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Trial Outcomes & Findings for A Phase II Study of Oral LDE225 in Patients With Hedge-Hog (Hh)-Pathway Activated Relapsed Medulloblastoma (MB) (NCT NCT01708174)

NCT ID: NCT01708174

Last Updated: 2017-08-11

Results Overview

ORR was defined as the percentage of participants with best overall response of complete response (CR) or partial response (PR) (as per tumor response guidelines and criteria for Medulloblastoma). The IRC evaluated all radiological images and applicable clinical data (i.e., neurological examination, steroid use and cerebrospinal fluid (CSF) results as applicable). Assessments after crossover were not included for TMZ participants.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

22 participants

Primary outcome timeframe

from date first participant randomized, 13-Sep-2013 to date of data cut-off, 15-Nov-2016

Results posted on

2017-08-11

Participant Flow

Analyses were performed by treatment and by age group.

Participant milestones

Participant milestones
Measure
Sonidegib (LDE225) Children
500 mg/m2 orally
Sonidegib (LDE225) Adults
600 mg orally
Temozolomide (TMZ)
150 to 200 mg/m2 for 5 sequential days every 4 weeks according to prescribing information until the study was amended to a single arm study.
Treatment Period
STARTED
2
16
4
Treatment Period
Pharmacokinetic Analysis Set
2
13
0
Treatment Period
COMPLETED
1
14
3
Treatment Period
NOT COMPLETED
1
2
1
Survival Follow-up
STARTED
1
10
0
Survival Follow-up
COMPLETED
1
10
0
Survival Follow-up
NOT COMPLETED
0
0
0

Reasons for withdrawal

Reasons for withdrawal
Measure
Sonidegib (LDE225) Children
500 mg/m2 orally
Sonidegib (LDE225) Adults
600 mg orally
Temozolomide (TMZ)
150 to 200 mg/m2 for 5 sequential days every 4 weeks according to prescribing information until the study was amended to a single arm study.
Treatment Period
Withdrawal by Subject
1
0
0
Treatment Period
Progressive disease
0
1
0
Treatment Period
Death
0
0
1
Treatment Period
Adverse Event
0
1
0

Baseline Characteristics

A Phase II Study of Oral LDE225 in Patients With Hedge-Hog (Hh)-Pathway Activated Relapsed Medulloblastoma (MB)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Sonidegib (LDE225) Children
n=2 Participants
500 mg/m2 orally
Sonidegib (LDE225) Adults
n=16 Participants
600 mg orally
Temozolomide (TMZ)
n=4 Participants
150 to 200 mg/m2 for 5 sequential days every 4 weeks according to prescribing information until the study was amended to a single arm study.
Total
n=22 Participants
Total of all reporting groups
Age, Continuous
8.5 Years
FULL_RANGE 6.36 • n=5 Participants
37.0 Years
FULL_RANGE 8.17 • n=7 Participants
35.5 Years
FULL_RANGE 3.16 • n=5 Participants
35.0 Years
FULL_RANGE 10.73 • n=4 Participants
Sex: Female, Male
Female
2 Participants
n=5 Participants
5 Participants
n=7 Participants
2 Participants
n=5 Participants
9 Participants
n=4 Participants
Sex: Female, Male
Male
0 Participants
n=5 Participants
11 Participants
n=7 Participants
2 Participants
n=5 Participants
13 Participants
n=4 Participants

PRIMARY outcome

Timeframe: from date first participant randomized, 13-Sep-2013 to date of data cut-off, 15-Nov-2016

Population: The full analysis set (FAS) was analyzed. The FAS included all randomized and non-randomized participants who received at least one dose of study treatment.

ORR was defined as the percentage of participants with best overall response of complete response (CR) or partial response (PR) (as per tumor response guidelines and criteria for Medulloblastoma). The IRC evaluated all radiological images and applicable clinical data (i.e., neurological examination, steroid use and cerebrospinal fluid (CSF) results as applicable). Assessments after crossover were not included for TMZ participants.

