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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
Compound Name RAAV.hCNGA3
Compound 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
Therapy Type 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
Delivery System Viral transduction
Vector Type AAV8
Editor Type none
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 Stage 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