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Phase II Study of Everolimus (RAD001) in Children and Adults With Neurofibromatosis Type 2

This study has been completed.
Sponsor:
Collaborators:
Novartis Pharmaceuticals
The Children's Tumor Foundation
Information provided by (Responsible Party):
New York University School of Medicine
ClinicalTrials.gov Identifier:
NCT01419639
First received: August 17, 2011
Last updated: June 14, 2016
Last verified: June 2016
Results First Received: July 8, 2015  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Neurofibromatosis Type II
Intervention: Drug: Everolimus (RAD001) , Afinitor®

  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
10 people enrolled; a total of 9 completed study

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
RAD001 RAD001 taken orally continuously until disease progression or unacceptable toxicity, dosed according to age

Participant Flow:   Overall Study
    RAD001  
STARTED     10  
COMPLETED     9  
NOT COMPLETED     1  



  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
RAD001 RAD001 taken orally continuously until disease progression or unacceptable toxicity, dosed according to age

Baseline Measures
    RAD001  
Number of Participants  
[units: participants]
  10  
Age  
[units: participants]
 
<=18 years     2  
Between 18 and 65 years     8  
>=65 years     0  
Age  
[units: years]
Median (Full Range)
  27  
  (12 to 44)  
Gender  
[units: participants]
 
Female     3  
Male     7  
Region of Enrollment  
[units: participants]
 
United States     10  



  Outcome Measures
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1.  Primary:   Radiographic Response   [ Time Frame: 1 Year ]

2.  Primary:   Change in Tumor Size From Baseline   [ Time Frame: 1 Year ]

3.  Secondary:   Audiologic Response   [ Time Frame: 1 Year ]


  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:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Matthias A. Karajannis
Organization: NYU Langone Medical Center
phone: 212 263 9959
e-mail: matthias.karajannis@nyumc.org



Responsible Party: New York University School of Medicine
ClinicalTrials.gov Identifier: NCT01419639     History of Changes
Other Study ID Numbers: RAD001 NF2 (11-00587)
2010-10-011 ( Other Grant/Funding Number: Children's Tumor Foundation )
Study First Received: August 17, 2011
Results First Received: July 8, 2015
Last Updated: June 14, 2016
Health Authority: United States: Institutional Review Board