EUCTR2010-023111-34-NL进行中（未招募）不适用
A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected SAR422459, Administered to Patients with Stargardt's Macular Degeneration.
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 46
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •-Signed and dated written informed consent obtained from the patient and/or the patient's legally acceptable representative.
- •-Diagnosis of Stargardt's Macular Degeneration, with at least one pathogenic mutant ABCA4 allele on each chromosome.
- •-Women of childbearing potential must have a negative pregnancy test at Day -1, and agree to use an effective form of contraception for at least three months, or be surgically sterile or postmenopausal, with the last menstrual period being over two years prior to enrollment .
- •-Males must agree with their partner to use two forms of contraception for at least three months following SAR422459 administration.
- •-Patients enrolled in France must be affiliated to or benefit from a social security regimen.
- •-Specific Inclusion Criteria Patient Group A:
- •-Patients (18 years or older) with Advanced Stargardt's Macular degeneration.
- •-Visual acuity =20/200 in the worst eye
- •-Severe cone-rod dysfunction with no detectable or severely abnormal full-field
- •electroretinogram responses.
- •-Specific Inclusion Criteria Patient Group B:
- •-Patients (18 years or older) with Stargardt’s Macular Degeneration.
- •-Visual Acuity =20/200 in the worst eye.
- •-Abnormal full-field electroretinogram responses.
- •-Specific Inclusion Criteria Patient Group C:
- •-Patients (18 years or older) with Stargardt’s Macular Degeneration.
- •-Visual acuity =20/100 in the worst eye.
- •-Abnormal full-field electroretinogram responses.
- •-Specific Inclusion Criteria Patient Group D:
- •-Symptomatic patients (6 years and older) with childhood or young adult onset
- •Stargardt's Macular Degeneration (eg, before age 26) with at least one
- •pathogenic mutant ABCA4 allele on each chromosome confirmed by direct
- •sequencing and co-segregation analysis within the patient’s family.
- •-Visual acuity of =20/200 in both eyes at the time of the screening visit.
- •-Patients are anticipated to experience rapid deterioration in visual function
- •and/or retinal structure in the opinion of the study Investigator.
- •NOTE: GROUP D WILL BE OPEN FOR RECRUITMENT ONLY AFTER AUTHORIZATION.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 14
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 32
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •-Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints.
- •-Cataract surgery with intraocular lens implantation within 6 months of enrolment.
- •-Aphakia or prior vitrectomy in the study eye.
- •-Concomitant systemic diseases including those in which the disease itself, or the treatment for the disease, can alter ocular function.
- •-Any intraocular surgery or laser in either eye planned within 6 months of Day 0.
- •-Any contraindication to pupil dilation in either eye.
- •-Any known allergy to any component of the delivery vehicle or diagnostic agents used during the study, or medications planned for use in the peri-operative period, particularly topical, injected or systemic corticosteroids.
- •-Any injectable intravitreal treatment to the treated eye or intravitreal device in the treated eye within 6 months prior to screening.
- •-Any periocular injections of corticosteroids to the treated eye within 4 months prior to screening.
- •-Laboratory test abnormalities or abnormalities in electrocardiogram, chest X rays that in the opinion of the principal investigator, would make the patient unsuitable for participation in the study.
- •-Significant intercurrent illness or infection during the 28 days prior to enrolment.
- •-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.
- •-Alcohol or other substance abuse.
- •-Contraindications to use of anaesthesia (local or general, as appropriate).
- •-Concurrent anti-retroviral therapy that would inactivate the investigational agent.
- •-History of any investigational agent within 28 days prior to SAR422459 administration.
- •-Participation in a prior ocular gene transfer therapy study.
- •-Enrollment in any other clinical treatment study throughout the duration of the SAR422459 study.
- •-Current or anticipated treatment with anticoagulant therapy or the use of anticoagulation therapy within the four weeks prior to surgery.
- •-A past medical history of human immunodeficiency virus (HIV) or hepatitis A, B or C infection.
- •-Women who are pregnant or are breastfeeding.
- •-History of or signs consistent with unilateral amblyopia (strabismic, anisometropic or stimulus deprivation).
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