Clinical Trial: CNTF Implants for CNGB3 Achromatopsia
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: A Phase I/II Study of the NT-501 Intraocular Implant Releasing Ciliary Neurotrophic Factor (CNTF) in Participants With CNGB3 Achromatopsia
Brief Summary:
Background:
- Achromatopsia is an inherited condition that causes vision loss because cells in the retina do not work properly. It causes loss of acuity, sensitivity to light, and loss of color vision. There are no effective treatments for achromatopsia.
- Four genes currently are known to cause achromatopsia. One of these, the cyclic nucleotide-gated channel beta 3 (CNGB3) gene, is the cause in about 50 percent of people.
- CNTF is a natural chemical found in the body that promotes survival and function of nerve cells. CNTF has been shown to be effective in treating retinal disease in animals and can slow vision loss.
- CNTF has also been studied in over 250 people with retinal disease other than achromatopsia. In these studies, a CNTF implant was placed into the eye during a simple surgery. The implant releases CNTF inside the eye, near the retina. These studies suggested that a CNTF implant might help vision in some eye diseases.
Objectives:
- To learn whether a CNTF implant is safe for people with CNGB3 achromatopsia.
- To learn whether CNTF can improve visual acuity or color vision, and whether it may reduce sensitivity to light in people with CNGB3 achromatopsia.
Eligibility:
You may be able to take part in this study if you:
- Are at least 18 years old.
- Test positive for mutations in the CNGB3 gene and have no mutations in another achromatopsia gene.
