Clinical Trial 19658

• Overview

  Study Title:

  A Phase 1, Open-Label, Dose-Escalation with Expansion Study of SX-682 in Subjects with Myelodysplastic Syndrome Who Had Disease Progression or are Intolerant to Prior Therapy

  Summary:

  This study will determine the safety profile, maximum tolerated dose (MTD), dose-limiting toxicities (DLT), and recommended Phase 2 dose (RP2D) of SX-682 in the treatment of patients with Myelodysplastic Syndromes (MDS).

  Objective:

  The primary objective is to determine the safety profile of SX-682 in subjects with MDS, including the maximum dose that can be administered until adverse effects prevent further dose increases (i.e., the MTD or recommended phase 2 dose), and the dose-limiting toxicity (DLT). The secondary objectives are to: 1. Estimate the overall best response rates of SX-682 using standard international working group (IWG) 2006 response criteria. 2. Determine the duration of response. 3. Determine the rate and time to acute myeloid leukemia (AML) transformation. 4. Determine the median overall survival (OS). 5. Characterize single-dose and multi-dose pharmacokinetic (PK) profile of SX-682 and its metabolite M1 at Day 1 and 15 of Cycle 1, respectively. For the 100 mg dose, additionally determine the effect of food at Day -3.

• Treatments

  Therapies:

  Chemotherapy (NOS); Therapy (NOS)

  Medications:

  Cedazuridine (); SX-682 (); decitabine (5-aza-2'-deoxycytidine)

• Inclusion Criteria

  • Diagnosis of MDS by World Health Organization criteria, and either International Prognostic Scoring System (IPSS) low risk or intermediate-1 risk without 5q deletion and failed treatment (no response, loss of response, progressive disease/treatment intolerance) following: 4 cycles hypomethylating agent; or ii. 4 cycles hypomethylating agent, or lenalidomide or erythropoietin stimulating agent (ESA)
  • IPSS low risk or intermediate-1 risk with 5q deletion and failed treatment following: 4 cycles of lenalidomide and hypomethylating agent; or ii. 4 cycles of lenalidomide
  • IPSS intermediate-2 risk or high risk and failed treatment following 4 cycles hypomethylating agent
  • Eastern Cooperative Oncology Group (ECOG) Performance Status ≤ 2
  • Screening laboratory values per protocol
  • No history of HIV being HIV positive
  • No active Hepatitis B or Hepatitis C infection
  • Life expectancy > 12 weeks
  • Women of childbearing potential must use study specified contraception
  • Women of childbearing potential demonstrate negative pregnancy test
  • Not breastfeeding
  • Men sexually active must use study specified contraception

• Exclusion Criteria

  • Use of chemotherapeutic agents or experimental agents for MDS within 14 days of the first day of study drug treatment
  • Use of erythroid stimulating agents, Granulocyte-colony stimulating factor (G-CSF), or Granulocyte-macrophage colony-stimulating factor (GM-CSF) within 14 days of the first day of study drug treatment, or during the study
  • Any of the following cardiac abnormalities:
  • QT interval > 480 msec corrected using Fridericia's formula;
  • Risk factors for Torsade de Pointes;
  • Use of concomitant medication that prolongs the QT interval with the exception of drugs that are considered absolutely essential for the care of the participant;
  • Myocardial infarction > Unstable angina pectoris or serious uncontrolled cardiac arrhythmia
  • Any serious or uncontrolled medical disorder
  • Prior malignancy within the previous 3 years except for local cancers that have been cured
  • Within 14 days of the first study drug treatment requiring systemic treatment with either corticosteroids or immunosuppressive medications. Corticosteroid adrenal replacement doses are permitted in the absence of active autoimmune disease
  • Use of other investigational drugs within 30 days of study drug administration
  • Major surgery within 4 weeks of study drug administration
  • Live-virus vaccination within 30 days of study drug administration
  • Allergy to study drug component

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