Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT02332902
• Date of registration: 12/12/2014
• Prospective Registration: Yes
• Primary sponsor: The University of Texas Health Science Center, Houston
• Public title: Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T DCLNF1
• Scientific title: Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1- CRAD001CUS232T
• Date of first enrolment: February 2015
• Target sample size: 24
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02332902
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Mary Kay Koenig, MD
• Affiliation: The University of Texas Health Science Center, Houston

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Patient is at least 18 years of age at the time of enrollment

2. Informed consent

3. Compliance with trial requirements (photography, lab draws, medication schedules, and
study visits)

4. Diagnosis of NF 1 and has cutaneous neurofibromas that are located in in a region
amenable to photography

5. Females of child bearing potential must not be pregnant as confirmed by a negative
pregnancy test (blood beta-hCG level) prior to study enrollment and must agree to use
appropriate contraceptive methods for the duration of the trial

6. Patient must have adequate liver function as shown by, total bilirubin ALT and AST
8. Patient must have adequate renal function, serum creatinine must have adequate lipid profile, fasting serum cholesterol mmol/L, fasting triglycerides
Exclusion Criteria:

1. Patients currently receiving anticancer therapies or who have received anticancer
therapies within 4 weeks of the start of Everolimus (including chemotherapy, radiation
therapy, antibody based therapy, etc.) 2. Known intolerance or hypersensitivity to
Everolimus or other rapamycin analogs (e.g. sirolimus, temsirolimus) 3. Known impairment of
gastrointestinal (GI) function or GI disease that may significantly alter the absorption of
oral Everolimus 4. Uncontrolled diabetes mellitus despite adequate therapy 5. Patients who
have any severe and /or uncontrolled medical conditions such as: unstable angina pectoris,
symptomatic congestive heart failure, myocardial infarction Everolimus, serious uncontrolled cardiac arrhythmia, or any other clinically significant
cardiac disease, symptomatic congestive heart failure of New York Hear Association Class
III or IV, known active (acute or chronic) or uncontrolled severe infection, liver disease
such as cirrhosis, decompensated liver disease, or chronic hepatitis, known severely
impaired lung function (spirometry and DLCO 50% or less of normal and O2 saturation 88% or
less at rest on room air), active, bleeding diathesis, chronic treatment with
corticosteroids, or other immunosuppressive agents, topical or inhaled corticosteroids are
allowed, know history of HIV seropositivity, patients who have received live attenuated
vaccines within 1 week of start of Everolimus. Patient would avoid close contact with
others who have received live attenuated vaccines during the study, patients who have a
history of primary malignancy, with the exceptions of mon-melanoma skin cancer, and
carcinoma in situ of the cervix, uteri, or breast from which the patient has been disease
free for >/- 3 years, patients with a history of non compliance to medical regimens or who
are considered potentially unreliable or will not be able to complete the entire study,
patients who are currently part of or have participated in any clinical investigation with
an investigational drug within 1 month prior to dosing, pregnant or nursing (lactating)
women, women of child-bearing potential (WOCBP), defined as all women physiologically
capable of becoming pregnant, unless they are using highly effective methods of
contraception during dosing of study treatment. highly effective contraception methods,male
patients whose sexual partner(s) are WOCBP who are not willing to use adequate
contraception, during the study and for 8 weeks after the end of treatment.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Neurofibromatosis 1

Intervention(s)

Drug: Everolimus

Primary Outcome(s)

3D Photographic Measurement of Surface Volume [Time Frame: 6 months]

Secondary Outcome(s)

Number of Participants With Grade 3-4 Adverse Events [Time Frame: 6 months]

Secondary ID(s)

HSC-MS-14-0758

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Texas Neurofibromatosis Foundation

Results

• Results available: Yes
• Date Posted: 06/06/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02332902