4DMT is accepting inquires from interested choroideremia (CHM) patients for their upcoming Natural History Study. In partnership with the Choroideremia Research Foundation, 4DMT seeks to develop a gene therapy product optimized for intravitreal administration to treat Choroideremia.
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.
Enrollment for 50 patients begins in fall 2016 at 5 study sites throughout the United States. We will be updating you as the study opens and patients are being accepted.
Eligibility requirements include but are not limited to: participants must be 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
- One visit every 6 months, for 24 months
- Medical history review
- Visual and eye exam
- Photography and imaging
Email questions to CHM@4Dmoleculartherapeutics.com