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Clinical Trial 21128

Cancer Type: Malignant Hematology
Study Type: Treatment
NCT#: NCT04638309

Phase: Phase I
Prinicipal Investigator: Sallman, David

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Overview

Study Title

Phase 1 Study to Evaluate Safety and Efficacy of APR-548 in Combination with Azacitidine for the Treatment of TP53-Mutant Myelodysplastic Syndromes

Summary

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

Objective

Part A: 1. To investigate the PK, safety and tolerability of APR-548 as monotherapy and in combination with azacitidine and determine the RP2D of APR-548 in subjects with TP53-mutant MDS. Part B: 1. To determine the complete remission (CR) rate, defined as the proportion of patients who achieve CR with APR-548 and azacitidine using modified IWG criteria (2006; Appendix V) Secondary objectives: 1. To assess preliminary clinical activity of APR-548 administered in combination with azacitidine - a. Duration of CR b. Overall response rate (ORR) c. Duration of ORR d. Rate of and time to development of acute myeloid leukemia (AML), as defined World Health Organization (WHO) criteria e. Overall survival (OS) f. Relapse-free survival (RFS) g. Rate of transition to hematopoietic stem cell transplant (HSCT) h. Rate of red blood cell and/or platelet transfusion independence for 8 weeks 2. To characterize PK of APR-548 after single and multiple dosing when given p.o.; 3. To evaluate the PK drug-drug interaction between APR-548 and azacitidine.

Treatments

Therapies

Therapy (NOS)

Medications

APR-548 (); Azacitidine (Oral) (); CC-486 (Azacitidine (Oral))

Inclusion Criteria

  • 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 protocol
  • Age >18 years at the time of signing the informed consent form
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2
  • Projected life expectancy of >12 weeks
  • Clear ocular media and adequate pupil dilation to permit fundus examination and retinal imaging

  • Exclusion Criteria

  • 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 > 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

  • If you are interested in learning more about clinical trials, our clinical trial navigators can discuss your options and recommend opportunities that may be suitable for you. Call 813-745-6100 or 1-800-679-0775 (toll-free) or submit a clinical trials inquiry form.