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
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).
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.
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
Men sexually active must use study specified contraception
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;
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
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