13 Participants Needed

Gene Therapy with 4D-110 for Choroideremia

Recruiting at 1 trial location
TA
XT
Overseen ByXuan Tran
Age: 18+
Sex: Male
Trial Phase: Phase 1
Sponsor: 4D Molecular Therapeutics
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

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).

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 gene therapy with 4D-110 for choroideremia safe?

Gene therapy using adeno-associated viral vectors for choroideremia has shown encouraging safety results in clinical trials. Most side effects were mild or moderate, and no serious toxicity was identified in preclinical studies. These findings suggest that the treatment is generally safe for humans.12345

How is the treatment 4D-110 for choroideremia different from other treatments?

The treatment 4D-110 is a gene therapy that uses an adeno-associated viral vector to deliver a functional copy of the gene responsible for producing Rab Escort Protein 1 (REP1), which is missing or defective in choroideremia patients. This approach is unique because it directly addresses the genetic cause of the disease, potentially restoring vision by improving REP1 function, unlike other treatments that may only manage symptoms.24567

What data supports the effectiveness of the treatment 4D-110 for choroideremia?

Research shows that gene therapy using a viral vector to deliver the REP1 protein can improve vision in patients with choroideremia. In a clinical trial, patients treated with this approach experienced a median gain of 4.5 letters in visual acuity, suggesting that this type of gene therapy can help sustain and improve vision.24567

Who Is on the Research Team?

SL

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

Both eyes must have ≥ 34 ETDRS letters (~20/200)
I have a confirmed CHM gene mutation.

Exclusion Criteria

My eyes are healthy enough for the treatment and won't affect the study's results.
I have received AAV treatment before.
I have an active eye infection or inflammation.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single intravitreal injection of 4D-110 at one of three dose levels

1 day
1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 months
Regular visits as per study protocol

What Are the Treatments Tested in This Trial?

Interventions

  • 4D-110
Trial Overview The study is testing a single injection into the eye of gene therapy called 4D-110 to see how safe and effective it is for treating Choroideremia. This involves using a virus (AAV) to deliver genetic material into cells.
How Is the Trial Designed?
3Treatment groups
Experimental Treatment
Group I: 4D-110 Dose 3Experimental Treatment1 Intervention
Group II: 4D-110 Dose 2Experimental Treatment1 Intervention
Group III: 4D-110 Dose 1Experimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

4D Molecular Therapeutics

Lead Sponsor

Trials
9
Recruited
850+

Published Research Related to This Trial

The long-term safety of AAV2-REP1 in choroideremia patients showed some adverse effects, including localized intraretinal immune responses and permanent retinal damage, raising concerns about its effectiveness in late-stage disease.
Antisense oligonucleotide (ASO) therapy demonstrated potential benefits, recovering significant amounts of normal RNA and protein in fibroblasts from trial patients, suggesting it may be a promising alternative treatment for certain subgroups of choroideremia patients.
AAV2-Mediated Gene Therapy for Choroideremia: 5-Year Results and Alternate Anti-sense Oligonucleotide Therapy.Zhai, Y., Xu, M., Radziwon, A., et al.[2023]

Citations

AAV-mediated gene therapy for choroideremia: preclinical studies in personalized models. [2022]
Beneficial effects on vision in patients undergoing retinal gene therapy for choroideremia. [2023]
Choroideremia Gene Therapy Phase 2 Clinical Trial: 24-Month Results. [2022]
AAV2-Mediated Gene Therapy for Choroideremia: 5-Year Results and Alternate Anti-sense Oligonucleotide Therapy. [2023]
Choroideremia: towards a therapy. [2018]
Two-Year Results After AAV2-Mediated Gene Therapy for Choroideremia: The Alberta Experience. [2022]
Subretinal timrepigene emparvovec in adult men with choroideremia: a randomized phase 3 trial. [2023]
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