RPT1G for adults with relapsed or refractory acute myeloid leukemia and high-risk MDS

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)

This study will test whether the drug RPT1G is safe and find the best dose for adults whose AML or high-risk MDS has come back or not responded to treatment.

Quick facts

• Phase: PHASE1
• Study type: Interventional
• Enrollment: 24 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Remedy Plan, Inc. (industry)
• Locations: 2 sites (New York, New York and 1 other locations)
• Trial ID: NCT07107126 on ClinicalTrials.gov

What this trial studies

This Phase 1, open-label dose-finding study gives RPT1G to adults with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS) to determine safety, tolerability, and an optimal dose and schedule. Eligible participants have had appropriate prior standard therapy (or declined it) and must meet performance status (ECOG ≤2) and organ-function criteria. Patients receive RPT1G on a protocol-defined schedule with frequent clinical and laboratory monitoring for adverse events and early signs of anti-leukemia activity. Dose-escalation and safety review procedures guide dose and schedule decisions at participating cancer centers.

Who should consider this trial

Good fit: Adults with relapsed or refractory AML or high-risk MDS who have received (or declined) appropriate prior standard therapy, have ECOG performance status ≤2, and adequate organ function are the intended candidates.

Not a fit: Patients with newly diagnosed, treatment-responsive disease, poor performance status (ECOG >2), or significant organ dysfunction are unlikely to benefit from or be eligible for this study.

Why it matters

Potential benefit: If successful, RPT1G could provide a new treatment option for patients with relapsed or refractory AML or high-risk MDS who have limited therapeutic choices.

How similar studies have performed: Early-phase trials of novel agents in AML/MDS have sometimes led to further development, but approaches like RPT1G remain investigational and proof-of-concept in humans is limited.

Eligibility criteria

Key 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.

Key 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

Where this trial is running

New York, New York and 1 other locations

• Memorial Sloan Kettering Cancer Center — New York, New York, United States (RECRUITING)
• MD Anderson Cancer Center — Houston, Texas, United States (RECRUITING)

Study contacts

• Study coordinator: Eunice Vukosavljevic, CP, Director of Clinical Operations

Conditions: Acute Myeloid Leukemia, Myelodysplastic Syndrome