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

  • STATUS
    Recruiting
  • participants needed
    15
  • sponsor
    4D Molecular Therapeutics
Updated on 19 February 2024

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).

Description

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 two dose levels in male patients with genetically-confirmed CHM.

Details
Condition Choroideremia
Age 18years - 100years
Treatment 4D-110
Clinical Study IdentifierNCT04483440
Sponsor4D Molecular Therapeutics
Last Modified on19 February 2024

Eligibility

Yes No Not Sure

Inclusion Criteria

Diagnosis of CHM defined as a pathogenic mutation in the CHM gene, confirmed by genetic testing
Both eyes must have 34 ETDRS letters (~20/200)

Exclusion Criteria

Clinically significant, active ocular or peri-ocular infection or inflammation in the study eye
Patient has previously received any AAV treatment
Ocular conditions or ocular media opacity in either eye that would preclude the planned treatment (i.e. IVT injection) or interfere with the interpretation of study endpoints (e.g. significant lens opacity)
Clear my responses

How to participate?

Step 1 Connect with a study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer  to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Investigator Avatar

Primary Contact

site

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider

Loading...

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer

Study AnnotationsStudy Notes

Notes added here are public and can be viewed by anyone. Notes added here are only available to you and those who you share with.

user name

Added by • 

 • 

Private

Reply by • Private
Loading...

Lorem ipsum dolor sit amet consectetur, adipisicing elit. Ipsa vel nobis alias. Quae eveniet velit voluptate quo doloribus maxime et dicta in sequi, corporis quod. Ea, dolor eius? Dolore, vel!

  The passcode will expire in None.
Loading...

No annotations made yet

Add a private note
  • abc Select a piece of text from the left.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.
Add a private note
  • abc Select a piece of text.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.