Outcome measures

Outcome measures
Measure
Temozolomide (TMZ)
n=4 Participants
150 to 200 mg/m2 for 5 sequential days every 4 weeks according to prescribing information until the study was amended to a single arm study.
Sonidegib (LDE225) Children
n=2 Participants
500 mg/m2 orally
Sonidegib (LDE225) Adults
n=16 Participants
600 mg orally
Percentage of Participants With Overall Response Rate (ORR) According to Independent Review Committee (IRC) From Date First Participant Randomized, 13-Sep-2013 to Date of Data Cut-off, 15-Nov-2016
0.0 Percentage of participants
0.0 Percentage of participants
18.8 Percentage of participants

SECONDARY outcome

Timeframe: from date first participant randomized, 13-Sep-2013 to date of data cut-off, 15-Nov-2016

Population: The FAS was analyzed. The FAS included all randomized and non-randomized participants who received at least one dose of study treatment.

PFS was defined as the time from date of randomization to the date of event defined as the first documented progression or death due to any cause (as per tumor response guidelines and criteria for Medulloblastoma). The IRC evaluated all radiological images and applicable clinical data (i.e., neurological examination, steroid use and cerebrospinal fluid (CSF) results as applicable). TMZ participants without event prior to crossover were censored.

Outcome measures

Outcome measures
Measure
Temozolomide (TMZ)
n=4 Participants
150 to 200 mg/m2 for 5 sequential days every 4 weeks according to prescribing information until the study was amended to a single arm study.
Sonidegib (LDE225) Children
n=2 Participants
500 mg/m2 orally
Sonidegib (LDE225) Adults
n=16 Participants
600 mg orally
Progression Free Survival (PFS) According to IRC From Date First Participant Randomized, 13-Sep-2013 to Date of Data Cut-off, 15-Nov-2016
2.9 months
Interval 0.9 to 4.0
1.6 months
The lower and upper 95% confidence intervals (CI) were not estimable because there were only 2 participants analyzed. With only 2 participants, it is not uncommon to have 'not estimable' for the limits.
3.3 months
Interval 1.7 to 17.1

SECONDARY outcome

Timeframe: from date first participant randomized, 13-Sep-2013 to date of data cut-off, 15-Nov-2016

Population: The FAS was analyzed. The FAS included all randomized and non-randomized participants who received at least one dose of study treatment.

PFS was defined as the time from date of randomization to the date of event defined as the first documented progression or death due to any cause. PFS was evaluated by local Investigator assessment per tumor response guidelines and criteria for Medulloblastoma.

Outcome measures

Outcome measures
Measure
Temozolomide (TMZ)
n=4 Participants
150 to 200 mg/m2 for 5 sequential days every 4 weeks according to prescribing information until the study was amended to a single arm study.
Sonidegib (LDE225) Children
n=2 Participants
500 mg/m2 orally
Sonidegib (LDE225) Adults
n=16 Participants
600 mg orally
PFS According to Local Investigator Assessment From Date First Participant Randomized, 13-Sep-2013 to Date of Data Cut-off, 15-Nov-2016
2.9 months
Interval 0.9 to 4.0
NA months
Interval 1.6 to
The median and upper limit of the 95% confidence interval were not estimable because there were too few events.
3.3 months
Interval 1.6 to 13.0

SECONDARY outcome

Timeframe: from date first participant randomized, 13-Sep-2013 to date of data cut-off, 15-Nov-2016

Population: The full analysis set (FAS) was analyzed. The FAS included all randomized and non-randomized participants who received at least one dose of study treatment.

ORR was defined as the percentage of participants with best overall response of complete response (CR) or partial response (PR). ORR was evaluated by local Investigator assessment per tumor response guidelines and criteria for Medulloblastoma. Assessments after crossover were not included for TMZ patients.

