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AchromatopsiaPhase 1/2NCT02935517

Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial)

A Phase 1/2 study testing AGTC-402 in people with Achromatopsia, run by Beacon Therapeutics at 6 sites in 2 countries.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-402, administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGA3 gene. The primary study endpoint will be safety and the secondary study endpoint will be efficacy. Read more about this study

Who is it for?
Ages 4 years and older
Conditions the study is for
Achromatopsia
What is being tested?
AGTC-402
Could I receive a placebo?
No, everyone receives an active treatment
How many people
24 participants
Where
6 sitesin 2 countries
Study dates
3 Aug 2017to Aug 2026
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 4 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Male or female subjects with documented mutations in both alleles of the CNGA3 gene;
  • Retinal disease consistent with a clinical diagnosis of achromatopsia;
  • At least 18 years of age for Groups 1, 2, 3, 4, and 5. At least 6 years of age for Group 3a, and and 4-8 years of age for Groups 4a and 6;
  • Able to perform tests of visual and retinal function;
  • 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;
  • Acceptable laboratory parameters;
  • For females of childbearing potential: A negative pregnancy test within 2 days before administration of study agent.

Who cannot join

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

Trial locations and contacts

6 sites in 2 countries.

6 sites in 2 countries

United States5 sites

  • Massachusetts Eye and Ear Infirmary

    Boston, Massachusetts, 02114

  • Cincinnati Eye Institute

    Cincinnati, Ohio, 45242

  • VitreoRetinal Associates

    Gainesville, Florida, 32607

  • Bascom Palmer Eye Institute

    Miami, Florida, 33136

  • Casey Eye Institute, Oregon Health and Sciences University

    Portland, Oregon, 97239

Other trials in United States

Israel1 site

  • Hadassah-Hebrew University Medical Center

    Jerusalem, 91120

Other trials in Israel

About this study

Official title: A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of AGTC 402, a Recombinant Adeno-associated Virus Vector Expressing CNGA3, in Patients With Congenital Achromatopsia Caused by Mutations in the CNGA3 Gene

This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-402, administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGA3 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-402 administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations in the CNGA3 gene. The primary study endpoint will be safety and the secondary study endpoint will be efficacy.

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

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.

Conditions
Achromatopsia
Being tested
AGTC-402
Keywords
Color vision deficiency, Color vision defects, Vision disorders, Sensation disorders, Neurologic manifestations, Nervous system diseases, Eye diseases, Signs and symptoms
Medical subject headings
Color Vision Defects, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Nervous System Diseases, Eye Diseases, Signs and Symptoms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AGTC-402Biological

    Also called rAAV2tYF-PR1.7-hCNGA3

    AGTC-402 is a non-replicating, rep/cap-deleted, recombinant adeno-associated virus vector that expresses the CNGA3 gene.

Study groups in NCT02935517
Study groupRoleWhat they receive
Group 1: 4.0 x 10^10 vg/mL of AGTC-402ExperimentalSubjects at least 18 y/o treated with 4.0 x 10^10 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Receives: Biological: AGTC-402
Group 2: 1.2 x 10^11 vg/mL of AGTC-402ExperimentalSubjects at least 18 y/o treated with 1.2 x 10^11 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Receives: Biological: AGTC-402
Group 3: 3.6 x 10^11 vg/mL of AGTC-402ExperimentalSubjects at least 18 y/o treated with 3.6 x 10^11 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Receives: Biological: AGTC-402
Group 3a: 3.6 x 10^11 vg/mL of AGTC-402ExperimentalSubjects 6 to 17 y/o treated with 3.6 x 10^11 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Receives: Biological: AGTC-402
Group 4: 1.1 x 10^12 vg/mL of AGTC-402ExperimentalSubjects at least 18 y/o treated with 1.1 x 10^12 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Receives: Biological: AGTC-402
Group 4a: 1.1 x 10^12 vg/mL of AGTC-402ExperimentalSubjects 4 to 8 y/o treated with 1.1 x 10^12 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Receives: Biological: AGTC-402
Group 5: 3.2 x 10^12 vg/mL of AGTC-402ExperimentalSubjects at least 18 y/o treated with 3.2 x 10^12 vg/mL of rAAV2tYF-PR1/7-hCNGA3 study drug.
Receives: Biological: AGTC-402
Group 6: MTD of AGTC-402ExperimentalSubjects 4 to 8 y/o treated with a maximum tolerated dose of rAAV2tYF-PR1/7-hCNGA3 study drug determined by Groups 1-5.
Receives: Biological: AGTC-402

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Adverse events

    Proportion of participants experiencing grade 3 or greater adverse events

    Measured: 1 year

Secondary outcomes (3)

  1. Visual acuity

    Changes in best corrected visual acuity compared to pre-treatment

    Measured: 1 year

  2. Light aversion

    Changes in light discomfort testing compared to pre-treatment

    Measured: 1 year

  3. Color vision

    Changes in color vision testing compared to pre-treatment

    Measured: 1 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Oct 2016 (estimated)
  2. Study start3 Aug 2017
  3. Primary completionAug 2022 (estimated)
  4. Study completionAug 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
24 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02935517
Sponsor's protocol ID
AGTC-CNGA3-002
Status last verified
Jul 2022
First submitted
13 Oct 2016
Last updated
22 Jul 2022
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Banin E, Gootwine E, Obolensky A, Ezra-Elia R, Ejzenberg A, Zelinger L, Honig H, Rosov A, Yamin E, Sharon D, Averbukh E, Hauswirth WW, Ofri R. Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia. Mol Ther. 2015 Sep;23(9):1423-33. doi: 10.1038/mt.2015.114. Epub 2015 Jun 19.

    PubMed 26087757

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing AGTC-402 in people with Achromatopsia, run by Beacon Therapeutics at 6 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 22 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in United States, Israel. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Adverse events (time frame: 1 year). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02935517, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jul 2022.

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