Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year



Status:Recruiting
Conditions:Ocular, Ocular
Therapuetic Areas:Ophthalmology
Healthy:No
Age Range:15 - Any
Updated:3/31/2019
Start Date:March 12, 2018
End Date:June 30, 2021
Contact:Barrett Katz, MD
Email:bkatz@gensight-biologics.com
Phone:+1 646 831 3799

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Efficacy and Safety of Bilateral Intravitreal Injection of GS010: A Randomized, Double-Masked, Placebo-Controlled Trial in Subjects Affected With G11778A ND4 Leber Hereditary Optic Neuropathy for Up to One Year

The goal of this clinical trial is to assess the safety and efficacy of GS010, a gene
therapy, in improving the retina functional & structural outcomes in subjects with LHON due
to the G11778A ND4 mitochondrial mutation when vision loss duration is present up to one
year.


Main Selection Criteria:

- Age 15 years or older on the date of signed informed consent.

- Clinically manifested vision loss due to ND4 LHON, to any extent, in at least one eye.

- Vision loss duration of ≤ 365 days (i.e. ≤ 1 year) in each affected eye at Inclusion
Visit (Visit 2).

Main Non-Selection Criteria:

- Contraindication to intravitreal injection in any eye.

- Subjects refusing to discontinue idebenone.

- Previous vitrectomy in either eye.

- Narrow angle in any eye contra-indicating pupillary dilation.

- Presence of known/documented mutations, other than the G11778A ND4 LHON-causing
mutation, which are known to cause pathology of the optic nerve, retina or afferent
visual system.

- History of recurrent uveitis (idiopathic or immune-related) or active ocular
inflammation.

Main Inclusion Criteria:

- Vision loss duration of ≤ 365 days (i.e. ≤ 1 year) in each affected eye at Inclusion
Visit (Visit 2).

- Each eye of the subject must maintain at least Hand Motion (HM) visual acuity, as
defined by the study's SOP for visual acuity testing.

- Documented results of genotyping showing the presence of the G11778A mutation in the
ND4 gene and the absence of the other primary LHON-associated mutations (ND1 or ND6)
in the subject's mitochondrial DNA.

Main Exclusion Criteria:

- Light Perception (LP) or No Light Perception (NLP) visual acuity in any eye, as
defined by the study's standard operating procedure (SOP) for visual acuity testing.

- Presence of active infectious conjunctivitis, keratitis, scleritis or endophthalmitis
in either eye.

- Presence of alcoholism, alcohol dependence, or alcohol or drug abuse (excluding
nicotine).
We found this trial at
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sites
1675 Aurora Court
Aurora, Colorado 80045
Principal Investigator: Prem Subramanian, MD
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Atlanta, Georgia 30322
Principal Investigator: Nancy Newman
Phone: 770-715-2555
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243 Charles St
Boston, Massachusetts 02114
(617) 523-7900
Principal Investigator: Dean Cestari
Phone: 617-573-3412
Massachusetts Eye & Ear Infirmary Whether you see our physicians at Mass. Eye and Ear's...
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Gent, 9000
Principal Investigator: Bart Leroy
Phone: 3293326344
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Nashville, Tennessee 37232
Principal Investigator: Sean Donahue
Phone: 615-936-1639
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1428 Madison Ave
New York, New York 10029
(212) 241-6500
Principal Investigator: Rudrani Banik
Phone: 212-979-4200
Icahn School of Medicine at Mount Sinai Icahn School of Medicine at Mount Sinai is...
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Pasadena, California 91105
Principal Investigator: Alfredo Sadun
Phone: 626-817-4728
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Philadelphia, Pennsylvania 19107
Principal Investigator: Mark Moster
Phone: 215-825-4725
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Philadelphia, PA
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