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

Stopped early · Phase 1

Conditions studied: MDS, Myelodysplastic Syndromes

In brief

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

Key facts

Study ID

NCT04638309

Run by

Aprea Therapeutics

People needed

4

Starts

2021-09-20

Expected to finish

2022-04-25

Last updated by the study team

2025-03-10

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Provision of signed and dated, written informed consent prior to any study specific procedures.
• Documented diagnosis of TP53-mutant MDS, according to WHO criteria that is relapsed/refractory or previously untreated MDS.
• Adequate organ function as defined by the following laboratory values:
• Creatinine clearance ≥60 mL/min (by Cockcroft-Gault method, Appendix I),
• Total serum bilirubin ≤1.5 × upper limit of normal (ULN) unless due to Gilbert's syndrome or MDS organ involvement,
• Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤2.5 × ULN, unless due to MDS organ involvement.
• Age ≥18 years at the time of signing the informed consent form.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2 (Appendix II).
• Projected life expectancy of ≥12 weeks.
• Clear ocular media and adequate pupil dilation to permit fundus examination and retinal imaging.

You may not qualify if…

• Cardiac abnormalities, which includes, but not limited to:
• Myocardial infarction within six months prior to enrollment
• New York Heart Association Class III or IV heart failure or known LVEF <40%
• Concomitant malignancies or previous malignancies with less than a 1 year disease-free interval at the time of signing informed consent.
• 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.
• Prior exposure to eprenetapopt (APR-246).
• A female subject who is pregnant or breast-feeding.
• Known history of human immunodeficiency virus (HIV), active hepatitis B or active hepatitis C infection.
• Malabsorption syndrome or other condition likely to affect gastrointestinal absorption of APR-548.
• Known history or current evidence of ocular disease in either eye

Where it is running

• H. Lee Moffitt Cancer Center — Tampa, Florida, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Dana Farber Cancer Institue — Boston, Massachusetts, United States
• MD Anderson Cancer Center — Houston, Texas, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.