Gene Therapy with 4D-110 for Choroideremia
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new gene therapy, 4D-110, to determine its safety and effectiveness in treating choroideremia, a genetic condition that gradually leads to vision loss. The study involves a single eye injection of 4D-110 and examines the efficacy of different doses. Men with choroideremia confirmed by genetic testing and some remaining vision (approximately 20/200 vision) may qualify as candidates. As a Phase 1 trial, this research aims to understand how the treatment works in people, providing participants the opportunity to be among the first to receive this new therapy.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications.
Is there any evidence suggesting that 4D-110 is likely to be safe for humans?
Research has shown that 4D-110 is well-tolerated in early tests. Initial safety data from studies indicated that 4D-110 did not cause serious side effects, even at higher doses, and found no major adverse reactions. The treatment employs a gene therapy method involving an injection into the eye, differing from some other gene therapy techniques. Overall, early findings suggest that 4D-110 is safe for further testing.12345
Why do researchers think this study treatment might be promising for Choroideremia?
Researchers are excited about 4D-110 because it offers a novel approach to treating choroideremia. Unlike current treatments, which primarily focus on managing symptoms, 4D-110 is a gene therapy designed to address the root cause of the disease by delivering a functional copy of the CHM gene directly into the eye. This innovative method has the potential to halt or even reverse vision loss, providing hope for long-term improvement rather than just temporary relief. Additionally, 4D-110 is administered via an intravitreal injection, offering a targeted approach that could enhance effectiveness and patient outcomes.
What evidence suggests that 4D-110 might be an effective treatment for Choroideremia?
Research has shown that 4D-110, a gene therapy, could help treat choroideremia, a genetic eye condition. In this trial, participants will receive one of the different doses of 4D-110. In previous studies, patients who received this treatment saw an average improvement in their vision, reading 4.5 more letters on an eye chart. Early results also suggest that lower doses of 4D-110 are safe, with no serious side effects reported. This therapy provides a healthy version of the gene that is faulty in choroideremia, aiming to improve vision by addressing the underlying issue. Overall, the initial findings are promising for people with this condition.24678
Who Is on the Research Team?
Schonmei Lee, MD
Principal Investigator
4D Molecular Therapeutics
Are You a Good Fit for This Trial?
This trial is for male patients with a confirmed genetic condition called Choroideremia. Participants must have a certain level of vision, roughly around 20/200 or better in both eyes. They can't join if they've had previous AAV treatments, active eye infections/inflammation, or other eye conditions that could affect the treatment's success.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single intravitreal injection of 4D-110 at one of three dose levels
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- 4D-110
Find a Clinic Near You
Who Is Running the Clinical Trial?
4D Molecular Therapeutics
Lead Sponsor