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Protocol No. UW17079

Principal Investigator Mattison, Ryan

Phase II

Age Group Adult

Scope National

Sponsor Type Industry

Title A Two-Stage, Open-Label Phase 2 Study of Pracinostat and Azacitidine in Patients with IPSS-R High and Very High Risk Myelodysplastic Syndromes Previously Untreated with Hypomethylating Agents

Objective The purpose of this research study is to learn how pracinostat works, including how safe it is when given with azacitidine. An investigational drug called pracinostat is being tested as an addition to Vidaza (azacitidine). Since pracinostat is investigational, it is only available to people taking part in a clinical research study.

Treatment Azacitidine is a standard treatment for MDS. Azacitidine is approved by the US Food and Drug Administration (FDA). Pracinstat is an investigational drug that is not approved by the FDA. Pracinostat has been shown in the laboratory to kill cancer cells. Animal studies have shown that pracinostat is active against many cancer cell types. Pracinostat has been tested in more than 460 humans. Different amounts of pracinostat have been given in previous research studies to subjects (participants) who have cancer and blood diseases.

Description This study has two stages. In the first stage (Stage 1), all research subjects will receive pracinostat combined with azacitidine. In the second stage (Stage 2), which begins after the first stage is completed, subjects will be randomized (assigned by chance, like flipping a coin) to receive pracinostat combined with azacitidine, or placebo (medicine free pills) combined with azacitidine. Azacitidine is given by injection under the skin, or by infusion into a vein. Pracinostat is taken by mouth.

Key Eligibility
  • Female or male subjects ≥18 years-of-age
  • Histologically or cytologically documented diagnosis of MDS according to the World Health Organization (WHO) classification
  • Classified as high or very high risk according to the Revised International Prognostic Scoring System
  • Bone marrow biopsy (BMBx) within 28 days prior to first study treatment
  • Clinical indication for treatment with azacitidine
  • Previously untreated with HMAs (prior therapy with transfusions, hematopoietic growth factors, or immunosuppressive therapy is allowed)
  • If patient has bone marrow blasts ≥20%, indicating a diagnosis of acute myeloid leukemia (AML), they are excluded
  • If patient has had major surgery within 28 days prior to first study treatment they are excluded
  • If patient has had prior treatment for MDS with the histone deacetylase (HDAC) inhibitors Zolinza (vorinostat), Belenodaq (belinostat), Farydak (panobinostat), Istodax (romidepsin/depsipetide), or investigational agent with significant action as an HDAC inhibitor, they are excluded
  • If patient has clinical evidence of central nervous system involvement, they are excluded
  • If patient has uncontrolled infection with human immunodeficiency virus (HIV) or chronic hepatitis B or C, they are excluded

Applicable Disease Sites Hematologic cancers, other

Therapies Involved Cytotoxic Chemotherapy

Drugs Involved 5-AC (azacitidine); 5-AZA (azacitidine); 5-azacitidine (azacitidine); Pracinostat; azacitidine; placebo

Status Open

Participating Institutions UW Hospital and Clinics; UWCCC 1 South Park