Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2014 by GenSight Biologics
Sponsor:
Information provided by (Responsible Party):
GenSight Biologics
ClinicalTrials.gov Identifier:
NCT02064569
First received: February 13, 2014
Last updated: February 14, 2014
Last verified: February 2014
  Purpose

The purpose of this study is to evaluate the safety and tolerability profile of ascending doses of GS010 in Leber Hereditary Optic Neuropathy (LHON) patients.


Condition Intervention Phase
Leber Hereditary Optic Neuropathy
Genetic: GS010
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Official Title: An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Tolerability of GS010 (rAAV2/2-ND4) in Patients With Leber Hereditary Optic Neuropathy Due to Mutations in the Mitochondrial NADH Dehydrogenase 4 Gene

Resource links provided by NLM:


Further study details as provided by GenSight Biologics:

Primary Outcome Measures:
  • Incidence of local and general adverse events and Serious Adverse Events [ Time Frame: Up to 48 weeks ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 18
Study Start Date: February 2014
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: GS010 Genetic: GS010

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Documented diagnosis of LHON based on a genetic test confirming the presence of the G11778A mutation in the mitochondrial ND4

Age 18 years old or older at the time of study entry (informed consent signature)

Visual acuity ≤ 1/10 of the less functional eye

Exclusion Criteria:

Any known allergy or hypersensibility to one of the product used during the trial

Contraindication to IVT surgery (anaemia Hb <8g/dl, severe cardiovascular disease, severe coagulopathy…)

Disorder of the ocular humors and of the internal retina involving visual disability

Glaucoma

Presence of other pathology whose symptoms or associated treatments might affect the retina or the optic nerve Vascular retinal occlusion

Narrow angle contra-indicating pupillary dilation

Other cause of optic neuropathy (inflammatory conditions or exposure to toxins...)

Patients presenting known mutation of other genes implicated in pathological retinal conditions

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02064569

Contacts
Contact: Serge Fitoussi, MD +33 (6) 65 75 78 41 sfitoussi@gensight-biologics.com

Locations
France
CIC du CHNO DES QUINZE-VINGTS Recruiting
Paris, France, 75012
Sponsors and Collaborators
GenSight Biologics
Investigators
Principal Investigator: CATHERINE VIGNAL, MD CIC CHNO DES QUINZE VINGTS
  More Information

Additional Information:
No publications provided

Responsible Party: GenSight Biologics
ClinicalTrials.gov Identifier: NCT02064569     History of Changes
Other Study ID Numbers: GS-LHON/CLIN/01
Study First Received: February 13, 2014
Last Updated: February 14, 2014
Health Authority: France: Agence Nationale de Sécurité du Médicament et des produits de santé

Additional relevant MeSH terms:
Optic Atrophy, Hereditary, Leber
Optic Atrophies, Hereditary
Eye Diseases, Hereditary
Optic Nerve Diseases
Peripheral Nervous System Diseases
Cranial Nerve Diseases
Nervous System Diseases
Eye Diseases
Neuromuscular Diseases
Optic Atrophy
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases

ClinicalTrials.gov processed this record on September 14, 2014