2024-518489-29-00招募中2 期
A Phase 1/2 Multicenter, Open-label, Dose Escalation, Safety and Efficacy Study of Subretinal Administration of Dual AAV8.MYO7A, AAVB-081 in Participants with Usher Syndrome Type 1B (USH1B) Retinitis Pigmentosa
Aavantgarde Bio S.r.l.1 个研究点 分布在 1 个国家目标入组 9 人开始时间: 2024年11月19日最近更新:
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- The incidence and severity of adverse events (AEs) including serious adverse events (SAEs), treatment emergent adverse events (TEAEs) and dose limiting toxicities (DLTs) at 24 months.
研究概览
简要总结
- To assess the safety and tolerability of subretinal administration of AAVB-081 in participants with USH1B retinitis pigmentosa
- To determine a well-tolerated dose with optimal risk-benefit profile (RBP)
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 years 至 64 years（18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •All participants: Informed consent signed by the participant or participant’s legally authorized representative (if applicable).
- •All participants: Male and female adults diagnosed with USH1B; ≥ 18 to 50 ≤ 60 years of age
- •All participants: Molecular diagnosis of USH1B due to MYO7A mutations (homozygotes or compound heterozygotes) by a Sponsor approved CLIA laboratory.
- •All participants: Residual central visual function evidenced by LLVA of ≥10 letters in the MVA subjects.
- •All participants: Female participants of childbearing potential must use a highly effective method of contraception (see Appendix 3 for examples) for 1 year after treatment (from Day 0). Male participants who have a partner of childbearing potential must use condoms (barrier contraception) for 1 year after treatment. For the purpose of this protocol, a female participant is considered of childbearing potential (FPOCBP), i.e., fertile, following menarche and until becoming post-menopausal unless permanently sterile. Permanent sterilization methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy. A postmenopausal state is defined as no menses for 12 months without an alternative medical cause.
排除标准
- •Unable or unwilling to meet the requirements of the study.
- •Previous participation in any other gene therapy trial.
- •Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints (including but not limited to glaucoma, steroid response, corneal or significant lenticular or media opacities, cystoid macular oedema, macular hole, uveitis, epiretinal membrane).
- •Any systemic condition that would preclude subretinal surgery.
- •Profound vision loss in one eye with visual acuity of counting fingers (CF) or worse on semiquantitative scale.
- •Complicating ocular and systemic diseases, medications, or clinically significant abnormal baseline laboratory values.
- •Prior ocular non-macular laser within 3 months, prior cataract surgery within 3 months, use of post-operative anti-inflammatory drops in the past month, presence of inflammatory complications of cataract surgery in the past month, any other non-retinal intra-ocular surgery in the past 6 months prior to Day 0; and prior macular laser or retinal surgery at any time.
- •Use of high dose vitamin A (>7500 retinol equivalent units or >3300 IU per day), tretinoin-containing skin crème (e.g., Retin-A), or isotretinoin within 3 months prior to Day 0, or intended use during the study.
- •Chronic use of Viagra (sildenafil) or any other phosphodiesterase type 5 inhibitors used to treat erectile dysfunction, defined as at least once per month over the 12 months prior to Screening and until completion of study participation.
- •Participants who are positive for hepatitis B, hepatitis C, HIV, tuberculosis (TB), or syphilis infection.
- •Suspected or laboratory confirmed SARS-CoV-2 infection within 14 days prior to Day
- •History of retinal detachment.
- •Has received a live, attenuated vaccine within 30 days prior to Day
- •Examples of live vaccines include, but are not limited, to the following: measles, mumps, rubella, varicella/zoster (chicken pox), yellow fever, rabies, Bacillus Calmette-Guerin (BCG), typhoid vaccine, and covid vaccine.
- •Poorly controlled diabetes mellitus (of any type), defined as HbA1c ≥7, in the 6 months prior to subretinal injection.
- •Presence of moderate or severe non-proliferative diabetic retinopathy or worse, diabetic macular edema (DME), retinal tumors, high axial myopia (>-6 Diopters), and micro/nanophthalmos.
- •Known sensitivity to medications planned for use in the peri- operative period.
- •Participants who are pregnant and/or breastfeeding at Screening.
- •Any other condition that would not allow the potential participant to complete follow-up examinations during the course of the study and in the opinion of the investigator, makes the potential participant unsuitable for the study.
- •Unable to communicate with suitable verbal/auditory and/or tactile sign language (in the opinion of the investigator).
- •Participation in a clinical study with an investigational drug in the past six months, or 5 half-lives, whichever is longer.
结局指标
主要结局
The incidence and severity of adverse events (AEs) including serious adverse events (SAEs), treatment emergent adverse events (TEAEs) and dose limiting toxicities (DLTs) at 24 months.
The incidence and severity of adverse events (AEs) including serious adverse events (SAEs), treatment emergent adverse events (TEAEs) and dose limiting toxicities (DLTs) at 24 months.
The incidence and nature of DLTs at each dose level at 24 months
The incidence and nature of DLTs at each dose level at 24 months
次要结局
未报告次要终点
研究者
-
Scientific
Aavantgarde Bio S.r.l.
研究点 (1)
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