A Phase 1/2 Open-label, Multi-centre, Dose-exploration Trial to Evaluate the Safety and Preliminary Efficacy of VG801 via Subretinal Injection in Treatment of Patients with Biallelic ABCA4 Mutation-Associated Retinal Dystrophy.

Conditions

Biallelic ABCA4 Mutation-Associated Retinal Dystrophy.

Brief summary

Safety Endpoint: Number of Adverse events (ocular and systemic), Safety Endpoint: Evaluation of the humoral immune responses against the AAV capsid, Safety Endpoint: Measurement of vector in blood, tears, and saliva (vector shedding) until two negative samples have been obtained, Safety Endpoint: Hematology, chemistry, and urinalysis, Safety Endpoint: Comprehensive ophthalmic assessments including slit lamp examination coupled with imaging including optical coherence tomography (OCT), fundus photography, intraocular pressure monitoring, and fundus autofluorescence imaging., Preliminary Efficacy Endpoint: Visual Function (BCVA, Pelli Robson contrast test, OCT, fundus photography, IOP, fundus autofluorescence imaging), Preliminary Efficacy Endpoint: Morphological retinal measures (Fundus autofluorescence imaging , Optical coherence tomography), Preliminary Efficacy EndPoint: Local retinal function measures for rods and cones ( Full-field stimulus threshold testing), Preliminary Efficacy Endpoint: Retinal function measures (Goldmann kinetic visual fields, Scotopic and photopic microperimetry), Preliminary Efficacy Endpoint: Mobility function (novel virtual reality mobility test)

Related papers

No related papers found yet.

Interventions

DRUGVG801

Eligibility

Sex/Gender

All

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Safety Endpoint: Number of Adverse events (ocular and systemic), Safety Endpoint: Evaluation of the humoral immune responses against the AAV capsid, Safety Endpoint: Measurement of vector in blood, tears, and saliva (vector shedding) until two negative samples have been obtained, Safety Endpoint: Hematology, chemistry, and urinalysis, Safety Endpoint: Comprehensive ophthalmic assessments including slit lamp examination coupled with imaging including optical coherence tomography (OCT), fundus photography, intraocular pressure monitoring, and fundus autofluorescence imaging., Preliminary Efficacy Endpoint: Visual Function (BCVA, Pelli Robson contrast test, OCT, fundus photography, IOP, fundus autofluorescence imaging), Preliminary Efficacy Endpoint: Morphological retinal measures (Fundus autofluorescence imaging , Optical coherence tomography), Preliminary Efficacy EndPoint: Local retinal function measures for rods and cones ( Full-field stimulus threshold testing), Preliminary Efficacy	—

Measure

Time frame

Safety Endpoint: Number of Adverse events (ocular and systemic), Safety Endpoint: Evaluation of the humoral immune responses against the AAV capsid, Safety Endpoint: Measurement of vector in blood, tears, and saliva (vector shedding) until two negative samples have been obtained, Safety Endpoint: Hematology, chemistry, and urinalysis, Safety Endpoint: Comprehensive ophthalmic assessments including slit lamp examination coupled with imaging including optical coherence tomography (OCT), fundus photography, intraocular pressure monitoring, and fundus autofluorescence imaging., Preliminary Efficacy Endpoint: Visual Function (BCVA, Pelli Robson contrast test, OCT, fundus photography, IOP, fundus autofluorescence imaging), Preliminary Efficacy Endpoint: Morphological retinal measures (Fundus autofluorescence imaging , Optical coherence tomography), Preliminary Efficacy EndPoint: Local retinal function measures for rods and cones ( Full-field stimulus threshold testing), Preliminary Efficacy

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Countries

Germany

Outcome results

None listed