A Phase 2/3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- argenx
- 入组人数
- 98
- 试验地点
- 129
- 主要终点
- Number of Participants With CRoff at Week 36
研究概览
简要总结
ARGX-113-2009 is an operationally seamless 2-part, phase 2/3, prospective, global, multicenter, randomized, double-blinded, placebo-controlled study to investigate the efficacy, safety, tolerability, immunogenicity, participant-reported outcome measures (including those assessing participant QoL), PK, and PD of efgartigimod PH20 SC administered via subcutaneous (SC) injection in adult participants with moderate to severe BP. This study intends to demonstrate that efgartigimod is an effective and safe treatment for BP, providing participants with control of disease activity (CDA) and eventually remission while reducing their cumulative exposure to OCS.
study will consist of 2 parts:
- Part A of the study is a phase 2 evaluation that intends to provide proof of concept for the therapeutic activity of efgartigimod PH20 SC in participants with BP.
- Part B of the study is a phase 3 evaluation that intends to confirm the results obtained from part A in a separate, larger group of participants with BP.
An interim analysis will be performed during part A (on data obtained through week 26 for all Part A participants) to assess the primary endpoint and several secondary endpoints, confirm the appropriate sample size for part B of the study, and determine whether the efficacy results observed through week 26 of part A warrant continued study of efgartigimod PH20 SC for the treatment of participants with BP (futility analysis).
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The participant is willing and able to do the following:
- •understand the requirements of the study
- •provide written informed consent
- •comply with the study protocol procedures.
- •The participant is male or female and has reached the age of consent at the time of signing the informed consent form (ICF).
- •Participants have clinical signs of BP.
- •Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies and: Women of childbearing potential must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before study intervention can be administered.
- •The full list of inclusion criteria can be found in the protocol.
排除标准
- •Other forms of pemphigoid or other autoimmune bullous diseases (AIBDs).
- •Received unstable dose of treatments known to cause or exacerbate BP for at least 4 weeks prior to the baseline visit
- •Use of BP treatments other than oral corticosteroids (OCS), topical corticosteroids (TCS), conventional immunosuppressants or dapsone.
- •Known contraindication to OCS therapy
- •Active, chronic or latent infection at screening
- •Positive COVID-19 test result at screening (testing performed if required per local regulations).
- •History of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of the IMP. Participants with the following cancers can be included at any time, provided they are adequately treated prior to their participation in the study: Basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast, Incidental histological finding of prostate cancer
- •Clinical evidence of other significant serious diseases, have had a recent surgery, or who have any other condition that, in the opinion of the investigator, could confound the results of the study or put the patient at undue risk or prevent participants from complying with protocol requirements
- •Use of an investigational product within 3 months before the first dose of IMP
- •Previously participated in a clinical study with efgartigimod or currently participating in another interventional clinical study
- •Known hypersensitivity to any of the components of the administered treatments
- •Positive serum test at screening for an active infection: HBV, HCV, HIV
- •Current or history (ie, within 12 months of screening) of alcohol, drug, or medication abuse as assessed by the investigator
- •Pregnant or lactating females and those who intend to become pregnant during the study
- •Live or live-attenuated vaccine received <4 weeks before baseline visit
- •The full list of exclusion criteria can be found in the protocol.
研究组 & 干预措施
efgartigimod PH20 SC
participants receiving efgartigimod PH20 SC on top of Prednisone
干预措施: efgartigimod PH20 SC (Biological)
efgartigimod PH20 SC
participants receiving efgartigimod PH20 SC on top of Prednisone
干预措施: Prednisone (Drug)
placebo PH20 SC
participants receiving placebo PH20 SC on top of Prednisone
干预措施: placebo (Other)
placebo PH20 SC
participants receiving placebo PH20 SC on top of Prednisone
干预措施: Prednisone (Drug)
结局指标
主要结局
Number of Participants With CRoff at Week 36
时间窗: at week 36
CRoff = complete remission while receiving efgartigimod PH20 SC or placebo and being off oral corticosteroid therapy for at least 8 weeks. Complete remission is defined as the absence of new lesions, complete healing of existing lesions and absence of pruritus.
次要结局
- Cumulative OCS Dose(up to week 36)
- Number of Participants Who Achieve an IGA-BP Score of 0 While Off OCS Therapy for ≥8 Weeks at Week 36(at week 36)
- Number of Participants With CDA Who Remained Free of Relapse Through Week 36(up to week 36)
- Number of Participants With CRmin at Week 36(at week 36)
- Change From Baseline to Week 36 in the 24-Hour Average Itch NRS Score(up to week 36)
- Changes From Baseline in the BPDAI Total Activity Score(up to week 36)
- Time to CDA(up to week 36)
- Time to CR(up to week 36)
- Time to CRmin(up to week 36)
- Time to CRoff/PRoff(up to week 36)
- Time to CRoff(up to week 36)
- Time From CDA to Achieve Relapse(up to week 36)
- Number of Participants Who Receive Rescue Therapy Before Week 36(at week 36)
- The AIS From the GTI(up to week 36)
- The CWS From the GTI(up to week 36)
- EQ-5D-5L VAS Scores Over Time(up to week 36)
- DLQI Scores Over Time(up to week 36)
- ABQoL Scores Over Time(up to week 36)
- Efgartigimod Serum Concentrations(up to week 36)
- Percent Change of Total IgG Serum Levels From Baseline Over Time(up to 36 weeks)
- Percent Change of Anti-BP180 and Anti-BP230 Antibodies From Baseline Over Time(up to 36 weeks)
- Incidence of Antidrug Antibodies Against Efgartigimod and Antibodies Produced Against rHuPH20(up to 46 weeks)
- Number of Participants (or Their Caregivers) Who Are Determined by Site Staff to be Sufficiently Competent in (Self-)Administering Efgartigimod PH20 SC(up to week 32)