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

NCT ID: NCT07107126

Last Updated: 2025-08-06

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 18 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-12-01
• Study Completion Date: 2028-12-01

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

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Cohort 1

Starting Dose of RPT1G

Group Type: EXPERIMENTAL

RPT1G

Intervention Type: DRUG

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

Cohort 2

2nd ascending dose of RPT1G

Group Type: EXPERIMENTAL

RPT1G

Intervention Type: DRUG

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

Cohort 3

3rd ascending dose of RPT1G

Group Type: EXPERIMENTAL

RPT1G

Intervention Type: DRUG

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

Interventions

RPT1G

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

Intervention Type: DRUG

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Remedy Plan, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Central Contacts

Gregory Crimmins, Ph.D.

Role: CONTACT

clinical@remedyplan.com

240-408-4854

Other Identifiers

RPT1G-1002

Identifier Type: -

Identifier Source: org_study_id