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Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC)

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ClinicalTrials.gov Identifier: NCT01070316
Recruitment Status : Completed
First Posted : February 18, 2010
Results First Posted : September 23, 2016
Last Update Posted : March 13, 2017
Sponsor:
Collaborator:
Novartis
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: Epilepsy
Tuberous Sclerosis Complex
Intervention: Drug: Everolimus

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
23 participants were screened; 3 participants were not eligible to begin treatment and were not enrolled

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
Everolimus

Subjects will be administered study drug if they meet study criteria after 4 weeks of baseline phase. The starting dose will be 5 mg/m2/day, rounded to the nearest 2.5 mg/dose, to be taken daily.

Everolimus: Everolimus is available in tablet form. The starting dose will be 5 mg/m2/day, rounded to the nearest 2.5 mg/dose, to be taken daily. After two weeks, serum trough level will be measured and dose adjusted according to the following algorithm If Blood trough level is less than 2.5 ng/ml than increase dose by 5 mg/m2/day; If Blood trough level is 2.5-5.0 ng/ml than increase dose by 2.5 mg/m2/day; If Blood trough level is 5.1-10.0 ng/ml than increase dose by 0 mg/m2/day (no change); If Blood trough level is 10.1-15.0 ng/ml than decrease dose by 2.5 mg/m2/day


Participant Flow for 2 periods

Period 1:   Main Phase
    Everolimus
STARTED   20 
COMPLETED   20 
NOT COMPLETED   0 

Period 2:   Extension Phase
    Everolimus
STARTED   18 [1] 
COMPLETED   14 
NOT COMPLETED   4 
Lack of Efficacy                3 
Withdrawal by Subject                1 
[1] Two participants did not qualify for Extension Phase as defined by protocol



  Baseline Characteristics

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.
20 study subjects were consented, screened, enrolled, and received study drug

Reporting Groups
  Description
Everolimus

Main Study Phase:

Subjects will be administered study drug if they meet study criteria after 4 weeks of baseline phase. The starting dose will be 5 mg/m2/day, rounded to the nearest 2.5 mg/dose, taken daily.

Everolimus: Everolimus is available in tablet form. The starting dose will be 5 mg/m2/day, rounded to the nearest 2.5 mg/dose, to be taken daily. After two weeks, serum trough level will be measured and dose adjusted according to the following algorithm If trough level is less than 2.5 ng/ml than increase dose by 5 mg/m2/day; If trough level is 2.5-5.0 ng/ml than increase dose by 2.5 mg/m2/day; If trough level is 5.1-10.0 ng/ml than increase dose by 0 mg/m2/day (no change); If trough level is 10.1-15.0 ng/ml than decrease dose by 2.5 mg/m2/day

Following the 4 week titration, subjects will continue in an 8 week maintenance period.

If subjects qualify for the Extension Phase of the study, they will continue on study drug and be followed through 48 months.


Baseline Measures
   Everolimus 
Overall Participants Analyzed 
[Units: Participants]
 20 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      18  90.0% 
Between 18 and 65 years      2  10.0% 
>=65 years      0   0.0% 
Age 
[Units: Years]
Mean (Standard Deviation)
 8.8  (5.3) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      10  50.0% 
Male      10  50.0% 
Region of Enrollment 
[Units: Participants]
 
United States   20 


  Outcome Measures

1.  Primary:   Reduction in Seizure Frequency   [ Time Frame: Baseline (Weeks 1-4), Week 16 ]

2.  Primary:   Number of Participants Continuing Study Medication Over Time   [ Time Frame: Individual subjects will be assessed every 6 months for up to 48 months; aggregate analysis will take place at end of study ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

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

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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.


Results Point of Contact:  
Name/Title: Elizabeth Hoskins
Organization: Cincinnati Children's Hospital Medical Center
phone: (513) 803-7263
e-mail: elizabeth.hoskins@cchmc.org


Publications of Results:

Responsible Party: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier: NCT01070316     History of Changes
Other Study ID Numbers: 2009-0998
First Submitted: February 15, 2010
First Posted: February 18, 2010
Results First Submitted: August 1, 2016
Results First Posted: September 23, 2016
Last Update Posted: March 13, 2017