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Acute Myeloid Leukemia, in RelapsePhase 1NCT04402541

Study of CB-5339 in Acute Myeloid Leukemia or Myelodysplastic Syndrome

A Phase 1 study testing CB-5339 in people with Acute Myeloid Leukemia, in Relapse and Myelodysplastic Syndromes, run by Cleave Therapeutics, Inc. at 10 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This is a multicenter, open-label Phase 1 study of orally administered CB-5339 in participants with R/R AML or participants with R/R intermediate- to high-risk MDS. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
CB-5339
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
55 participants
Where
10 sitesin 2 countries
Study dates
8 Jun 2020to 23 Jul 2023
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Male or female and ≥ 18 years of age at the time of signing the consent form
  • One of the following advanced hematologic malignancies including:
  • Relapsed or refractory AML as defined by 2016 WHO criteria and are not candidates for curative therapies such as allogeneic hematopoietic cell transplant or for whom there is no standard of care therapy available that is likely to lead to disease remission according the investigator
  • MDS high-very high risk by the revised international scoring system for evaluating prognosis in myelodysplastic syndromes that is recurrent or refractory or the participant is intolerant to established therapy known to provide clinical benefit for their condition (e.g., relapsed following treatment with hypomethylating agent or lack of response after > 4 cycles), according to treating physician. Potential participants who meet the criteria for intermediate risk may be considered with approval by the medical monitor if the participant has severe cytopenia(s) and/or elevated bone marrow blast counts.
  • Adequate organ function defined as:
  • Serum creatinine ≤1.5 mg/dL or an estimated glomerular filtration rate of ≥60 mL/min as calculated by the Cockcroft-Gault glomerular filtration rate equation
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  • Total bilirubin ≤ 1.5 × the upper limit of normal (ULN) unless considered due to Gilbert's disease or leukemic disease
  • Aspartate aminotransferase (AST) ≤3 × the ULN; alanine aminotransferase (ALT) ≤3 × the ULN. Levels of AST and/or ALT ≤5 × the ULN may be acceptable for participants with known leukemic involvement of the liver after discussion with the study medical monitor
  • Eastern Cooperative Oncology Group (ECOG) performance status ≤2.
  • Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. If of childbearing potential, agree to use an effective barrier method of birth control (i.e., latex condom, diaphragm, cervical cap, etc.) to avoid pregnancy during the study and 90 days after the last dose of CB-5339. Female participants of childbearing potential need a negative serum or urine pregnancy test within 7 days of study enrollment. Non-childbearing is defined as ≥ 1 year postmenopausal or surgically sterilized
  • Capable of giving signed informed consent as described in Appendix 1 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol

Who cannot join

  • Acute promyelocytic leukemia with t(15;17)(q22;q12); or abnormal promyelocytic leukemia/retinoic acid receptor alpha (PML-RARA).
  • Participants with clinical symptoms suggestive of active central nervous system (CNS) leukemia or known CNS leukemia. Evaluation of cerebrospinal fluid is only required if there is a clinical suspicion of CNS involvement by leukemia during screening.
  • Participants with immediately life-threatening, severe complications of leukemia such as uncontrolled bleeding, pneumonia with hypoxia or shock, and/or disseminated intravascular coagulation.
  • Concomitant malignancy, requiring active treatment, except for basal-cell or squamous cell carcinoma of the skin, carcinoma-in-situ of the uterine cervix, or localized prostate cancer.
  • Adjuvant therapy for breast cancer or prostate cancer is allowed.
  • Active, uncontrolled, systemic infection or severe localized infection during screening or prior to Cycle 1 Day 1 (C1D1; unless considered due to tumor by the investigator).
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  • Note, participants receiving prophylactic anti-infectives are allowed on study.
  • Known human immunodeficiency virus (HIV) infection with CD4+ T cell counts <350 cells/μL, initiation of antiretroviral therapy within 4 weeks before C1D1, or acquired immunodeficiency syndrome (AIDS)-related infection within 12 months before C1D1.
  • Hepatitis B virus (HBV) or hepatitis C virus (HCV) infection with viral load above the limit of quantification
  • Major cardiac abnormalities as defined but not limited to the following: uncontrolled angina or unstable life-threatening arrhythmias, history of myocardial infarction within 12 weeks prior to Baseline, Class 3 or higher New York Heart Association (NYHA) congestive heart failure, or left ventricular ejection fraction (LVEF) <45% as measured by echocardiogram (ECHO) within 28 days of C1D1
  • Persistent (3 consecutive ECGs performed ≥5 minutes apart) prolongation of the corrected QT interval by Fredericia's method (QTcF) to > 480 msec
  • Gastrointestinal conditions that may interfere with the absorption of orally-administered drugs including but not limited to short gut syndrome, gastroparesis, inflammatory bowel disease, or acute pancreatitis.
  • Any other severe, acute, or chronic medical or psychiatric condition, or laboratory abnormality that may increase the risk associated with study participation or CB-5339 administration, may interfere with the informed consent process and/or with compliance with the requirements of the study, may interfere with the interpretation of the study results and, in the Investigator's opinion, or would make the participant inappropriate for entry into this study
  • A condition that is expected to require concomitant use of any medication listed as prohibited while on study.
  • Known hypersensitivity to any components of CB-5339.
  • Use of chemotherapy (except hydroxyurea), radiation or monoclonal antibodies within 14 days or 5 half-lives for small molecule inhibitors prior to first dose of CB-5339
  • Participants who have undergone a hematopoietic cell transplant (HCT) within 100 days of the first dose of CB-5339, or participants on immunosuppressive therapy post-HCT at the time of screening, use of calcineurin inhibitors within 4 weeks prior to first dose of CB-5339, or with clinically significant graft-versus-host disease (GVHD).
  • Note: The use of topical steroids or <10mg oral prednisone for ongoing skin GVHD is permitted.
  • Major surgery within 4 weeks prior to first dose of CB-5339. Participant must have recovered from surgery and be without current complications of infection or dehiscence
  • Enrollment in other clinical trials unless approved by Medical Monitor

