NCT02610582: Colourbridge

Safety and Efficacy of a Bilateral Single Subretinal Injection of rAAV.hCNGA3 in Adult and Minor Patients With CNGA3-linked Achromatopsia Investigated in a Randomized, Wait List Controlled, Observer-masked Trial

Active, enrolled Phase 1/Phase 2 Last updated 17 April 2024

What this trial tests

Phase 1/Phase 2 trial testing rAAV.hCNGA3 in Achromatopsia in 13 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

1 November 2015

Primary endpoint
1 June 2027

1 June 2027

Quick facts

Lead sponsor	STZ eyetrial
Phase	Phase 1/Phase 2
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	randomized
Design	sequential
Masking	none
Primary purpose	treatment
Enrollment	13
Start date	1 November 2015
Primary completion	1 June 2027
Estimated completion	1 June 2027
Sites	1 location across Germany

Drugs / interventions tested

• rAAV.hCNGA3 — full drug profile →

Conditions studied

• Achromatopsia — all drugs for Achromatopsia →

Sponsor

STZ eyetrial — full company profile →

Who can join

6 and older, any sex, with Achromatopsia. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• 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, fundus photography. Systemic safety will be assessed by vital signs, routine clinical chemistry testing (including CRP, ESR) and full/differential blood counts. Immunopathology essays will include specific enzyme-linked immunosorbent assays for humoral antibodies against

Sponsor's own description

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

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Current Clinical Applications of In Vivo Gene Therapy with AAVs.
   Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, Goodspeed K, et al · · 2021 · cited 544× · PMID 33309881 · DOI 10.1016/j.ymthe.2020.12.007
2. Progress in treating inherited retinal diseases: Early subretinal gene therapy clinical trials and candidates for future initiatives.
   Garafalo AV, Cideciyan AV, Héon E, Sheplock R, et al · · 2020 · cited 136× · PMID 31899291 · DOI 10.1016/j.preteyeres.2019.100827
3. Safety and Vision Outcomes of Subretinal Gene Therapy Targeting Cone Photoreceptors in Achromatopsia: A Nonrandomized Controlled Trial.
   Fischer MD, Michalakis S, Wilhelm B, Zobor D, et al · · 2020 · cited 127× · PMID 32352493 · DOI 10.1001/jamaophthalmol.2020.1032
4. Residual Foveal Cone Structure in CNGB3-Associated Achromatopsia.
   Langlo CS, Patterson EJ, Higgins BP, Summerfelt P, et al · · 2016 · cited 94× · PMID 27479814 · DOI 10.1167/iovs.16-19313
5. Taking Stock of Retinal Gene Therapy: Looking Back and Moving Forward.
   Bennett J. · · 2017 · cited 84× · PMID 28391961 · DOI 10.1016/j.ymthe.2017.03.008
6. Advancing Clinical Trials for Inherited Retinal Diseases: Recommendations from the Second Monaciano Symposium.
   Thompson DA, Iannaccone A, Ali RR, Arshavsky VY, et al · · 2020 · cited 82× · PMID 32832209 · DOI 10.1167/tvst.9.7.2
7. A nonhuman primate model of inherited retinal disease.
   Moshiri A, Chen R, Kim S, Harris RA, et al · · 2019 · cited 81× · PMID 30667376 · DOI 10.1172/jci123980
8. Gene Therapy in Retinal Dystrophies.
   Ziccardi L, Cordeddu V, Gaddini L, Matteucci A, et al · · 2019 · cited 71× · PMID 31739639 · DOI 10.3390/ijms20225722