Targeting the Mechanisms Underlying Cutaneous Neurofibroma Formation in NF1: A Clinical Translational Approach.
|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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02777775|
Recruitment Status : Recruiting
First Posted : May 19, 2016
Last Update Posted : May 19, 2016
|Condition or disease||Intervention/treatment|
|Cutaneous Neurofibromas Neurofibromatosis Type 1 Plexiform Neurofibromas||Procedure: Skin Biopsy|
Skin tumors in Neurofibromatosis Type 1 (NF1) are very common and diminish quality of life. Apart from surgery, very few treatment options exist. In comparison to other types of tumors in NF1, skin tumors receive less attention in the research world because they almost never turn into cancer. Under the microscope, skin tumors and plexiform tumors look similar, however they do not share the same growth potential, nor do they appear at the same time during development. These differences suggest that skin tumors are driven by different factors than plexiform tumors.
In this study we will be harvesting CNFs from 3 groups of subjects in order to obtain a minimum of 18 tumors in three size categories: <5mm, 5-10mm, and >1cm. These sizes were selected based on the variable natural history of CNF progression where incipient lesions (typically <5mm) are more numerous and demonstrate a less aggressive growth pattern, whereas the intermediate group (5-10mm) represent a transition state towards growth to larger (>1cm) lesions. Interestingly, CNF growth tends to stall out at 3cm. We plan to do experiments that identify which factors are important to CNF progression, and then find matching drugs that can shrink the tumors.
|Study Type :||Observational|
|Estimated Enrollment :||74 participants|
|Official Title:||Targeting the Mechanisms Underlying Cutaneous Neurofibroma Formation in NF1: A Clinical Translational Approach.|
|Study Start Date :||May 2016|
|Estimated Primary Completion Date :||August 2018|
|Estimated Study Completion Date :||August 2018|
- Procedure: Skin Biopsy
The procedure to sample the cutaneous neurofibroma involves a skin incision and will take approximately 10 to 15 minutes. The tissue will be taken in the following way:
- The skin area will be cleaned
- The skin area will be numbed (by either a numbing cream or a tiny numbing shot)
- Cutaneous (skin) Neurofibroma: will be taken via a small incision and removal of the neurofibroma whenever possible.
- A small piece of normal (skin) tissue will be numbed as above and taken using a special skin biopsy device.
Afterward, the area will be cleaned and the skin put back together with medical super glue or 1 to 2 dissolvable stitches that will dissolve as the incision heals, they do not require removal. The purpose of taking this piece of skin is to compare normal skin with the skin of a cutaneous neurofibroma.
- Harvesting Up To 74 CNFs From Patients to obtain 8 tumors in three size categories: <5mm, 5-10mm, and >1cm. [ Time Frame: 2 Years ]Identify which factors are important to CNF progression, and then find matching drugs that can shrink the tumors and improve quality of life.
- UV Exposure and NF1 History Questionaires [ Time Frame: 2 Years ]Measuring the relationship between sun exposure and CNF growth and development.
Biospecimen Retention: Samples With DNA
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 ClinicalTrials.gov identifier (NCT number): NCT02777775
|Contact: Heidi A De Vries, BSemail@example.com|
|Contact: Mathew R Steensma, MDfirstname.lastname@example.org|
|United States, Michigan|
|Spectrum Health Hospital Group||Recruiting|
|Grand Rapids, Michigan, United States, 49503|
|Contact: Heidi De Vries, BS 616-486-2076 email@example.com|
|Principal Investigator: Matthew Steensma, MD|
|Principal Investigator:||Matthew R Steensma, MD||Spectrum Health Hospitals|