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CPI-613 Given With Metformin in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML)

Phase 2
Not yet recruiting
Conditions
Acute Myeloid Leukemia, in Relapse
Acute Myeloid Leukemia Refractory
Granulocytic Sarcoma
Interventions
Biological: Blood draws
Procedure: Bone marrow biopsy
Registration Number
NCT05854966
Lead Sponsor
Wake Forest University Health Sciences
Brief Summary

The purpose of this research study is to find out what effects (the good and bad) the combination treatment of metformin and CPI-613 has in treating participants with acute myeloid leukemia or granulocytic sarcoma that has either returned after treatment or did not respond to treatment.

Detailed Description

Primary Objective: To establish the feasibility of delivering the combination of CPI-613 and metformin in patients with relapsed or refractory acute myeloid leukemia (AML).

Secondary Objectives: To determine the response rate of CPI-613 and metformin in relapsed or refractory AML defined as Complete remission (CR) + Complete remission with incomplete count recovery (CRi) + Morphologic Leukemia-Free State (MLFS).

* To determine the overall survival of patients with relapsed or refractory AML treated with CPI-613 and metformin.

* To determine the safety of CPI-613 and metformin in patients with relapsed or refractory acute myeloid leukemia (AML).

Recruitment & Eligibility

Status
NOT_YET_RECRUITING
Sex
All
Target Recruitment
17
Inclusion Criteria
  • Patients must have histologically or cytologically documented relapsed and/or refractory Acute Myeloid Leukemia or granulocytic sarcoma.
  • Eastern Cooperative Oncology Group (ECOG) Performance Status of ≤ 3.
  • Must be ≥ 18 years of age.
  • Persons of child-bearing potential (i.e., women who are pre-menopausal or not surgically sterile) must use accepted contraceptive methods (abstinence, intrauterine device [IUD], oral contraceptive or double barrier device), and must have a negative serum or urine pregnancy test within 1 week prior to treatment initiation.
  • Persons who are having sexual relationships in which their partner may become pregnant must practice effective contraceptive methods during the study treatment and for 60 days after the last dose of study treatment, unless documentation of infertility exists.
  • Mentally competent, ability to understand and willingness to sign the informed consent form.
  • Patients with persisting, non-hematologic, non-infectious toxicities from prior treatment must be ≤ Grade 2 and must be documented as such.
  • Laboratory values ≤ 2 weeks prior to the start of study treatment must be the following:
  • Aspartate aminotransferase [AST/SGOT] ≤ 5x upper normal limit [UNL],
  • Alanine aminotransferase [ALT/SGPT] ≤ 5x UNL
  • Bilirubin ≤ 3x UNL
  • Albumin ≥ 2.0 g/dL or ≥ 20 g/L
  • Serum creatinine ≤ 2.0 mg/dL
  • Presence of central venous catheter or willing to have central venous access placed.
Exclusion Criteria
  • Patients with active central nervous system (CNS) or epidural tumor.
  • Pregnant persons, or persons of child-bearing potential not using reliable means of contraception (because the teratogenic potential of CPI-613 is unknown).
  • Breastfeeding individuals because the potential of excretion of CPI-613 into breast milk. (Note: Breastfeeding individuals are excluded because the effects of CPI-613 on a nursing child are unknown).
  • Any condition or abnormality which may, in the opinion of the investigator, compromise the safety of patient.
  • Unwilling or unable to follow protocol requirements.
  • Patients receiving any other standard or investigational treatment for their cancer, or any other investigational agent for any indication within the past 1 week prior to initiation of CPI-613 treatment with the following exceptions:
  • The use of Hydrea or any targeted oral agent is allowed up to the day before initiation of treatment.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Treatment - CPI-613 with MetforminBlood drawsInduction therapy with CPI-613 and Metformin (ideally 2 hours prior to start of CPI-613 infusions on days 1-5) for two cycles of treatment. Maintenance therapy with CPI-613 and Metformin (ideally 2 hours prior to start of CPI-613 infusions on days 1-5) until progression, intolerable toxicity of withdrawal of consent.
Treatment - CPI-613 with MetforminBone marrow biopsyInduction therapy with CPI-613 and Metformin (ideally 2 hours prior to start of CPI-613 infusions on days 1-5) for two cycles of treatment. Maintenance therapy with CPI-613 and Metformin (ideally 2 hours prior to start of CPI-613 infusions on days 1-5) until progression, intolerable toxicity of withdrawal of consent.
Treatment - CPI-613 with MetforminCPI 613Induction therapy with CPI-613 and Metformin (ideally 2 hours prior to start of CPI-613 infusions on days 1-5) for two cycles of treatment. Maintenance therapy with CPI-613 and Metformin (ideally 2 hours prior to start of CPI-613 infusions on days 1-5) until progression, intolerable toxicity of withdrawal of consent.
Treatment - CPI-613 with MetforminMetforminInduction therapy with CPI-613 and Metformin (ideally 2 hours prior to start of CPI-613 infusions on days 1-5) for two cycles of treatment. Maintenance therapy with CPI-613 and Metformin (ideally 2 hours prior to start of CPI-613 infusions on days 1-5) until progression, intolerable toxicity of withdrawal of consent.
Primary Outcome Measures
NameTimeMethod
Number of Participants to Receive at Least One Cycle of Maintenance Therapy - FeasibilityAfter the completion of cycle 1 of maintenance therapy (maintenance cycle is 21 days)

Feasibility is defined as the ability to deliver at least 1 cycle of maintenance therapy in 50% or more of patients who complete induction therapy.

Secondary Outcome Measures
NameTimeMethod
Response Rate - Efficacy (Acute Myeloid Leukemia European LeukemiaNet 2022)After the completion of cycle 2 (each cycle is 14 days), then every three months up to 12 months

Efficacy will be assessed in the first 9 evaluable participants using a Simon's two-stage design to examine efficacy in terms of response where response is defined as:

* Complete remission (CR) (Bone marrow blasts, 5%; absence of circulating blasts; absence of extramedullary disease; ANC ≤ 1.0 × 109/L (1,000/μL); plate) or

* Complete remission with incomplete count recovery (CRi) (All CR criteria except for residual neutropenia \< 1.0 × 109/L (1,000/μL) or thrombocytopenia \< 100 × 109/L (100,000/μL) or

* Morphologically leukemia free state (MLFS) (Bone marrow blasts \< 5%; absence of circulating blasts; absence of extramedullary disease; no hematologic recovery required).

Number of Reported Adverse Events - SafetyUp to 30 days after last dose of study treatment

Safety will be evaluated by describing the nature and frequency of adverse events as defined by Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.

Overall SurvivalEvery 3 months after last dose of study treatment, up to 2 years

Overall survival is calculated in days from date of study treatment initiation to date of death due to any cause. Patients last known to be alive are censored at date of last contact.

Trial Locations

Locations (1)

Wake Forest Baptist Comprehensive Cancer Center

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Winston-Salem, North Carolina, United States

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