RPT1G for AML and High-Risk MDS

This study is testing a drug called RPT1G in adults with acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS). RPT1G is a selective hyperbolic NAMPT inhibitor and is taken by mouth. The main goals are to find out if RPT1G is safe and well-tolerated, and to determine the best dose and schedule. You may be able to join if you have AML that has come back or not responded to previous treatment, or high-risk MDS, and meet other health criteria. The study will look at any side effects and serious side effects over three years to understand its safety. The current status of this study is unclear, and it plans to enroll 24 participants.

Study design
This is an interventional study with an unclear phase, planning to enroll 24 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The study will track side effects and serious adverse events for three years.

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NCT07107126

Safety and Proof-of-Concept Study of RPT1G in Adults With Acute Myeloid Leukemia and High-Risk Myelodysplastic Syndromes

Recruiting
PHASE1Ages 18+InterventionalTreatment
Remedy Plan, Inc.
~24 participants
Updated 2026-03-25 on ClinicalTrials.gov
What's tested:RPT1G

At a glance

Recruiting sites
2 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Incidence and nature of dose-limiting toxicity(ies) (DLTs)
Measured over 3 years
+1 more outcome measured
Acute Myeloid Leukemia
Myelodysplastic Syndrome
2 sites across 2 states
New York1
Texas1
Eunice Vukosavljevic, CP, Director of Clinical Operations
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Eligibility criteria

Inclusion

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.

Exclusion

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
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
  • Incidence and nature of dose-limiting toxicity(ies) (DLTs)3 years
  • Incidence of adverse events, serious adverse events, and treatment-emergent adverse events3 years