Clinical Trials Logo

Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT02599922
Other study ID # AGTC_CNGB3-001
Secondary ID R24EY022023
Status Active, not recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date April 11, 2016
Est. completion date July 2026

Study information

Verified date July 2022
Source Applied Genetic Technologies Corp
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


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.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 32
Est. completion date July 2026
Est. primary completion date July 2022
Accepts healthy volunteers No
Gender All
Age group 4 Years and older
Eligibility Inclusion Criteria include: 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 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.

Study Design


Related Conditions & MeSH terms


Intervention

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

Locations

Country Name City State
United States Boston Children's Hospital Boston Massachusetts
United States Massachusetts Eye and Ear Infirmary Boston Massachusetts
United States Pangere Center for Inherited Retinal Diseases, The Chicago Lighthouse for People Who Are Blind or Visually Imp Chicago Illinois
United States Cincinnati Eye Institute Cincinnati Ohio
United States Duke Eye Center, Duke University Medical Center Durham North Carolina
United States VitreoRetinal Associates Gainesville Florida
United States Bascom Palmer Eye Institute Miami Florida
United States Casey Eye Institute, Oregon Health and Sciences University Portland Oregon

Sponsors (2)

Lead Sponsor Collaborator
Applied Genetic Technologies Corp National Eye Institute (NEI)

Country where clinical trial is conducted

United States, 

References & Publications (1)

Komáromy 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. — View Citation

Outcome

Type Measure Description Time frame Safety issue
Primary Adverse events Proportion of participants experiencing grade 3 or greater adverse events 1 year
Secondary Visual acuity Changes in best corrected visual acuity compared to pre-treatment 1 year
Secondary Light aversion Changes in light discomfort testing compared to pre-treatment 1 year
Secondary Color vision Changes in color vision testing compared to pre-treatment 1 year
See also
  Status Clinical Trial Phase
Completed NCT04124185 - Natural History Study for Achromatopsia
Active, not recruiting NCT02610582 - Safety and Efficacy of rAAV.hCNGA3 Gene Therapy in Patients With CNGA3-linked Achromatopsia Phase 1/Phase 2
Not yet recruiting NCT04041232 - PBA Use for Treatment of ATF6-/- Patients Early Phase 1
Completed NCT03278873 - Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3 Phase 1/Phase 2
Recruiting NCT02435940 - Inherited Retinal Degenerative Disease Registry
Completed NCT01846052 - Clinical and Genetic Characterization of Individuals With Achromatopsia N/A
Active, not recruiting NCT01648452 - CNTF Implants for CNGB3 Achromatopsia Phase 1/Phase 2
Completed NCT03001310 - Gene Therapy for Achromatopsia (CNGB3) Phase 1/Phase 2
Active, not recruiting NCT02935517 - Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial) Phase 1/Phase 2
Completed NCT03758404 - Gene Therapy for Achromatopsia (CNGA3) Phase 1/Phase 2

External Links