Safety and Proof-of-Concept Study of RPT1G in Adults With Acute Myeloid Leukemia and High-Risk Myelodysplastic Syndromes
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: RPT1G.
- Who it may be relevant to
- Registry conditions: Acute Myeloid Leukemia, Myelodysplastic Syndrome. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
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)
Overview
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).
Interventions
- Drug RPT1G
RPT1G is a selective hyperbolic NAMPT inhibitor. RPT1G is administered orally.
Primary outcome measures
- 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]
Eligibility criteria
Inclusion criteria
- 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 criteria
- 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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 2 centers
- Memorial Sloan Kettering Cancer Center — New York
- MD Anderson Cancer Center — Houston
Identifiers
NCT: NCT07107126 · RPT1G-1002