CTX-712 for Acute Myeloid Leukemia and Myelodysplastic Syndrome

Recruiting at 5 trial locations

Overseen By Haris Durutlic

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Chordia Therapeutics, Inc.

Disqualifiers: CNS leukemia, Other malignancy, Heart failure, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

225 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new drug, CTX-712 (also known as rogocekib), to determine its safety and effectiveness for people with specific blood cancers, including acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). In its early stages, the trial seeks to find the right dose and assess patient response to the treatment. It seeks participants with these conditions who have tried 1-4 other treatments without success. The study aims to gather more information about CTX-712's impact on these cancers and ensure the dosage is both safe and effective for future use. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the chance to be among the first to receive this new drug.

Do I need to stop taking my current medications to join the trial?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor to get a clear answer.

Is there any evidence suggesting that CTX-712 is likely to be safe for humans?

Research has shown that CTX-712 is generally safe for patients with relapsed or hard-to-treat acute myeloid leukemia (AML) and more serious myelodysplastic syndromes (MDS). Previous studies found that most patients tolerated the treatment well, with manageable side effects. Although specific side effects are not detailed, the progression to this trial phase suggests it is considered safe enough for further testing. Overall, CTX-712 appears promising, but it is important to monitor for any possible side effects during the trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

CTX-712 is unique because it targets acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) in a different way from most standard treatments like chemotherapy, such as cytarabine and daunorubicin. While traditional therapies attack rapidly dividing cells, CTX-712 may offer a more targeted approach, potentially improving precision in attacking cancer cells while reducing harm to healthy cells. Researchers are excited about CTX-712 because of its innovative mechanism of action, which could lead to improved effectiveness and fewer side effects, offering new hope for patients with these challenging conditions.

What evidence suggests that CTX-712 might be an effective treatment for acute myeloid leukemia and myelodysplastic syndrome?

Research has shown that CTX-712, a pill designed to block a protein involved in cell growth, has produced promising results in patients with relapsed or hard-to-treat acute myeloid leukemia (AML). Studies indicate that CTX-712 is generally safe and well-tolerated. Early findings suggest it can slow cancer cell growth in some patients with blood cancers. Although research is still in its early stages, these results offer hope that CTX-712 might effectively treat AML and certain high-risk blood disorders. Participants in this trial will receive CTX-712 in different dosing regimens to further evaluate its safety and effectiveness. ^{1 2 3 4 6}

Who Is on the Research Team?

Guillermo Garcia-Manero

Principal Investigator

M.D. Anderson Cancer Center

Are You a Good Fit for This Trial?

Adults (18+) with relapsed/refractory acute myeloid leukemia or high-risk myelodysplastic syndromes, who have undergone 1-4 prior treatments. Participants must have adequate organ function and blood counts, agree to contraception if of childbearing potential, and not be suffering from severe lung disease or recent major surgery.

Inclusion Criteria

I agree to use effective contraception or practice abstinence during and for four months after the study.

My kidney, liver, and blood tests are within normal ranges.

I am not pregnant, can take a test to prove it, and will use effective birth control during and up to 4 months after the study.

Exclusion Criteria

I have not had a heart attack in the last six months.

I have heart rhythm problems not managed by medicine.

I have not had major surgery in the last 4 weeks.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose Escalation

Participants receive ascending doses of CTX-712 to determine the recommended phase 2 dose (RP2D)

28 days per cycle

Weekly visits for dose administration

Dose Expansion

Additional patients are treated with CTX-712 at the RP2D to gain further confidence in the selected dose level

Duration not specified

Phase 2 Treatment

Evaluation of therapeutic activity and confirmation of the safety profile in R/R AML or R/R HR-MDS

Duration not specified

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• CTX-712

Trial Overview The trial is testing CTX-712's safety and effectiveness in two phases. Phase 1 determines the best dose through a '3+3' method where small groups receive increasing doses. Phase 2 evaluates its therapeutic activity at this established dose in patients with specific types of leukemia.

How Is the Trial Designed?

Group I: Phase 2 Experimental Treatment 1 Intervention

CTX-712 administered at the recommended dose by the expansion cohort

Group II: Initial Expansion Cohort Experimental Treatment 1 Intervention

Drug: CTX-712 administered at a dose to be determined from the data of dose escalation cohort

Group III: Dose Escalation Cohort Experimental Treatment 1 Intervention

Drug: CTX-712 administered at 20 mg, 40 mg, 80 mg, 100 mg, 140 mg weekly, or 60 mg, 80 mg, 100 mg twice a week

Who Is Running the Clinical Trial?

Chordia Therapeutics, Inc.

Lead Sponsor

Trials

1

Recruited

170+

Theradex

Industry Sponsor

Trials

34

Recruited

1,600+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05732103

A Study of CTX-712 in Relapsed/Refractory Acute Myeloid ... The goal of this phase 1/2 multicenter, open-label, single-arm dose escalation and expansion study is to assess the safety, tolerability, ...

2. chordiatherapeutics.com chordiatherapeutics.com/en/ir/news/auto_20250925562212/pdfFile.pdf

Chordia Therapeutics Inc. Rogocekib (CTX-712) is an oral, selective inhibitor of CDC2-like kinase (CLK), a key regulator of RNA splicing, that was evaluated for relapsed ...

3. mayo.edu mayo.edu/research/clinical-trials/cls-20548490

A Study of CTX-712 in Relapsed/Refractory Acute Myeloid ... The purpose of Phase I of this study is to evaluate the safety and tolerability of CTX-712 in patients with relapsed/refractory (R/R) acute myeloid leukemia ( ...

4. aacrjournals.org aacrjournals.org/cancerres/article/84/7_Supplement/CT115/742613/Abstract-CT115-A-first-in-human-phase-I-study-of

Abstract CT115: A first-in-human phase I study of CTX-712 in ... Conclusions: CTX-712 demonstrated a manageable and tolerable safety profile and showed anti-tumor efficacy in patients with hematologic ...

5. msn.com msn.com/en-us/health/other/chordia-therapeutics-ctx-712-study-a-potential-breakthrough-in-aml-and-hr-mds-treatment/ar-AA1PiwfW

A Potential Breakthrough in AML and HR-MDS Treatment ' The study aims to evaluate the safety, tolerability, and efficacy of CTX-712 in patients with relapsed/refractory acute myeloid leukemia (AML) and higher risk ...

6. fredhutch.org fredhutch.org/en/research/clinical-trials/trial-details.fh_trial_id_15192.a-study-of-ctx-712-in-relapsed-refractory-acute-myeloid-leukemia-and-higher-risk-myelodysplastic-syndromes.html

A Study of CTX-712 in Relapsed/?Refractory Acute ... The goal of this phase 1/2 multicenter, open-label, singe arm dose escalation and expansion study is to assess the safety, tolerability, ...