A Randomized, Double-masked, Placebo-controlled Safety, Tolerability, and Efficacy Study of VRDN-001, a Humanized Monoclonal Antibody Directed Against the IGF-1 Receptor, in Participants With Chronic Thyroid Eye Disease (TED)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 188
- 试验地点
- 110
- 主要终点
- Proptosis Responder Rate (PRR) in the Most Proptotic Eye as Measured by Exophthalmometer
研究概览
简要总结
This is a clinical trial assessing the efficacy, safety, and tolerability of an investigational drug, veligrotug (VRDN-001), in participants with chronic thyroid eye disease (TED).
详细描述
This is a randomized (meaning participants will be assigned to study arms by chance), double-masked (meaning study doctor and participant will not know which study arm participant is assigned to), placebo-controlled study that will include participants with chronic TED. The key objectives of this study are to determine if veligrotug (VRDN-001) is efficacious, safe, and tolerable when administered as 5 IV infusions given every 3 weeks for a total of 12 weeks in participants with chronic TED.
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
- Triple (Participant, 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
- •Must have moderate to severe chronic TED with documented evidence of ocular symptoms or signs that began greater than 15 months prior to screening
- •Must have had a clinical diagnosis of TED, with any CAS (0-7)
- •Must agree to use highly effective contraception as specified in the protocol
- •Female TED participants must have a negative serum pregnancy test at screening
排除标准
- •Must not have received prior treatment with another anti-IGF-1R therapy
- •Must not have received systemic corticosteroids for any condition, including TED, or selenium within 2 weeks prior to first dose
- •Must not have received other immunosuppressive drugs or another investigational agent for any condition, including TED, or any other therapy for TED, within 8 weeks prior to first dose
- •Must not have received radioactive iodine (RAI) treatment within 8 weeks prior to first dose
- •Must not have a pre-existing ophthalmic condition in the study eye that in the opinion of the study doctor would confound interpretation of the study results
- •Must not have had previous orbital irradiation or decompression surgery for TED to the study eye's orbit
- •Must not have inflammatory bowel disease
- •Must not have abnormal baseline audiometry Pure Tone Average (PTA) assessment or history of significant (as determined by the Investigator) ear pathology, relevant ear surgery or hearing loss.
- •Female TED participants must not be pregnant or lactating
研究组 & 干预措施
Placebo
Participants will receive placebo matched to veligrotug as an IV infusion Q3W for 12 weeks.
干预措施: Placebo (Drug)
Veligrotug
Participants will receive veligrotug 10 mg/kg as an intravenous (IV) infusion every 3 weeks (Q3W) for 12 weeks.
干预措施: Veligrotug (Drug)
结局指标
主要结局
Proptosis Responder Rate (PRR) in the Most Proptotic Eye as Measured by Exophthalmometer
时间窗: Baseline to Week 15
Proptosis response in the most proptotic eye was defined as a reduction of proptosis of ≥2 millimeters (mm) from baseline in the most proptotic eye (without a corresponding increase of ≥2 mm in the other eye) as measured by exophthalmometer. Missing data were imputed with the Multiple Imputation (MI) method.
Proptosis Responder Rate in the most proptotic eye
时间窗: Week 15
Proptosis Responder Rate in the most proptotic eye (i.e., reduction of proptosis of ≥2 mm from baseline \[without a corresponding increase of ≥2 mm in the other eye\]) as measured by exophthalmometer.
次要结局
- Diplopia Responder Rate(Week 15)
- Diplopia Resolution Rate(Week 15)
- Change From Baseline in Proptosis in the Most Proptotic Eye as Measured by Exophthalmometer(Baseline, Week 15)
- PRR in the Most Proptotic Eye, as Measured by Magnetic Resonance Imaging (MRI)/Computed Tomography (CT)(Baseline to Week 15)
- Change From Baseline in Proptosis in the Most Proptotic Eye as Measured by MRI/CT(Baseline, Week 15)
- Clinical Activity Responder Rate in the Most Proptotic Eye as Measured by Exophthalmometer(Week 15)
- Overall Responder Rate (ORR) Comprising PRR and Clinical Activity Responder Rate in the Most Proptotic Eye as Measured by Exophthalmometer(Baseline to Week 15)
- Change from baseline in proptosis in the most proptotic eye(Week 15)
- Proptosis Responder Rate in the most proptotic eye(Week 15)
- Clinical Activity Responder Rate in the most proptotic eye(Week 15)
- Overall Responder Rate in the most proptotic eye(Week 15)
- Diplopia Responder Rate(Week 15)
- Diplopia Resolution Rate(Week 15)