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

Phase 1

Recruiting

• Conditions: CNGA3-linked achromatopsia; Therapeutic area: Diseases [C] - Eye Diseases [C11]; MedDRA version: 20.0Level: LLTClassification code: 10000454Term: Achromatopsia Class: 10010331

• Registration Number: CTIS2024-515553-17-00
• Lead Sponsor: niversitaetsklinikum Tuebingen AöR

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-07-01
• Last Update Posted: 21 August 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 14

Inclusion Criteria

clinical diagnosis of achromatopsia, Female patients of childbearing potential must agree to use an effective method ofbirth control during the first 6 months post treatment., negative pregnancy test in women with childbearing potential (a woman who istwo years post-menopausal or surgically sterile is not considered to be ofchildbearing potential), 6-12 years of age, = 18 years of age, bi-allelic pathogenic or likely pathogenic mutation in CNGA3, BCVA = 20/400, a minimal outer nuclear layer thickness of 10µm at 3° eccentricity (normal =38±6µm), ability to understand and willingness to consent to study protocol, no infection with Human Immundeficiency Virus (HIV), Male patients must agree to use condoms during the first 6 months post treatment.

Exclusion Criteria

any other retinopathy due to other diseases e.g. (but not limited to) arterialhypertension, trauma or acquired inflammatory diseases (uveitis serology) ,retinopathy of the premature, causal mutations in other genes for hereditary retinal diseases, contraindications in view of the planned surgery (e.g. anaemia Hb<8g/dl, severecoagulopathy, severe blood pressure fluctuations) including intolerance andcontraindications to general anaesthesia, ocular opacity and mature cataract, ocular infection with herpes simplex virus in medical history, history of ocular malignancies, disorders of the inner retina (e.g. retinal vascular occlusions in the patients history), glaucoma defined as damage of the optic nerve, history of poorly controlled (HbA1c > 7%) Diabetes Mellitus type 1 or type 2, patients treated with systemic corticoids within 14 days prior inclusion, systemic illness or medically significant abnormal laboratory values >3 UNL in bloodanalysis including renal and hepatic functions at inclusion, systemic conditions (e.g. coronary heart disease, congenital/genetic conditions,autoimmune disorders) which may affect study participation or outcome measures, absence of vision on the contralateral eye, contraindication to pharmacological mydriasis (e.g. history of angle blockglaucoma), current or recent participation in other study or administration of biologic agentwithin the last three months, recent (within the last 6 months) ocular surgery, intravitreal or subretinalimplantation of a medical device, known sensitivity to any compound used in the study, contraindications to systemic immunosuppression, subject/partner of childbearing potential unwilling to use adequate contraceptionfor six months after dosing, nursing or pregnant female subject, any other cause that, in the investigator‘s opinion, renders potential subjects notsuitable for the study

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified