of RPT1G in Patients With Acute Myeloid Leukemia (AML) and High-Risk Myelodysplastic Syndromes (MDS)
- ClinicalTrials.gov ID
- NCT07107126
- Acronym / sponsor ID
- RPT1G-1002
- Conditions
- Acute Myeloid Leukemia, Myelodysplastic Syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Remedy Plan, Inc.Industry (pharma, biotech, devices)
- Study start
- 13 Feb 2026 (actual)
- Primary completion
- 1 Dec 2028 (estimated)
- Study completion
- 1 Dec 2028 (estimated)
- First posted
- 6 Aug 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.
The main goals of this study are to learn if RPT1G is safe and tolerable and to determine the best dose and schedule of RPT1G for patients with relapsed or refractory acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Drug: RPT1GStarting Dose of RPT1G |
| Cohort 2 | Experimental | Drug: RPT1G2nd ascending dose of RPT1G |
| Cohort 3 | Experimental | Drug: RPT1G3rd ascending dose of RPT1G |
| Cohort 4 | Experimental | Drug: RPT1G4th ascending dose of RPT1G |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Eastern Cooperative Oncology Group (ECOG) performance status ≤2
- Histological confirmation of AML (ELN 2022 criteria) that relapsed and/or refractory AML or high-risk MDS as defined by International Consortium for MDS (icMDS) 2023 criteria that have received appropriate standard of care therapy, in the opinion of the investigator or declined receipt of these
- Organ function/reserve as per the following laboratory criteria:
- Hepatic: Aspartate aminotransferase (AST) ≤2.5 x upper limit of normal (ULN), alanine aminotransferase (ALT) ≤2.5 x ULN, and total bilirubin <2 x ULN (except for study patients with known Gilbert's where 1.5 x UL of the subject's baseline or in the case of suspected Gilbert's syndrome where a maximum total bilirubin level of 4.0 mg/dL is acceptable) for the local laboratory. If due to disease, higher values may be approved after discussion with medical monitor.
- Renal: Adequate renal function as defined by calculated creatinine clearance >50 mL/min for the CLIA certified local laboratory. Creatinine clearance must be calculated by Cockcroft-Gault equation.
Key exclusion criteria (9 listed)
- Patients without evidence of blood or marrow involvement
- Acute promyelocytic leukemia
- Symptomatic central nervous system involvement by AML
- Clinical signs/symptoms of leukostasis requiring urgent therapy
- Active infections
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 |
|---|---|---|
| RPT1GRPT1G is a selective hyperbolic NAMPT inhibitor. RPT1G is administered orally. | Drug | Cohort 1, Cohort 2, Cohort 3, Cohort 4 |
Primary outcome
Incidence and nature of dose-limiting toxicity(ies) (DLTs)
Time frame: 3 years
Incidence of adverse events, serious adverse events, and treatment-emergent adverse events
Time frame: 3 years
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 25 Mar 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.
Eunice Vukosavljevic, CP, Director of Clinical Operations contact
2 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 RPT1G in people with Acute Myeloid Leukemia and Myelodysplastic Syndrome, run by Remedy Plan, Inc. at 2 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 5 inclusion and 9 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 25 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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?
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 Eunice Vukosavljevic, CP, Director of Clinical Operations (240-408-4854, clinical@remedyplan.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and nature of dose-limiting toxicity(ies) (DLTs) (time frame: 3 years). 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 25 Mar 2026.