Outcome measures

Outcome measures
Measure
Temozolomide (TMZ)
n=4 Participants
150 to 200 mg/m2 for 5 sequential days every 4 weeks according to prescribing information until the study was amended to a single arm study.
Sonidegib (LDE225) Children
n=2 Participants
500 mg/m2 orally
Sonidegib (LDE225) Adults
n=16 Participants
600 mg orally
Percentage of Participants With ORR According to Local Investigator Assessment From Date First Participant Randomized, 13-Sep-2013 to Date of Data Cut-off, 15-Nov-2016
0.0 Percentage of participants
0.0 Percentage of participants
25.0 Percentage of participants

SECONDARY outcome

Timeframe: from date first participant randomized, 13-Sep-2013 to date of data cut-off, 15-Nov-2016

Population: The full analysis set (FAS) was analyzed. The FAS included all randomized and non-randomized participants who received at least one dose of study treatment.

DoR was defined as the time from the first documented onset of confirmed PR or CR to the date of PD/relapse or death due to medulloblastoma. DoR was evaluated by local Investigator assessment per tumor response guidelines and criteria for Medulloblastoma. TMZ participants without an event prior to crossover were censored.

Outcome measures

Outcome measures
Measure
Temozolomide (TMZ)
n=4 Participants
150 to 200 mg/m2 for 5 sequential days every 4 weeks according to prescribing information until the study was amended to a single arm study.
Sonidegib (LDE225) Children
n=2 Participants
500 mg/m2 orally
Sonidegib (LDE225) Adults
n=16 Participants
600 mg orally
Duration of Response (DoR) According to Local Investigator Assessment From Date First Participant Randomized, 13-Sep-2013 to Date of Data Cut-off, 15-Nov-2016
NA months
The median and lower and upper 95% CI could not be calculated because there were too few events.
NA months
The median and lower and upper 95% CI could not be calculated because there were too few events.
8.5 months
Interval 4.1 to 16.6

SECONDARY outcome

Timeframe: from date first participant randomized, 13-Sep-2013 to date of data cut-off, 15-Nov-2016

Population: The full analysis set (FAS) was analyzed. The FAS included all randomized and non-randomized participants who received at least one dose of study treatment.

OS was defined as the time from date of randomization to date of death due to any cause. All deaths are considered, including deaths occurred after crossover for TMZ participants.

Outcome measures

Outcome measures
Measure
Temozolomide (TMZ)
n=4 Participants
150 to 200 mg/m2 for 5 sequential days every 4 weeks according to prescribing information until the study was amended to a single arm study.
Sonidegib (LDE225) Children
n=2 Participants
500 mg/m2 orally
Sonidegib (LDE225) Adults
n=16 Participants
600 mg orally
Overall Survival (OS) From Date First Participant Randomized, 13-Sep-2013 to Date of Data Cut-off, 15-Nov-2016
NA months
Interval 1.2 to
The median and upper 95% CI could not be calculated because there were too few events.
NA months
Interval 4.5 to
The median and upper 95% CI could not be calculated because there were too few events.
9.5 months
Interval 4.9 to 15.6

SECONDARY outcome

Timeframe: Weeks 1, 3, 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49 and 53

Population: The full analysis set (FAS) was analyzed. The FAS included all randomized and non-randomized participants who received at least one dose of study treatment.

Blood samples were collected for assessment. The children's group was analyzed up until week 25 only.

