Clinical Trial: Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Pilot Study of Ranibizumab (Lucentis) for Neurofibromas Associated With Neurofibromatosis 1

Brief Summary: The purpose of this research study is to determine if ranibizumab can prevent the growth of neurofibromas. We will also be collecting extra blood and serum samples to help us learn more about NF1. Ranibizumab is a drug that affects the development of blood vessels that feed tumors. It targets a substance in the body called VEGF (Vascular Endothelial Growth Factor). VEGF helps tumors to grow and survive by supporting the growth of blood vessels that bring nutrients to the tumor. VEGF is made by cancerous tumors and also by non-cancerous tumors such as neurofibromas.

Detailed Summary:

  • Participants will receive one injection of ranibizumab into 3 tumors on their skin (one dose per tumor) on day 1 of treatment. One other tumor will be injected with normal saline solution. The saline solution-called a control-is necessary to determine whether injections (without medicine) can cause a tumor to shrink. Tumors will be measured and photographed prior to treatment. Ranibizumab tumors will be removed on days 8, 15, and 29; the saline treated tumor will be removed on day 29.
  • Participants will come into the clinic once a week for a total of 4 weeks and then again on Days 35. 57 and 85 for post-treatment visits. Some of the following tests and procedures will be performed: physical examination (including photographs of tumors), review of current medications, vital signs, routine blood tests, serum chemistry blood tests, interstitial fluid pressure measurements and tumor samples.

Sponsor: Massachusetts General Hospital

Current Primary Outcome: Determine the effect of inhibiting VEGF signaling on tumor volume and tumor interstitial fluid pressure by local injection of ranibizumab into cutaneous neurofibromas. [ Time Frame: 2 years ]

Original Primary Outcome: Same as current

Current Secondary Outcome: To identify angiogenic molecules upregulated in neurofibromas treated with ranibizumab. [ Time Frame: 2 years ]

Original Secondary Outcome: Same as current

Information By: Massachusetts General Hospital

Dates:
Date Received: March 27, 2008
Date Started: March 2008
Date Completion:
Last Updated: July 6, 2016
Last Verified: July 2016