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

NCT07107126 | Recruiting Phase 1 FDA Drug

• 1 other identifier: RPT1G-1002
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 2

Brief 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).

Conditions

Acute Myeloid Leukemia; Myelodysplastic Syndrome

Outcome Measures

Primary Outcomes (2)

• Incidence and nature of dose-limiting toxicity(ies) (DLTs)

  3 years

• Incidence of adverse events, serious adverse events, and treatment-emergent adverse events

  3 years

Study Arms (4)

Cohort 1

EXPERIMENTAL

Starting Dose of RPT1G

Drug: RPT1G

Cohort 2

EXPERIMENTAL

2nd ascending dose of RPT1G

Drug: RPT1G

Cohort 3

EXPERIMENTAL

3rd ascending dose of RPT1G

Drug: RPT1G

Cohort 4

EXPERIMENTAL

4th ascending dose of RPT1G

Drug: RPT1G

Interventions

RPT1G DRUG

RPT1G is a selective hyperbolic NAMPT inhibitor. RPT1G is administered orally.

Cohort 1; Cohort 2; Cohort 3; Cohort 4

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Remedy Plan, Inc.: lead

Study Sites (2)

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

RECRUITING

MD Anderson Cancer Center

Houston, Texas, 77030, United States

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases

Central Study Contacts

Eunice Vukosavljevic, CP, Director of Clinical Operations

CONTACT

240-408-4854; clinical@remedyplan.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 30, 2025
• First Posted: August 6, 2025
• Study Start: February 13, 2026
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2028
• Last Updated: March 25, 2026

Record last verified: 2026-03

Locations

US (2)