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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01708174
Recruitment Status : Completed
First Posted : October 16, 2012
Results First Posted : June 14, 2017
Last Update Posted : August 11, 2017
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Medulloblastoma
Interventions Drug: LDE225
Drug: TMZ
Enrollment 22
Recruitment Details  
Pre-assignment Details Analyses were performed by treatment and by age group.
Arm/Group Title Sonidegib (LDE225) Children Sonidegib (LDE225) Adults Temozolomide (TMZ)
Hide Arm/Group Description 500 mg/m2 orally 600 mg orally 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.
Period Title: Treatment Period
Started 2 16 4
Pharmacokinetic Analysis Set 2 13 0
Completed 1 14 3
Not Completed 1 2 1
Reason Not Completed
Withdrawal by Subject             1             0             0
Progressive disease             0             1             0
Death             0             0             1
Adverse Event             0             1             0
Period Title: Survival Follow-up
Started 1 10 0
Completed 1 10 0
Not Completed 0 0 0
Arm/Group Title Sonidegib (LDE225) Children Sonidegib (LDE225) Adults Temozolomide (TMZ) Total
Hide Arm/Group Description 500 mg/m2 orally 600 mg orally 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 of all reporting groups
Overall Number of Baseline Participants 2 16 4 22
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 2 participants 16 participants 4 participants 22 participants
8.5
(4.0 to 13.0)
37.0
(24.0 to 51.0)
35.5
(31.0 to 38.0)
35.0
(4.0 to 51.0)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants 16 participants 4 participants 22 participants
Female
2
 100.0%
5
  31.3%
2
  50.0%
9
  40.9%
Male
0
   0.0%
11
  68.8%
2
  50.0%
13
  59.1%
1.Primary Outcome
Title 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
Hide Description 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.
Time Frame from date first participant randomized, 13-Sep-2013 to date of data cut-off, 15-Nov-2016
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Sonidegib (LDE225) Children Sonidegib (LDE225) Adults Temozolomide (TMZ)
Hide Arm/Group Description:
500 mg/m2 orally
600 mg orally
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.
Overall Number of Participants Analyzed 2 16 4
Measure Type: Number
Unit of Measure: Percentage of participants
0.0 18.8 0.0
2.Secondary Outcome
Title Progression Free Survival (PFS) According to IRC From Date First Participant Randomized, 13-Sep-2013 to Date of Data Cut-off, 15-Nov-2016
Hide Description 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.
Time Frame from date first participant randomized, 13-Sep-2013 to date of data cut-off, 15-Nov-2016
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS was analyzed. The FAS included all randomized and non-randomized participants who received at least one dose of study treatment.
Arm/Group Title Sonidegib (LDE225) Children Sonidegib (LDE225) Adults Temozolomide (TMZ)
Hide Arm/Group Description:
500 mg/m2 orally
600 mg orally
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.
Overall Number of Participants Analyzed 2 16 4
Median (95% Confidence Interval)
Unit of Measure: months
1.6 [1] 
(NA to NA)
3.3
(1.7 to 17.1)
2.9
(0.9 to 4.0)
[1]
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.Secondary Outcome
Title PFS According to Local Investigator Assessment From Date First Participant Randomized, 13-Sep-2013 to Date of Data Cut-off, 15-Nov-2016
Hide Description 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.
Time Frame from date first participant randomized, 13-Sep-2013 to date of data cut-off, 15-Nov-2016
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS was analyzed. The FAS included all randomized and non-randomized participants who received at least one dose of study treatment.
Arm/Group Title Sonidegib (LDE225) Children Sonidegib (LDE225) Adults Temozolomide (TMZ)
Hide Arm/Group Description:
500 mg/m2 orally
600 mg orally
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.
Overall Number of Participants Analyzed 2 16 4
Median (95% Confidence Interval)
Unit of Measure: months
NA [1] 
(1.6 to NA)
3.3
(1.6 to 13.0)
2.9
(0.9 to 4.0)
[1]
The median and upper limit of the 95% confidence interval were not estimable because there were too few events.
4.Secondary Outcome
Title 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
Hide Description 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.
Time Frame from date first participant randomized, 13-Sep-2013 to date of data cut-off, 15-Nov-2016
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Sonidegib (LDE225) Children Sonidegib (LDE225) Adults Temozolomide (TMZ)
Hide Arm/Group Description:
500 mg/m2 orally
600 mg orally
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.
