APR-548 in Combination With Azacitidine for the Treatment of TP53 Myelodysplastic Syndromes (MDS)

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1

Total Enrollment

4 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-09-20

Study Completion Date

2022-04-25

Brief Summary

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Phase 1 study evaluating the safety and efficacy of APR-548 in combination with Azacitidine for the treatment of TP53-Mutant Myelodysplastic Syndromes.

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Detailed Description

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Open-label first-in-human (FIH) phase 1 clinical trial assessing the safety, pharmacokinetics (PK), and clinical activity of orally (p.o.) administered APR-548 alone and in combination with azacitidine for the treatment of TP53-mutant myelodysplastic syndromes (MDS).

Conditions

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MDS Myelodysplastic Syndromes

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1

Dose level 1

Group Type EXPERIMENTAL

APR-548 + Azacitidine

Intervention Type DRUG

APR-548 monotherapy period followed by APR-548 in combination with Azacitidine

Cohort 2

Dose level 2

Group Type EXPERIMENTAL

APR-548 + Azacitidine

Intervention Type DRUG

APR-548 monotherapy period followed by APR-548 in combination with Azacitidine

Cohort 3

Dose level 3

Group Type EXPERIMENTAL

APR-548 + Azacitidine

Intervention Type DRUG

APR-548 monotherapy period followed by APR-548 in combination with Azacitidine

Interventions

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APR-548 + Azacitidine

APR-548 monotherapy period followed by APR-548 in combination with Azacitidine

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Provision of signed and dated, written informed consent prior to any study specific procedures.
2. Documented diagnosis of TP53-mutant MDS, according to WHO criteria that is relapsed/refractory or previously untreated MDS.
3. Adequate organ function as defined by the following laboratory values:

1. Creatinine clearance ≥60 mL/min (by Cockcroft-Gault method, Appendix I),
2. Total serum bilirubin ≤1.5 × upper limit of normal (ULN) unless due to Gilbert's syndrome or MDS organ involvement,
3. Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤2.5 × ULN, unless due to MDS organ involvement.
4. Age ≥18 years at the time of signing the informed consent form.
5. Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2 (Appendix II).
6. Projected life expectancy of ≥12 weeks.
7. Clear ocular media and adequate pupil dilation to permit fundus examination and retinal imaging.

Exclusion Criteria

1. Cardiac abnormalities, which includes, but not limited to:

1. Myocardial infarction within six months prior to enrollment
2. New York Heart Association Class III or IV heart failure or known LVEF \<40%
2. Concomitant malignancies or previous malignancies with less than a 1 year disease-free interval at the time of signing informed consent.
3. Use of cytotoxic chemotherapeutic agents, or experimental agents for the treatment of MDS within 14 days or 5 half-lives of the product (whichever is shorter) of the first day of study drug treatment.
4. Prior exposure to eprenetapopt (APR-246).
5. A female subject who is pregnant or breast-feeding.
6. Known history of human immunodeficiency virus (HIV), active hepatitis B or active hepatitis C infection.
7. Malabsorption syndrome or other condition likely to affect gastrointestinal absorption of APR-548.
8. Known history or current evidence of ocular disease in either eye

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No