RightFit
RecruitingPhase 1

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

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)

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests how safe RPT1G is, how well people handle it, and the best dose and treatment schedule. It is for adults with acute myeloid leukemia (AML), a blood cancer, that came back or did not respond to treatment, or high-risk myelodysplastic syndromes (MDS), a disease of the bone marrow.

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).

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Have AML that came back or did not respond to treatment, or high-risk MDS, confirmed by testing.
  • Have received standard treatment that the study doctor considers appropriate, or have chosen not to receive it.
  • Be able to care for yourself and be out of bed or a chair for more than half of your waking hours (ECOG score of 0–2).
  • Have liver blood tests within the study’s limits. Special limits may apply for Gilbert’s syndrome, which affects bilirubin levels, or liver changes caused by the disease.
  • Have kidney function above the study’s required level, based on a calculation using a blood test.

Who can't join

  • Have no signs of the disease in your blood or bone marrow.
  • Have acute promyelocytic leukemia, a type of AML.
  • Have AML in the brain or spinal cord that causes symptoms.
  • Have signs of leukemia cells blocking blood flow that need urgent treatment.
  • Have an active infection.
  • Had radiation treatment less than 14 days before the first RPT1G dose.
  • Have ongoing problems from past treatment, or have a past or current cancer.
  • Have another health problem or treatment that makes the study not in your best interest, in the study doctor’s judgment.

Simplified for readability. The official criteria below are what the study team uses.

Official 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

Study sites (2)

NY

TX