4DMT Now Recruiting for Natural History Studies!
February 10th, 2017 by Cory MacDonald
4D Molecular Therapeutics (4DMT), a leader in gene therapy product discovery and development, is accepting patients for enrollment in its Choroideremia Natural History Study (NHS). This study is an important step in developing a groundbreaking gene therapy product optimized for intravitreal administration to treat Choroideremia (CHM) patients. 4DMT has deployed its proprietary AAV vector discovery platform, Therapeutic Vector Evolution, to create and optimize a proprietary AAV vector for intravitreal delivery to the retina. This vector is designated to be the basis for the first 4DMT experimental gene therapeutic targeted for the treatment of CHM. 4DMT is working in close collaboration with the Choroideremia Research Foundation on CHM product development and the Natural History Study.
Choroideremia Natural History Study (NHS) Purpose
The NHS is a multi-center US-based study designed to evaluate disease progression in a wide variety of individuals with CHM. Understanding more about clinical endpoints as the disease progresses will aid in identifying both potential participants and the best clinical measures for upcoming 4DMT clinical trials. Participants in the study will be assessed every six months over two years. At each visit, they will undergo a series of photographic, imaging and clinical evaluations.
The study is accepting males with a confirmed CHM diagnosis aged 14 and up. There is no exclusion for good visual acuity. Additional information about the NHS study criteria, locations, and contact information may be found at clinicaltrials.gov
Two clinical sites are actively recruiting for the study:
Retina Foundation of the Southwest
9600 N Central Expressway, Suite 200
Dallas, TX 75231
Contact: Kirsten Locke
Tel: 214-363-3911 ext. 114
Principal Investigator: David Birch, PhD
Retina-Vitreous Associates Medical Group
1127 Wilshire Boulevard, #1620
Los Angeles, CA 90017
Contact: Janet Kurokouchi
Tel: 310-289-2478 – Clinical Trial Department
Principal Investigator: David Liao, MD
A third site for the NHS, the Moran Eye Center at the University of Utah, is not currently enrolling patients but expects to do so in the near future. They are currently compiling a list of interested CHM patients. If you are interested in enrolling at this study site contact:
John A. Moran Eye Center – University of Utah
65 Mario Capecchi Drive
Salt Lake City, UT 84132
Contact: Katie Rogers
Tel: 801-581-2352 – Ask for Clinical Study Staff Office
Principal Investigator: Paul Bernstein, MD
“The Choroideremia Research Foundation and its community are thrilled at the initiation of 4DMT’s Natural History Study. This milestone brings us one step closer to a treatment that could end blindness from CHM,” said Christopher Moen, MD, President of the Choroideremia Research Foundation.
“This clinical study is a significant milestone for the company and a critical step forward for our lead program to treat choroideremia. We are convinced the data generated in this study will accelerate the clinical development of our lead product,” said Dr. David Kirn, co-founder and CEO, 4D Molecular Therapeutics.