Pharmacokinetic Guided Dose Escalation and Dose Confirmation With Oral Decitabine and Oral Cytidine Deaminase Inhibitor (CDAi) in Patients With Myelodysplastic Syndromes (MDS)

NCT ID: NCT02103478

Last Updated: 2024-08-27

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

130 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-10-28

Study Completion Date

2019-12-04

Brief Summary

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This first-in-human, 3-stage, open-label study evaluated the safety and pharmacokinetics of ASTX727, as well as determined the dose for later stages.

Detailed Description

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The trial was designed to define daily doses of the individual components (cedazuridine \[E7727\] or decitabine) so that decitabine exposure after oral administration would be comparable to exposure after IV decitabine at the approved daily dose of 20 mg/m\^2. The main objective of Phases 1 and 2 was to establish and confirm the doses of the 2 components to be used in the final fixed-dose combination (FDC) product (ASTX727) using mainly pharmacokinetics and pharmacodynamics as endpoints.

Conditions

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Myelodysplastic Syndrome MDS

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Phase 1 Dose Escalation

Starting cohort was administered 40 mg oral cedazuridine and 20 mg oral decitabine. Participants were enrolled into successive cohorts in which either the cedazuridine or decitabine oral dose was varied in Course 1 Day 2 through Course 1 Day 5 for comparison with a single dose of IV decitabine at 20 mg/m\^2 administered on Day 1 by continuous IV infusion over 1 hour (28 days per course).

Group Type EXPERIMENTAL

ASTX727 Dose Escalation

Intervention Type DRUG

Oral investigational product and approved IV decitabine

Phase 2 Dose Confirmation

Participants were randomized in a 1:1 ratio to receive either oral cedazuridine (E7727) (100 mg) + decitabine (35 mg) capsules Dailyx5 in Course 1 followed by IV decitabine (20 mg/m\^2) Dailyx5 in Course 2 (28 days per course) or the converse. In Courses ≥ 3, participants received cedazuridine and decitabine capsules Dailyx5 in 28-day courses until disease progression, unacceptable toxicity, withdrawal of consent or withdrawal from the study.

Group Type EXPERIMENTAL

ASTX727 Dose Confirmation

Intervention Type DRUG

Randomization cross over design for courses 1 and 2

Phase 2 Fixed-Dose Combination

Participants were randomized in a 1:1 ratio to receive either the fixed-dose combination (FDC) tablet (100 mg cedazuridine (E7727)/35 mg decitabine) Dailyx5 in Course 1 followed by IV decitabine (20 mg/m\^2) Dailyx5 in Course 2 (28 days per course) or the converse. In Courses ≥ 3, all participants received the FDC tablet Dailyx5 in 28-day courses until disease progression, unacceptable toxicity, withdrawal of consent or withdrawal from the study.

Group Type EXPERIMENTAL

ASTX727 Fixed-Dose Combination

Intervention Type DRUG

Fixed-dose investigational product

Interventions

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ASTX727 Dose Escalation

Oral investigational product and approved IV decitabine

Intervention Type DRUG

ASTX727 Dose Confirmation

Randomization cross over design for courses 1 and 2

Intervention Type DRUG

ASTX727 Fixed-Dose Combination

Fixed-dose investigational product

Intervention Type DRUG

Other Intervention Names

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cedazuridine (E7727) oral decitabine IV decitabine ASTX727 oral (combination of oral E7727 and oral decitabine) IV decitabine ASTX727 oral (combination of oral E7727 and oral decitabine)

Eligibility Criteria

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Inclusion Criteria

* International Prognostic Scoring System (IPSS) low, intermediate -1, intermediate-2, or high risk MDS (including chronic myelomonocytic leukemia; CMML) in Dose Escalation and Dose Confirmation-Randomization; only intermediate-2, or high risk MDS in Dose Confirmation-Open Label
* Eastern Cooperative Oncology Group (ECOG) 0 to 2
* No major surgery within 2 weeks of starting study treatment
* No cytotoxic chemotherapy within 2 weeks of starting study treatment
* Able to swallow pills

Exclusion Criteria

* Previous treatment with 2 or more courses of decitabine (all stages) or azacitidine (Dose Confirmation stage only)
* Treatment with investigational therapy within 2 weeks of study treatment
* Uncontrolled medical disease(s) or active, uncontrolled infection
* Diagnosed with acute myeloid leukemia (AML)
* Active uncontrolled gastric or duodenal ulcer
* Known history of HIV or hepatitis C or B
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Astex Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Mohammad Azab, MD

Role: STUDY_DIRECTOR

Astex Pharmaceuticals, Inc.

James Lowder, MD

Role: STUDY_CHAIR

Astex Pharmaceuticals, Inc.

Locations

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Mayo Clinic

Phoenix, Arizona, United States

Site Status

University of Southern California

Los Angeles, California, United States

Site Status

University of Chicago

Chicago, Illinois, United States

Site Status

Horizon Oncology

Lafayette, Indiana, United States

Site Status

Johns Hopkins

Baltimore, Maryland, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Dana Farber Cancer Institute

Boston, Massachusetts, United States

Site Status

John Theurer Cancer Center/ Hackensack University Medical Center

Hackensack, New Jersey, United States

Site Status

Roswell Park Cancer Institute

Buffalo, New York, United States

Site Status

Weill Cornell Medical College - New York Presbyterian Hospital

New York, New York, United States

Site Status

Vanderbilt Ingram Cancer Center

Nashville, Tennessee, United States

Site Status

M. D. Anderson

Houston, Texas, United States

Site Status

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

University of Alberta Hospital

Edmonton, Alberta, Canada

Site Status

Sunnybrook Health Sciences Centre, Odette Cancer Centre

Toronto, Ontario, Canada

Site Status

Princess Margaret Cancer Center

Toronto, Ontario, Canada

Site Status

Hôpital Maisonneuve-Rosemont

Montreal, Quebec, Canada

Site Status

Countries

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United States Canada

References

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Garcia-Manero G, Griffiths EA, Steensma DP, Roboz GJ, Wells R, McCloskey J, Odenike O, DeZern AE, Yee K, Busque L, O'Connell C, Michaelis LC, Brandwein J, Kantarjian H, Oganesian A, Azab M, Savona MR. Oral cedazuridine/decitabine for MDS and CMML: a phase 2 pharmacokinetic/pharmacodynamic randomized crossover study. Blood. 2020 Aug 6;136(6):674-683. doi: 10.1182/blood.2019004143.

Reference Type DERIVED
PMID: 32285126 (View on PubMed)

Savona MR, Odenike O, Amrein PC, Steensma DP, DeZern AE, Michaelis LC, Faderl S, Harb W, Kantarjian H, Lowder J, Oganesian A, Azab M, Garcia-Manero G. An oral fixed-dose combination of decitabine and cedazuridine in myelodysplastic syndromes: a multicentre, open-label, dose-escalation, phase 1 study. Lancet Haematol. 2019 Apr;6(4):e194-e203. doi: 10.1016/S2352-3026(19)30030-4.

Reference Type DERIVED
PMID: 30926081 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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ASTX727-01

Identifier Type: -

Identifier Source: org_study_id

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