Overall Number of Participants Analyzed 2 16 4
Measure Type: Number
Unit of Measure: Percentage of participants
0.0 25.0 0.0
5.Secondary Outcome
Title 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
Hide Description 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.
Time Frame from date first participant randomized, 13-Sep-2013 to date of data cut-off, 15-Nov-2016
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Sonidegib (LDE225) Children Sonidegib (LDE225) Adults Temozolomide (TMZ)
Hide Arm/Group Description:
500 mg/m2 orally
600 mg orally
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.
Overall Number of Participants Analyzed 2 16 4
Median (95% Confidence Interval)
Unit of Measure: months
NA [1] 
(NA to NA)
8.5
(4.1 to 16.6)
NA [1] 
(NA to NA)
[1]
The median and lower and upper 95% CI could not be calculated because there were too few events.
6.Secondary Outcome
Title Overall Survival (OS) From Date First Participant Randomized, 13-Sep-2013 to Date of Data Cut-off, 15-Nov-2016
Hide Description 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.
Time Frame from date first participant randomized, 13-Sep-2013 to date of data cut-off, 15-Nov-2016
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Sonidegib (LDE225) Children Sonidegib (LDE225) Adults Temozolomide (TMZ)
Hide Arm/Group Description:
500 mg/m2 orally
600 mg orally
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.
Overall Number of Participants Analyzed 2 16 4
Median (95% Confidence Interval)
Unit of Measure: months
NA [1] 
(4.5 to NA)
9.5
(4.9 to 15.6)
NA [1] 
(1.2 to NA)
[1]
The median and upper 95% CI could not be calculated because there were too few events.
7.Secondary Outcome
Title Pharmacokinetics (PK): Summary of Plasma Trough Concentrations for Sonidegib (LDE225)
Hide Description Blood samples were collected for assessment. The children's group was analyzed up until week 25 only.
Time Frame Weeks 1, 3, 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49 and 53
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Sonidegib (LDE225) Children Sonidegib (LDE225) Adults
Hide Arm/Group Description:
500 mg/m2 orally
600 mg orally
Overall Number of Participants Analyzed 2 13
Mean (Standard Deviation)
Unit of Measure: ng/mL
Week 1 (n=2,11) 0.00  (0.00) 0.00  (0.00)
Week 3 (n=2,10) 2890  (1240) 761  (519)
Week 5 (n=2,8) 4930  (1380) 1090  (700)
Week 7 (n=1,8) 2810 [1]   (NA) 1450  (842)
Week 9 (n=1,8) 4670 [2]   (NA) 1530  (682)
Week 13 (n=1,4) 3680 [2]   (NA) 2330  (1100)
Week 17 (n=1,6) 3060 [2]   (NA) 1880  (730)
Week 21 (n=1,5) 3770 [2]   (NA) 2270  (915)
Week 25 (n=1,4) 1890 [2]   (NA) 2050  (625)
Week 29 (n=NA,4) NA [3]   (NA) 2050  (906)
Week 33 (n=NA,4) NA [3]   (NA) 2180  (512)
Week 37 (n=NA,3) NA [3]   (NA) 2850  (306)
Week 41 (n=NA,4) NA [3]   (NA) 2370  (916)
Week 45 (n=NA,2) NA [3]   (NA) 2890  (290)
Week 49 (n=NA,2) NA [3]   (NA) 2330  (990)
Week 53 (n=NA,2) NA [3]   (NA) 2080  (764)
[1]
Standard deviation (SD) does not apply when n=1.
[2]
SD does not apply when n=1.
[3]
PK analysis for the Children group was done up until week 25 only.
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Sonidegib (LDE225) Children Sonidegib (LDE225) Adults Sonidegib (Total) Temozolomide (TMZ)
Hide Arm/Group Description 500 mg/m2 orally 600 mg orally Sonidegib (Total) 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.
