Trial Outcomes & Findings for Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T (NCT NCT02332902)
NCT ID: NCT02332902
Last Updated: 2023-12-20
Results Overview
Photographs of selected lesions to measure surface volume.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
24 participants
Primary outcome timeframe
6 months
Results posted on
2023-12-20
Participant Flow
2 of the 24 enrolled participants withdrew consent immediately and did not receive study drug.
Participant milestones
| Measure |
Intervention
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.
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|---|---|
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Overall Study
STARTED
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24
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Overall Study
Received Study Drug
|
22
|
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Overall Study
COMPLETED
|
17
|
|
Overall Study
NOT COMPLETED
|
7
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T
Baseline characteristics by cohort
| Measure |
Intervention
n=24 Participants
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.
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|---|---|
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Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
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Age, Categorical
Between 18 and 65 years
|
23 Participants
n=5 Participants
|
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Age, Categorical
>=65 years
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1 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
17 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
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Region of Enrollment
United States
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24 participants
n=5 Participants
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PRIMARY outcome
Timeframe: 6 monthsPhotographs of selected lesions to measure surface volume.
Outcome measures
| Measure |
Intervention
n=17 Participants
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.
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|---|---|
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3D Photographic Measurement of Surface Volume of Cutaneous Neurofibroma Lesion
Baseline
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32.54 mm^3
Interval 14.3 to 50.77
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3D Photographic Measurement of Surface Volume of Cutaneous Neurofibroma Lesion
6 months
|
33.93 mm^3
Interval 13.28 to 54.58
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SECONDARY outcome
Timeframe: 6 monthsPopulation: The 2 participants who withdrew consent and did not receive study drug were not analyzed for adverse events.
Determination if orally administered Afinitor is safe in patients a indicated by lack of Grade 3-4 adverse events during the trial period.
Outcome measures
| Measure |
Intervention
n=22 Participants
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.
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|---|---|
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Number of Participants With Grade 3-4 Adverse Events
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0 Participants
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OTHER_PRE_SPECIFIED outcome
Timeframe: 6 monthsQuantification via immunohistochemical staining of PTEN, pS6, p4EBP-1, TSC2, mTOR, NF-1, pAKT, VEGF-A and IGF-IR expression in biopsied neurofibroma tissue samples.
Outcome measures
Outcome data not reported
Adverse Events
Intervention
Serious events: 0 serious events
Other events: 22 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Intervention
n=22 participants at risk
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.
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|---|---|
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Skin and subcutaneous tissue disorders
Stomatitis
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90.9%
20/22 • Number of events 20 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Upper Respiratory Infection/Cough/Sinus Infection/Pneumonia/Pharyngitis
|
54.5%
12/22 • Number of events 12 • 6 months
|
|
Gastrointestinal disorders
Gastrointestinal (GI) Upset
|
40.9%
9/22 • Number of events 9 • 6 months
|
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Skin and subcutaneous tissue disorders
Folliculitis/Impetigo/Rash/Itching
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40.9%
9/22 • Number of events 9 • 6 months
|
|
Gastrointestinal disorders
Decreased Per Oral Intake/Weight loss
|
22.7%
5/22 • Number of events 5 • 6 months
|
|
Nervous system disorders
Headache
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13.6%
3/22 • Number of events 3 • 6 months
|
|
Infections and infestations
Tooth Infection
|
13.6%
3/22 • Number of events 3 • 6 months
|
|
Infections and infestations
Urinary Tract Infection
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13.6%
3/22 • Number of events 3 • 6 months
|
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Nervous system disorders
Fatigue
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13.6%
3/22 • Number of events 3 • 6 months
|
|
Skin and subcutaneous tissue disorders
Erythema Nodosum
|
9.1%
2/22 • Number of events 2 • 6 months
|
|
Infections and infestations
Vaginitis
|
9.1%
2/22 • Number of events 2 • 6 months
|
|
Skin and subcutaneous tissue disorders
Thrush
|
4.5%
1/22 • Number of events 1 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
4.5%
1/22 • Number of events 1 • 6 months
|
|
Renal and urinary disorders
Bladder Leakage
|
4.5%
1/22 • Number of events 1 • 6 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Pheochromocytoma
|
4.5%
1/22 • Number of events 1 • 6 months
|
|
Skin and subcutaneous tissue disorders
Ingrown Toenail
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4.5%
1/22 • Number of events 1 • 6 months
|
|
Vascular disorders
Nose Bleed
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4.5%
1/22 • Number of events 1 • 6 months
|
|
Metabolism and nutrition disorders
Hypercholesterolemia
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4.5%
1/22 • Number of events 1 • 6 months
|
|
Nervous system disorders
Left Side Numbness
|
4.5%
1/22 • Number of events 1 • 6 months
|
|
Skin and subcutaneous tissue disorders
Biopsy Infection
|
4.5%
1/22 • Number of events 1 • 6 months
|
|
Ear and labyrinth disorders
Foggy Hearing
|
4.5%
1/22 • Number of events 1 • 6 months
|
|
Reproductive system and breast disorders
Ammenorrhea
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4.5%
1/22 • Number of events 1 • 6 months
|
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Reproductive system and breast disorders
Ovarian Cyst
|
4.5%
1/22 • Number of events 1 • 6 months
|
Additional Information
Dr. Mary Kay Koenig
The University of Texas Health Science Center at Houston
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place