Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T (DCLNF1)
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|ClinicalTrials.gov Identifier: NCT02332902|
Recruitment Status : Completed
First Posted : January 7, 2015
Results First Posted : June 6, 2017
Last Update Posted : June 6, 2017
|Condition or disease||Intervention/treatment||Phase|
|Neurofibromatosis 1||Drug: Everolimus||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||24 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1- CRAD001CUS232T|
|Study Start Date :||February 2015|
|Actual Primary Completion Date :||March 2016|
|Actual Study Completion Date :||March 2016|
This is a single arm intervention using Everolimus
Everyone in the study will receive Everolimus at a starting dose of 10 mg daily and will be adjusted up or down by 2.5 mg at 2 week intervals to attain a trough concentration of 5-15 ng/ml
Other Name: Afinitor
- 3D Photographic Measurement of Surface Volume [ Time Frame: 6 months ]Photographs of selected lesions to measure surface volume.
- Number of Participants With Grade 3-4 Adverse Events [ Time Frame: 6 months ]Determination if orally administered Afinitor is safe in patients a indicated by lack of Grade 3-4 adverse events during the trial period.
- Determine How Orally Administered Everolimus Effects mTOR Signaling in NF-1 Tissues [ Time Frame: 6 months ]Quantification via immunohistochemical staining of PTEN, pS6, p4EBP-1, TSC2, mTOR, NF-1, pAKT, VEGF-A and IGF-IR expression in biopsied neurofibroma tissue samples.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02332902
|United States, Texas|
|The University of Texas Health Science Center at Houston|
|Houston, Texas, United States, 77030|
|Principal Investigator:||Mary Kay Koenig, MD||The University of Texas Health Science Center, Houston|