Outcome measures

Outcome measures
Measure
Temozolomide (TMZ)
150 to 200 mg/m2 for 5 sequential days every 4 weeks according to prescribing information until the study was amended to a single arm study.
Sonidegib (LDE225) Children
n=2 Participants
500 mg/m2 orally
Sonidegib (LDE225) Adults
n=13 Participants
600 mg orally
Pharmacokinetics (PK): Summary of Plasma Trough Concentrations for Sonidegib (LDE225)
Week 41 (n=NA,4)
—
NA ng/mL
Standard Deviation NA
PK analysis for the Children group was done up until week 25 only.
2370 ng/mL
Standard Deviation 916
Pharmacokinetics (PK): Summary of Plasma Trough Concentrations for Sonidegib (LDE225)
Week 1 (n=2,11)
—
0.00 ng/mL
Standard Deviation 0.00
0.00 ng/mL
Standard Deviation 0.00
Pharmacokinetics (PK): Summary of Plasma Trough Concentrations for Sonidegib (LDE225)
Week 3 (n=2,10)
—
2890 ng/mL
Standard Deviation 1240
761 ng/mL
Standard Deviation 519
Pharmacokinetics (PK): Summary of Plasma Trough Concentrations for Sonidegib (LDE225)
Week 5 (n=2,8)
—
4930 ng/mL
Standard Deviation 1380
1090 ng/mL
Standard Deviation 700
Pharmacokinetics (PK): Summary of Plasma Trough Concentrations for Sonidegib (LDE225)
Week 7 (n=1,8)
—
2810 ng/mL
Standard Deviation NA
Standard deviation (SD) does not apply when n=1.
1450 ng/mL
Standard Deviation 842
Pharmacokinetics (PK): Summary of Plasma Trough Concentrations for Sonidegib (LDE225)
Week 9 (n=1,8)
—
4670 ng/mL
Standard Deviation NA
SD does not apply when n=1.
1530 ng/mL
Standard Deviation 682
Pharmacokinetics (PK): Summary of Plasma Trough Concentrations for Sonidegib (LDE225)
Week 13 (n=1,4)
—
3680 ng/mL
Standard Deviation NA
SD does not apply when n=1.
2330 ng/mL
Standard Deviation 1100
Pharmacokinetics (PK): Summary of Plasma Trough Concentrations for Sonidegib (LDE225)
Week 17 (n=1,6)
—
3060 ng/mL
Standard Deviation NA
SD does not apply when n=1.
1880 ng/mL
Standard Deviation 730
Pharmacokinetics (PK): Summary of Plasma Trough Concentrations for Sonidegib (LDE225)
Week 21 (n=1,5)
—
3770 ng/mL
Standard Deviation NA
SD does not apply when n=1.
2270 ng/mL
Standard Deviation 915
Pharmacokinetics (PK): Summary of Plasma Trough Concentrations for Sonidegib (LDE225)
Week 25 (n=1,4)
—
1890 ng/mL
Standard Deviation NA
SD does not apply when n=1.
2050 ng/mL
Standard Deviation 625
Pharmacokinetics (PK): Summary of Plasma Trough Concentrations for Sonidegib (LDE225)
Week 29 (n=NA,4)
—
NA ng/mL
Standard Deviation NA
PK analysis for the Children group was done up until week 25 only.
2050 ng/mL
Standard Deviation 906
Pharmacokinetics (PK): Summary of Plasma Trough Concentrations for Sonidegib (LDE225)
Week 33 (n=NA,4)
—
NA ng/mL
Standard Deviation NA
PK analysis for the Children group was done up until week 25 only.
2180 ng/mL
Standard Deviation 512
Pharmacokinetics (PK): Summary of Plasma Trough Concentrations for Sonidegib (LDE225)
Week 37 (n=NA,3)
—
NA ng/mL
Standard Deviation NA
PK analysis for the Children group was done up until week 25 only.
2850 ng/mL
Standard Deviation 306
Pharmacokinetics (PK): Summary of Plasma Trough Concentrations for Sonidegib (LDE225)
Week 45 (n=NA,2)
—
NA ng/mL
Standard Deviation NA
PK analysis for the Children group was done up until week 25 only.
2890 ng/mL
Standard Deviation 290
Pharmacokinetics (PK): Summary of Plasma Trough Concentrations for Sonidegib (LDE225)
Week 49 (n=NA,2)
—
NA ng/mL
Standard Deviation NA
PK analysis for the Children group was done up until week 25 only.
2330 ng/mL
Standard Deviation 990
Pharmacokinetics (PK): Summary of Plasma Trough Concentrations for Sonidegib (LDE225)
Week 53 (n=NA,2)
—
NA ng/mL
Standard Deviation NA
PK analysis for the Children group was done up until week 25 only.
2080 ng/mL
Standard Deviation 764

