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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

Phase 2
18 Years
Not Enrolling
Primary Myelofibrosis, Post-Polycythemia Vera, Post-Essential Thrombocytopenia

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Trial Information

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

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 -

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

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.

Outcome Time Frame:

Upon enrollment of 13 participants into each cohort of the study and at the end of the study.

Safety Issue:



United States: Food and Drug Administration

Study ID:




Start Date:

July 2009

Completion Date:

Related Keywords:

  • Primary Myelofibrosis
  • Post-Polycythemia Vera
  • Post-Essential Thrombocytopenia
  • Bone marrow
  • Myelofibrosis
  • JAK2mutation
  • Post-Polycythemia Vera
  • Post-Essential Thrombocytopenia
  • Primary Myelofibrosis
  • Polycythemia
  • Polycythemia Vera
  • Thrombocythemia, Essential
  • Thrombocytopenia
  • Purpura, Thrombocytopenic



University of Michigan Ann Arbor, Michigan  48109-0624
City of Hope National Medical Center Los Angeles, California  91010
Medical College of Georgia Augusta, Georgia  30912
Dana Farber Cancer Institute Boston, Massachusetts  02115
Northwestern University Feinberg School of Medicine Chicago, Illinois  60611
Stanford Comprehensive Cancer Center Stanford, California  94305
Mayo Clinic - Rochester Rochester, Minnesota  55905
Mayo Clinic - Scottsdale Scottsdale, Arizona  85259
New York Prebyterian Hospital - Weill Cornell Medical College New York, New York  10021