REVEAL-1 - A Phase 3, Randomized, Double-masked, Placebo-controlled, Efficacy, Safety, and Tolerability Study of VRDN-003 in Participants With Active Thyroid Eye Disease (TED)
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 132
- 试验地点
- 82
- 主要终点
- Proptosis Responder Rate in the study eye
研究概览
简要总结
This is a clinical trial assessing the efficacy, safety, and tolerability of an investigational drug, VRDN-003, in participants with active 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 active TED. The key objectives of this study are to determine if VRDN-003 is efficacious, safe and tolerable when administered as a series of subcutaneous/SC injections given every 4 weeks or every 8 weeks compared to placebo in participants with active TED.
Participants who do not have a meaningful response at Week 24 (irrespective of the initial treatment arm) may be eligible to receive additional subcutaneous injections of VRDN-003.
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)
盲法说明
Participants, Investigator, Outcomes Assessor and Sponsor will be masked.
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 active TED with documented evidence of ocular symptoms or signs that began within 15 months prior to screening
- •Must have a Clinical Activity Score (CAS) of ≥ 3 on the 7-item scale for the study eye
- •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 or steroid eye drops for any condition, including TED, or selenium within 2 weeks prior to first dose
- •Must not have received other immunosuppressive drugs for any condition, including TED, or any other therapy for TED within 12 weeks prior to first dose.
- •Must not have received an investigational agent for any condition, including TED, within 8 weeks or longer duration (depending on the type of investigational agent) prior to first dose
- •Must not have received radioactive iodine (RAI) treatment within 8 weeks prior to first dose
- •Must not have had previous orbital irradiation or decompression surgery for TED to the study eye's orbit
- •Must not have a pre-existing ophthalmic condition in the study eye which in the study doctor's opinion, would interfere with interpretation of study results
- •Must not have abnormal hearing test before first dose. Must also not have a history of ear conditions considered significant by study doctor
- •Must not have a history of inflammatory bowel disease
- •Female TED participants who must not be pregnant or breastfeeding
- •NOTE: There are additional eligibility criteria for participants who do not have a meaningful response at Week 24 (irrespective of initial treatment arm) who may receive additional injections of VRDN-
- •These are described in the protocol.
研究组 & 干预措施
VRDN-003 every 4 weeks
6 subcutaneous administrations of VRDN-003
干预措施: VRDN-003 (Drug)
VRDN-003 every 8 weeks
3 subcutaneous administrations of VRDN-003 and 3 subcutaneous administrations of placebo
干预措施: VRDN-003 (Drug)
VRDN-003 every 8 weeks
3 subcutaneous administrations of VRDN-003 and 3 subcutaneous administrations of placebo
干预措施: Placebo (Drug)
Placebo every 4 weeks
6 subcutaneous administrations of placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Proptosis Responder Rate in the study eye
时间窗: At Week 24
Proportion of participants with a ≥2 mm reduction from baseline in proptosis in the study eye \[without a corresponding increase of ≥2 mm in the fellow eye\]
Proptosis Responder Rate in the most proptotic eye as measured by exophtalmometer
时间窗: At Week 24
Proportion of participants with a ≥2 mm reduction from baseline in proptosis in the most proptotic eye as measured by exophtalmometer \[without a corresponding increase of ≥2 mm in the other eye\]
次要结局
- Change from baseline in proptosis in the study eye(At Week 24)
- Diplopia Responder Rate for participants with baseline Diplopia Score greater than 0(At Week 24)
- Clinical Activity Responder Rate in the study eye(At Week 24)
- Overall Responder Rate in the study eye(At Week 24)
- Change from baseline in Clinical Activity Score (CAS) in the study eye(At Week 24)
- Proportion of participants with a Clinical Activity Score (CAS) of 0 or 1 in the study eye(At Week 24)
- Diplopia Resolution Rate for participants with baseline Diplopia Score greater than 0(At Week 24)
- Change from baseline in proptosis in the most proptotic eye as measured by exophthalmometer(At Week 24)
- Proptosis Responder Rate in the most proptotic eye as measured by MRI(At Week 24)
- Change from baseline in proptosis in the most proptotic eye as measured by MRI(At Week 24)
- Overall Responder Rate in the most proptotic eye as measured by exophthalmometer(At Week 24)
- Change from baseline in Clinical Activity Score (CAS) in the most proptotic eye (as measured by exophthalmometer)(At Week 24)
- Proportion of participants with a Clinical Activity Score (CAS) of 0 or 1 in the most proptotic eye (as measured by exophthalmometer)(At Week 24)
- Clinical Activity Responder Rate in the most proptotic eye (as measured by exophthalmometer)(At Week 24)