Blood and Lymph Conditions

All Conditions

Neoplasms

Rare Diseases

Thrombocytopenia

Polycythemia Vera

Polycythemia

Primary Myelofibrosis

Thrombocytosis

Thrombocythemia, Essential

Blood Platelet Disorders

Hematologic Diseases

Cytopenia

Myeloproliferative Disorders

Bone Marrow Diseases

Bone Marrow Neoplasms

Hematologic Neoplasms

Essential Thrombocythemia

Chronic Myeloproliferative Disorders

Antineoplastic Agents

All Drugs and Chemicals

Panobinostat

Histone Deacetylase Inhibitors

Enzyme Inhibitors

Participants received panobinostat 40 mg, capsules, orally, with or without food, three times a week on Monday, Wednesday, and Friday for up to 6 treatment cycles (each cycle of 28-days) with dose adjustments possible.

Novartis Pharmaceuticals

This study assessed the safety and efficacy of Panobinostat as a single agent in the treatment of Primary Myelofibrosis, Post-Polycythemia Vera and Post-Essential Thrombocythemia. There were two cohorts - participants with JAK2 mutation and participants without JAK2 mutation.

A Study to Evaluate Safety and Efficacy of Panobinostat in Participants With Primary Myelofibrosis

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

Overall response (OR) = Complete remission (CR) + Partial remission (PR) + Clinical improvement (CI). CR: complete resolution of disease-related symptoms and signs with hemoglobin level 110 grams per deciliter (g/L), platelet count 100 x 10\^9/liter (L), and absolute neutrophil count (ANC) 1.0 x 10\^9/L, all 3 blood counts \< upper normal limit (UNL), normal leukocyte differential, and bone marrow histologic remission. PR= all of the criteria for CR except the requirement for bone marrow histologic remission. CI= minimum 20 g/L increase in hemoglobin for transfusion or becoming transfusion independent or reduction in splenomegaly ≥ 50% or 100% increase in platelet count and absolute platelet count of 50 x 10\^9/L and 100% increase in ANC of at least 0.5 x 10\^9/L.

The MF-SAF consists of seven questions about key symptoms and impact of MF. Questions were scored on a scale of 0-10, with higher scores indicating more severe symptoms and greater inactivity. Questions 1-6 investigate the following symptoms: night sweats, pruritus/itching, abdominal discomfort, pain under the ribs, early satiety and bone/muscle pain. Question 7 asked participants to report levels of inactivity. Negative change from baseline indicates improvement.

An adverse event is the appearance or worsening of any undesirable sign, symptom, or medical condition occurring after starting the study drug even if the event is not considered to be related to study drug. SAEs was an undesirable sign, symptom or medical condition which is: life-threatening, persistent or significant disability/incapacity, congenital anomaly/birth defect, requires hospitalizations or prolongation of hospitalizations, or is medically significant

Safety set population included all participants who were randomized and received at least 1 dose of study drug.

Age, Continuous

Sex: Female, Male

Full Analysis set (FAS) population included all participants who received at least one dose of study drug.

Study Director

FAS population included all participants who received at least one dose of study drug.

Overall Response (OR) Rate Per International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) Criteria

Cycle 6, Day 1: Fatigue Right now

Cycle 6, Day 1: Fatigue Usual Past 24 Hours

Cycle 6, Day 1: Fatigue Worst Past 24 Hours

Cycle 6, Day 1: General Activity

Cycle 6, Day 1: Mood

Cycle 6, Day 1: Walking Ability

Cycle 6, Day 1: Normal Work

Cycle 6, Day 1: Relations with Other People

Cycle 6, Day 1: Enjoyment of Life

Cycle 6, Day 1: Filling up quickly when you eat

Cycle 6, Day 1: Abdominal pain or Discomfort

Cycle 6, Day 1: Inactivity

Cycle 6, Day 1: Cough

Cycle 6, Day 1: Night Sweats

Cycle 6, Day 1: Itching

Cycle 6, Day 1: Bone Pain

Cycle 6, Day 1: What is Overall Quality of Life?

FAS population included all participants who received at least one dose of study drug.

All 35 participants were evaluated for this Outcome. The Number Analyzed represented in each row differs from the Overall Number of Participants Analyzed as scores that are presented are for the participants who reported the measured event at the time evaluated.

Change From Baseline in Myelofibrosis Symptom Assessment Form (MF-SAF) Scale Scores At Cycle 6

Cycle 2, Day 1: Fatigue Right now

Cycle 2, Day 1: Fatigue Usual Past 24 Hours

Cycle 2, Day 1: Fatigue Worst Past 24 Hours

Cycle 2, Day 1: General Activity

Cycle 2, Day 1: Mood

Cycle 2, Day 1: Walking Ability

Cycle 2, Day 1: Normal Work

Cycle 2, Day 1: Relations with Other People

Cycle 2, Day 1: Enjoyment of Life

Cycle 2, Day 1: Filling up quickly when you eat

Cycle 2, Day 1: Abdominal Pain or Discomfort

Cycle 2, Day 1: Inactivity

Cycle 2, Day 1: Cough

Cycle 2, Day 1: Night Sweats

Cycle 2, Day 1: Itching

Cycle 2, Day 1: Bone Pain

Cycle 2, Day 1: What is Overall Quality of Life?

Cycle 4, Day 1: Fatigue Right now

Cycle 4, Day 1: Fatigue Usual Past 24 Hours

Cycle 4, Day 1: Fatigue Worst Past 24 Hours

Cycle 4, Day 1: General Activity

Cycle 4, Day 1: Mood

Cycle 4, Day 1: Walking Ability

Cycle 4, Day 1: Normal Work

Cycle 4, Day 1: Relations with Other People

Cycle 4, Day 1: Enjoyment of Life

Cycle 4, Day 1: Filling up quickly when you eat

Cycle 4, Day 1: Abdominal Pain or Discomfort

Cycle 4, Day 1: Inactivity

Cycle 4, Day 1: Cough

Cycle 4, Day 1: Night Sweats

Cycle 4, Day 1: Itching

Cycle 4, Day 1: Bone Pain

Cycle 4, Day 1: What is Overall Quality of Life?

Change From Baseline in Myelofibrosis Symptom Assessment Form (MF-SAF) Scale Scores

SAEs

Safety set population included all participants who received at least one dose of study drug and had at least one post-baseline safety assessment.

Number of Participants With Adverse Events (AEs), and Serious Adverse Events (SAEs)

Monthly Capsule Counts to Assess Compliance to Panobinostat Treatment

To Compare the Response to Panobinostat in Patients With the JAK2 V617F Mutation to Those Without the JAK2 V617F Mutation

Overall Study

Spots Global Cancer Trial Database for A Study to Evaluate Safety and Efficacy of Panobinostat in Participants With Primary Myelofibrosis

Trial Identification

Conditions

Interventions

Study Description

Keywords

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Useful links and downloads for this trial