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

Remedy Plan, Inc.

Status and phase: Not yet enrolling; Phase 1

Conditions: Myelodysplastic Syndrome; Acute Myeloid Leukemia

Treatments: Drug: RPT1G

Study type: Interventional

Funder types: Industry

Identifiers

NCT07107126

RPT1G-1002

Details and patient eligibility

About

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).

Enrollment: 18 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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18 participants in 3 patient groups

Cohort 1

Experimental group

Description:

Starting Dose of RPT1G

Treatment:

Drug: RPT1G

Cohort 2

Experimental group

Description:

2nd ascending dose of RPT1G

Treatment:

Drug: RPT1G

Cohort 3

Experimental group

Description:

3rd ascending dose of RPT1G

Treatment:

Drug: RPT1G