Adverse Events

Sonidegib (LDE225) Children

Serious events: 2 serious events
Other events: 2 other events
Deaths: 0 deaths

Sonidegib (LDE225) Adults

Serious events: 9 serious events
Other events: 16 other events
Deaths: 0 deaths

Sonidegib (Total)

Serious events: 11 serious events
Other events: 18 other events
Deaths: 0 deaths

Temozolomide (TMZ)

Serious events: 1 serious events
Other events: 4 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Sonidegib (LDE225) Children
n=2 participants at risk
500 mg/m2 orally
Sonidegib (LDE225) Adults
n=16 participants at risk
600 mg orally
Sonidegib (Total)
n=18 participants at risk
Sonidegib (Total)
Temozolomide (TMZ)
n=4 participants at risk
150 to 200 mg/m2 for 5 sequential days every 4 weeks according to prescribing information until the study was amended to a single arm study.
Gastrointestinal disorders
NAUSEA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Gastrointestinal disorders
VOMITING
0.00%
0/2
12.5%
2/16
11.1%
2/18
0.00%
0/4
General disorders
GENERAL PHYSICAL HEALTH DETERIORATION
50.0%
1/2
0.00%
0/16
5.6%
1/18
0.00%
0/4
General disorders
PAIN
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Infections and infestations
BACTERIAL SEPSIS
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Infections and infestations
CLOSTRIDIAL INFECTION
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Infections and infestations
LUNG INFECTION
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Infections and infestations
SEPSIS
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Investigations
ASPARTATE AMINOTRANSFERASE INCREASED
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Investigations
BLOOD CREATINE PHOSPHOKINASE INCREASED
0.00%
0/2
12.5%
2/16
11.1%
2/18
0.00%
0/4
Metabolism and nutrition disorders
DEHYDRATION
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Metabolism and nutrition disorders
HYPERNATRAEMIA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Metabolism and nutrition disorders
HYPONATRAEMIA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Musculoskeletal and connective tissue disorders
BACK PAIN
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Musculoskeletal and connective tissue disorders
NECK PAIN
0.00%
0/2
0.00%
0/16
0.00%
0/18
25.0%
1/4
Nervous system disorders
SEIZURE
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Product Issues
THROMBOSIS IN DEVICE
50.0%
1/2
0.00%
0/16
5.6%
1/18
0.00%
0/4
Psychiatric disorders
CONFUSIONAL STATE
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Psychiatric disorders
DRUG DEPENDENCE
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Psychiatric disorders
HALLUCINATION
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Respiratory, thoracic and mediastinal disorders
COUGH
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Respiratory, thoracic and mediastinal disorders
DYSPNOEA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Vascular disorders
EMBOLISM
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4

