A Phase 3, Randomized, Double-Masked, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Efgartigimod PH20 SC Administered by Prefilled Syringe in Adult Participants With Thyroid Eye Disease
概览
- 阶段
- 3 期
- 干预措施
- Efgartigimod PH20 SC
- 疾病 / 适应症
- 未指定
- 发起方
- argenx
- 入组人数
- 102
- 试验地点
- 154
- 主要终点
- Percentage of participants who were proptosis responders
- 状态
- 终止
- 最后更新
- 上个月
概览
简要总结
This study aims to evaluate the efficacy, safety and tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of efgartigimod PH20 SC in participants with active, moderate-to-severe TED, compared with placebo PH20 SC.
After the screening period, eligible participants will be randomized in a 2:1 ratio to receive efgartigimod PH20 SC or placebo PH20 SC, respectively during the double-blinded treatment period (DBTP). At the end of the DBTP, participants may enter a follow-up observational period while off study drug. Some participants may also enter the open-label treatment period with efgartigimod PH20 SC. The study duration varies from approximately 60 to 110 weeks.
An alternative list of clinical sites open for recruitment could be found in the other UplighTED study record (https://www.clinicaltrials.gov/study/NCT06307626).
This study was terminated early on 15 December 2025 as the pre-defined interim analysis concluded that continuing the trials is unlikely to demonstrate the intended efficacy. This decision is not related to safety concerns, and the safety profile of efgartigimod remains unchanged. End-of-study and Safety-Follow-Up visits are ongoing for the participants of this trial.
研究者
入排标准
入选标准
- •The participant is at least 18 years of age
- •The participant is capable of providing signed informed consent and following with protocol requirements
- •The investigator determines active, moderate-to-severe thyroid eye disease (TED) associated with autoimmune thyroid conditions (Graves' disease or Hashimoto's thyroiditis) for the most severely affected eye
- •The participant has first onset of active TED symptoms within 12 months before screening
- •The participant must have normal thyroid function with the baseline disease under control or have mild hypo or hyperthyroidism at screening. Every effort should be made to correct the mild hypo or hyperthyroidism promptly and to maintain the normal thyroid function for the full duration of the study
- •The participant agrees to use birth control consistent with local regulations and the people of child-bearing potential must have a negative blood pregnancy test at screening and a negative urine pregnancy test before receiving the study drug
排除标准
- •Optic neuropathy (damage to optic nerve), defined as new visual field defect (blind spot), relative afferent pupillary defect (pupils respond differently to light), or color defect secondary to optic nerve involvement within the 6 months before screening
- •Corneal decompensation (swelling of the cornea) unresponsive to medical management
- •Previous orbital irradiation or surgery for TED
- •Use of some medications before screening (more information is found in the protocol)
- •Known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of TED or puts the participant at undue risk
- •History of malignancy, cancer, unless considered cured by adequate treatment with no evidence of recurrence for ≥3 years. Adequately treated participants with the following cancers can be included at any time: Basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast, Incidental histological findings of prostate cancer
- •Clinically significant active infection that is not sufficiently resolved in the investigator's opinion or positive serum test at screening for active infection with any of the following: Hepatitis B virus (HBV), Hepatitis C virus (HCV), HIV
- •Current participation in another interventional clinical study or previous participation in an efgartigimod clinical study and at least 1 dose of study drug received or has received at least 1 dose of commercially available efgartigimod
- •Known hypersensitivity to study drug or one of its excipients (inactive ingredients)
- •History of or current alcohol, drug, or medication abuse within 12 months before screening as assessed by the investigator
研究组 & 干预措施
Efgartigimod arm
Participants with active, moderate-to-severe TED receiving Efgartigimod PH20 SC (subcutaneously) via pre-filled syringe (PFS)
干预措施: Efgartigimod PH20 SC
Placebo arm
Participants with active, moderate-to-severe TED receiving Placebo PH20 SC (subcutaneously) via pre-filled syringe (PFS)
干预措施: Placebo PH20 SC
结局指标
主要结局
Percentage of participants who were proptosis responders
时间窗: At week 24 of the Double-Blinded Treatment Period
次要结局
- Change in proptosis measurement in the study eye from baseline(Up to week 24 of the Double-Blinded Treatment Period)
- Change in the total Graves' Orbitopathy Quality of Life (GO-QoL) score from baseline(Up to week 24 of the Double-Blinded Treatment Period)
- Percentage of participants with a resolution of diplopia (responders)(At week 24 of the Double-Blinded Treatment Period)