Pilot Study of Ranibizumab (Lucentis) for Neurofibromas Associated With Neurofibromatosis 1
- 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.
Endpoint Classification: Pharmacodynamics Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Determine the effect of inhibiting VEGF signaling on tumor volume and tumor interstitial fluid pressure by local injection of ranibizumab into cutaneous neurofibromas.
Scott Plotkin, MD, PhD
Massachusetts General Hospital
United States: Institutional Review Board
|Massachusetts General Hospital||Boston, Massachusetts 02114-2617|