Everolimus for Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis1 CRAD001CUS232T

NCT02332902 | Completed Phase 2 Results

• 1 other identifier: HSC-MS-14-0758
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

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.

Conditions

Neurofibromatosis 1

Keywords

Neurofibroma; NF-1; Everolimus; mTOR inhibitor

Outcome Measures

Primary Outcomes (1)

• 3D Photographic Measurement of Surface Volume of Cutaneous Neurofibroma Lesion

  Photographs of selected lesions to measure surface volume.

  6 months

Secondary Outcomes (1)

• Number of Participants With Grade 3-4 Adverse Events

  6 months

Other Outcomes (1)

• Determine How Orally Administered Everolimus Effects mTOR Signaling in NF-1 Tissues

  6 months

Study Arms (1)

Intervention

EXPERIMENTAL

This is a single arm intervention using Everolimus

Drug: Everolimus

Interventions

Everolimus DRUG

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

Also known as: Afinitor

Intervention

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• \. 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

You may not qualify if:

• \. 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.

Sponsors & Collaborators

• The University of Texas Health Science Center, Houston: lead
• Texas Neurofibromatosis Foundation: collaborator

Study Sites (1)

The University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

Location

Related Publications (1)

• Slopis JM, Arevalo O, Bell CS, Hebert AA, Northrup H, Riascos RF, Samuels JA, Smith KC, Tate P, Koenig MK. Treatment of Disfiguring Cutaneous Lesions in Neurofibromatosis-1 with Everolimus: A Phase II, Open-Label, Single-Arm Trial. Drugs R D. 2018 Dec;18(4):295-302. doi: 10.1007/s40268-018-0248-6.

  PMID: 30284154 RESULT

MeSH Terms

Conditions

Neurofibromatosis 1; Neurofibroma

Interventions

Everolimus

Condition Hierarchy (Ancestors)

Neurofibromatoses; Nerve Sheath Neoplasms; Neoplasms, Nerve Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Peripheral Nervous System Neoplasms; Nervous System Neoplasms

Intervention Hierarchy (Ancestors)

Sirolimus; Macrolides; Lactones; Organic Chemicals

Results Point of Contact

• Title: Dr. Mary Kay Koenig
• Organization: The University of Texas Health Science Center at Houston

Mary.K.Koenig@uth.tmc.edu

Study Officials

• Mary Kay Koenig, MD

  The University of Texas Health Science Center, Houston

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Study Record Dates

• First Submitted: December 12, 2014
• First Posted: January 7, 2015
• Study Start: February 1, 2015
• Primary Completion: March 1, 2016
• Study Completion: March 1, 2016
• Last Updated: December 20, 2023
• Results First Posted: June 6, 2017

Record last verified: 2023-11

Locations

US (1)