Study to Allow Access to Single Agent Panobinostat for Patients Who Are on s.a. Panobinostat Treatment in a Novartis-sponsored Study and Continue to Benefit From the Treatment as Judged by the Investigator

An Open-label Multi-center Single Agent Panobinostat Roll-over Protocol for Patients Who Have Completed a Previous Novartis-sponsored Panobinostat Study and Are Judged by the Investigator to Benefit From Continued Single Agent Panobinostat Treatment

The study allowed continued use of single agent panobinostat in patients who were on single agent panobinostat treatment in a Novartis-sponsored study which had met its endpoint and were benefiting from the treatment as judged by the investigator.

Study Overview

• Status: Completed
• Conditions: Hematologic Neoplasms
• Intervention / Treatment: Drug: Panobinostat

Detailed Description

This was a multi-center open label study to provide continued use of single agent oral panobinostat to patients treated in a Novartis-sponsored study (parent study) which had met its endpoint and were benefiting from continuation of the treatment with single-agent panobinostat as judged by the investigator. Patients from multiple parent studies were transferred over to this protocol and continued to receive single agent panobinostat at the last assigned dose and regimen of the parent protocol. There was no screening period, and patients had to visit the study center at least on a quarterly basis. During these visits limited information on study treatment and occurrence of SAEs was collected for the clinical database. SAEs were only reported to the Novartis safety database. Other assessments and possibly more frequent visits occurred as per standard of care at the site. Patients continued treatment until they were no longer benefiting from panobinostat treatment, developed unacceptable toxicities, withdrew consent, were non-compliant with the protocol, the investigator believed it was no longer in the best interest to continue, the patient died, or for other administrative reasons. An end of treatment visit and a safety follow-up for 30 days after the last dose was performed. The study was expected to remain open for 5 years or until such time that enrolled patients no longer needed treatment with panobinostat, whichever came earlier.

• Study Type: Interventional
• Enrollment (Actual): 9
• Phase: Phase 2

Contacts and Locations

Study Locations

• Israel
  • Jerusalem, Israel, 91120
    • Novartis Investigative Site
• Netherlands
  • Leiden, Netherlands, 2300 RC
    • Novartis Investigative Site
• Spain
  • Castilla Y Leon
    • Salamanca, Castilla Y Leon, Spain, 37007
      • Novartis Investigative Site
• United States
  • California
    • Duarte, California, United States, 91010 3000
      • City of Hope National Medical Center Dept.ofCityofHopeMedicalCtr(1)
  • Georgia
    • Augusta, Georgia, United States, 30912
      • Georgia Regents University SC-2
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Dana Farber Cancer Institute Reg. Ped
  • Utah
    • Salt Lake City, Utah, United States, 84103
      • University of Utah / Huntsman Cancer Institute SC-2

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• patient had been enrolled in a Novartis-sponsored, Oncology OGD&GMA study receiving s.a. oral panobinostat and had fulfilled all their requirements in the parent study
• patient had been benefiting from the treatment with s.a. oral panobinostat as determined by the guidelines of the parent protocol and according to the Investigator's clinical judgment
• patient had demonstated compliance
• patient had given written informed consent.

Exclusion Criteria:

• patient had been permanently discontinued from s.a. oral panobinostat study treatment in the parent study due to unacceptable toxicity, withdrawal of consent, non-compliance to study procedures or any other reason (including progression of disease).
• patient had participated in a Novartis sponsored combincation trial where panobinostat was dispensed in combination with another study medication and was still receiving combination therapy
• patient was pregnant or nursing at the time of entry
• women of child-bearing potential and male patients with sexual partners of child-bearing potential who were unwilling to use highly effective methods of contraception during dosing and for a specified duration after stopping study treatment

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Panobinostat - 10 to 40 mg/day TIW QoW; 10 to 40mg/day TIW QoW (3 times/week every other week) as per parent protocol design	Drug: Panobinostat; Panobinostat was provided as 5, 10 and 20 mg hard gelatin capsules to be taken orally. Patients started on dose from parent protocol and dose modifications were at the discretion of the investigator based on guidance provided in the protocol and IB.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overview of Adverse Events (Safety Set)	Adverse events were collected from baseline up to 30 days post treatment at scheduled visits. Severity of adverse events was assessed according to the current version of Common Terminology Criteria for Adverse Events (CTCAE). If CTCAE grading did not exist for an adverse event, the severity of mild, moderate, severe, and life-threatening, corresponding to Grades 1 - 4, was used	Baseline up to approximately 60 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Patients With Clinical Benefit as Assessed by the Investigator.	Patients were assessed by investigators at scheduled visits to determine if patient continued to benefit from panobinostat therapy.	baseline up to approximate 5 years

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): June 24, 2013
• Primary Completion (Actual): November 19, 2018
• Study Completion (Actual): November 19, 2018

Study Registration Dates

• First Submitted: February 26, 2013
• First Submitted That Met QC Criteria: February 28, 2013
• First Posted (Estimate): March 4, 2013

Study Record Updates

• Last Update Posted (Actual): December 6, 2019
• Last Update Submitted That Met QC Criteria: November 18, 2019
• Last Verified: November 1, 2019

More Information

Terms related to this study

• Keywords: Hematologic Neoplasms, LBH589,; Panobinostat,
• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Site; Hematologic Diseases; Hematologic Neoplasms; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antineoplastic Agents; Histone Deacetylase Inhibitors; Panobinostat
• Other Study ID Numbers: CLBH589B2402B; 2012-005252-41 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No