2023-508645-40-00已完成3 期
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
- 3 期
- 状态
- 已完成
- 发起方
- Argenx
- 入组人数
- 90
- 试验地点
- 46
- 主要终点
- Proportion of participants who are in complete remission (CR) while receiving efgartigimod PH20 SC or placebo and have been off oral corticosteroid (OCS) therapy for ≥8 weeks at week 36
研究概览
简要总结
To evaluate the efficacy of efgartigimod PH20 SC on achieving sustained remission in the treatment of participants with bullous pemphigoid (BP)
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Treatment-free follow-up period
- 盲法
- None
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •The participant is willing and able to do the following: a. understand the requirements of the study b. provide written informed consent c. comply with the study protocol procedures.
- •The participant is male or female and has reached the local legal age of consent at the time of signing the informed consent form (ICF)
- •Participants have clinical signs of BP.
- •The participant agrees to use contraceptive measures consistent with local regulations. WOCBP must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before receiving IMP.
排除标准
- •Other forms of pemphigoid or other autoimmune bullous diseaes (AIBDs)
- •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 with any of the following conditions: a. HBV b. HCV c. 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
- •Received unstable dose of treatments known to cause or exacerbate BP for at least 4 weeks prior 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 before their participation in the study: a. Basal cell or squamous cell skin cancer b. Carcinoma in situ of the cervix c. Carcinoma in situ of the breast d. 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 or 5 half-lives (whichever is longer) before the first dose of IMP
结局指标
主要结局
Proportion of participants who are in complete remission (CR) while receiving efgartigimod PH20 SC or placebo and have been off oral corticosteroid (OCS) therapy for ≥8 weeks at week 36
Proportion of participants who are in complete remission (CR) while receiving efgartigimod PH20 SC or placebo and have been off oral corticosteroid (OCS) therapy for ≥8 weeks at week 36
次要结局
- Cumulative dose of OCS from baseline to week 36
- Proportion of participants who achieve an Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) score of 0 while receiving efgartigimod PH20 SC or placebo and have been off OCS therapy for ≥8 weeks at week 36
- Proportion of participants who achieve an Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) score of 0 or 1 while receiving efgartigimod PH20 SC or placebo and have been off OCS therapy for ≥8 weeks at week 36
- Proportion of participants who achieve an IGA-BP score of 0 or 1 while receiving efgartigimod PH20 SC or placebo at any time through week 36
- Proportion of participants who receive rescue therapy before week 36
- Proportion of participants who achieve control of disease activity (CDA) while receiving efgartigimod PH20 SC or placebo and remain free of relapse through week 36
- Proportion of participants who are in CR while receiving efgartigimod PH20 SC or placebo and have been receiving minimal OCS therapy for ≥8 weeks at week 36. (Minimal OCS therapy is defined as ≤0.1 mg/kg/day of prednisone [or an equivalent dose of another OCS].)
- Changes from baseline in the BPDAI activity score
- Changes from baseline in the 24-hour average itch score from the Itch Numerical Rating Scale (NRS)
- Changes from baseline in the 24-hour worst itch score from the Itch Numerical Rating Scale (NRS)
- Time to achieve the following: - CDA - CR - CR while on minimal OCS therapy for ≥8 weeks - CR/PR while off OCS therapy for ≥8 weeks - CR while off OCS therapy for ≥8 weeks - Relapse
- Cumulative OCS dose for the participant at the time points when they exhibit the following: - CDA - CR - CR while on minimal OCS therapy for ≥8 weeks - CR/PR while off OCS therapy for ≥8 weeks - CR while off OCS therapy for ≥8 weeks - Relapse
- Incidence and severity of TEAEs, AESIs, and SAEs
- The Aggregate Improvement Score (AIS) from the GTI
- The Cumulative Worsening Score (CWS) from the GTI
- The GTI Specific List (GTI-SL)
- EQ-5D-5L scores over time
- DLQI scores over time
- ABQoL scores over time
- EFG serum concentrations
- Percentage change of total IgG serum levels from baseline over time
- Percentage change of anti-BP180 and anti-BP230 antibodies from baseline over time
- Incidence and prevalence of antidrug antibodies (ADA) against efgartigimod (in serum) and antibodies against rHuPH20 (in plasma)
- Number and percentage of participants (or their caregivers) who complete the (self-)administration training at study sites
- Number and percentage of participants (or their caregivers) who are determined by site staff to be sufficiently competent in (self-)administering efgartigimod PH20 SC
- Number and percentage of participants (or their caregivers) who successfully (self-)administer efgartigimod PH20 SC under site staff supervision
研究者
Peter Ulrichts (Chief Scientific Officer)
Scientific
Argenx
研究点 (46)
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