EUCTR2017-002204-27-GB进行中（未招募）1 期
A Phase 1/2a, Open-Label, Non-Randomized, Dose-Escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects with Retinitis Pigmentosa
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 18
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
入选标准
- •- Signed informed consent form
- •- Age =18 years to =75 years at the time of ICF signature
- •- Diagnosis of non-syndromic RP defined as: Clinical diagnosis of non-syndromic RP based on history, mid-peripheral visual dysfunction, and fundoscopic appearance, or diagnosis of non-syndromic RP is confirmed on full-field ERG
- •- Visual acuity: Visual acuity in the dose-escalation cohorts of no better than LP, or Visual acuity in the extension cohort of no better than CF pending review of dose-escalation cohort data by the DSMB
- •- Relatively preserved ganglion cell layer volume and retinal nerve fiber layer thickness, as measured with spectral domain optical coherence tomography (SD-OCT)
- •- Retains memory of former useful vision
- •- Ability to obtain adequate pupillary dilation to permit thorough ocular examination and testing
- •- Negative serum pregnancy test for women of childbearing age only
- •- Female subjects (if of childbearing potential) must agree to use highly effective methods of birth control up to 12 months after GS030-DP IVT, and male subjects must agree to use condoms for up to 12 months after GS030-DP IVT. (Highly effective methods of birth control include: combined (estrogen and progesterone containing) hormonal contraception (oral, injectable or transdermal) associated with inhibition of ovulation; progesterone-only hormonal contraception (oral, injectable or transdermal) associated with inhibition of ovulation; intrauterine device (IUD); intrauterine hormone-releasing system (IUS); bilateral tubal occlusion; vasectomized partner (provided that partner is the sole sexual partner of the woman of childbearing potential and that the vasectomized partner has received medical assessment of the surgical success); true sexual abstinence, when consistent with the preferred and usual lifestyle of the subject (sexual abstinence defined as refraining from heterosexual intercourse during the entire period of risk associated with study treatments).
- •- Ability to wear, utilize, and follow all instructions on proper use of the GS030-MD
- •- Interpupillary distance of =51 mm and =72 mm
- •- Refractive error of the study eye between -9 diopters and +6 diopters
- •- Review of all selection criteria to ensure continued compliance from Visit 2 through Visit 4
- •- Have a negative urine pregnancy test at Visit 4 for women of childbearing potential (women who are 2 years post-menopausal or surgically sterile are not considered to be of childbearing potential)
- •- Have a negative test result for infection with HIV (results from test performed at Visit 1)
- •- Ability to tolerate repeated light stimuli produced by the GS030-MD, as assessed in the inclusion phase (Visit 2 through Visit 4)
- •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 14
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 4
排除标准
- •- Prior receipt of any gene therapy
- •- Subjects participating in another clinical trial and receiving an investigational medicinal product within 90 days prior to Visit 1
- •- Subjects with systemic disease or other pathology other than that related to diagnosis of non-syndromic RP whose symptoms or associated treatments may affect vision, for example cancers or pathology of the central nervous system
- •- Subjects with systemic disease or other medical or psychiatric conditions that preclude safe participation in the study
- •- Subjects who are taking photosensitizing drugs used in psoralen plus ultraviolet light (PUVA) therapy for psoriasis, eczema, vitiligo, and other similar diseases, or photosensitizing drugs used for photodynamic therapy in the treatment of cancer or eye diseases
- •- Subjects receiving immunosuppressive therapies, other than the immune modulating regimen described in this protocol
- •- Subjects of reproductive potential unwilling to use effective contraception for the 12 months after administration of GS030-DP
- •- Subjects who are pregnant or breastfeeding
- •- Subjects who are unwilling or unable to comply with the study protocol
- •- Subjects with any condition that would not allow them to complete follow-up examinations during the study and, in the opinion of the investigator, would make them unsuitable for the study
- •- Subject who are human immunodeficiency virus (HIV) positive
- •- Subject with known allergy to corticosteroids, or who will be unable to tolerate the corticosteroid regimen, regimen, or with an active intercurrent infection contraindicating treatment.
- •- Subjects who have undergone significant ocular surgery (per investigator determination) within 3 months prior to Visit 1
- •- Presence of narrow iridocorneal angles contraindicating pupillary dilation
- •- Presence of disorders of the ocular media which interfere with visual acuity and other ocular assessments, including SD-OCT, during the study period
- •- Presence of any systemic or ocular diseases, or pathologies, other than non-syndromic RP, or their associated therapies, that can cause or have the potential to cause vision loss
- •- Prior vitrectomy or vitreomacular surgery
- •- Presence of vitreo-macular adhesion or traction, epiretinal membrane macular pucker and macular hole, evident by ophthalmoscopy and/or by SD-OCT examinations and assessed by the investigator to significantly affect central vision
- •- Current evidence of retinal detachment assessed by the investigator to significantly affect central vision
- •- Active ocular inflammation or recurrent history of idiopathic or autoimmune-associated uveitis
- •- Hypersensitivity to GS030-DP or to any of the ingredients
- •- Presence of an Active Implantable Medical Device.
- •- Subjects who have undergone thermal laser procedure to the retina within 3 months of trial entry, or any prior thermal laser procedure to the macular region.
- •- Any non-selection criteria which become applicable after the selection visit
- •- Presence, at the time of study inclusion, of infectious conjunctivitis, keratitis, scleritis, or endophthalmitis in either eye
研究者
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