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Trial record 2 of 20 for:    "Neurofibromatosis, Type I" | "Anti-Infective Agents"

Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T (DCLNF1)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02332902
Recruitment Status : Completed
First Posted : January 7, 2015
Results First Posted : June 6, 2017
Last Update Posted : June 6, 2017
Sponsor:
Collaborator:
Texas Neurofibromatosis Foundation
Information provided by (Responsible Party):
Mary Kay Koenig, The University of Texas Health Science Center, Houston

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Neurofibromatosis 1
Intervention Drug: Everolimus
Enrollment 24
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Intervention
Hide Arm/Group Description

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.

Period Title: Overall Study
Started 24
Completed 17
Not Completed 7
Arm/Group Title Intervention
Hide Arm/Group Description

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.

Overall Number of Baseline Participants 24
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 24 participants
<=18 years
0
   0.0%
Between 18 and 65 years
23
  95.8%
>=65 years
1
   4.2%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 24 participants
Female
17
  70.8%
Male
7
  29.2%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 24 participants
24
1.Primary Outcome
Title 3D Photographic Measurement of Surface Volume
Hide Description Photographs of selected lesions to measure surface volume.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intervention
Hide Arm/Group Description:

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.

Overall Number of Participants Analyzed 17
Mean (95% Confidence Interval)
Unit of Measure: mm^3
Baseline
32.54
(14.30 to 50.77)
6 months
33.93
(13.28 to 54.58)
2.Secondary Outcome
Title Number of Participants With Grade 3-4 Adverse Events
Hide Description Determination if orally administered Afinitor is safe in patients a indicated by lack of Grade 3-4 adverse events during the trial period.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intervention
Hide Arm/Group Description:

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.

Overall Number of Participants Analyzed 24
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
3.Other Pre-specified Outcome
Title Determine How Orally Administered Everolimus Effects mTOR Signaling in NF-1 Tissues
Hide Description 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.
Time Frame 6 months
Outcome Measure Data Not Reported
Time Frame 6 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Intervention
Hide Arm/Group Description

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.

All-Cause Mortality
Intervention
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Intervention
Affected / at Risk (%) # Events
Total   0/24 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Intervention
Affected / at Risk (%) # Events
Total   22/22 (100.00%)    
Ear and labyrinth disorders   
Foggy Hearing *  1/22 (4.55%)  1
Gastrointestinal disorders   
Gastrointestinal (GI) Upset *  9/22 (40.91%)  9
Decreased Per Oral Intake/Weight loss *  5/22 (22.73%)  5
Infections and infestations   
Tooth Infection *  3/22 (13.64%)  3
Urinary Tract Infection *  3/22 (13.64%)  3
Vaginitis *  2/22 (9.09%)  2
Metabolism and nutrition disorders   
Hypercholesterolemia *  1/22 (4.55%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Pheochromocytoma *  1/22 (4.55%)  1
Nervous system disorders   
Headache *  3/22 (13.64%)  3
Fatigue *  3/22 (13.64%)  3
Left Side Numbness *  1/22 (4.55%)  1
Renal and urinary disorders   
Bladder Leakage *  1/22 (4.55%)  1
Reproductive system and breast disorders   
Ammenorrhea *  1/22 (4.55%)  1
Ovarian Cyst *  1/22 (4.55%)  1
Respiratory, thoracic and mediastinal disorders   
Upper Respiratory Infection/Cough/Sinus Infection/Pneumonia/Pharyngitis *  12/22 (54.55%)  12
Pneumonitis *  1/22 (4.55%)  1
Skin and subcutaneous tissue disorders   
Stomatitis *  20/22 (90.91%)  20
Folliculitis/Impetigo/Rash/Itching *  9/22 (40.91%)  9
Erythema Nodosum *  2/22 (9.09%)  2
Thrush *  1/22 (4.55%)  1
Ingrown Toenail *  1/22 (4.55%)  1
Biopsy Infection *  1/22 (4.55%)  1
Vascular disorders   
Nose Bleed *  1/22 (4.55%)  1
*
Indicates events were collected by non-systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Mary Kay Koenig
Organization: The University of Texas Health Science Center at Houston
EMail: Mary.K.Koenig@uth.tmc.edu
Layout table for additonal information
Responsible Party: Mary Kay Koenig, The University of Texas Health Science Center, Houston
ClinicalTrials.gov Identifier: NCT02332902     History of Changes
Other Study ID Numbers: HSC-MS-14-0758
First Submitted: December 12, 2014
First Posted: January 7, 2015
Results First Submitted: May 9, 2017
Results First Posted: June 6, 2017
Last Update Posted: June 6, 2017