Clinical Trial: Safety and Efficacy of a Single Subretinal Injection of rAAV.hCNGA3 in Patients With CNGA3-linked Achromatopsia

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: Safety and Efficacy of a Single Subretinal Injection of rAAV.hCNGA3 in Patients With CNGA3-linked Achromatopsia Investigated in an Exploratory, Dose-escalation Trial

Brief Summary: The purpose of this study is to proof the safety and efficacy of a single subretinal injection of rAAV.hCNGA3 in patients with CNGA3-linked achromatopsia.

Detailed Summary:

Title: Safety and efficacy of a single subretinal injection of rAAV.hCNGA3 in patients with CNGA3-linked achromatopsia investigated in an exploratory, dose-escalation trial

Phase: I/II

Indication: CNGA3-linked achromatopsia

Aim: To proof the safety and efficacy of rAAV.hCNGA3 in patients with achromatopsia

Study design: open, mono-center trial with fellow-eye comparison

Study Population:

Inclusion Criteria (Study Eye)

  • clinical diagnosis of achromatopsia
  • ≥ 18 years of age
  • confirmed mutation in CNGA3
  • BCVA ≥ 20/400
  • a minimal outer nuclear layer thickness of 10µm at 3° eccentricity in the study eye (normal = 38±6µm)
  • ability to understand and willingness to consent to study protocol
  • no infection with Human Immunodeficiency Virus (HIV)
  • negative pregnancy test in women with childbearing potential (a woman who is two years post-menopausal or surgically sterile is not considered to be of childbearing potential)

Exclusion Criteria

  • additional interfering eye conditions (e.g. uveitis, advanced cataract) in the study eye
  • systemic conditions (e.g. coronary heart disease, autoimmune disorders) which may affect study pa
    Sponsor: STZ eyetrial

    Current Primary Outcome: Safety (AE). Number of Participants With Abnormal Laboratory Values and/or Adverse Events That Are Related to Treatment [ Time Frame: Day 0 - Day 365 ]

    Safety as the primary endpoint will be assessed by clinical examination of ocular inflammation (slit lamp, fundus biomicroscopy, angiography, perimetry or electrophysiology). Systemic safety will be assessed by vital signs, routine clinical chemistry testing (including C reactive protein, ESR) and full/differential blood counts. Immunopathology essays will include specific enzyme-linked immunosorbent assays for humoral antibodies against rAAV8 capsid protein and/or CNGA3 gene product and specific enzyme-linked immunosorbent spot assays to monitor cellular immune reactivity against rAAV8 capsid protein and/or CNGA3 gene product. Biodistribution will be monitored by polymerase chain reaction studies on rAAV8 genome in blood, urine, saliva and lachrymal fluid.


    Original Primary Outcome: Same as current

    Current Secondary Outcome: Efficacy measures. Number of Participants With improved Visual Function. [ Time Frame: Day 14 - Day 365 ]

    Efficacy data (improvement in visual function) and patient reported outcomes will be investigated exploratively as well as retinal imaging. They are all secondary endpoints in this trial.


    Original Secondary Outcome: Same as current

    Information By: STZ eyetrial

    Dates:
    Date Received: September 18, 2015
    Date Started: November 2015
    Date Completion: November 2017
    Last Updated: May 3, 2017
    Last Verified: May 2017