Trial locations and contacts

10 sites in 2 countries.

10 sites in 2 countries

United States7 sites

  • Cleveland Clinic, Case Comprehensive Cancer Center

    Cleveland, Ohio, 44122

  • KU Clinical Research Center

    Fairway, Kansas, 66205

  • Saint Francis Hospital Cancer Center

    Greenville, North Carolina, 29607

  • MD Anderson Cancer Cancer

    Houston, Texas, 77030

  • USC Norris Comprehensive Cancer Center

    Los Angeles, California, 90033

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10021

  • Weill Cornel Medical Center

    New York, New York, 10065

Other trials in United States

Australia3 sites

  • St. Vincent Hospital, Sydney, NSW

    Darlinghurst, New South Wales, 2010

  • Epworth Healthcare

    Melbourne, Victoria, 3002

  • Haematology Clinical Trials Unit, Royal Perth Hospital

    Perth, Western Australia, 6000

Other trials in Australia

About this study

Official title: A Phase 1 Study to Evaluate the Safety and Pharmacokinetic Profiles of CB-5339 in Participants With Relapsed/Refractory Acute Myeloid Leukemia or Relapsed/Refractory Intermediate or High Risk Myelodysplastic Syndrome

This is a multicenter, open-label Phase 1 study of orally administered CB-5339 in participants with R/R AML or participants with R/R intermediate- to high-risk MDS.

Read the detailed description

This is a multicenter, open-label Phase 1 study of orally administered CB-5339 in participants with R/R AML or participants with R/R intermediate- to high-risk MDS. The study will include two parts:1) a Dose Escalation phase in participants with R/R AML, or R/R intermediate- to high-risk MDS and 2) a Dose Expansion phase in participants with R/R AML for whom there is no standard of care therapy available that is likely to lead to disease remission. Additional cohorts for participants with R/R intermediate- to high-risk MDS following hypomethylating agents or other AML cohorts may be added at a later time.

Being tested
CB-5339
Medical subject headings
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CB-5339Drug

    Also called CB-5339 Tosylate

    25mg and 75mg capsules

Study groups in NCT04402541
Study groupRoleWhat they receive
CB-5339ExperimentalOrally administered CB-5339
Receives: Drug: CB-5339

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs).

    Measured: 28 Days

  2. Define the MTD and/or RP2D and schedule for CB-5339

    Incidence and nature of DLTs with CB-5339 monotherapy for MTD determination. All available safety, efficacy and PK, for RP2D determination

    Measured: 28 Days

Secondary outcomes (6)

  1. Peak plasma concentration (Cmax)

    Measured: Day 1 and Day 4

  2. Time to reach peak plasma concentration (Tmax)

    Measured: Day 1 and Day 4

  3. Area under the plasma concentration time curve (AUC0-t)

    Measured: Day 1 and Day 4

  4. Elimination half-life (t½)

    Measured: Day 1 and Day 4

  5. Accumulation ratio

    Measured: Day 1 and Day 4

  6. Antitumor effects

    CR/CRi rate at the RP2D in participants with R/R AML, as assessed by the investigator, based on 2017 ELN response criteria.

    Objective response rate (ORR) at the RP2D in participants with R/R AML, as assessed by the investigator based on 2017 ELN response criteria.

    DoCR in participants with R/R AML who attain CR or CRi

    Measured: End of each cycle (cycle is 28 days)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted27 May 2020
  2. Study start8 Jun 2020
  3. Primary completion23 Jul 2023
  4. Study completion23 Jul 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
55 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04402541
Sponsor's protocol ID
CTX-001
Status last verified
Aug 2023
First submitted
18 May 2020
Last updated
31 Aug 2023
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Wang F, Li S, Rosencrans WM, Cheng KW, Stott GM, Mroczkowski B, Chou TF. Sulforaphane is Synergistic with CB-5083 and Inhibits Colony Formation of CB-5083-Resistant HCT116 Cells. ChemMedChem. 2022 Jun 3;17(11):e202200030. doi: 10.1002/cmdc.202200030. Epub 2022 Apr 22.

    PubMed 35451199

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing CB-5339 in people with Acute Myeloid Leukemia, in Relapse and Myelodysplastic Syndromes, run by Cleave Therapeutics, Inc. at 10 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 11 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 31 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 10 sites in United States, Australia. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs). (time frame: 28 Days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04402541, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Aug 2023.

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