Ora Secunda Cerclage Laser Retinopexy To Prevent Retinal Detachment In Stickler Syndrome (OSC/SS): A Prospective Historically Controlled Study
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 500
- 试验地点
- 6
- 主要终点
- Number of Participants with SS who Develop RD After Undergoing the OSC/SS Procedure as Assessed by One of the Following Photographic Fundus Mapping and IDO Examinations
研究概览
简要总结
The goal of this clinical trial is to prospectively document to what extent the OSC/SS prophylactic laser retinopexy procedure works to prevent retinal detachment in SS in children and adults.
Researchers will compare the OSC/SS procedure in SS to the natural progression of SS to see to what extent the OSC/SS procedure works to prevent retinal detachment.
Participants will:
- Have the OSC/SS procedure in one or both eyes
- Have eye tests
- Have genetic testing for SS as needed
- Visit the study center 9 times over 5 years for checkups and tests
- Have data for the untreated fellow-eye collected and used as study data if available
详细描述
Prophylactic treatment for RD has been well-documented to be safe and effective for specific predisposing conditions, but parameters are not standardized and are poorly documented in the literature for Stickler syndrome. There is significant, but only retrospective, evidence in the scientific literature that non-invasive, encircling (360 degree) laser prophylaxis (cerclage prophylaxis) reduces the rate of retinal detachment in patients with Stickler syndrome by approximately five-fold. In February 2025, the American Academy of Ophthalmology (AAO) endorsed 360-degree cerclage laser prophylaxis for Stickler syndrome based on retrospective evidence. A statistical review of pertinent articles concluded that prophylactic laser therapy was associated with a significantly lower risk of retinal detachment in patients with Stickler syndrome compared to no laser prophylaxis. This clinical trial is designed to offer OSC/SS as the optimal form of such prevention, and to prospectively document, in a standardized manner, the extent to which it prevents retinal detachment in patients with Stickler syndrome.
This clinical trial aims to address the lack of standardized documentation in previous literature and studies by providing standardized treatment parameters and rigorously documenting the OSC/SS technique. By evaluating the safety and efficacy of OSC/SS in preventing RD in Stickler syndrome patients, this study seeks to establish a benchmark for future clinical practice and research.
The primary objective is to demonstrate the efficacy of the OSC/SS procedure compared to the natural progression of SS to prevent RD for individuals with SS. This comparison will be made using historical control data provided by Cambridge University, which documents the rate of occurrence of RD in the absence of prior prophylactic treatment. Cambridge University maintains a large SS database, consisting of decades of clinical and research data on Stickler syndrome populations, providing the necessary control raw data for this study population. If the OSC/SS procedure proves to be adequately successful in the study, then it may become the gold standard treatment used to prevent RD in patients with SS and, with modifications, other populations at high risk for RD.
Enrollment is competitive.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •No contraindications to laser retinopexy surgery per local site criteria.
- •Capable of giving signed informed consent, and assent (as appropriate)
- •Signature of research informed consent form from participant, or participant's legally authorized representative (LAR)/parent(s)/legal guardian
- •Male or female of any age
- •SS type 1 or type 2 confirmed by genetic testing
排除标准
- •Presence of media opacity (for example, dense cataract, corneal scar) or poor pupillary dilation that precludes adequate OSC/SS performance, laser treatment, or imaging.
- •Prior prophylactic laser treatment that cannot be supplemented to achieve a complete OSC/SS pattern or exceeds the designated OSC/SS pattern.
- •RD in the eye to be treated.
- •Significant intraocular hemorrhage in the eye to be treated.
- •Uncontrolled ocular conditions (e.g., glaucoma) that may be worsened by the procedure or preclude follow-up.
- •Any other ocular condition that precludes the ability to perform adequate laser treatment under scleral depression.
- •Any systemic medical contraindication where the risk of intervention outweighs the potential benefit.
- •Known pathogenic mutations in genes associated with other inherited retinal detachment syndromes, including but not limited to: FEVR-related genes: FZD4, LRP5, TSPAN12, NDPX-linked retinoschisis (RS1) Wagner Syndrome (VCAN).
- •Other syndromes associated with increased risk of retinal detachment (for example, Knobloch syndrome, Marfan syndrome, Pierson syndrome).
- •History of Coats disease.
- •History of Retinopathy of Prematurity (with or without laser treatment).
- •Prior laser photocoagulation in the study eye for any indication other than the following: retinal detachment prophylaxis for Stickler syndrome, treatment of lattice degeneration, or treatment of retinal tears.
- •Laser photocoagulation, for the indications permitted in exclusion criteria 12, that cannot be supplemented to meet the OSC/SS criteria.
- •Previous cryopexy.
- •Previous scleral buckle.
- •Previous vitrectomy in the study eye for any reason.
- •Significant peripheral retinal pathology unrelated to Stickler that increases the risk of retinal detachment including but not limited to: retinoschisis, hemorrhages, angiomatous lesions, pars planitis.
- •History of ocular trauma with significant trauma to the posterior segment of the eye and may increase the risk of retinal detachment including Ruptured globe, Vitreous hemorrhage, Commotio retinae, Traumatic macular hole, Intraocular foreign body.
- •Inability or unwillingness to comply with required follow-up schedule (clinical visits, imaging).
- •Anticipated relocation, foster care instability, or any factor significantly limiting longitudinal follow-up.
- •Investigator judgment: Any condition not listed above that could interfere with the trial's integrity or subject safety.
研究组 & 干预措施
Children, Adolescents, and Adults with SS treated with Prophylactic Laser Retinopexy
Intervention: Children, Adolescents, and Adults with SS treated with prophylactic laser retinopexy procedure.
干预措施: Prophylactic (non-invasive) Laser Retinopexy (Procedure)
结局指标
主要结局
Number of Participants with SS who Develop RD After Undergoing the OSC/SS Procedure as Assessed by One of the Following Photographic Fundus Mapping and IDO Examinations
时间窗: From enrollment to post initial procedure at 5 years
Efficacy of the OSC/SS procedure in preventing RD in participants with type 1 (STL1) and type 2 (STL2) SS will be assessed annually by tracking the number of participants who develop RD after undergoing the OSC/SS procedure, using data from one of the following: photographic fundus mapping and IDO examinations.
次要结局
- Frequency of Adverse Events as Assessed by Recurring Eye Evaluations (photographic fundus mapping, OCT, intraocular pressure, pupil measurements, slit lamp biomicroscopy, motility assessments, and vision acuity testing)(From enrollment to post initial procedure at 5 years)
- Size of Retinal Defects(From enrollment to post initial procedure at 5 years)
- Number of Occurrences of Retinal Defects(From enrollment to post initial procedure at 5 years)
- Measurement of Visual Acuity in Participants with SS Before and After the OSC/SS Procedure as Assessed by Using Standard of Care Methods(From enrollment to post initial procedure at 5 years)
- Number of Participants Experiencing Retinal Defects(From enrollment to post initial procedure at 5 years)
- Number of Surgeries to Repair Retinal Detachments or Retinal Defects(From enrollment to post initial procedure at 5 years)
- Number of Procedure Deviations by Treating Physicians as Assessed by Photographic Fundus Mapping(3 to 6 months post OSC/SS procedure)
- Size of Retinal Defects(From enrollment to post initial procedure at 5 years)
- Number of Occurrences of Retinal Defects(From enrollment to post initial procedure at 5 years)