A Phase III gene therapy clinical trial in LHON Subjects Affected for 6 Months or Less
- Conditions
- eber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 geneTherapeutic area: Diseases [C] - Eye Diseases [C11]
- Registration Number
- EUCTR2015-001265-11-DE
- Lead Sponsor
- GENSIGHT-BIOLOGICS
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 40
Selection criteria:
1 Age 15 years or older
2 Onset of vision loss based on medically documented history or
subject testimony, in at least one eye for =180 days in duration and ifboth eyes are affected the duration of vision loss in both eyes must be = 180 days in duration
3 Each eye of the subject maintaining visual ability to allow at least for
counting of the examiner's fingers at any distance
4 Female subjects (if of childbearing potential) must agree to use
effective methods of birth control up to 6 months after IVT injection and male subjects must agree to use condoms for up to 6 months after IVT injection
5 Ability to obtain adequate pupillary dilation to permit thorough
ocular examination and testing
6 Signed written informed consent
Inclusion Criteria:
Subjects included in the study must satisfy all the following criteria at
the Inclusion Visit (Visit 2).
1 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
2 Review of all selection criteria to ensure continued compliance
3 Have a negative test for infection with human immunodeficiency
virus (HIV)
4 Have a negative pregnancy test for women of childbearing potential
(a woman who is two years post-menopausal or surgically sterile is not
considered to be of childbearing potential)
Are the trial subjects under 18? yes
Number of subjects for this age range: 7
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 38
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2
Non selection criteria:
Subjects who meet at least one of the following criteria at the Screening Visit (Visit 1) will not be included into the study
1 Any known allergy or hypersensitivity to GS010 or its constituents
2 Contraindication to IVT injection
3 IVT drug delivery to either eye within 30 days prior to the Screening
Visit (Visit 1)
4 Previous vitrectomy in either eye
5 Narrow angle in either eye contra-indicating pupillary dilation
6 Presence of disorders of the ocular media, such as the cornea and
lens, which may interfere with visual acuity and other ocular
assessments during the study period
7 Vision disorders, other than LHON, involving visual disability or with
the potential to cause further vision loss during the trial period.
8 Causes of optic neuropathy other than LHON and glaucoma.
9 Subjects with known mutations of other genes involved in
pathological retinal or optic nerve conditions
10 Presence of ocular or systemic disease, other than LHON and well-controlled glaucoma, whose pathology or associated treatments might affect the retina or the optic nerve
11 History of amblyopia associated with a Snellen visual acuity
equivalent of worse than 20/80 (equivalent to 6/24 at 6 meters, decimal acuity 0.25, LogMAR +0.6) in the affected eye
12 Presence of ocular conditions, which in the opinion of the
Investigator will prevent good quality SD-OCT imaging from being
obtained
13. Presence, in either eye, of uncontrolled glaucoma, defined as an
IOP greater than 25 mmHg, despite maximal medical therapy with IOP lowering agents
14 Active ocular inflammation or history of idiopathic or autoimmune associated uveitis
15 Subjects participating in another clinical trial and receiving an IMP
within 90 days prior to the Screening Visit (Visit 1)
16 Previous treatment with an ocular gene therapy product
17 Subjects who have undergone ocular surgery of clinical relevance
(per Investigator opinion) within 90 days preceding the Screening Visit
(Visit 1)
18 Female Subjects who are or who intend to breast feed during the
trial period
Subjects who meet at least one of the following criteria at the Inclusion Visit (Visit 2) will not be included in the study
1 Any non-selection criteria which may have appeared after the
screening visit
2 Subjects taking idebenone who have not completely discontinued the idebenone at least 7 days prior to Visit 2. If the subject has not
discontinued idebenone at least 7 days prior to Visit 2, the visit may be delayed until the 7-day period is complete
3 Presence, at the time of study inclusion, of infectious conjunctivitis,
keratitis, scleritis or endophthalmitis in either eye
4 Presence of systemic illness, including alcohol and drug abuse
(except nicotine), or medically significant abnormal laboratory values
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method