All-Cause Mortality
Sonidegib (LDE225) Children Sonidegib (LDE225) Adults Sonidegib (Total) Temozolomide (TMZ)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/--   --/-- 
Hide Serious Adverse Events
Sonidegib (LDE225) Children Sonidegib (LDE225) Adults Sonidegib (Total) Temozolomide (TMZ)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/2 (100.00%)   9/16 (56.25%)   11/18 (61.11%)   1/4 (25.00%) 
Gastrointestinal disorders         
NAUSEA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
VOMITING  1  0/2 (0.00%)  2/16 (12.50%)  2/18 (11.11%)  0/4 (0.00%) 
General disorders         
GENERAL PHYSICAL HEALTH DETERIORATION  1  1/2 (50.00%)  0/16 (0.00%)  1/18 (5.56%)  0/4 (0.00%) 
PAIN  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
Infections and infestations         
BACTERIAL SEPSIS  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
CLOSTRIDIAL INFECTION  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
LUNG INFECTION  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
SEPSIS  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
Investigations         
ASPARTATE AMINOTRANSFERASE INCREASED  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
BLOOD CREATINE PHOSPHOKINASE INCREASED  1  0/2 (0.00%)  2/16 (12.50%)  2/18 (11.11%)  0/4 (0.00%) 
Metabolism and nutrition disorders         
DEHYDRATION  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
HYPERNATRAEMIA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
HYPONATRAEMIA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
Musculoskeletal and connective tissue disorders         
BACK PAIN  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
NECK PAIN  1  0/2 (0.00%)  0/16 (0.00%)  0/18 (0.00%)  1/4 (25.00%) 
Nervous system disorders         
SEIZURE  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
Product Issues         
THROMBOSIS IN DEVICE  1  1/2 (50.00%)  0/16 (0.00%)  1/18 (5.56%)  0/4 (0.00%) 
Psychiatric disorders         
CONFUSIONAL STATE  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
DRUG DEPENDENCE  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
HALLUCINATION  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
Respiratory, thoracic and mediastinal disorders         
COUGH  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
DYSPNOEA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
Vascular disorders         
EMBOLISM  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (19.0)
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Sonidegib (LDE225) Children Sonidegib (LDE225) Adults Sonidegib (Total) Temozolomide (TMZ)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/2 (100.00%)   16/16 (100.00%)   18/18 (100.00%)   4/4 (100.00%) 
Blood and lymphatic system disorders         
ANAEMIA  1  0/2 (0.00%)  2/16 (12.50%)  2/18 (11.11%)  0/4 (0.00%) 
THROMBOCYTOPENIA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
Cardiac disorders         
ATRIOVENTRICULAR BLOCK FIRST DEGREE  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
SINUS TACHYCARDIA  1  0/2 (0.00%)  2/16 (12.50%)  2/18 (11.11%)  0/4 (0.00%) 
Ear and labyrinth disorders         
EAR CONGESTION  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
VERTIGO  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
Endocrine disorders         
DIABETES INSIPIDUS  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
Eye disorders         
DIPLOPIA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
PHOTOPHOBIA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
VISION BLURRED  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
Gastrointestinal disorders         
ABDOMINAL DISTENSION  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
ABDOMINAL PAIN  1  0/2 (0.00%)  2/16 (12.50%)  2/18 (11.11%)  1/4 (25.00%) 
ABDOMINAL PAIN UPPER  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
AEROPHAGIA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
ANAL INCONTINENCE  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
CONSTIPATION  1  0/2 (0.00%)  6/16 (37.50%)  6/18 (33.33%)  1/4 (25.00%) 
DIARRHOEA  1  1/2 (50.00%)  3/16 (18.75%)  4/18 (22.22%)  0/4 (0.00%) 
DYSPHAGIA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
HAEMATOCHEZIA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
NAUSEA  1  0/2 (0.00%)  5/16 (31.25%)  5/18 (27.78%)  3/4 (75.00%) 
PARAESTHESIA ORAL  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
