Hematologic Neoplasms
Conditions
Keywords
Hematologic Neoplasms, LBH589,, Panobinostat,
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.
Interventions
DRUGPanobinostat
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.
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overview of Adverse Events (Safety Set) | Baseline up to approximately 60 months | 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Patients With Clinical Benefit as Assessed by the Investigator. | baseline up to approximate 5 years | Patients were assessed by investigators at scheduled visits to determine if patient continued to benefit from panobinostat therapy. |
Countries
Israel, Netherlands, Spain, United States
Participant flow
Pre-assignment details
There was no screening period. Patients enrolled into trial directly from the parent protocol.
Participants by arm
| Arm | Count |
|---|---|
| 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 | 8 |
| Total | 8 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Administrative problems | 2 |
| Overall Study | Disease progression | 6 |
Baseline characteristics
| Characteristic | Panobinostat - 10 to 40 mg/Day TIW QoW | — |
|---|---|---|
| Age, Continuous | 54 years STANDARD_DEVIATION 14.5 | — |
| Parent protocol participants CLBH589B2201 | 2 participants | — |
| Parent protocol participants CLBH589B2207 | 3 participants | — |
| Parent protocol participants CLBH589E2214 | 1 participants | — |
| Parent protocol participants CLBH589X2105 | 2 participants | — |
| Race and Ethnicity Not Collected | — | — Participants |
| Sex: Female, Male Female | 4 Participants | — |
| Sex: Female, Male Male | 4 Participants | — |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 8 |
| other Total, other adverse events | 4 / 8 |
| serious Total, serious adverse events | 2 / 8 |
Outcome results
Primary
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
Time frame: Baseline up to approximately 60 months
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Panobinostat - 10 to 40 mg/Day TIW QoW | Overview of Adverse Events (Safety Set) | Any adverse event (AE) | 6 participants |
| Panobinostat - 10 to 40 mg/Day TIW QoW | Overview of Adverse Events (Safety Set) | Any treatment related AE | 2 participants |
| Panobinostat - 10 to 40 mg/Day TIW QoW | Overview of Adverse Events (Safety Set) | Any serious adverse event (SAE) | 2 participants |
| Panobinostat - 10 to 40 mg/Day TIW QoW | Overview of Adverse Events (Safety Set) | Grade 3 or 4 AE | 3 participants |
| Panobinostat - 10 to 40 mg/Day TIW QoW | Overview of Adverse Events (Safety Set) | Grade 3 or 4 AE - suspected to be related | 1 participants |
| Panobinostat - 10 to 40 mg/Day TIW QoW | Overview of Adverse Events (Safety Set) | AEs leading discontinuation | 0 participants |
| Panobinostat - 10 to 40 mg/Day TIW QoW | Overview of Adverse Events (Safety Set) | AEs leading to dose adjust/ temp dose interruption | 2 participants |
| Panobinostat - 10 to 40 mg/Day TIW QoW | Overview of Adverse Events (Safety Set) | On-treatment death | 0 participants |
Secondary
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.
Time frame: baseline up to approximate 5 years
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Panobinostat - 10 to 40 mg/Day TIW QoW | Percentage of Patients With Clinical Benefit as Assessed by the Investigator. | Participants with clinical benefit | 7 Participants |