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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. Identifier: NCT02332902
Recruitment Status : Completed
First Posted : January 7, 2015
Results First Posted : June 6, 2017
Last Update Posted : June 6, 2017
Texas Neurofibromatosis Foundation
Information provided by (Responsible Party):
Mary Kay Koenig, The University of Texas Health Science Center, Houston

Brief Summary:
This trial is evaluating the use of oral Everolimus to determine if there is a reduction in the size of the disfiguring cutaneous lesions in patients with Neurofibromatosis 1 over a 6 month period. The evaluation will be done by 3D photography measuring volume with the LIFEVIZ Micro system.

Condition or disease Intervention/treatment Phase
Neurofibromatosis 1 Drug: Everolimus Phase 2

Detailed Description:
Qualifying subjects will have a diagnosis of Neurofibromatosis 1 and have disfiguring cutaneous lesions that can be measured by photography. The subjects will have photographs of the target lesions,biopsies of the lesions,and safety blood laboratory tests at 3 time points (baseline, 3 months and 6 months which is end of treatment). Everolimus will be taken orally for 6 months. Subjects will visit the clinic monthly for an exam and adverse event evaluation. Laboratory testing will be done at these visits if determined necessary by the PI.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 24 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Arm Intervention/treatment
Experimental: Intervention
This is a single arm intervention using Everolimus
Drug: 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

Primary Outcome Measures :
  1. 3D Photographic Measurement of Surface Volume [ Time Frame: 6 months ]
    Photographs of selected lesions to measure surface volume.

Secondary Outcome Measures :
  1. 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.

Other Outcome Measures:
  1. 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.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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 </= 2.0 mg/dL, ALT and AST </= 2.5 X ULN, INR </= 2

8. Patient must have adequate renal function, serum creatinine </= 1.5 X ULN 9. Patient must have adequate lipid profile, fasting serum cholesterol </= 300 mg/dL OR </= 7.75 mmol/L, fasting triglycerides </= X ULN

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 </= prior to start of 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.


Information from the National Library of Medicine

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 identifier (NCT number): NCT02332902

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United States, Texas
The University of Texas Health Science Center at Houston
Houston, Texas, United States, 77030
Sponsors and Collaborators
The University of Texas Health Science Center, Houston
Texas Neurofibromatosis Foundation
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Principal Investigator: Mary Kay Koenig, MD The University of Texas Health Science Center, Houston

Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Mary Kay Koenig, Associate Professor, The University of Texas Health Science Center, Houston Identifier: NCT02332902     History of Changes
Other Study ID Numbers: HSC-MS-14-0758
First Posted: January 7, 2015    Key Record Dates
Results First Posted: June 6, 2017
Last Update Posted: June 6, 2017
Last Verified: May 2017
Keywords provided by Mary Kay Koenig, The University of Texas Health Science Center, Houston:
mTOR inhibitor
Additional relevant MeSH terms:
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Neurofibromatosis 1
Anti-Infective Agents
Nerve Sheath Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Neoplastic Syndromes, Hereditary
Neurocutaneous Syndromes
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Peripheral Nervous System Diseases
Neuromuscular Diseases
Peripheral Nervous System Neoplasms
Nervous System Neoplasms
Antineoplastic Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Anti-Bacterial Agents
Antibiotics, Antineoplastic
Antifungal Agents