An Open-Label, Pilot Study of Bevacizumab in Subjects With Choroidal Neovascularization Secondary to Diseases Other Than Age-Related Macular Degeneration
Bevacizumab is a recombinant humanized monoclonal antibody against VEGF. It has been
approved by the FDA for the treatment of metastatic colon cancer 18. We hypothesize that
VEGF also plays a role in the development of CNV in pathologic myopia. Therefore, employing
a mode of therapy that would decrease the risks posed to eyes with attenuated sclera, we
have treated, through special approval by the Pharmacy and Therapeutic Committee of the
Johns Hopkins University School of Medicine, two patients with persistent myopic CNV with
intravenous bevacizumab 19. Despite multiple treatments with PDT, the CNV remained active
and vision continued to decline in these two index patients. After four infusions of
bevacizumab, the CNV became inactive. Six months after the last infusion in each patient,
the CNV showed no evidence of activity or leakage on fluorescein angiography. Vision also
improved in the diseased eyes of both patients. The two patients tolerated the infusions
well, with no adverse events detected. In particular, blood pressure remained stable and no
proteinuria was noted on serial analyses of 24-hour urine collections. The Bascom Palmer
Eye Institute at the University of Miami has also recently reported favorable outcomes of
bevacizumab administered in repeated doses to 14 patients (age > 65 years) with CNV
secondary to AMD that has been refractory to other therapies.
We propose a non-randomized, open-label pilot study to evaluate the effect of bevacizumab in
patients with CNV due to any cause other than AMD. This design will allow us to closely
monitor safety and tolerability of bevacizumab while we evaluate 3 bioactivity outcomes.
Based upon dramatic responses in two patients with CNV due to myopic degeneration, we
hypothesize that treatment with bevacizumab may have major advantages over current standard
Abbreviated: Contact Coordinator or Principal investigator for expanded criteria.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Visual acuity: In this pilot study, the sample size is not powered for clinical response. However, preliminary data on visual acuity will aid in the design of future randomized control trial.
each visit for study duration up to 24 mos
Quan D Nguyen, MD, MSc
Johns Hopkins University
United States: Food and Drug Administration