Phase 2 Study Adding Pracinostat to a Hypomethylating Agent (HMA) in Patients With MDS Who Failed to Respond to Single Agent HMA

NCT ID: NCT01993641

Last Updated: 2017-02-23

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

45 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-12-31

Study Completion Date

2016-06-30

Brief Summary

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The purpose of this open label study is to determine whether combining pracinostat (study drug) with Vidaza (azacitidine) or Dacogen (decitabine) will improve clinical responses in Myelodysplastic Syndrome (MDS) patients who have failed an initial single agent hypomethylating agent (HMA), and to provide additional safety and efficacy data.

Detailed Description

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Conditions

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Myelodysplastic Syndrome MDS

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Pracinostat added to HMA

Pracinostat in combination with HMA treatment (either azacitidine or decitabine) used in initial single agent treatment for that patient

Group Type EXPERIMENTAL

pracinostat

Intervention Type DRUG

Histone deacetylase inhibitor (HDACi) Pracinostat is to be taken before HMA administration 3 times/week (e.g., Monday, Wednesday, and Friday) for 3 weeks, followed by 1 week of rest as a 28-day cycle. Pracinostat administration will be at the clinic on Day 1 of Cycles 1 and 2 and subject will self-administer at home on all other days

Azacitidine

Intervention Type DRUG

All patients will receive the dose and schedule of azacitadine to which they previously failed to respond. (e.g. 75 mg/m2 via subcutaneous (SC) injection or intravenous infusion if SC injections are intolerable; 7 days of each 28 day cycle, either Days 1-7, or Days 1-5, rest on Days 6-7, and azacitadine dosing on Days 8-9)

Decitabine

Intervention Type DRUG

All patients will receive the dose and schedule of decitabine to which they previously failed to respond. Common 28 day treatment regimens include: 20 mg/m2 IV for either 5 or 10 days of each 28-day cycle, 10 mg/m2 given intravenously daily for first 10 days of each 28 day cycle, or 20 mg/m2 given subcutaneously daily for the first 5 days of each 28 day cycle.

The 6-week regimen utilizes a dose of 15 mg/m2 by continuous intravenous infusion over 3 hours repeated every 8 hours for 3 days repeated every 6 weeks.

Interventions

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pracinostat

Histone deacetylase inhibitor (HDACi) Pracinostat is to be taken before HMA administration 3 times/week (e.g., Monday, Wednesday, and Friday) for 3 weeks, followed by 1 week of rest as a 28-day cycle. Pracinostat administration will be at the clinic on Day 1 of Cycles 1 and 2 and subject will self-administer at home on all other days

Intervention Type DRUG

Azacitidine

All patients will receive the dose and schedule of azacitadine to which they previously failed to respond. (e.g. 75 mg/m2 via subcutaneous (SC) injection or intravenous infusion if SC injections are intolerable; 7 days of each 28 day cycle, either Days 1-7, or Days 1-5, rest on Days 6-7, and azacitadine dosing on Days 8-9)

Intervention Type DRUG

Decitabine

All patients will receive the dose and schedule of decitabine to which they previously failed to respond. Common 28 day treatment regimens include: 20 mg/m2 IV for either 5 or 10 days of each 28-day cycle, 10 mg/m2 given intravenously daily for first 10 days of each 28 day cycle, or 20 mg/m2 given subcutaneously daily for the first 5 days of each 28 day cycle.

The 6-week regimen utilizes a dose of 15 mg/m2 by continuous intravenous infusion over 3 hours repeated every 8 hours for 3 days repeated every 6 weeks.

Intervention Type DRUG

Other Intervention Names

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SB939 Vidaza Dacogen

Eligibility Criteria

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Inclusion Criteria

1. Voluntary written informed consent
2. Histologically or cytologically documented diagnosis of MDS (any French-American-British classification \[FAB\] subtype)
3. Bone marrow blasts \>5% and \<30% and a peripheral white blood cell (WBC) count of \<20,000 /µL
4. Bone marrow biopsy, aspirates, and peripheral blood smears within 28 days of first study treatment
5. Group 1:

Primary failures: Progression after their most recent HMA therapy according to IWG criteria after receiving single agent azacitidine and/or single agent decitabine, or has worsening cytopenias (increased transfusion requirement), increased BM blasts, progression to a higher FAB type, or develops additional clinically significant cytogenetic abnormalities; Secondary failures: Relapse after any initial CR, PR, HI, or development of clinically significant cytogenetic abnormalities at any time according to IWG criteria after receiving single agent azacitidine or decitabine

Group 2:

Failure to achieve a response (any CR, PR or HI) according to IWG criteria definition of stable disease after the most recent HMA therapy (at least 6 cycles of azacitidine or 4 cycles of decitabine)
6. Must have demonstrated tolerability to single agent HMA
7. Able to start combination therapy within 3 months of the last single agent HMA dose with no other therapy for disease under study received during this interval
8. Not a candidate for hematopoietic stem cell transplant within 4 months of screening
9. ECOG performance status of 0, 1, or 2
10. Adequate organ function as evidenced by:

* Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤2.5 x the upper limit of normal (ULN)
* Total bilirubin ≤1.5 x ULN or total bilirubin of ≤2 mg/dL, whichever is higher
* Serum creatinine \<2 mg/dL, or creatinine clearance ≥60 mL/min
* QTcF interval ≤470 msec
11. Female or male patients ≥18 years-of-age
12. Male patients with female partners are required to use two forms of acceptable contraception; Female patients of childbearing potential must have a negative pregnancy test ≤7 days before first study treatment.
13. Willingness and ability to understand the nature of this trial and to comply

Exclusion Criteria

1. Received any of the following within the specified time frame after the last single agent HMA dose until the first administration of study medication:

* Any therapy for malignancy between the time of single agent HMA and first on-study treatment
* Hydroxyurea within 48 hours prior to first study treatment
* Hematopoietic growth factors: erythropoietin, granulocyte colony stimulating factor (G-CSF), granulocyte macrophage colony stimulating factor (GM-CSF), or thrombopoietin receptor agonists within 7 days (14 days for Aranesp) prior to first study treatment
* Major surgery within 28 days of study day 1
2. Patients who are candidates for aggressive chemotherapy (e.g. typical AML induction therapy)
3. Cardiopulmonary function criteria:

* Current unstable arrhythmia requiring treatment
* History of symptomatic congestive heart failure (New York Heart Association Class III or IV)
* History of myocardial infarction within 6 months of enrollment
* Current unstable angina
4. Concomitant treatment with agents that have activity against HDAC inhibitors is not permitted
5. Clinical evidence of CNS involvement
6. Patients with gastrointestinal (GI) tract disease, uncontrolled inflammatory GI disease (e.g., Crohn's disease, ulcerative colitis)
7. Active infection with human immunodeficiency virus or chronic hepatitis B or C
8. Life-threatening illness unrelated to cancer or any serious medical or psychiatric illness that could potentially interfere with participation in this study
9. Presence of a malignant disease within the last 12 months, with the exception of adequately treated in-situ carcinomas, basal or squamous cell carcinoma, or non-melanomatous skin cancer and other concurrent malignancies will be considered on a case by case basis
10. Inability or unwillingness (including psychological, familial, sociological, or geographical conditions) to comply
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Helsinn Healthcare SA

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Guillermo Garcia-Manero, MD

Role: PRINCIPAL_INVESTIGATOR

M.D. Anderson Cancer Center

Locations

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Southern Cancer Center

Mobile, Alabama, United States

Site Status

City of Hope

Duarte, California, United States

Site Status

USC Norris Comprehensive Cancer Center

Los Angeles, California, United States

Site Status

Sutter Medical Group

Sacramento, California, United States

Site Status

Colorado Blood Cancer Institute

Denver, Colorado, United States

Site Status

Yale School of Medicine

New Haven, Connecticut, United States

Site Status

Florida Cancer Specialist South

Fort Myers, Florida, United States

Site Status

Florida Cancer Specialist North

St. Petersburg, Florida, United States

Site Status

Northwestern University

Chicago, Illinois, United States

Site Status

University of Kansas Cancer Center

Westwood, Kansas, United States

Site Status

University of Kentucky

Lexington, Kentucky, United States

Site Status

John Theurer Cancer Center

Hackensak, New Jersey, United States

Site Status

Oncology Hematology Care

Cincinati, Ohio, United States

Site Status

Cleveland Clinic

Cleveland, Ohio, United States

Site Status

University of Oklahoma Health Science Center

Oklahoma City, Oklahoma, United States

Site Status

Tennessee Oncology-Chattanooga

Chattanooga, Tennessee, United States

Site Status

Tennessee Oncology

Nashville, Tennessee, United States

Site Status

Baylor University Medical Center

Dallas, Texas, United States

Site Status

University of Texas Southwestern

Dallas, Texas, United States

Site Status

MD Anderson Cancer Center

Houston, Texas, United States

Site Status

Cancer Care Centers of South Texas

San Antonio, Texas, United States

Site Status

Countries

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United States

Related Links

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http://MEIPharma.com

Sponsor website

Other Identifiers

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MEI-005

Identifier Type: -

Identifier Source: org_study_id

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