Other adverse events

Other adverse events
Measure
Sonidegib (LDE225) Children
n=2 participants at risk
500 mg/m2 orally
Sonidegib (LDE225) Adults
n=16 participants at risk
600 mg orally
Sonidegib (Total)
n=18 participants at risk
Sonidegib (Total)
Temozolomide (TMZ)
n=4 participants at risk
150 to 200 mg/m2 for 5 sequential days every 4 weeks according to prescribing information until the study was amended to a single arm study.
Blood and lymphatic system disorders
ANAEMIA
0.00%
0/2
12.5%
2/16
11.1%
2/18
0.00%
0/4
Blood and lymphatic system disorders
THROMBOCYTOPENIA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Cardiac disorders
ATRIOVENTRICULAR BLOCK FIRST DEGREE
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Cardiac disorders
SINUS TACHYCARDIA
0.00%
0/2
12.5%
2/16
11.1%
2/18
0.00%
0/4
Ear and labyrinth disorders
EAR CONGESTION
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Ear and labyrinth disorders
VERTIGO
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Endocrine disorders
DIABETES INSIPIDUS
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Eye disorders
DIPLOPIA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Eye disorders
PHOTOPHOBIA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Eye disorders
VISION BLURRED
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Gastrointestinal disorders
ABDOMINAL DISTENSION
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Gastrointestinal disorders
ABDOMINAL PAIN
0.00%
0/2
12.5%
2/16
11.1%
2/18
25.0%
1/4
Gastrointestinal disorders
ABDOMINAL PAIN UPPER
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Gastrointestinal disorders
AEROPHAGIA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Gastrointestinal disorders
ANAL INCONTINENCE
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Gastrointestinal disorders
CONSTIPATION
0.00%
0/2
37.5%
6/16
33.3%
6/18
25.0%
1/4
Gastrointestinal disorders
DIARRHOEA
50.0%
1/2
18.8%
3/16
22.2%
4/18
0.00%
0/4
Gastrointestinal disorders
DYSPHAGIA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Gastrointestinal disorders
HAEMATOCHEZIA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Gastrointestinal disorders
NAUSEA
0.00%
0/2
31.2%
5/16
27.8%
5/18
75.0%
3/4
Gastrointestinal disorders
PARAESTHESIA ORAL
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Gastrointestinal disorders
REGURGITATION
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Gastrointestinal disorders
TOOTH DISCOLOURATION
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Gastrointestinal disorders
TOOTH LOSS
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Gastrointestinal disorders
VOMITING
100.0%
2/2
43.8%
7/16
50.0%
9/18
25.0%
1/4
General disorders
ASTHENIA
0.00%
0/2
12.5%
2/16
11.1%
2/18
25.0%
1/4
General disorders
FATIGUE
0.00%
0/2
43.8%
7/16
38.9%
7/18
50.0%
2/4
General disorders
GAIT DISTURBANCE
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
General disorders
NON-CARDIAC CHEST PAIN
0.00%
0/2
0.00%
0/16
0.00%
0/18
25.0%
1/4
General disorders
OEDEMA PERIPHERAL
0.00%
0/2
12.5%
2/16
11.1%
2/18
25.0%
1/4
General disorders
PAIN
0.00%
0/2
12.5%
2/16
11.1%
2/18
0.00%
0/4
General disorders
PYREXIA
0.00%
0/2
12.5%
2/16
11.1%
2/18
0.00%
0/4
Immune system disorders
ALLERGY TO ARTHROPOD BITE
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Infections and infestations
ACINETOBACTER INFECTION
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Infections and infestations
FOLLICULITIS
0.00%
0/2
12.5%
2/16
11.1%
2/18
0.00%
0/4
Infections and infestations
ORAL HERPES
50.0%
1/2
0.00%
0/16
5.6%
1/18
0.00%
0/4
Infections and infestations
RHINITIS
0.00%
0/2
0.00%
0/16
0.00%
0/18
25.0%
1/4
Infections and infestations
UPPER RESPIRATORY TRACT INFECTION
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Infections and infestations
URINARY TRACT INFECTION
0.00%
0/2
25.0%
4/16
22.2%
4/18
0.00%
0/4
Investigations
ACTIVATED PARTIAL THROMBOPLASTIN TIME PROLONGED
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Investigations
ALANINE AMINOTRANSFERASE INCREASED
0.00%
0/2
18.8%
3/16
16.7%
3/18
0.00%
0/4
Investigations
ASPARTATE AMINOTRANSFERASE INCREASED
