NCT00657202

Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1

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

• Status: Completed
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: Massachusetts General Hospital · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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 description

* 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.

Conditions

• Neurofibromatosis Type 1
• Cutaneous Neurofibromas

Interventions

Type	Name	Description
DRUG	Ranibizumab	Given as an injection into three cutaneous neurofibromas on day 1 of the study treatment.

Timeline

• Start date: 2008-03-01
• Primary completion: 2009-10-01
• Completion: 2013-12-01
• First posted: 2008-04-14
• Last updated: 2016-07-07

Locations

1 site across 1 country: United States