- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02332902
Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T (DCLNF1)
Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1- CRAD001CUS232T
Study Overview
Detailed Description
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
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Texas
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Houston, Texas, United States, 77030
- The University of Texas Health Science Center at Houston
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- Patient is at least 18 years of age at the time of enrollment
- Informed consent
- Compliance with trial requirements (photography, lab draws, medication schedules, and study visits)
- Diagnosis of NF 1 and has cutaneous neurofibromas that are located in in a region amenable to photography
- 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
- 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.
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Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Intervention
This is a single arm intervention using Everolimus
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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 Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
3D Photographic Measurement of Surface Volume of Cutaneous Neurofibroma Lesion
Time Frame: 6 months
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Photographs of selected lesions to measure surface volume.
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6 months
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Participants With Grade 3-4 Adverse Events
Time Frame: 6 months
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Determination if orally administered Afinitor is safe in patients a indicated by lack of Grade 3-4 adverse events during the trial period.
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6 months
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Other Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Determine How Orally Administered Everolimus Effects mTOR Signaling in NF-1 Tissues
Time Frame: 6 months
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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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6 months
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Collaborators and Investigators
Collaborators
Investigators
- Principal Investigator: Mary Kay Koenig, MD, The University of Texas Health Science Center, Houston
Publications and helpful links
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimated)
Study Record Updates
Last Update Posted (Estimated)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Nervous System Diseases
- Neoplasms by Histologic Type
- Neoplasms
- Genetic Diseases, Inborn
- Neuromuscular Diseases
- Neurodegenerative Diseases
- Neoplasms, Nerve Tissue
- Peripheral Nervous System Diseases
- Nervous System Neoplasms
- Heredodegenerative Disorders, Nervous System
- Neoplastic Syndromes, Hereditary
- Nerve Sheath Neoplasms
- Neurocutaneous Syndromes
- Peripheral Nervous System Neoplasms
- Neurofibromatoses
- Neurofibromatosis 1
- Neurofibroma
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Enzyme Inhibitors
- Antineoplastic Agents
- Immunosuppressive Agents
- Immunologic Factors
- Protein Kinase Inhibitors
- MTOR Inhibitors
- Everolimus
Other Study ID Numbers
- HSC-MS-14-0758
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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