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

Conditions

Leber Hereditary Optic Neuropathy

Brief summary

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.

José‐Alain Sahel, Nancy J. Newman, Patrick Yu‐Wai‐Man, Catherine Vignal, Valério Carelli et al. (13 authors)

International Ophthalmology Clinics2021-09-2827 citations

10.1097/iio.0000000000000364

Relationship between immune response and ocular inflammation after intravitreal injection of rAAV2/2–ND4 (GS010) in Leber hereditary optic neuropathy (LHON) patients

C. Vignal Clermont, Céline Bouquet, Scott Uretsky, Anne Galy, Nitza Thomasson et al. (8 authors)

Acta Ophthalmologica2016-09-14

10.1111/j.1755-3768.2016.0682

267. Phase I Gene Therapy Preliminary Clinical Results for Treatment of ND4 Leber Hereditary Optic Neuropathy with rAAV2-2-ND4

Scott Uretsky, Catherine Vignal, Samuel Bidot, Serge Fitoussi, Anne Galy et al. (11 authors)

Molecular Therapy2016-05-011 citations

10.1016/s1525-0016(16)33076-3

83. Relationship Between Immune Responses and Ocular Inflammation: What Is Learnt from Intravitreal Injection of rAAV2-2-ND4 (GS010) in Non-Human Primates and Leber Hereditary Optic Neuropathy (LHON) Patients

Céline Bouquet, Anne Galy, Serge Fitoussi, Sandrine Meunier, Roxane Noel et al. (10 authors)

Molecular Therapy2016-05-01

10.1016/s1525-0016(16)32892-1

Interventions

GENETICGS010

Study design

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

No

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

Design outcomes

Primary

Measure	Time frame
Incidence of local and general adverse events and Serious Adverse Events	Up to 48 weeks

Countries

France

Contacts

PRINCIPAL_INVESTIGATORCATHERINE J. VIGNAL, MD

CIC CHNO DES QUINZE VINGTS

Outcome results

None listed