A Study of CTX-712 in Relapsed/Refractory Acute Myeloid Leukemia and Higher Risk Myelodysplastic Syndromes

NCT ID: NCT05732103

Last Updated: 2025-09-22

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

225 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-04-25

Study Completion Date

2029-02-28

Brief Summary

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The goal of this phase 1/2 multicenter, open-label, single-arm dose escalation and expansion study is to assess the safety, tolerability, pharmacokinetic and pharmacodynamic profile of CTX-712 in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) and higher risk myelodysplastic syndromes (HR-MDS), or MDS/MPN (including CMML).

The phase 1 part of the study consists of sequential standard 3 + 3 dose escalation, where patients will receive ascending doses of CTX-712 to determine the recommended phase 2 dose (RP2D) for further clinical development. This is followed by initial expansion cohorts in AML and/or HR-MDS where patients will be treated with CTX-712 at the RP2D to gain further confidence in the selected dose level. Additional expansion cohorts may be initiated if considered necessary. After RP2D is determined, Drug-Drug-Interaction cohorts will be started.

The phase 2 part of the study will commence after the RP2D has been identified and confirmed and will evaluate therapeutic activity in R/R AML or R/R HR-MDS, in addition to confirmation of the safety profile.

Detailed Description

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Conditions

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Acute Myeloid Leukemia Myelodysplastic Syndromes

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Dose Escalation Cohort

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

Group Type EXPERIMENTAL

CTX-712

Intervention Type DRUG

CTX-712 will be provided as a 20 mg tablet for oral administration. Patients will take CTX-712 once or twice weekly, depending on their dose level assignment, during each 28-day cycle.

Initial Expansion Cohort

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

Group Type EXPERIMENTAL

CTX-712

Intervention Type DRUG

CTX-712 will be provided as a 20 mg tablet for oral administration. Patients will take CTX-712 once or twice weekly, depending on their dose level assignment, during each 28-day cycle.

Phase 2

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

Group Type EXPERIMENTAL

CTX-712

Intervention Type DRUG

CTX-712 will be provided as a 20 mg tablet for oral administration. Patients will take CTX-712 once or twice weekly, depending on their dose level assignment, during each 28-day cycle.

Interventions

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CTX-712

CTX-712 will be provided as a 20 mg tablet for oral administration. Patients will take CTX-712 once or twice weekly, depending on their dose level assignment, during each 28-day cycle.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Age ≥18 years.
2. Diagnosis of AML, HR-MDS, or high marrow blast MDS/MPN (including CMML).
3. Prior treatment history must include 1-4 prior lines of therapy.
4. Adequate organ function evidenced by the following laboratory values:

Creatinine clearance (CL) ≥60 mL/min Total serum bilirubin \< 1.5 × upper limit of normal (ULN) Alanine aminotransferase (ALT) Aspartate aminotransferase(AST) \< 2.5 × ULN White blood cell count at the time of the first dose \<10 k/μL
5. Eastern Cooperative Oncology Group performance status ≤2.
6. Female patients of childbearing potential must have a negative pregnancy test within 7 days before study treatment initiation and if sexually active, agree to use a highly effective form of contraception throughout their participation during study treatment and up to 4 months after the last dose of study drug
7. Male patients with female partners of childbearing potential must, even if surgically sterilized, agree to practice effective barrier contraception during the entire study treatment period and through four months after the last dose of study drug, or practice true abstinence, when this is in line with the preferred and usual lifestyle of the participant.

Exclusion Criteria

1. Diagnosis of acute promyelocytic leukemia.
2. Isolated extramedullary relapse (phase 2 only).
3. Active central nervous system (CNS) leukemia.
4. History of other malignancy.
5. Any of the following cardiopulmonary abnormalities:

1. Myocardial infarction within six months prior to registration.
2. New York Heart Association Class III or IV heart failure or known left ventricular ejection fraction \< 50%.
3. A history of familial long QT syndrome.
4. Symptomatic atrial or ventricular arrhythmias not controlled by medications.
5. QTcF ≥ 470 msec calculated according to institutional guidelines, unless due to underlying bundle branch block and/or pacemaker and with approval of the medical monitor.
6. Known moderate to severe and clinically significant chronic obstructive pulmonary disease, interstitial lung disease and/or pulmonary fibrosis (e.g., requiring home oxygen therapy).
6. Pregnancy and/or lactation.
7. Major surgery (excluding placement of vascular access) within 4 weeks prior to first dose of CTX-712.
8. History of allogeneic organ transplantation (excluding cornea).
9. History of allogenic hematopoietic stem cell transplantation within 6 months of planned study treatment initiation and/or graft-versus host disease grade ≥ 1 following allogenic hematopoietic stem cell transplantation.
10. History of or chimeric antigen receptor T-cell therapy or other modified T cell therapy.
11. Active, uncontrolled bacterial, fungal, or viral infection, including hepatitis B virus, hepatitis C virus, known human immunodeficiency virus, or acquired immunodeficiency syndrome related illness. Infections controlled with oral anti-infective agents, including prophylactic treatments, are allowed. Patient must be viral load negative.
12. Psychological, familial, sociological, or geographical conditions that do not permit compliance with the protocol and/or follow-up procedures outlined in the protocol.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Theradex

INDUSTRY

Sponsor Role collaborator

Chordia Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Guillermo Garcia-Manero, MD

Role: PRINCIPAL_INVESTIGATOR

M.D. Anderson Cancer Center

Locations

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Mayo Clinic Arizona

Phoenix, Arizona, United States

Site Status RECRUITING

Mayo Clinic Florida

Jacksonville, Florida, United States

Site Status RECRUITING

Mayo Clinic Comprehensive Cancer Center

Rochester, Minnesota, United States

Site Status RECRUITING

The University of Texas MD Anderson Cancer Center

Houston, Texas, United States

Site Status RECRUITING

University of Virginia

Charlottesville, Virginia, United States

Site Status RECRUITING

Fred Hutchinson Cancer Center

Seattle, Washington, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Laurie F Graham, RN, MSN

Role: CONTACT

(609) 608-2152

Haris Durutlic

Role: CONTACT

(781) 560-4419

Facility Contacts

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Clinical Trials Referral Office

Role: primary

855-776-0015

Clinical Trials Referral Office

Role: primary

855-776-0015

Clinical Trials Referral Office

Role: primary

855-776-0015

Kristy Bodden

Role: primary

713-563-4412

Avani Hopkins

Role: primary

434-243-8108

Issac Miller

Role: primary

206-606-1894

References

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Yokoyama H, Fukuhara N, Ando K, Iida H, Yamauchi T, Fukuhara S, Izutsu K, Tanoue Y, Yamamoto M, Tozaki H, Takahara E, Shoji S, Mizutani A, Morishita D, Oda RW, Miyake H, Yamamoto N. Phase I Study of Rogocekib in Patients with Relapsed or Refractory Hematologic Malignancies. Blood Adv. 2025 Oct 7:bloodadvances.2025017601. doi: 10.1182/bloodadvances.2025017601. Online ahead of print.

Reference Type DERIVED
PMID: 41056522 (View on PubMed)

Other Identifiers

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CTX-712-CL-02

Identifier Type: OTHER

Identifier Source: secondary_id

C22-11236

Identifier Type: -

Identifier Source: org_study_id

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