Phase 1 Study of AUTX-703 in Relapsed/Refractory AML and MDS

NCT ID: NCT06846606

Last Updated: 2025-11-18

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

Total Enrollment

69 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-05-01

Study Completion Date

2028-06-30

Brief Summary

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This Phase 1, multicenter, open-label, dose escalation and dose optimization study is designed to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of AUTX-703 administered orally in subjects with advanced hematologic malignancies.

Detailed Description

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This is a first-in-human, Phase 1, multicenter study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of AUTX-703, an orally bioavailable lysine acetyltransferase 2A (KAT2A) and lysine acetyltransferase 2B (KAT2B) degrader, in participants with relapsed/refractory acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). The study consists of two parts: Part A (Dose Escalation) to determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D), and Part B (Dose Optimization) to further evaluate safety, PK, PD and efficacy at selected dosages.

Conditions

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Relapsed Acute Myeloid Leukemia (AML) Refractory Acute Myeloid Leukemia (AML) Relapsed/Refractory AML Relapsed Myelodysplastic Syndromes Refractory Myelodysplastic Syndromes Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML)

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

This study consists of two parts: Part A (Dose Escalation) and Part B (Dose Optimization). In Part A, participants are assigned sequentially to escalating dosages of AUTX-703 to determine the maximum tolerated dose (MTD) and/or the recommended Phase 2 dose (RP2D). In Part B, participants are randomly assigned to receive one of two dosages of AUTX-703 for safety, pharmacokinetics, pharmacodynamics, and preliminary clinical activity.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Dose Escalation - Part A

Participants will receive escalating dosages of AUTX-703 orally in tablet form once, twice or three times weekly to determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D).

Group Type EXPERIMENTAL

AUTX-703

Intervention Type DRUG

AUTX-703 administered orally

Dose Optimization - Part B, Dosage 1

Participants will receive AUTX-703 at the first selected dosage determined from Part A, administered orally in tablet form either once, twice or three times weekly to further evaluate safety, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity at this specified dose.

Group Type EXPERIMENTAL

AUTX-703

Intervention Type DRUG

AUTX-703 administered orally

Dose Optimization - Part B, Dosage 2

Participants will receive AUTX-703 at the second selected dosage determined from Part A, administered orally in tablet form either once, twice or three times weekly to further evaluate safety, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity at this specified dose.

Group Type EXPERIMENTAL

AUTX-703

Intervention Type DRUG

AUTX-703 administered orally

Interventions

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AUTX-703

AUTX-703 administered orally

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Participant must be ≥18 years of age
2. Participant must have confirmed diagnosis as follows:

R/R AML and has not achieved adequate response to, cannot tolerate, or refused all approved therapies known to be active for treatment of their disease OR R/R MDS with over 10% blasts in the bone marrow and has not achieved an adequate response to at least 4 cycles of a hypomethylating agent (HMA)- containing regimen or other treatment known to be active for their disease OR R/R AML or R/R MDS that has relapsed after a hematopoietic stem cell transplant (HSCT)
3. Participant must be willing and able to comply with scheduled study visits and treatment plans.
4. Participant must be willing to undergo all study procedures unless contraindicated due to medical risk.
5. Participant must have an Eastern Cooperative Oncology Group (ECOG) performance status (PS) of ≤2
6. Participant must have adequate hepatic function
7. Participant must have adequate renal function
8. Participant must have adequate cardiovascular function
9. Participant must have a white blood cell (WBC) count ≤20 × 10⁹/L (with stable hydroxyurea use allowed)
10. Participant must meet timing requirements with respect to prior therapy and surgery
11. Participant must agree to use effective contraception during the study and for the required post-treatment period: Males: Use condoms (even if vasectomized) during the study and for 90 days post-treatment. Females of childbearing potential: Use a combination of 1 highly effective and 1 effective method of contraception during the study and for 180 days post-treatment.

Exclusion Criteria

1. Participant is unable to provide informed consent and/or to follow protocol requirements.
2. Participant has undergone chimeric antigen receptor T cell therapy or HSCT within 60 days of the first dose of study treatment or has active clinically significant graft-versus-host disease (GVHD)
3. Participant has another malignancy that may interfere with diagnosis and treatment of R/R AML or R/R MDS.
4. Participant has an active severe infection that requires anti-infective therapy or has an unexplained temperature of \>38.5°C during screening visits or on their first day of study treatment.
5. Participant has a known sensitivity to AUTX-703 or any of its components.
6. Participant is taking systemic strong CYP3A4 inhibitors or inducers within 14 days of the first dose of study treatment.
7. Participant who are taking proton pump inhibitors should be switched to another acid-reducing agent such as an antacid or H2 blocker
8. Participant is taking P-gp and breast cancer resistance protein (BCRP) inhibitors or inducers within 14 days of first dose of study treatment.
9. Participant has active hepatitis B virus (HBV) or hepatitis C virus (HCV) infections with detectable viral load
10. Participant has experienced AIDS related illness within the past 6 months or have detectable HIV viral load.
11. Participant has an uncontrolled intercurrent illness
12. Participant has active Class III or IV cardiovascular disease within 6 months prior to the start of study treatment
13. Participant is unable to tolerate the administration of oral medication or has GI dysfunction that would preclude adequate absorption, distribution, metabolism, or excretion of an oral medication
14. Participant is pregnant or breastfeeding or is planning to become pregnant within 1 year of the start of study treatment
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Auron Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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City of Hope National Medical Center

Duarte, California, United States

Site Status RECRUITING

H Lee Moffitt Cancer Center and Research Institute

Tampa, Florida, United States

Site Status RECRUITING

Roswell Park Comprehensive Cancer Center

Buffalo, New York, United States

Site Status RECRUITING

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Site Status RECRUITING

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Site Status RECRUITING

Ohio State University, The James Comprehensive Cancer

Columbus, Ohio, United States

Site Status RECRUITING

UPENN Perelman Center for Advanced Medicine

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Sarah Cannon Center for Blood Cancer at TriStar Centennia

Nashville, Tennessee, United States

Site Status RECRUITING

University of Texas MD Anderson Cancer Center

Houston, Texas, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Auron Clinical Trials

Role: CONTACT

617-514-2356

Other Identifiers

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AUTX-703-001

Identifier Type: -

Identifier Source: org_study_id

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