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Gene Therapy

Gene Therapy with 4D-110 for Choroideremia

Phase 1
Waitlist Available
Research Sponsored by 4D Molecular Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Diagnosis of CHM defined as a pathogenic mutation in the CHM gene, confirmed by genetic testing
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 24 months
Awards & highlights

Study Summary

This trial will test a gene therapy for safety and effectiveness in treating Choroideremia, a disease that causes blindness.

Who is the study for?
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.Check my eligibility
What is being tested?
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.See study design
What are the potential side effects?
Potential side effects may include discomfort at the injection site, inflammation inside the eye, changes in vision, and possible immune reactions to the viral vector used in gene therapy.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have a confirmed CHM gene mutation.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Frequency and severity of ocular and systemic adverse events (AEs)

Trial Design

2Treatment groups
Experimental Treatment
Group I: 4D-110 Dose 2Experimental Treatment1 Intervention
4D-110 IVT injection
Group II: 4D-110 Dose 1Experimental Treatment1 Intervention
4D-110 IVT injection

Find a Location

Who is running the clinical trial?

4D Molecular TherapeuticsLead Sponsor
7 Previous Clinical Trials
360 Total Patients Enrolled
1 Trials studying Choroideremia
57 Patients Enrolled for Choroideremia
Robert Kim, MDStudy Director4D Molecular Therapeutics
2 Previous Clinical Trials
78 Total Patients Enrolled
1 Trials studying Choroideremia
57 Patients Enrolled for Choroideremia
Schonmei Lee, MDStudy Director4D Molecular Therapeutics
4 Previous Clinical Trials
300 Total Patients Enrolled
1 Trials studying Choroideremia
57 Patients Enrolled for Choroideremia

Media Library

4D-110 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04483440 — Phase 1
Choroideremia Research Study Groups: 4D-110 Dose 1, 4D-110 Dose 2
Choroideremia Clinical Trial 2023: 4D-110 Highlights & Side Effects. Trial Name: NCT04483440 — Phase 1
4D-110 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04483440 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What potential harms does 4D-110 pose to individuals?

"Limited data on the safety and efficacy of 4D-110 resulted in it receiving a score of 1."

Answered by AI

Are there still openings for participants in this investigation?

"Clinicaltrials.gov states that this experiment is in the process of gathering participants, having been first posted on June 2nd 2020 and updated most recently on September 17th 2021."

Answered by AI

How many individuals are being accepted into this medical experiment?

"Correct. Clinicaltrials.gov corroborates that this clinical trial, originally posted on June 2nd 2020, is actively recruiting. This trial needs to enroll a total of 15 patients across two medical sites."

Answered by AI

Who else is applying?

What state do they live in?
Texas
How old are they?
18 - 65
What site did they apply to?
Retina Foundation of the Southwest
What portion of applicants met pre-screening criteria?
Did not meet criteria
Recent research and studies
clinicaltrials.govStudy
Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia
2020. "Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04483440.
All > Choroideremia
~0 spots leftby Jun 2024
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