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
- 发起方
- 入组人数
- 170
- 试验地点
- 28
- 主要终点
- Overall Response Rate comprised of Proptosis Responder Rate in the most proptotic eye at 3 weeks post the fifth IV infusion and Clinical Activity Responder Rate in the most proptotic eye at 3 weeks post the fifth IV infusion.
研究概览
简要总结
To determine the safety, tolerability, and efficacy of VRDN- 001 when given as 5 infusions of 10 mg/kg compared to placebo in participants with chronic TED.
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 否
入选标准
- •Males and/or females of age ≥18 to ≤75 years of age and older who have a clinical diagnosis of TED with any degree of disease severity and symptoms as measured by the CAS scale (0-7).
- •Participants must have moderate to severe chronic TED with proptosis (eye bulging) of at least 3 mm and >17 mm measurement at pre-dose baseline (Day -1 or Day 1) in the study eye.
- •Have experienced eye-related symptoms or signs that began more than 15 months before study screening.
- •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
- •Participants who have previously received treatment with other anti-IGF-1R therapy
- •Participants who have taken corticosteroids (except topically) or selenium (except multivitamins) for any condition, including TED, within 2 weeks before starting study medication. Also exclusionary are injections of corticosteroids into the eye within 3 months prior to the first dose of study medication or having received greater than 3 injections of corticosteroids into the eye at any time.
- •Participants who have received immunosuppressive agents, radioactive iodine, any other therapy for TED, or an investigational agent for any condition within 8 weeks prior to the first dose of study medication.
- •Participants who have a decrease in disease severity and symptoms as measured by CAS scale of at least 2 points in the study eye between screening and one day before treatment, for participants who had a CAS greater than 2 at screening.
- •Participants who have a decrease of at least 2 mm in proptosis (eye bulging) in the study eye from screening to one day before treatment.
- •Participants who require immediate surgical intervention and/or have other eye conditions as evaluated by the study doctor that are not responding to treatment.
- •Female participants who are pregnant or breastfeeding.
- •Have abnormal baseline hearing assessment
研究组 & 干预措施
VRDN-001 (Insulin-like growth factor-1 receptor [IGF-1R] inhibitor)
干预措施: VRDN-001 (Insulin-like growth factor-1 receptor [IGF-1R] inhibitor) (Drug)
SODIUM CHLORIDE
干预措施: SODIUM CHLORIDE (Drug)
结局指标
主要结局
Overall Response Rate comprised of Proptosis Responder Rate in the most proptotic eye at 3 weeks post the fifth IV infusion and Clinical Activity Responder Rate in the most proptotic eye at 3 weeks post the fifth IV infusion.
Overall Response Rate comprised of Proptosis Responder Rate in the most proptotic eye at 3 weeks post the fifth IV infusion and Clinical Activity Responder Rate in the most proptotic eye at 3 weeks post the fifth IV infusion.
Safety Endpoint: Adverse Events (AEs) and Serious Adverse Events (SAEs) will be monitored and recorded throughout the duration of the study. All clinically significant changes in other safety measurements will be recorded as AEs.
Safety Endpoint: Adverse Events (AEs) and Serious Adverse Events (SAEs) will be monitored and recorded throughout the duration of the study. All clinically significant changes in other safety measurements will be recorded as AEs.
次要结局
- Change from baseline in proptosis for the most proptotic eye at Week 15.
- Proptosis change from baseline, general and measured by exophtalmometer and responder rate at Week 15.
- Diplopia Responder and Resolution Rate (i.e., reduction in Diplopia Score to 0 from baseline for participants with baseline Diplopia Score >0) at Week 15.
研究者
Chief Development Officer
Scientific
Viridian Therapeutics S.à.r.l.