0.00%
0/2
12.5%
2/16
11.1%
2/18
0.00%
0/4
Investigations
BLOOD ALKALINE PHOSPHATASE INCREASED
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Investigations
BLOOD CREATINE PHOSPHOKINASE INCREASED
50.0%
1/2
43.8%
7/16
44.4%
8/18
25.0%
1/4
Investigations
BLOOD CREATINE PHOSPHOKINASE MB INCREASED
50.0%
1/2
6.2%
1/16
11.1%
2/18
25.0%
1/4
Investigations
BLOOD CREATININE INCREASED
0.00%
0/2
18.8%
3/16
16.7%
3/18
0.00%
0/4
Investigations
BLOOD PHOSPHORUS INCREASED
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Investigations
BODY TEMPERATURE INCREASED
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Investigations
C-REACTIVE PROTEIN INCREASED
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Investigations
CRYSTAL URINE PRESENT
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Investigations
GRANULOCYTE COUNT INCREASED
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Investigations
LYMPHOCYTE COUNT DECREASED
0.00%
0/2
31.2%
5/16
27.8%
5/18
25.0%
1/4
Investigations
OXYGEN SATURATION DECREASED
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Investigations
PLATELET COUNT DECREASED
0.00%
0/2
6.2%
1/16
5.6%
1/18
25.0%
1/4
Investigations
PROTHROMBIN TIME PROLONGED
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Investigations
RED BLOOD CELL COUNT DECREASED
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Investigations
RED BLOOD CELL SEDIMENTATION RATE INCREASED
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Investigations
WEIGHT DECREASED
0.00%
0/2
12.5%
2/16
11.1%
2/18
25.0%
1/4
Investigations
WHITE BLOOD CELL COUNT DECREASED
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Metabolism and nutrition disorders
DECREASED APPETITE
0.00%
0/2
25.0%
4/16
22.2%
4/18
0.00%
0/4
Metabolism and nutrition disorders
FAILURE TO THRIVE
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Metabolism and nutrition disorders
HYPERCALCAEMIA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Metabolism and nutrition disorders
HYPERGLYCAEMIA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Metabolism and nutrition disorders
HYPERURICAEMIA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Metabolism and nutrition disorders
HYPOALBUMINAEMIA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Metabolism and nutrition disorders
HYPOCALCAEMIA
0.00%
0/2
18.8%
3/16
16.7%
3/18
0.00%
0/4
Metabolism and nutrition disorders
HYPOKALAEMIA
0.00%
0/2
25.0%
4/16
22.2%
4/18
0.00%
0/4
Metabolism and nutrition disorders
HYPOMAGNESAEMIA
0.00%
0/2
12.5%
2/16
11.1%
2/18
0.00%
0/4
Metabolism and nutrition disorders
HYPONATRAEMIA
0.00%
0/2
18.8%
3/16
16.7%
3/18
0.00%
0/4
Metabolism and nutrition disorders
HYPOPHOSPHATAEMIA
0.00%
0/2
12.5%
2/16
11.1%
2/18
0.00%
0/4
Musculoskeletal and connective tissue disorders
ARTHRALGIA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Musculoskeletal and connective tissue disorders
BACK PAIN
0.00%
0/2
25.0%
4/16
22.2%
4/18
0.00%
0/4
Musculoskeletal and connective tissue disorders
MUSCLE SPASMS
0.00%
0/2
25.0%
4/16
22.2%
4/18
0.00%
0/4
Musculoskeletal and connective tissue disorders
MYALGIA
50.0%
1/2
31.2%
5/16
33.3%
6/18
0.00%
0/4
Musculoskeletal and connective tissue disorders
NECK PAIN
0.00%
0/2
18.8%
3/16
16.7%
3/18
25.0%
1/4
Musculoskeletal and connective tissue disorders
PAIN IN EXTREMITY
50.0%
1/2
12.5%
2/16
16.7%
3/18
25.0%
1/4
Nervous system disorders
APHASIA
0.00%
0/2
12.5%
2/16
11.1%
2/18
0.00%
0/4
Nervous system disorders
ATAXIA
0.00%
0/2
6.2%
1/16
5.6%
1/18
25.0%
1/4
Nervous system disorders
CEREBROSPINAL FLUID LEAKAGE
0.00%
0/2
0.00%
0/16
0.00%
0/18
25.0%
1/4
Nervous system disorders
DEPRESSED LEVEL OF CONSCIOUSNESS
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Nervous system disorders
DIZZINESS
0.00%
0/2
18.8%
3/16
16.7%
3/18
25.0%
1/4
Nervous system disorders
DROOLING
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Nervous system disorders
DYSARTHRIA
0.00%
0/2
6.2%
1/16
5.6%
1/18
25.0%
1/4
Nervous system disorders
DYSGEUSIA
0.00%
0/2
31.2%
5/16
27.8%
5/18
0.00%
0/4
Nervous system disorders
FACIAL NERVE DISORDER
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Nervous system disorders
