Contact SCGE

Gene Therapy Trial Report

Summary

Safety and Efficacy of rAAV.hCNGA3 Gene Therapy in Patients With CNGA3-linked Achromatopsia


NCTID NCT02610582 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Achromatopsia
Disease Ontology Term DOID:0110007
Drug Product Name RAAV.hCNGA3
Drug Product Description AAV8-hArr3-hCNGA3-WPREm
Sponsor STZ eyetrial
Funder Type Other
Recruitment Status
Active not recruiting
Enrollment Count 13 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant CNGA3
Regulatory Element ARR3 (promoter)
Therapeutic Modality Gene transfer
Therapy Route in vivo
Mechanism of Action Functional gene replacement
Route of Administration Subretinal
Drug Product Type Viral vector
Target Tissue/Cell Cone cells
Gene Delivery System Type Viral transduction
Vector Type 🔄 Adeno-associated virus
Viral Vector Subtype AAV8
Dose 1 1E10 vg/dose (n=3)
Dose 2 5E10 vg/dose (n=3)
Dose 3 1E11 vg/dose (n=3)

Study Record Dates


Current Phase Phase1, Phase2
Submit Date 2015-09-18
Completion Date 2027-06
Last Update 2024-04-18

Participation Criteria


Eligible Age >=6 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria (Both Eyes) * clinical diagnosis of achromatopsia * 6-12 years of age * ≥ 18 years of age * confirmed 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. * Female patients of childbearing potential must agree to use an effective method of birth control during the first 6 months post treatment. * negative pregnancy test in women with childbearing potential (a woman who is two years post-menopausal or surgically sterile is not considered to be of childbearing potential) Exclusion Criteria * any other retinopathy due to other diseases e.g. (but not limited to) arterial hypertension, trauma or acquired inflammatory diseases (uveitis serology) , retinopathy of the premature * systemic conditions (e.g. coronary heart disease, congenital/genetic conditions, autoimmune disorders) which may affect study participation or outcome measures * current or recent participation in other study or administration of biologic agent within the last three months * recent (6 months) ocular surgery, intravitreal or subretinal implantation 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 contraception for six months after dosing * nursing or pregnant female subject women * any other cause that, in the investigator's opinion, renders potential subjects not suitable for the study * mutations in another achromatopsia gene * contraindications in view of the planned surgery (e.g. anaemia Hb\<8g/dl, severe coagulopathy, severe blood pressure fluctuations) including intolerance and contraindications to general anaesthesia * ocular opacity and mature cataract * ocular infection with herpes simplex virus in medical history * history of ocular malignancies * disorders of the internal retina (e.g. retinal detachment in the patients history) * glaucoma defined as damage of the optic nerve * vascular retinal occlusion * 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 blood analysis including renal and hepatic functions at inclusion * absence of vision on the other contralateral eye * contraindication to pharmacological mydriasis (e.g. history of angle block glaucoma)
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations Germany

Regulatory Information


Has US IND
FDA Designations
Recent Updates

Resources/Links