A Multicenter, Double-Masked, Placebo-Controlled, Efficacy And Safety Study Of RV 001, An Insulin-Like Growth Factor-1 Receptor (IGF-1R) Antagonist Antibody (Fully Human), Administered Every 3 Weeks (q3W) By Intravenous (IV) Infusion In Patients Suffering From Active Thyroid Eye Disease (TED)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Amgen
- 入组人数
- 88
- 试验地点
- 15
- 主要终点
- Responder Status at Week 24
研究概览
简要总结
The primary objective of this study is to investigate the efficacy, safety, and tolerability of RV 001 (teprotumumab), a fully human anti-IGF1R antibody, administered q3W for 6 months, in comparison to placebo, in the treatment of participants suffering from active TED.
"Funding Source - FDA OOPD"
详细描述
Study with completed results acquired from Horizon in 2024.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 75 Years（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 TED and a clinical activity score of ≥ 4
- •Fewer than 9 months from onset of TED
- •No previous medical or surgical treatment, excluding local supportive measures and oral steroids if the maximum cumulative dose is less than 1000 mg methylprednisolone or equivalent with at least 6 weeks between last administration of oral steroids and randomization
- •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 (every effort should be made to correct the mild hypo- or hyperthyroidism promptly)
排除标准
- •Optic neuropathy
- •Corneal decompensation unresponsive to medical management
- •Oral or IV steroid treatment for any non-TED reason in the preceding 3 months
- •Poorly controlled diabetes
- •Platelets < 100 x 10^9/L
- •Hemoglobin concentration > 2 g/dL below the lower limit of normal
研究组 & 干预措施
Placebo
A placebo infusion (normal saline) administered q3W by IV infusion over a period of 24 weeks for a total of 8 infusions.
干预措施: normal saline (Drug)
Teprotumumab
Teprotumumab administered q3W by IV infusion over a period of 24 weeks for a total of 8 infusions. All participants start treatment at a dose of 10 mg/kg. At Week 3, the dose is escalated to 20 mg/kg and kept constant for the remainder of the study.
干预措施: teprotumumab (Drug)
结局指标
主要结局
Responder Status at Week 24
时间窗: Week 24
Number of participants classified as responders and non-responders at Week 24. Responders were defined as participants with a reduction in clinical activity score (CAS, see Outcome Measure 4 description for details) of ≥ 2 points, and a reduction in proptosis (amount of protrusion of the eye from the orbital rim) of ≥ 2 mm in the study eye, and no deterioration (increase in CAS of ≥ 2 points or increase in proptosis of ≥ 2 mm) in the non-study eye. Participants who had no assessment at 24 weeks were considered non-responders.
次要结局
- Overall Average Change From Baseline in Graves' Ophthalmopathy Quality of Life (GO-QOL) Scale - Overall to Week 24 (Mixed-Model Repeated Measures [MMRM])(Baseline to Week 24)
- Overall Average Change From Baseline in GO-QOL Scale - Visual Functioning to Week 24 (MMRM)(Baseline to Week 24)
- Overall Average Change From Baseline in Proptosis of the Study Eye to Week 24 (MMRM)(Baseline to Week 24)
- Overall Average Change From Baseline in CAS to Week 24 (MMRM)(Baseline to Week 24)
- Overall Average Change From Baseline in GO-QOL Scale - Appearance to Week 24 (MMRM)(Baseline to Week 24)