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

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ClinicalTrials.gov Identifier: NCT01419639
Recruitment Status : Completed
First Posted : August 18, 2011
Results First Posted : August 7, 2015
Last Update Posted : July 18, 2017
Sponsor:
Collaborators:
Novartis Pharmaceuticals
The Children's Tumor Foundation
Information provided by (Responsible Party):
New York University School of Medicine

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Neurofibromatosis Type II
Intervention Drug: Everolimus (RAD001) , Afinitor®
Enrollment 10
Recruitment Details 10 people enrolled; a total of 9 completed study
Pre-assignment Details  
Arm/Group Title RAD001
Hide Arm/Group Description RAD001 taken orally continuously until disease progression or unacceptable toxicity, dosed according to age
Period Title: Overall Study
Started 10
Completed 9
Not Completed 1
Arm/Group Title RAD001
Hide Arm/Group Description RAD001 taken orally continuously until disease progression or unacceptable toxicity, dosed according to age
Overall Number of Baseline Participants 10
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
<=18 years
2
  20.0%
Between 18 and 65 years
8
  80.0%
>=65 years
0
   0.0%
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 10 participants
27
(12 to 44)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
Female
3
  30.0%
Male
7
  70.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 10 participants
10
1.Primary Outcome
Title Radiographic Response
Hide Description To estimate the objective response rates to RAD001 in patients with NF2-related tumors including cranial nerve schwannomas, meningiomas and ependymomas. Radiographic response for study purposes = greater than or equal to 15% reduction in tumor volume in any of the target tumors (partial response). Complete disappearance of any of the target tumors = complete response. MRI of the brain and spine will be performed every 3 months. If an objective response (15% reduction in tumor volume compared to baseline) is observed in any target tumor or stable disease, drug will be continued.
Time Frame 1 Year
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title RAD001
Hide Arm/Group Description:
RAD001 taken orally continuously until disease progression or unacceptable toxicity, dosed according to age
Overall Number of Participants Analyzed 9
Overall Number of Units Analyzed
Type of Units Analyzed: Tumors
12
Measure Type: Number
Unit of Measure: participants
0
2.Primary Outcome
Title Change in Tumor Size From Baseline
Hide Description [Not Specified]
Time Frame 1 Year
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title RAD001
Hide Arm/Group Description:
RAD001 taken orally continuously until disease progression or unacceptable toxicity, dosed according to age
Overall Number of Participants Analyzed 9
Overall Number of Units Analyzed
Type of Units Analyzed: Tumors
12
Median (Full Range)
Unit of Measure: % of change in tumor size from baseline
5.72
(-14 to 25.71)
3.Secondary Outcome
Title Audiologic Response
Hide Description

Defined as improvement in speech discrimination score (SDS), defined as an improvement in the score above the 95% critical difference threshold, compared to baseline audiogram at initiation of treatment. Audiologic worsening: decrease in SDS score below the 95% critical difference threshold, compared to baseline audiogram at initiation of treatment.

Patients with vestibular schwannomas will receive baseline audiograms within 28 days before enrollments and subsequent audiograms at the time of each MRI.

Time Frame 1 Year
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title RAD001
Hide Arm/Group Description:
RAD001 taken orally continuously until disease progression or unacceptable toxicity, dosed according to age
Overall Number of Participants Analyzed 9
Measure Type: Number
Unit of Measure: participants
0
Time Frame Continuously throughout 1 year of study
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title RAD001
Hide Arm/Group Description RAD001 taken orally continuously until disease progression or unacceptable toxicity, dosed according to age
All-Cause Mortality
RAD001
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
RAD001
Affected / at Risk (%)
Total   0/10 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
RAD001
Affected / at Risk (%)
Total   10/10 (100.00%) 
Blood and lymphatic system disorders   
anemia   5/10 (50.00%) 
Gastrointestinal disorders   
constipation   1/10 (10.00%) 
diarrhea   4/10 (40.00%) 
mucositis oral   10/10 (100.00%) 
nausea   1/10 (10.00%) 
toothache   1/10 (10.00%) 
vomiting   1/10 (10.00%) 
General disorders   
chills   1/10 (10.00%) 
fatigue   6/10 (60.00%) 
fever   1/10 (10.00%) 
non-cardiac chest pain   1/10 (10.00%) 
Infections and infestations   
skin infection   1/10 (10.00%) 
Injury, poisoning and procedural complications   
bruising   1/10 (10.00%) 
Investigations   
alkaline phosphatase increased   2/10 (20.00%) 
AST increased   3/10 (30.00%) 
cholesterol high   7/10 (70.00%) 
lymphocyte count decreased   5/10 (50.00%) 
neutrophil count decreased   3/10 (30.00%) 
platelet count decreased   6/10 (60.00%) 
white blood cell decreased   4/10 (40.00%) 
Metabolism and nutrition disorders   
hyperglycemia   5/10 (50.00%) 
hypernatremia   5/10 (50.00%) 
hypertriglyceridemia   4/10 (40.00%) 
hypoglycemia   1/10 (10.00%) 
hyponatremia   1/10 (10.00%) 
hypophosphatemia   2/10 (20.00%) 
Musculoskeletal and connective tissue disorders   
arthralgia   1/10 (10.00%) 
muscle weakness- right sided   1/10 (10.00%) 
neck pain   1/10 (10.00%) 
pain (to bone; right clavicula)   1/10 (10.00%) 
pain (to extremity; left 4th finger)   1/10 (10.00%) 
pain (to right flank)   1/10 (10.00%) 
Nervous system disorders   
dysgeusia   1/10 (10.00%) 
headache   6/10 (60.00%) 
Psychiatric disorders   
anxiety   1/10 (10.00%) 
depression   1/10 (10.00%) 
Reproductive system and breast disorders   
azoospermia   1/10 (10.00%) 
irregular menstruation   1/10 (10.00%) 
Respiratory, thoracic and mediastinal disorders   
cough   1/10 (10.00%) 
dyspnea   1/10 (10.00%) 
epistaxis   1/10 (10.00%) 
nasal congestion   1/10 (10.00%) 
Skin and subcutaneous tissue disorders   
dry skin   3/10 (30.00%) 
pruritis   4/10 (40.00%) 
rash   5/10 (50.00%) 
rash acneiform   1/10 (10.00%) 
rash maculo-papular   1/10 (10.00%) 
scalp pain   1/10 (10.00%) 
Vascular disorders   
hypertension   2/10 (20.00%) 
Indicates events were collected by systematic assessment
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
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 )
First Submitted: August 17, 2011
First Posted: August 18, 2011
Results First Submitted: July 8, 2015
Results First Posted: August 7, 2015
Last Update Posted: July 18, 2017