A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected StarGen, Administered to Patients with Stargardt's Macular Degeneration.
- Conditions
- Stargardt Macular Dystrophy, also known as fundus flavimaculatus or Stargardt disease.
- Registration Number
- EUCTR2010-023111-34-FR
- Lead Sponsor
- Oxford BioMedica (UK) Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 27
1.Signed and dated written informed consent obtained from the patient in accordance with the local regulations.
2.Diagnosis of moderate to severe SMD, with at least one pathogenic mutation in the ABCA4 gene on each chromosome, previously confirmed by direct sequencing and co-segregation analysis within the patient’s family.
3.Women of childbearing potential must have a negative pregnancy test at screening and at baseline, and agreement to using an effective form of contraception such as the contraceptive pill or intra uterine device, or be surgically sterile or postmenopausal, with the last menstrual period being over two years ago. Partners of study patients must agree to use barrier contraception for at least three months after StarGen administration.
4.Males must agree with their partner to use two forms of contraception, including one barrier method for at least three months following StarGen administration if their partner is of child-bearing potential, or must be surgically sterile.
5.Affiliated with the French social security healthcare system (French patients only).
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
1.Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints: glaucoma or other primary optic neuropathy that has resulted in significant visual field loss, corneal or significant lens opacities, active uveitis, retinopathy and maculopathy (other than that from Stargardt disease) that in the opinion of the investigator is causing significant visual loss, myopia greater than eight diopters spherical equivalent.
2.Cataract surgery with intraocular lens implantation within 6 months of enrolment.
3.Aphakia or prior vitrectomy in the study eye.
4.Concomitant systemic diseases including those in which the disease itself, or the treatment for the disease, can alter ocular function. For instance malignancies whose treatment could affect central nervous system function, diabetes, juvenile rheumatoid arthritis or sickle cell disease.
5.Any intraocular surgery or laser in either eye within 6 months of Day 0.
6.Any contraindication to pupil dilation in either eye.
7.Any known allergy to any component of the delivery vehicle or diagnostic agents used during the study (e.g., fluorescein, dilation drops), or medications planned for use in the peri-operative period, particularly topical, injected or systemic cortico-steroids.
8.Any injectable intravitreal treatment to the treated eye or intravitreal device in the treated eye within 6 months prior to screening.
9.Any periocular injections of corticosteroids to the treated eye within 4 months prior to screening.
10.Clinically significant laboratory test abnormalities, including full blood count, chemistry panel, liver function tests, electrocardiogram and chest X rays, that in the opinion of the principal investigator, would make the patient unsuitable for participation in the study.
11.Significant intercurrent illness or infection during the 28 days prior to enrolment.
12.Pre-menopausal or non-surgically sterile women who are unwilling to use an effective form of contraception such as the contraceptive pill or intrauterine device.
13.Men or women who do not agree to use contraception as specified in the inclusion criteria.
14.Alcohol or other substance abuse.
15.Contraindications to use of anaesthesia (local or general, as appropriate).
16.Concurrent anti-retroviral therapy that would inactivate the investigational agent.
17.History of any investigational agent within 28 days prior to StarGen administration.
18.Participation in a prior ocular gene transfer therapy study.
19.Enrolment in any other clinical treatment study, for any condition, including those relating to SMD, throughout the duration of the StarGen study.
20.Current or anticipated treatment with anticoagulant therapy or the use of anticoagulation therapy within the four weeks prior to surgery.
21.Past medical history of HIV or Hepatitis A, B or C.
22.Inability to comply with the demands of the study.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To assess the safety and tolerability of ascending doses of StarGen in adult patients with Stargardt Macular Degeneration.;Secondary Objective: To determine a delay in retinal degeneration following subretinal injection of StarGen, through changes in function relative to he untreated contralateral eye utilising retinal analytical techniques.;Primary end point(s): The incidence and severity of treatment emergent adverse events.<br>Clinically important changes in the following safety assessments:<br>Best corrected visual acuity (BCVA)<br>Slit-lamp biomicroscopy<br>Indirect ophthalmoscopy<br>Fundus photography <br>Measurement of intraocular pressure<br>Visual field - microperimetry<br>Ocular Coherence Tomography (OCT)<br>Laboratory parameters<br>Vital signs<br>Concomitant medications<br>
- Secondary Outcome Measures
Name Time Method