REGURGITATION  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
TOOTH DISCOLOURATION  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
TOOTH LOSS  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
VOMITING  1  2/2 (100.00%)  7/16 (43.75%)  9/18 (50.00%)  1/4 (25.00%) 
General disorders         
ASTHENIA  1  0/2 (0.00%)  2/16 (12.50%)  2/18 (11.11%)  1/4 (25.00%) 
FATIGUE  1  0/2 (0.00%)  7/16 (43.75%)  7/18 (38.89%)  2/4 (50.00%) 
GAIT DISTURBANCE  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
NON-CARDIAC CHEST PAIN  1  0/2 (0.00%)  0/16 (0.00%)  0/18 (0.00%)  1/4 (25.00%) 
OEDEMA PERIPHERAL  1  0/2 (0.00%)  2/16 (12.50%)  2/18 (11.11%)  1/4 (25.00%) 
PAIN  1  0/2 (0.00%)  2/16 (12.50%)  2/18 (11.11%)  0/4 (0.00%) 
PYREXIA  1  0/2 (0.00%)  2/16 (12.50%)  2/18 (11.11%)  0/4 (0.00%) 
Immune system disorders         
ALLERGY TO ARTHROPOD BITE  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
Infections and infestations         
ACINETOBACTER INFECTION  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
FOLLICULITIS  1  0/2 (0.00%)  2/16 (12.50%)  2/18 (11.11%)  0/4 (0.00%) 
ORAL HERPES  1  1/2 (50.00%)  0/16 (0.00%)  1/18 (5.56%)  0/4 (0.00%) 
RHINITIS  1  0/2 (0.00%)  0/16 (0.00%)  0/18 (0.00%)  1/4 (25.00%) 
UPPER RESPIRATORY TRACT INFECTION  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
URINARY TRACT INFECTION  1  0/2 (0.00%)  4/16 (25.00%)  4/18 (22.22%)  0/4 (0.00%) 
Investigations         
ACTIVATED PARTIAL THROMBOPLASTIN TIME PROLONGED  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
ALANINE AMINOTRANSFERASE INCREASED  1  0/2 (0.00%)  3/16 (18.75%)  3/18 (16.67%)  0/4 (0.00%) 
ASPARTATE AMINOTRANSFERASE INCREASED  1  0/2 (0.00%)  2/16 (12.50%)  2/18 (11.11%)  0/4 (0.00%) 
BLOOD ALKALINE PHOSPHATASE INCREASED  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
BLOOD CREATINE PHOSPHOKINASE INCREASED  1  1/2 (50.00%)  7/16 (43.75%)  8/18 (44.44%)  1/4 (25.00%) 
BLOOD CREATINE PHOSPHOKINASE MB INCREASED  1  1/2 (50.00%)  1/16 (6.25%)  2/18 (11.11%)  1/4 (25.00%) 
BLOOD CREATININE INCREASED  1  0/2 (0.00%)  3/16 (18.75%)  3/18 (16.67%)  0/4 (0.00%) 
BLOOD PHOSPHORUS INCREASED  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
BODY TEMPERATURE INCREASED  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
C-REACTIVE PROTEIN INCREASED  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
CRYSTAL URINE PRESENT  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
GRANULOCYTE COUNT INCREASED  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
LYMPHOCYTE COUNT DECREASED  1  0/2 (0.00%)  5/16 (31.25%)  5/18 (27.78%)  1/4 (25.00%) 
OXYGEN SATURATION DECREASED  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
PLATELET COUNT DECREASED  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  1/4 (25.00%) 
PROTHROMBIN TIME PROLONGED  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
RED BLOOD CELL COUNT DECREASED  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
RED BLOOD CELL SEDIMENTATION RATE INCREASED  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
WEIGHT DECREASED  1  0/2 (0.00%)  2/16 (12.50%)  2/18 (11.11%)  1/4 (25.00%) 
WHITE BLOOD CELL COUNT DECREASED  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
Metabolism and nutrition disorders         
DECREASED APPETITE  1  0/2 (0.00%)  4/16 (25.00%)  4/18 (22.22%)  0/4 (0.00%) 
FAILURE TO THRIVE  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
HYPERCALCAEMIA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
HYPERGLYCAEMIA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
HYPERURICAEMIA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
HYPOALBUMINAEMIA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
HYPOCALCAEMIA  1  0/2 (0.00%)  3/16 (18.75%)  3/18 (16.67%)  0/4 (0.00%) 
HYPOKALAEMIA  1  0/2 (0.00%)  4/16 (25.00%)  4/18 (22.22%)  0/4 (0.00%) 
HYPOMAGNESAEMIA  1  0/2 (0.00%)  2/16 (12.50%)  2/18 (11.11%)  0/4 (0.00%) 
HYPONATRAEMIA  1  0/2 (0.00%)  3/16 (18.75%)  3/18 (16.67%)  0/4 (0.00%) 
HYPOPHOSPHATAEMIA  1  0/2 (0.00%)  2/16 (12.50%)  2/18 (11.11%)  0/4 (0.00%) 
Musculoskeletal and connective tissue disorders         
ARTHRALGIA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
BACK PAIN  1  0/2 (0.00%)  4/16 (25.00%)  4/18 (22.22%)  0/4 (0.00%) 
MUSCLE SPASMS  1  0/2 (0.00%)  4/16 (25.00%)  4/18 (22.22%)  0/4 (0.00%) 
MYALGIA  1  1/2 (50.00%)  5/16 (31.25%)  6/18 (33.33%)  0/4 (0.00%) 
NECK PAIN  1  0/2 (0.00%)  3/16 (18.75%)  3/18 (16.67%)  1/4 (25.00%) 
PAIN IN EXTREMITY  1  1/2 (50.00%)  2/16 (12.50%)  3/18 (16.67%)  1/4 (25.00%) 
Nervous system disorders         
APHASIA  1  0/2 (0.00%)  2/16 (12.50%)  2/18 (11.11%)  0/4 (0.00%) 
ATAXIA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  1/4 (25.00%) 
CEREBROSPINAL FLUID LEAKAGE  1  0/2 (0.00%)  0/16 (0.00%)  0/18 (0.00%)  1/4 (25.00%) 
DEPRESSED LEVEL OF CONSCIOUSNESS  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
DIZZINESS  1  0/2 (0.00%)  3/16 (18.75%)  3/18 (16.67%)  1/4 (25.00%) 
DROOLING  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
DYSARTHRIA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  1/4 (25.00%) 
DYSGEUSIA  1  0/2 (0.00%)  5/16 (31.25%)  5/18 (27.78%)  0/4 (0.00%) 
FACIAL NERVE DISORDER  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
HEADACHE  1  2/2 (100.00%)  3/16 (18.75%)  5/18 (27.78%)  1/4 (25.00%) 
HEMIPARESIS  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
IIIRD NERVE DISORDER  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
LETHARGY  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
MUSCLE SPASTICITY  1  1/2 (50.00%)  1/16 (6.25%)  2/18 (11.11%)  0/4 (0.00%) 
MYOCLONUS  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
NEUROPATHY PERIPHERAL  1  0/2 (0.00%)  2/16 (12.50%)  2/18 (11.11%)  0/4 (0.00%) 
NYSTAGMUS  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
PARAESTHESIA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
PERIPHERAL MOTOR NEUROPATHY  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
PYRAMIDAL TRACT SYNDROME  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
SCIATICA  1  0/2 (0.00%)  0/16 (0.00%)  0/18 (0.00%)  1/4 (25.00%) 
SOMNOLENCE  1  0/2 (0.00%)  3/16 (18.75%)  3/18 (16.67%)  0/4 (0.00%) 
SYNCOPE  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
Psychiatric disorders         
CONFUSIONAL STATE  1  0/2 (0.00%)  2/16 (12.50%)  2/18 (11.11%)  0/4 (0.00%) 
DELIRIUM  1  0/2 (0.00%)  2/16 (12.50%)  2/18 (11.11%)  0/4 (0.00%) 
DEPRESSION  1  0/2 (0.00%)  2/16 (12.50%)  2/18 (11.11%)  0/4 (0.00%) 
INSOMNIA  1  0/2 (0.00%)  3/16 (18.75%)  3/18 (16.67%)  0/4 (0.00%) 
IRRITABILITY  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
Renal and urinary disorders         
HAEMATURIA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
MICTURITION URGENCY  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
PROTEINURIA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
URINARY RETENTION  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
Respiratory, thoracic and mediastinal disorders         
APNOEA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
ATELECTASIS  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
COUGH  1  0/2 (0.00%)  2/16 (12.50%)  2/18 (11.11%)  0/4 (0.00%) 
DYSPNOEA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
EPISTAXIS  1  0/2 (0.00%)  0/16 (0.00%)  0/18 (0.00%)  1/4 (25.00%) 
PNEUMONITIS  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
PRODUCTIVE COUGH  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
Skin and subcutaneous tissue disorders         
ALOPECIA  1  2/2 (100.00%)  6/16 (37.50%)  8/18 (44.44%)  0/4 (0.00%) 
DERMATITIS ACNEIFORM  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  1/4 (25.00%) 
DRY SKIN  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
ERYTHEMA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
HYPERHIDROSIS  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
PRURITUS  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
RASH  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
RASH MACULO-PAPULAR  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
Vascular disorders         
HAEMATOMA  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
HYPERTENSION  1  0/2 (0.00%)  1/16 (6.25%)  1/18 (5.56%)  0/4 (0.00%) 
HYPOTENSION  1  0/2 (0.00%)  2/16 (12.50%)  2/18 (11.11%)  0/4 (0.00%) 
THROMBOSIS  1  1/2 (50.00%)  0/16 (0.00%)  1/18 (5.56%)  0/4 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (19.0)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
Phone: 862-778-8300
Layout table for additonal information
Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01708174    
Other Study ID Numbers: CLDE225C2301
First Submitted: October 11, 2012
First Posted: October 16, 2012
Results First Submitted: March 31, 2017
Results First Posted: June 14, 2017
Last Update Posted: August 11, 2017