HEADACHE
100.0%
2/2
18.8%
3/16
27.8%
5/18
25.0%
1/4
Nervous system disorders
HEMIPARESIS
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Nervous system disorders
IIIRD NERVE DISORDER
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Nervous system disorders
LETHARGY
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Nervous system disorders
MUSCLE SPASTICITY
50.0%
1/2
6.2%
1/16
11.1%
2/18
0.00%
0/4
Nervous system disorders
MYOCLONUS
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Nervous system disorders
NEUROPATHY PERIPHERAL
0.00%
0/2
12.5%
2/16
11.1%
2/18
0.00%
0/4
Nervous system disorders
NYSTAGMUS
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Nervous system disorders
PARAESTHESIA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Nervous system disorders
PERIPHERAL MOTOR NEUROPATHY
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Nervous system disorders
PYRAMIDAL TRACT SYNDROME
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Nervous system disorders
SCIATICA
0.00%
0/2
0.00%
0/16
0.00%
0/18
25.0%
1/4
Nervous system disorders
SOMNOLENCE
0.00%
0/2
18.8%
3/16
16.7%
3/18
0.00%
0/4
Nervous system disorders
SYNCOPE
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Psychiatric disorders
CONFUSIONAL STATE
0.00%
0/2
12.5%
2/16
11.1%
2/18
0.00%
0/4
Psychiatric disorders
DELIRIUM
0.00%
0/2
12.5%
2/16
11.1%
2/18
0.00%
0/4
Psychiatric disorders
DEPRESSION
0.00%
0/2
12.5%
2/16
11.1%
2/18
0.00%
0/4
Psychiatric disorders
INSOMNIA
0.00%
0/2
18.8%
3/16
16.7%
3/18
0.00%
0/4
Psychiatric disorders
IRRITABILITY
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Renal and urinary disorders
HAEMATURIA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Renal and urinary disorders
MICTURITION URGENCY
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Renal and urinary disorders
PROTEINURIA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Renal and urinary disorders
URINARY RETENTION
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Respiratory, thoracic and mediastinal disorders
APNOEA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Respiratory, thoracic and mediastinal disorders
ATELECTASIS
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Respiratory, thoracic and mediastinal disorders
COUGH
0.00%
0/2
12.5%
2/16
11.1%
2/18
0.00%
0/4
Respiratory, thoracic and mediastinal disorders
DYSPNOEA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Respiratory, thoracic and mediastinal disorders
EPISTAXIS
0.00%
0/2
0.00%
0/16
0.00%
0/18
25.0%
1/4
Respiratory, thoracic and mediastinal disorders
PNEUMONITIS
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Respiratory, thoracic and mediastinal disorders
PRODUCTIVE COUGH
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Skin and subcutaneous tissue disorders
ALOPECIA
100.0%
2/2
37.5%
6/16
44.4%
8/18
0.00%
0/4
Skin and subcutaneous tissue disorders
DERMATITIS ACNEIFORM
0.00%
0/2
6.2%
1/16
5.6%
1/18
25.0%
1/4
Skin and subcutaneous tissue disorders
DRY SKIN
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Skin and subcutaneous tissue disorders
ERYTHEMA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Skin and subcutaneous tissue disorders
HYPERHIDROSIS
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Skin and subcutaneous tissue disorders
PRURITUS
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Skin and subcutaneous tissue disorders
RASH
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Skin and subcutaneous tissue disorders
RASH MACULO-PAPULAR
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Vascular disorders
HAEMATOMA
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Vascular disorders
HYPERTENSION
0.00%
0/2
6.2%
1/16
5.6%
1/18
0.00%
0/4
Vascular disorders
HYPOTENSION
0.00%
0/2
12.5%
2/16
11.1%
2/18
0.00%
0/4
Vascular disorders
THROMBOSIS
50.0%
1/2
0.00%
0/16
5.6%
1/18
0.00%
0/4

Additional Information

Study Director

Novartis Pharmaceuticals

Phone: 862-778-8300

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 (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
  • Publication restrictions are in place

Restriction type: OTHER