Clinical Trials Announced in Miami


The Bascom Palmer Eye Institute at the University of Miami has announced the opening of a clinical trial using gene therapy on Choroideremia patients.  The clinical trial, titled An Open Label Phase 2 Clinical Trial of Retinal Gene Therapy for Choroideremia Using an Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein 1 (REP1), was officially posted on the website on September 17th.  Led by Dr. Byron Lam, the phase II clinical trial will treat 6 male patients with a sub-retinal injection of the AAV2-REP1 gene therapy vector in one eye, using the second eye for comparison.  Patients will be followed for a total of 11 visits over 24 months with an additional 3 year follow-up period.  All patients are required to be 18 years or older, have a genetic diagnosis of Choroideremia, and have visible disease in their retina on examination.  The clinical trial team will be evaluating both the safety of the treatment as well as its effectiveness in treating the diseased eye.

“The Choroideremia Research Foundation is excited to report the announcement of clinical trials for Choroideremia at the Bascom Palmer Eye Institute,” says Christopher Moen, CRF President.  “Bascom Palmer is an internationally recognized leader in ophthalmology care, and we are excited that they and Dr. Lam have chosen to study Choroideremia.  This clinical trial will expand access to gene therapy to individuals with Choroideremia in the United States, and will continue progress toward an approved treatment.  We believe in the potential for a successful gene therapy treatment for Choroideremia, and this study will bring us even closer to that goal.”

The Bascom Palmer study marks the fourth clinical trial using gene therapy to treat Choroideremia around the globe.  Clinical trials are currently underway at the University of Oxford by Dr. Robert Maclaren, the University of Alberta by Dr. Ian Macdonald, and at Children’s Hospital of Philadelphia and the University of Pennsylvania by Spark Therapeutics. In 2014, Dr. Maclaren reported the preliminary results of the University of Oxford clinical trial in the medical journal The Lancet.  In the six months after treatment with this therapy, the first six patients showed improvement in their vision in dim light and the two patients who had impaired visual acuity at the start of the trial were able to read more lines on the eye chart.

Enrollment for this study is underway currently.  Individuals who are interested in learning more about the clinical trial, or who are considering enrolling as a patient, can go to the study page on the website for more details and contact information.

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The Choroideremia Research Foundation Inc. is an international, non-profit organization dedicated to raising funds to find a treatment or cure for Choroideremia, a rare inherited retinal degenerative disease that causes blindness.

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