CClinicalTrials.gg
Sign In
Active, not recruitingNCT02599922Updated Jul 22, 2022

Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial)

A Phase 1/2 interventional study of rAAV2tYF-PR1.7-hCNGB3 in Achromatopsia, sponsored by Beacon Therapeutics. Active, not recruiting at 8 sites in United States. Open to participants aged 4 Years and older. Per ClinicalTrials.gov, last updated 2022-07-22.

Sponsored by Beacon Therapeutics · Phase 1/2, Interventional, and Treatment

From the registry’s dates

  • Primary completion was expected by Jul 2022, 4 years 3 months ago, but the record still lists the study as active, not recruiting.
Phase
Phase 1/2
Study type
Interventional
Enrollment
32
Allocation
Non-randomized
Ages
4 Years and older
Sex
All
01

Study summary

This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-401 administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGB3 gene. The primary study endpoint will be safety and the secondary study endpoint will be efficacy.

Read the detailed description

This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-401 administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGB3 gene. The primary study endpoint will be safety and the secondary study endpoint will be efficacy.

Subjects will be enrolled sequentially in seven dosing groups. Subjects in Groups 1, 2, 3, 4, 5, and 6 will be at least 18 years of age and will receive varying dose levels of study agent. Subjects in Group 4a will be 6 to 17 years of age and will receive the same dose as Group 4. Subjects in Groups 5a and 7 will be between 4 and 8 years of age. Subjects in Group 5a will receive the same dose as Group 5, and subjects in Group 7 will receive the maximum tolerated dose identified in Groups 1, 2, 3, 4, 4a, 5, 5a, and 6.

Safety will be monitored by evaluation of ocular and non-ocular adverse events and hematology and clinical chemistry parameters. Efficacy parameters will include visual acuity, light discomfort testing, color vision, static visual field, ERG, adaptive optics retinal imaging, functional MRI (fMRI), color brightness test and OCT.

02

Conditions studied

  • Achromatopsia

Browse trials for

03

In context

Color Vision Defects

38 studies on the registry are indexed under Color Vision Defects; 11 are open to participants now.

This study's enrollment of 32 is below the median of 40 across 25 interventional studies indexed under Color Vision Defects.

Browse Color Vision Defects studies →

Lead sponsor

Beacon Therapeutics is the lead sponsor of 13 studies on the registry; 1 is open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
4 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  1. Male or female subjects with documented mutations in both alleles of the CNGB3 gene;
  2. Retinal disease consistent with a clinical diagnosis of achromatopsia;
  3. At least 18 years of age for Groups 1, 2, 3, 4, 5 and 6. At least 6 years of age for Group 4a, and 4-8 years of age for Groups 5a and 7;
  4. Able to perform tests of visual and retinal function;
  5. Visual acuity in the study eye not better than 55 ETDRS letters (Snellen equivalent 20/80) based on the average of two examinations at the baseline visit;
  6. Acceptable laboratory parameters;
  7. For females of childbearing potential: A negative pregnancy test within 2 days before administration of study agent.

Exclusion criteria

Exclusion Criteria include:

  1. Best-corrected visual acuity difference between the two eyes of > 15 ETDRS letters (3 lines);
  2. Evidence of degenerative myopia in the study eye;
  3. Pre-existing eye conditions that would contribute to vision loss in either eye or increase the risk of subretinal injection in the study eye.
05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
32 participants (actual)

Study arms

  • Experimental
    Group 1: 2.0 x 10^11 vg/mL of AGTC-401

    Subjects at least 18 y/o treated with 2.0 x 10\^11 vg/mL of rAAV2tYF-PR1/7-hCNGB3 study drug.

    Biological: rAAV2tYF-PR1.7-hCNGB3

  • Experimental
    Group 2: 4.0 x 10^10 vg/mL of AGTC-401

    Subjects at least 18 y/o treated with 4.0 x 10\^10 vg/mL of rAAV2tYF-PR1/7-hCNGB3 study drug.

    Biological: rAAV2tYF-PR1.7-hCNGB3

  • Experimental
    Group 3: 1.2 x 10^11 vg/mL of AGTC-401

    Subjects at least 18 y/o treated with 1.2 x 10\^11 vg/mL of rAAV2tYF-PR1/7-hCNGB3 study drug.

    Biological: rAAV2tYF-PR1.7-hCNGB3

  • Experimental
    Group 4: 3.6 x 10^11 vg/mL of AGTC-401

    Subjects at least 18 y/o treated with 3.6 x 10\^11 vg/mL of rAAV2tYF-PR1/7-hCNGB3 study drug.

