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Safety and Efficacy of Panobinostat in Patients With Primary Myelofibrosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00931762
Recruitment Status : Completed
First Posted : July 2, 2009
Last Update Posted : November 18, 2016
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Brief Summary:
This study will assess the safety and efficacy of Panobinostat as a single agent in the treatment of Primary Myelofibrosis, Post-Polycythemia Vera and Post-Essential Thrombocythemia. There will be two cohorts - patients with JAK2 mutation and patients without JAK2 mutation.

Condition or disease Intervention/treatment Phase
Primary Myelofibrosis Post-Polycythemia Vera Post-Essential Thrombocytopenia Drug: Panobinostat Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 35 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Trial of Oral Panobinostat (LBH589), a Novel Deacetylase Inhibitor (DACi) in Patients With Primary Myelofibrosis (PMF), Post Essential Thrombocythemia (ET) Myelofibrosis and Post- Polycythemia Vera (PV) Myelofibrosis
Study Start Date : July 2009
Actual Primary Completion Date : August 2011
Actual Study Completion Date : August 2011

Arm Intervention/treatment
Experimental: Panobinostat
Panobinostat will be administered orally once a day on Monday, Wednesday and Friday at a fixed dose of 40 mg.
Drug: Panobinostat
Panobinostat will be administered orally once a day on Monday, Wednesday and Friday at a fixed dose of 40 mg. A cycle is defined as 28 days of treatment.
Other Names:
  • LBH589
  • H-DAC Inhibitor

Primary Outcome Measures :
  1. To evaluate the overall response (CR, PR, and clinical improvement) to oral panobinostat as a single agent at 40 mg daily every Monday, Wednesday and Friday in patients with myelofibrosis. [ Time Frame: Upon enrollment of 13 participants into each cohort of the study and at the end of the study. ]

Secondary Outcome Measures :
  1. To compare the response to panobinostat in patients with the JAK2 V617F mutation to those without the JAK2 V617F mutation [ Time Frame: Upon enrollment of 13 participants into the study and at the end of the study ]
  2. To evaluate the symptomatic improvement of myelofibrosis patients treated with panobinostat using the Myelofibrosis Symptom Assessment Form (MF-SAF) at baseline and after 2 and 4 months of treatment [ Time Frame: Upon enrollment of 13 participants in each cohort and at the end of the study ]
  3. To evaluate the symptomatic improvement of myelofibrosis patients treated with panobinostat using the Myelofibrosis Symptom Assessment Form (MF-SAF) at baseline and after 2 and 4 months of treatment [ Time Frame: throughout the study ]
  4. To assess compliance to panobinostat treatment as assessed by monthly capsule counts [ Time Frame: at the end of the study ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

1. Diagnosis of myelofibrosis, either PMF, post-PV or post-ET MF with IPSS score of 2 (intermediate risk) or 3 (high risk) plus at least one of the following: Symptomatic spenomegaly (≥10cm BCM) Hemoglobin < 10 or red cell transfusion dependent. (The presence of a JAK2 V617F mutation is not required for study entry) 2. Patients must meet the following laboratory criteria:

  • Patients can be either JAK2 V617F mutated or wild type
  • Serum potassium, magnesium, phosphorous, sodium, total calcium (corrected for serum albumin) or ionized calcium within normal limits (WNL) for the institution Note: Potassium, magnesium, phosphorous, sodium, and/or calcium supplements maybe given to correct values that are < LLN. Post correction values must not be deemed to be a clinically significant abnormality prior to patients being dosed.
  • Creatinine < 1.5 X ULN or Calculated CrCl ≥ 50 mL/min (MDRD Formula)
  • AST and ALT ≤ 2.5 x ULN
  • Serum total bilirubin ≤ 1.5 x ULN 3. Eastern Cooperative Oncology Group (ECOG) Performance Status of ≤ 2 4. Clinically euthyroid. Note: Patients are permitted to receive thyroid hormone supplements to treat underlying hypothyroidism.

Exclusion Criteria:

  1. Prior HDAC, DAC, HSP90 inhibitors or valproic acid for the treatment of cancer
  2. Previous treatment with JAK2 inhibitors
  3. Any patient who has previously received radiation therapy to ≥ 30% of the bone marrow
  4. Impaired cardiac function or clinically significant cardiac diseases
  5. Patient with unresolved diarrhea ≥ grade 2
  6. Patients using medications that have a relative risk of prolonging the QT interval or inducing Torsade de pointes if treatment cannot be discontinued or switched to a different medication prior to starting study drug
  7. Patients who have undergone major surgery ≤ 4 weeks prior to starting study drug or who have not recovered from side effects of surgery
  8. Women who are pregnant or breast feeding or women of childbearing potential (WOCBP) not using an effective method of birth control. WOCBP are defined as sexually mature women who have not undergone a hysterectomy or who have not been naturally postmenopausal for at least 12 consecutive months (i.e., who has had menses any time in the preceding 12 consecutive months). Women of childbearing potential must have a negative serum pregnancy test at screening and at baseline.
  9. Male patients whose sexual partners are WOCBP not using effective birth control
  10. Patients with a prior malignancy with in the last 5 years (except for basal or squamous cell carcinoma, or in situ cancer of the cervix)
  11. Patients with known positivity for human immunodeficiency virus (HIV) or hepatitis C; baseline testing for HIV and hepatitis C is not required Other protocol-defined inclusion/exclusion criteria may apply -

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00931762

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United States, Arizona
Mayo Clinic - Scottsdale
Scottsdale, Arizona, United States, 85259
United States, California
City of Hope National Medical Center
Duate, California, United States, 91010
Stanford Comprehensive Cancer Center
Stanford, California, United States, 94305
United States, Georgia
Medical College of Georgia
Augusta, Georgia, United States, 30912
United States, Illinois
Northwestern University Feinberg School of Medicine
Chicago, Illinois, United States, 60611
United States, Massachusetts
Dana Farber Cancer Institute
Boston, Massachusetts, United States, 02115
United States, Michigan
University of Michigan
Ann Arbor, Michigan, United States, 48109
United States, Minnesota
Mayo Clinic - Rochester
Rochester, Minnesota, United States, 55905
United States, New York
New York Prebyterian Hospital - Weill Cornell Medical College
New York, New York, United States, 10021
Sponsors and Collaborators
Novartis Pharmaceuticals
Additional Information:
Publications of Results:
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Responsible Party: Novartis Pharmaceuticals Identifier: NCT00931762    
Other Study ID Numbers: CLBH589BUS58
First Posted: July 2, 2009    Key Record Dates
Last Update Posted: November 18, 2016
Last Verified: November 2016
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Bone marrow
Post-Polycythemia Vera
Post-Essential Thrombocytopenia
Additional relevant MeSH terms:
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Polycythemia Vera
Primary Myelofibrosis
Blood Platelet Disorders
Hematologic Diseases
Myeloproliferative Disorders
Bone Marrow Diseases
Bone Marrow Neoplasms
Hematologic Neoplasms
Neoplasms by Site
Antineoplastic Agents
Histone Deacetylase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action