Single-centre, Safety and Efficacy, Open-label Study Evaluating Antithymocyte Globulin Treatment in Subjects With Graves Orbitopathy
试验速览
- 阶段
- 不适用
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- ≥2 point change in Clinical Activity Score from baseline
研究概览
简要总结
The overall objective of the study is to evaluate the safety and efficacy of rabbit antithymocyte globulin in the treatment of Graves orbitopathy (GO) after ineffective treatment with moderate-to-high doses of glucocorticoids.
详细描述
This is a prospective interventional, single-center study examining the safety and efficacy of rabbit antithymocyte globulin (rATG) in adult patients with active moderate-to-severe GO after ineffective treatment with moderate-to-high doses of glucocorticoids. All enrolled participants will receive 0.8 - 1.0 mg/kg of rATG (cumulative dose of 150-200 mg given intravenously in two or three divided doses, 24 hours apart) after premedication with methylprednisolone i.v. (in total dose of 375 mg), 1 mg of antihistaminic agent clemastine and 1000 mg paracetamol i.v.
In order to assess efficacy and safety of the treatment, patients will be evaluated at baseline and at 6, 12, 24 and 48 weeks. Baseline and subsequent evaluation will involve medical history, physical examination, including detailed eye examination, laboratory assessment (thyroid-stimulating hormone [TSH], flow cytometry, TSH-receptor antibodies, CBC) and orbital magnetic resonance imaging (MRI).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of Graves' disease associated with active thyroid eye disease and a clinical activity score of ≥ 3
- •Euthyroid or with mild hypo- or hyperthyroidism defined as free thyroxine (FT4) and free triiodothyronine (FT3) levels less than 50% above or below the normal limits
- •previous ineffective treatment (partial response, recurrence or progression of symptoms) with moderate-to-high doses of glucocorticoids (at least 4.5 g of methylprednisolone)
排除标准
- •hypersensitivity to rabbit proteins or to any product excipients
- •active acute or chronic infections
- •latent tuberculosis
- •leucopenia below 3000/μl
- •lymphopenia below 400/μl
- •thrombocytopenia below 75000/μl
- •coagulation disorders
- •active malignancy and pregnancy
研究组 & 干预措施
Rabbit antithymocyte globulin (rATG)
干预措施: rabbit anti-thymocyte globulin (Drug)
结局指标
主要结局
≥2 point change in Clinical Activity Score from baseline
时间窗: Week 6, 12, 24, 48
change in proptosis
时间窗: 48 weeks
a diplopia response
时间窗: 48 weeks
change of distant best-corrected visual acuity
时间窗: Week 6, 12, 24, 48
change of mean retinal sensitivity
时间窗: Week 6, 12, 24, 48
次要结局
- changes in TSH-receptor antibodies level(Week 6, 12, 24, 48)
- changes in CD4/CD8 ratio(Week 6, 12, 24, 48)
- increase in 1 degree amplitude in Pattern visual evoked potential (VEP) by 1 µV(Week 6, 12, 24, 48)
研究者
Gabriela Handzlik
Principal Investigator
Medical University of Silesia