    Biological: rAAV2tYF-PR1.7-hCNGB3

  • Experimental
    Group 4a: 3.6 x 10^11 vg/mL of AGTC-401

    Subjects 6 to 17 y/o treated with 3.6 x 10\^11 vg/mL of rAAV2tYF-PR1/7-hCNGB3 study drug.

    Biological: rAAV2tYF-PR1.7-hCNGB3

  • Experimental
    Group 5: 1.1 x 10^12 vg/mL of AGTC-401

    Subjects at least 18 y/o treated with 1.1 x 10\^12 vg/mL of rAAV2tYF-PR1/7-hCNGB3 study drug.

    Biological: rAAV2tYF-PR1.7-hCNGB3

  • Experimental
    Group 5a: 1.1 x 10^12 vg/mL of AGTC-401

    Subjects 4 to 8 y/o treated with 1.1 x 10\^12 vg/mL of rAAV2tYF-PR1/7-hCNGB3 study drug.

    Biological: rAAV2tYF-PR1.7-hCNGB3

  • Experimental
    Group 6: 3.2 x 10^12 vg/mL of AGTC-401

    Subjects at least 18 y/o treated with 3.2 x 10\^12 vg/mL of rAAV2tYF-PR1/7-hCNGB3 study drug.

    Biological: rAAV2tYF-PR1.7-hCNGB3

  • Experimental
    Group 7: MTD of AGTC-401

    Subjects 4 to 8 y/o treated with a maximum tolerated dose of rAAV2tYF-PR1/7-hCNGB3 study drug determined by Groups 1-6.

    Biological: rAAV2tYF-PR1.7-hCNGB3

Interventions

  • BiologicalrAAV2tYF-PR1.7-hCNGB3

    rAAV2tYF-PR1.7-hCNGB3 is a non-replicating, rep/cap-deleted, recombinant adeno-associated virus vector that expresses the CNGB3 gene.

    Also known as: AGTC-401

06

What researchers measure

Primary outcomes

  1. Adverse events

    Proportion of participants experiencing grade 3 or greater adverse events

    Time frame: 1 year

Secondary outcomes

  1. Visual acuity

    Changes in best corrected visual acuity compared to pre-treatment

    Time frame: 1 year

  2. Light aversion

    Changes in light discomfort testing compared to pre-treatment

    Time frame: 1 year

  3. Color vision

    Changes in color vision testing compared to pre-treatment

    Time frame: 1 year

07

Study locations

8 sites
  • VitreoRetinal Associates
    Gainesville, Florida 32607, United States
  • Bascom Palmer Eye Institute
    Miami, Florida 33136, United States
  • Pangere Center for Inherited Retinal Diseases, The Chicago Lighthouse for People Who Are Blind or Visually Imp
    Chicago, Illinois 60608, United States
  • Massachusetts Eye and Ear Infirmary
    Boston, Massachusetts 02114, United States
  • Boston Children's Hospital
    Boston, Massachusetts 02115, United States
  • Duke Eye Center, Duke University Medical Center
    Durham, North Carolina 27710, United States
  • Cincinnati Eye Institute
    Cincinnati, Ohio 45242, United States
  • Casey Eye Institute, Oregon Health and Sciences University
    Portland, Oregon 97239, United States
08

References and documents

Publications

  • Komaromy AM, Alexander JJ, Rowlan JS, Garcia MM, Chiodo VA, Kaya A, Tanaka JC, Acland GM, Hauswirth WW, Aguirre GD. Gene therapy rescues cone function in congenital achromatopsia. Hum Mol Genet. 2010 Jul 1;19(13):2581-93. doi: 10.1093/hmg/ddq136. Epub 2010 Apr 8. Erratum In: Hum Mol Genet. 2011 Dec 15;20(24):5024. PubMed 20378608 ↗
  • Davis JL. The Blunt End: Surgical Challenges of Gene Therapy for Inherited Retinal Diseases. Am J Ophthalmol. 2018 Dec;196:xxv-xxix. doi: 10.1016/j.ajo.2018.08.038. Epub 2018 Sep 5. PubMed 30194931 ↗

Related links

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jul 22, 2022, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT02599922
Lead sponsor
Beacon Therapeutics
Collaborators
National Eye Institute (NEI)
Responsible party
Sponsor
First posted
Nov 9, 2015
Start date
Apr 11, 2016
Primary completion
Jul 2022 (estimated)
Completion
Jul 2026 (estimated)
Last update
Jul 22, 2022

Study contacts

David Jacobs, MD, MBA
study director · Applied Genetic Technologies Corporation

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in Jul 2022. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Color Vision Defects→

Other studies from this sponsor

Beacon Therapeutics

Start the discussion