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Report for Clinical Trial NCT02161380

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

An Open-label Dose Escalation Study of an Adeno-associated Virus Vector (scAAV2-P1ND4v2) for Gene Therapy of Leber's Hereditary Optic Neuropathy (LHON) Caused by the G11778A Mutation in Mitochondrial DNA

Hypotheses: The primary hypothesis being tested is that there will be no toxicity resulting in loss of vision to no light perception in injected eyes.

NCT02161380 Leber's Hereditary Optic Neuropathy
MeSH: Optic Nerve Diseases Optic Atrophy, Hereditary, Leber
HPO: Leber optic atrophy

4 Interventions

Name: injection of scAAV2-P1ND4v2 1.18x10e9 vg (Low),

Description: injection of Total Volume of each intravitreal injection is 200 µL

Type: Drug

1(Chronic) 2(Acute) 3(Presymptomatic)

Name: injection of scAAV2-P1ND4v2 5.81 X10e9 vg (Med)

Description: injection of Total Volume of each intravitreal injection is 200 µL

Type: Drug

1(Chronic) 2(Acute) 3(Presymptomatic)

Name: injection of scAAV2-P1ND4v2 2.4 X10e10vg (High)

Description: injection of Total Volume of each intravitreal injection is 100 µL

Type: Drug

1(Chronic)

Name: injection of scAAV2-P1ND4v2 1.0 X10e11vg (Higher)

Description: injection of Total Volume of each intravitreal injection is 100 µL

Type: Drug

1(Chronic) 2(Acute) 3(Presymptomatic)


Primary Outcomes

Description: Incidence of local and general adverse events and Serious Adverse Events

Measure: Assessment of Primary Endpoint - Toxicity

Time: 1 year

Purpose: Treatment

Allocation: Non-Randomized

Parallel Assignment


There is one SNP

SNPs


1 G11778A

An Open-label Dose Escalation Study of an Adeno-associated Virus Vector (scAAV2-P1ND4v2) for Gene Therapy of Leber's Hereditary Optic Neuropathy (LHON) Caused by the G11778A Mutation in Mitochondrial DNA. --- G11778A ---

Inclusion Criteria: 1. Age 15 or older; 2. Patients with LHON and the G11778A mitochondrial DNA mutation. --- G11778A ---

A previous CLIA certified genetic lab result showing the LHON G11778A mutation will be accepted for inclusion; 3. Ability to perform tests of visual and retinal function; 4. Ability to comply with research procedures; 5. Able and willing to provide informed consent before undergoing any study related procedures. --- G11778A ---



HPO Nodes


HPO:
Leber optic atrophy
Genes 11
ND2 ATP6 ND4L ND5 CYTB ND1 TBC1D24 ND4 CPLX1 ND6 COX3