Choroideremia Research Foundation (CRF)
In partnership with the Choroideremia Research Foundation, 4DMT seeks to develop a gene therapy product optimized for intravitreal administration to treat Choroideremia.
4DMT is conducting a Natural History Study for Choroideremia (CHM).
Natural History Studies track disease progression and are an essential first step in developing efficient clinical stages of testing in rare diseases. The purpose of this study is to evaluate disease progression as well as determine the feasibility of measuring endpoints for subsequent clinical trials in the hopes of providing medical benefit through gene therapy to all choroideremia patients and their families.
Two clinical sites are actively recruiting for the study:
This website will be updated as additional study sites open throughout the United States.
Eligibility requirements include but are not limited to: participants must be male, age 14 years or older, and have a CLIA Certified Genetic Diagnosis with visual acuity ≥ 20/200 or better.
Study participation requirements:
Attend Study Site Visits
Email Questions to CHM@4Dmoleculartherapeutics.com
For more information about Choroideremia, visit the Choroideremia Research Foundation (CRF) online. Additional clinical trial information including contact numbers may be found at clinicaltrials.gov, NCT 02994368.
Saturday April 22, 2017, Country Inn and Suites, Brookfield, Wisconsin. Join the CRF for a free, one day seminar / meet and greet for all choroideremia patients and family members. This family friendly event will include speakers from the choroideremia patient community who will provide the latest updates on current and upcoming research, studies, clinical trials, free genetic testing and low vision resources.
Registration is free, lunch and refreshments will be provided. Discounted hotel rates are available for this event for those who wish to come early or stay beyond. Visit www.crfmidwest.org for more information and to register.
Will this meeting help you and your family members?
This event will bring to attendees the spirit of family and hope that we have come to know. You will also receive information on CHM research and therapy development advances and how to prepare to participate in clinical trials and studies.
Looking forward to seeing you in Wisconsin!