What side effects are associated with this type of intervention?

Common treatments for leukemia, such as vincristine, daunorubicin, dexamethasone, and cytarabine, are associated with a variety of side effects. These can include neurotoxicity (e.g., peripheral neuropathy), myelosuppression (leading to increased risk of infection and anemia), gastrointestinal disturbances (e.g., nausea, vomiting, diarrhea), and organ-specific toxicities (e.g., cardiotoxicity with daunorubicin). Additionally, patients may experience general symptoms such as fatigue, hair loss, and mucositis. These side effects result from the non-selective nature of these chemotherapeutic agents, which damage both cancerous and healthy cells.

What prior FDA approvals exist?

The FDA has approved several drugs for the treatment of leukemia, including venetoclax and ivosidenib, which are used in combination with hypomethylating agents (HMAs) like azacitidine and decitabine. Venetoclax targets BCL-2, and ivosidenib targets IDH1 mutations. While these drugs do not specifically inhibit mitochondrial TCA cycle enzymes like CPI-613, they represent significant advancements in targeted leukemia therapies. Additionally, IDH inhibitors and gemtuzumab ozogamicin are other notable FDA-approved treatments for leukemia.

What other types of research exist?

Promising novel alternatives to CPI-613 for leukemia patients include: <ol> <li>Venetoclax: A BCL-2 inhibitor used in combination with hypomethylating agents (HMAs) like azacitidine or decitabine, showing efficacy in acute myeloid leukemia (AML).</li> <li></li> </ol> Ivosidenib: An IDH1 inhibitor used in combination with HMAs for AML. 3. Nelarabine: A prodrug converted to ara-GTP, effective in relapsed or refractory T-cell acute lymphoblastic leukemia (ALL). 4. CAR-T cell therapies (e.g., Axicabtagene ciloleucel and Tisagenlecleucel): CD19-directed immunotherapies for relapsed or refractory B-cell leukemias.

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Enzyme Inhibitor

CPI-613 for Lymphoma

Phase 2

Waitlist Available

Led By Ariela Noy, MD

Research Sponsored by Memorial Sloan Kettering Cancer Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Failure of at least one previous line of therapy

Must be ≥ 12 years of age

Must not have

Patients that have received a chemotherapy regimen with stem cell support in the previous 2 months

Serious medical illness, such as significant cardiac disease (e.g. symptomatic congestive heart failure, unstable angina pectoris, coronary artery disease, myocardial infarction within the past 3 months, uncontrolled cardiac arrhythmia, pericardial disease or New York Heart Association Class III or IV), or severe debilitating pulmonary disease, that would potentially increase patient's risk for toxicity

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 3 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new medication called CPI-613. It aims to see if this drug can help patients who might benefit from it. The drug works by targeting specific pathways in the body to treat diseases.

Who is the study for?

This trial is for patients aged 12 or older with relapsed/refractory Burkitt Lymphoma/Leukemia, high-grade B-cell lymphoma with high-risk translocations, and adequate organ function. Participants must not have HIV complications, recent allogeneic stem cell transplant, uncontrolled conditions, or recent other cancer treatments. Women of childbearing potential must use contraception and have a negative pregnancy test.

What is being tested?

The study is testing the effects of CPI-613 on individuals with specific types of lymphomas and leukemias that are resistant to previous treatments. The drug's effectiveness as well as any adverse reactions will be monitored throughout the trial.

What are the potential side effects?

While the exact side effects are not listed here, participants can expect possible adverse reactions typical for oncology drugs such as nausea, fatigue, blood count changes, liver enzyme alterations which will be closely monitored during the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have tried at least one treatment that didn't work for my condition.

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I am 12 years old or older.

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I have recovered from serious side effects of my previous cancer treatments.

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I can care for myself but spend more than half of my waking hours in bed or a chair.

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I am using birth control and have a negative pregnancy test.

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My kidneys are functioning well enough for treatment.

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My diagnosis is a specific type of lymphoma with certain genetic changes.

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I am willing to get a central venous access device if I don't already have one.

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I agree not to breastfeed during the study.

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My previous bone marrow transplant for Burkitt Lymphoma/Leukemia or DHL/THL was unsuccessful, or I am ineligible for or chose not to undergo one.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I had chemotherapy with stem cell support in the last 2 months.

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I do not have serious heart or severe lung conditions that could worsen with treatment.

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I have HIV but it's under control, and I'm not on certain HIV medications that could interfere with the study drug.

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I do not have any ongoing serious bleeding issues.

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I had a stem cell transplant from a donor within the last 2 months.

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I do not have any uncontrolled medical conditions.

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I am not on immunosuppressive therapy for graft-versus-host disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 3 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~3 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 3 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

overall response rate of CPI-613

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: CPI-613Experimental Treatment1 Intervention

CPI-613 IV induction (Days 1-5 for first 2 Cycles \[14-day cycles\]), followed by CPI-613 IV maintenance (Days 1-5 for all Cycles thereafter \[21-day cycles\].

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

CPI-613

2008

Completed Phase 1

~140

0 / 17Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Leukemia treatments often target the metabolic pathways essential for cancer cell survival and proliferation. CPI-613, for example, inhibits mitochondrial TCA cycle enzymes, disrupting the energy production and biosynthesis processes crucial for leukemia cell survival. This mechanism is significant as it targets the metabolic vulnerabilities of cancer cells, potentially leading to their death while sparing normal cells. Other common treatments include chemotherapy agents like cytarabine and clofarabine, which interfere with DNA synthesis and repair, and targeted therapies like tyrosine kinase inhibitors, which block specific proteins involved in cancer cell signaling and growth. Understanding these mechanisms helps in designing effective treatment regimens and improving patient outcomes.

Red blood cell-encapsulated L-asparaginase: potential therapy of patients with asparagine synthetase deficient acute myeloid leukemia.