EMAIL SIGNUP

DONATE NOW

CONTACT US

Ongoing Trials

Dr. Ian MacDonald in the lab.

Ongoing clinical trials play an important role in advancing potential treatments for choroideremia. These studies allow researchers to track long-term safety and effectiveness, gather real-world data, and better understand how emerging therapies may benefit individuals with CHM. Even when enrollment is closed, the progress of active trials provides valuable insight that helps shape future research efforts. Patients who may want to participate in upcoming studies are encouraged to pursue genetic testing to confirm a CHM diagnosis, as it is a key requirement for eligibility.

Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia

NCT: NCT04483440
URL:  https://clinicaltrials.gov/study/NCT04483440
Intervention: BIOLOGICAL: Injection of AAV2-REP1 (10e11 vg)
Sponsor: 4D Molecular Therapeutics

NOTE: Enrollment for this study is now closed.

Technical Summary: This study will evaluate safety, tolerability, and preliminary efficacy of a single intravitreal (IVT) injection of a recombinant adeno-associated virus (AAV) gene therapy, 4D-110, in male patients with genetically-confirmed Choroideremia (CHM).  This is an open-label, Phase 1 study to evaluate safety and tolerability as well as preliminary efficacy of a single IVT injection of 4D-110 at three dose levels in male patients with genetically-confirmed CHM. 13 patients were enrolled and are currently being monitored. The study is estimated to be completed in 2027.

Lay Language Summary: This study is testing the safety and early signs of effectiveness of a gene therapy called 4D-110. The treatment is given as a single injection into the eye. Only males with genetically confirmed choroideremia are included. This is a Phase 1 study, which means the main goal is to understand whether the treatment is safe and how well people tolerate it. Researchers are also watching for any early improvements or changes in vision. Thirteen participants have enrolled and continue to be monitored. The study is expected to finish in 2027.