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Report for Clinical Trial NCT03406104

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

Long-term Follow-up of ND4 LHON Subjects Treated With GS010 Ocular Gene Therapy in the RESCUE or REVERSE Phase III Clinical Trials

The goal of this clinical trial is to assess the long-term safety and efficacy of GS010, a gene therapy, and assess the quality of life in subjects with LHON due to the G11778A ND4 mitochondrial mutation and who were treated in the Rescue or Reverse studies.

NCT03406104 Leber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber)
MeSH:Optic Nerve Diseases Optic Atrophy, Hereditary, Leber Atrophy
HPO:Leber optic atrophy

2 Interventions

Name: GS010

Description: Different procedures will be performed in order to assess the safety and efficacy of GS010 up to 5 years post-treatment
Type: Genetic

Name: Sham Intravitreal Injection

Description: Different procedures will be performed in order to assess the safety and efficacy of GS010 up to 5 years post-treatment
Type: Other


Primary Outcomes

Description: AEs or SAEs (ocular or systemic) related to IMP or administration procedure, as judged by the Investigator, reported during the long-term follow-up visits (2, 2.5, 3, 4, and 5 years) from the period of 96 weeks up to 5 years post-treatment and summarized descriptively by type, frequency (number, percentage), severity, causal relationship, and seriousness

Measure: Adverse events (AEs) or serious adverse events (SAEs) (ocular or systemic)

Time: Up to 5-Year post-treatment

Secondary Outcomes

Description: Change in best BCVA reported with LogMAR

Measure: Best-Corrected Visual Acuity (BCVA) reported with LogMAR

Time: Up to 5-Year post-treatment

Description: Change of parameters measured with HVF 30-2

Measure: HumphreyTM visual field (HVF) 30-2 parameters

Time: Up to 5-Year post-treatment

Description: Change of parameters measured with SD-OCT

Measure: Spectral domain optical coherence tomography (SD-OCT) parameters

Time: Up to 5-Year post-treatment

Description: Response status of eyes treated with GS010 IVT injection compared to eyes treated with sham IVT injection (An improvement of at least 15 ETDRS letters/Eyes that lose less than the 15 ETDRS letters)

Measure: Responder Analysis

Time: Up to 5-Year post-treatment

Description: Time course of the response in eyes treated with GS010 IVT injection compared to eyes treated with sham IVT injection, for the BCVA reported with LogMAR, for parameters measured with HVF 30-2 and SD-OCT

Measure: Time course of the response

Time: Up to 5-Year post-treatment

Description: Visual improvement as measured by LogMAR by analysis of covariance (ANCOVA)

Measure: Visual improvement

Time: Up to 5-Year post-treatment

Description: Change of GCL thickness/volume and topographical map and other parameters measured by SD-OCT using a mixed model of ANCOVA

Measure: Change of ganglion cell layer (GCL) thickness/volume and topographical map and other parameters measured by SD-OCT

Time: Up to 5-Year post-treatment

Description: QOL as measured with VFQ-25 subject-rated instrument

Measure: Quality of Life: Visual Functioning Questionnaire 25 (VFQ-25)

Time: Up to 5-Year post-treatment

Description: QOL as measured with SF-36v2 subject-rated instrument

Measure: Quality of Life: 36-Item Short Form Health Survey, version 2 (SF-36-v2)

Time: Up to 5-Year post-treatment

Time Perspective: Prospective

Cohort


There is one SNP

SNPs


1 G11778A

RESCUE and REVERSE Long-term Follow-up The goal of this clinical trial is to assess the long-term safety and efficacy of GS010, a gene therapy, and assess the quality of life in subjects with LHON due to the G11778A ND4 mitochondrial mutation and who were treated in the Rescue or Reverse studies. --- G11778A ---



HPO Nodes


HPO: