Clinical trial NCT01802879: 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
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hematologic Neoplasms
Keywords: Hematologic Neoplasms, LBH589,
Panobinostat,
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2013-03-04
Enrollment: 9
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Overview of Adverse Events (Safety Set)
timeFrame: Baseline up to approximately 60 months
Secondary outcomes:
measure: Percentage of Patients With Clinical Benefit as Assessed by the Investigator.
timeFrame: baseline up to approximate 5 years
Other outcomes:
Locations:
city: Duarte
country: United States
facility: City of Hope National Medical Center Dept.ofCityofHopeMedicalCtr(1)
state: California
city: Augusta
country: United States
facility: Georgia Regents University SC-2
state: Georgia
city: Boston
country: United States
facility: Dana Farber Cancer Institute Reg. Ped
state: Massachusetts
city: Salt Lake City
country: United States
facility: University of Utah / Huntsman Cancer Institute SC-2
state: Utah
city: Jerusalem
country: Israel
facility: Novartis Investigative Site
city: Leiden
country: Netherlands
facility: Novartis Investigative Site
city: Salamanca
country: Spain
facility: Novartis Investigative Site
state: Castilla Y Leon
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: