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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01708174
First received: October 11, 2012
Last updated: August 7, 2017
Last verified: August 2017
Results First Received: March 31, 2017  
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

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Analyses were performed by treatment and by age group.

Reporting Groups
  Description
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.

Participant Flow for 2 periods

Period 1:   Treatment Period
    Sonidegib (LDE225) Children   Sonidegib (LDE225) Adults   Temozolomide (TMZ)
STARTED   2   16   4 
Pharmacokinetic Analysis Set   2   13   0 
COMPLETED   1   14   3 
NOT COMPLETED   1   2   1 
Withdrawal by Subject                1                0                0 
Progressive disease                0                1                0 
Death                0                0                1 
Adverse Event                0                1                0 

Period 2:   Survival Follow-up
    Sonidegib (LDE225) Children   Sonidegib (LDE225) Adults   Temozolomide (TMZ)
STARTED   1   10   0 
COMPLETED   1   10   0 
NOT COMPLETED   0   0   0 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
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.
Total Total of all reporting groups

Baseline Measures
   Sonidegib (LDE225) Children   Sonidegib (LDE225) Adults   Temozolomide (TMZ)   Total 
Overall Participants Analyzed 
[Units: Participants]
 2   16   4   22 
Age 
[Units: Years]
Median (Full Range)
 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 
[Units: Participants]
Count of 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% 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   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   [ Time Frame: from date first participant randomized, 13-Sep-2013 to date of data cut-off, 15-Nov-2016 ]

2.  Secondary:   Progression Free Survival (PFS) According to IRC From Date First Participant Randomized, 13-Sep-2013 to Date of Data Cut-off, 15-Nov-2016   [ Time Frame: from date first participant randomized, 13-Sep-2013 to date of data cut-off, 15-Nov-2016 ]

3.  Secondary:   PFS According to Local Investigator Assessment From Date First Participant Randomized, 13-Sep-2013 to Date of Data Cut-off, 15-Nov-2016   [ Time Frame: from date first participant randomized, 13-Sep-2013 to date of data cut-off, 15-Nov-2016 ]

4.  Secondary:   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   [ Time Frame: from date first participant randomized, 13-Sep-2013 to date of data cut-off, 15-Nov-2016 ]

5.  Secondary:   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   [ Time Frame: from date first participant randomized, 13-Sep-2013 to date of data cut-off, 15-Nov-2016 ]

6.  Secondary:   Overall Survival (OS) From Date First Participant Randomized, 13-Sep-2013 to Date of Data Cut-off, 15-Nov-2016   [ Time Frame: from date first participant randomized, 13-Sep-2013 to date of data cut-off, 15-Nov-2016 ]

7.  Secondary:   Pharmacokinetics (PK): Summary of Plasma Trough Concentrations for Sonidegib (LDE225)   [ Time Frame: Weeks 1, 3, 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49 and 53 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information
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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 agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
phone: 862-778-8300



Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01708174     History of Changes
Other Study ID Numbers: CLDE225C2301
Study First Received: October 11, 2012
Results First Received: March 31, 2017
Last Updated: August 7, 2017