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Completed

NCT Number: NCT03406104

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.

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Key information

Conditions

Leber Hereditary Optic Neuropathy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Cranial Nerve Diseases Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolic Diseases Mitochondrial Diseases Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases Optic Atrophies, Hereditary Optic Atrophy Optic Atrophy, Hereditary, Leber Optic Nerve Diseases

Age range

15 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

CHNO Les Quinze Vingts, Paris, France

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About this study

Seven investigational centers from the RESCUE and REVERSE studies also participated in the RESTORE study, located in the European union and in the USA.

Primary objectives: to assess the long term safety of intravitreal injection up to 5 years of post treatment in subjects who were treated in the RESCUE or REVERSE studies.

Secondary objectives: (1) to assess the long-term efficacy of intravitreal GS010 administration up to 5 years post-treatment in subjects who were treated in the RESCUE or REVERSE studies; and (2) to assess the quality of life (QoL) in subjects who were treated with GS010 in the RESCUE or REVERSE studies for up to 5 years post-treatment.

Methodology: This was a Phase III prospective long-term follow-up (LTFU) clinical study of subjects previously treated with GS010 and Sham during 2 Phase III studies-RESCUE and REVERSE. The LTFU study followed subjects for an additional 3 years, for a total of 5 years post-injection. The LTFU study included 5 visits at 2, 2.5, 3, 4, and 5 years after the investigational medicinal product (IMP) injection. Safety, efficacy, and QoL variables were assessed during each of the 5 LTFU visits, and descriptive summaries and statistical testing were used for the analysis of the data.

This report presents the final analysis of results at Year 5 after treatment administration in RESCUE and REVERSE.

Number of analysed subjects: all subjects completing RECUE and REVERSE studies who provided consent for the RESTORE study: 62 subjects.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Subject was treated with GS010 IVT injection in either of the RESCUE or REVERSE Phase III clinical studies
  • Subject of legal consent age has provided informed consent; subjects that are not of legal consent age have undergone their country-approved clinical trial enrollment consent process

Exclusion criteria

  • Subject is unwilling or unable to comply with the protocol requirements
  • Subject has any medical or psychological condition that, in the opinion of the Investigator, may compromise his or her safe participation in the study
  • Subject is taking or intending to take idebenone during the long-term follow-up study period

Treatment and study plan

GS010

Genetic

Lenadogene nolparvovec Intravitreal ocular unilateral Injection

Other names: Lenadogene Nolparvovec

Sham intravitreal injection

Other

Lenadogene nolparvovec Intravitreal ocular unilateral Injection

Primary outcomes

  1. Ocular Adverse Events (AEs)

    Time frame: from year 2 to year 5 post treatment

    Number of Eyes with Ocular Adverse events related to study treatment or study procedures as judged by the investigator reported from year 2 to year 5 post treatment

Secondary outcomes

  1. Visual Acuity

    Time frame: Nadir to 5 Years post-treatment, Assessed at Baseline in RESCUE and REVERSE studies to year 5 post-treatment

    Change from Nadir to 5 Years post-treatment, Assessed at Baseline in RESCUE and REVERSE studies to year 5 post-treatment, expressed in LogMAR.

    On chart visual acuity: Visual acuity inferior or equal to LogMAR +1.6 Off chart visual acuity: Visual acuity superior to LogMar +1.7

    Normal vision LogMar: 0 and less than 0

  2. Responder Analysis: Clinically Relevant Recovery

    Time frame: Nadir to 5 Years post-treatment, Assessed at Baseline in RESCUE and REVERSE studies to year 5 post-treatment

    CRR clinically relevant recovery from Nadir defined as i/ for eyes on chart at Nadir, an improvement of at least -0.2 LogMar from Nadir and ii/ for eyes off chart at Nadir eyes that became on chart.

    Off chart visual acuity expressed in LogMar: more than +1.7 On chart visual acuity expressed in LogMar: less than 1.6 Normal vision LogMar: 0 and less than 0

Other outcomes

  1. Eyes on Chart

    Time frame: Year 5 post-treatment

    Definition: Visual acuity inferior or equal to LogMAR +1.6 at 5 Years post treatment

Sponsors and collaborators

Lead sponsor

GenSight Biologics

Industry

Registry information

Official study title

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

Acronym: RESTORE

Important dates

Study start
2018
Primary completion
2022
Study completion
2022
First posted
Jan 23, 2018
Registry last updated
Mar 2, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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