Trial Outcomes & Findings for 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 (NCT NCT01802879)
NCT ID: NCT01802879
Last Updated: 2019-12-06
Results Overview
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
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
9 participants
Primary outcome timeframe
Baseline up to approximately 60 months
Results posted on
2019-12-06
Participant Flow
There was no screening period. Patients enrolled into trial directly from the parent protocol.
Participant milestones
| Measure |
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
|
|---|---|
|
Overall Study
STARTED
|
8
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
8
|
Reasons for withdrawal
| Measure |
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
|
|---|---|
|
Overall Study
Administrative problems
|
2
|
|
Overall Study
Disease progression
|
6
|
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
Panobinostat - 10 to 40 mg/Day TIW QoW
n=8 Participants
10 to 40mg/day TIW QoW (3 times/week every other week) as per parent protocol design
|
|---|---|
|
Age, Continuous
|
54 years
STANDARD_DEVIATION 14.5 • n=8 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=8 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=8 Participants
|
|
Parent protocol participants
CLBH589B2201
|
2 participants
n=8 Participants
|
|
Parent protocol participants
CLBH589B2207
|
3 participants
n=8 Participants
|
|
Parent protocol participants
CLBH589E2214
|
1 participants
n=8 Participants
|
|
Parent protocol participants
CLBH589X2105
|
2 participants
n=8 Participants
|
PRIMARY outcome
Timeframe: Baseline up to approximately 60 monthsAdverse 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
Outcome measures
| Measure |
Panobinostat - 10 to 40 mg/Day TIW QoW
n=8 Participants
10 to 40mg/day TIW QoW (3 times/week every other week) as per parent protocol design
|
|---|---|
|
Overview of Adverse Events (Safety Set)
Any adverse event (AE)
|
6 participants
|
|
Overview of Adverse Events (Safety Set)
Any treatment related AE
|
2 participants
|
|
Overview of Adverse Events (Safety Set)
Any serious adverse event (SAE)
|
2 participants
|
|
Overview of Adverse Events (Safety Set)
Grade 3 or 4 AE
|
3 participants
|
|
Overview of Adverse Events (Safety Set)
Grade 3 or 4 AE - suspected to be related
|
1 participants
|
|
Overview of Adverse Events (Safety Set)
AEs leading discontinuation
|
0 participants
|
|
Overview of Adverse Events (Safety Set)
AEs leading to dose adjust/ temp dose interruption
|
2 participants
|
|
Overview of Adverse Events (Safety Set)
On-treatment death
|
0 participants
|
SECONDARY outcome
Timeframe: baseline up to approximate 5 yearsPatients were assessed by investigators at scheduled visits to determine if patient continued to benefit from panobinostat therapy.
Outcome measures
| Measure |
Panobinostat - 10 to 40 mg/Day TIW QoW
n=8 Participants
10 to 40mg/day TIW QoW (3 times/week every other week) as per parent protocol design
|
|---|---|
|
Percentage of Patients With Clinical Benefit as Assessed by the Investigator.
Participants with clinical benefit
|
7 Participants
|
Adverse Events
Panobinostat - 10 to 40 mg/Day TIW QoW
Serious events: 2 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Panobinostat - 10 to 40 mg/Day TIW QoW
n=8 participants at risk
10 to 40mg/day TIW QoW (3 times/week every other week) as per parent protocol design
|
|---|---|
|
General disorders
Non-cardiac chest pain
|
12.5%
1/8 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 84 weeks
AE additional description
|
|
Nervous system disorders
Cerebrovascular accident
|
12.5%
1/8 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 84 weeks
AE additional description
|
Other adverse events
| Measure |
Panobinostat - 10 to 40 mg/Day TIW QoW
n=8 participants at risk
10 to 40mg/day TIW QoW (3 times/week every other week) as per parent protocol design
|
|---|---|
|
Blood and lymphatic system disorders
Neutropenia
|
12.5%
1/8 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 84 weeks
AE additional description
|
|
Gastrointestinal disorders
Diarrhoea
|
12.5%
1/8 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 84 weeks
AE additional description
|
|
Gastrointestinal disorders
Gastric disorder
|
12.5%
1/8 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 84 weeks
AE additional description
|
|
General disorders
Asthenia
|
12.5%
1/8 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 84 weeks
AE additional description
|
|
Infections and infestations
Nasopharyngitis
|
12.5%
1/8 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 84 weeks
AE additional description
|
|
Investigations
Blood creatinine increased
|
12.5%
1/8 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 84 weeks
AE additional description
|
|
Investigations
Platelet count decreased
|
12.5%
1/8 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 84 weeks
AE additional description
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
12.5%
1/8 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 84 weeks
AE additional description
|
|
Musculoskeletal and connective tissue disorders
Osteoarthritis
|
12.5%
1/8 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 84 weeks
AE additional description
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Benign muscle neoplasm
|
12.5%
1/8 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 84 weeks
AE additional description
|
|
Nervous system disorders
Neuropathy peripheral
|
25.0%
2/8 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 84 weeks
AE additional description
|
|
Psychiatric disorders
Insomnia
|
12.5%
1/8 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 84 weeks
AE additional description
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
12.5%
1/8 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 84 weeks
AE additional description
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.
- Publication restrictions are in place
Restriction type: OTHER