NCT04138485撤回2 期
A Randomized, Multicenter, Double-Blind, Placebo Controlled, Phase 2 Study to Evaluate the Efficacy and Safety of IgPro10 (Intravenous Immunoglobulin, Privigen®) for the Treatment of Adults With Systemic Sclerosis
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 发起方
- CSL Behring
- 试验地点
- 77
- 主要终点
- Response on American College of Rheumatology Combined Response Index in Diffuse Systemic Sclerosis (ACR CRISS) score in IgPro10 vs Placebo
研究概览
简要总结
This randomized, multicenter, double-blind (DB), placebo controlled, phase 2 study will evaluate the efficacy and safety of IgPro10. The DB Treatment Period will be followed by a 24-week Open-label (OL) Treatment Period.
Eligible subjects will be randomized at Baseline in a 2:1 ratio of treatment IgPro10 or placebo in the DB Treatment Period. All subjects who enter OL Treatment Period will receive IgPro10.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Age ≥18 years (male or female) at time of providing written informed consent
- •Documented diagnosis of SSc according to ACR / EULAR criteria 2013
- •mRSS ≥ 15 and ≤ 45
- •Disease duration ≤ 5 years defined as the time from the first non-Raynaud's phenomenon manifestation
- •Subjects within first 18 months of disease duration from first non-Raynaud's phenomenon manifestation.
排除标准
- •Primary rheumatic autoimmune disease other than dcSSc, including but not limited to rheumatoid arthritis, systemic lupus erythematosus, mixed connective tissue disorder, polymyositis, and dermatomyositis, as determined by the investigator Note: Subjects with fibromyalgia, secondary Sjogren's syndrome, and scleroderma-associated myopathy or myositis at Screening are not excluded
- •Positive anti-centromere autoantibodies at Screening
- •Evidence of severe chronic kidney disease with estimated glomerular filtration rate < 45 mL/min/1.73 m2 (as calculated by the Chronic Kidney Disease Epidemiology Collaboration equation) or receiving dialysis. Additionally, subjects with current confirmed diagnosis of diabetes mellitus and requiring medication, with eGFR < 90 mL/min/1.73m2 will be excluded from the study.
- •History of documented thrombotic episode eg, PE, DVT, myocardial infarction, thromboembolic stroke at any time Note: past superficial thrombophlebitis more than two years from Screening is not exclusionary
- •Documented thrombophilic abnormalities including blood hyperviscosity, protein S or protein C deficiency, anti-thrombin-3 deficiency, plasminogen deficiency, antiphospholipid syndrome, Factor V Leiden mutation, dysfibrinogenemia, or prothrombin G20210A mutation
- •Greater than 3 specified current risk factors for TEEs (documented and currents conditions): atrial fibrillation, coronary disease, diabetes mellitus, dyslipidemia, hypertension, obesity (Body Mass Index ≥ 30 kg/m2), recent significant trauma, and immobility (wheelchair-bound or bedridden)
- •Ongoing active serious infection at Screening (including, but not limited to, pneumonia, bacteremia/septicemia, osteomyelitis/septic arthritis, bacterial meningitis, or visceral abscess)
- •Malignancy in the past 2 years, except for non-melanoma skin cancer, cervical carcinoma in situ, or other in situ cancer if it has been excised and treated within in the past year
- •Known hypoalbuminemia, protein-losing enteropathies, and any proteinuria (defined by total urine protein concentration > 0.2 g/L)
- •Known IgA deficiency or serum IgA level < 5% lower limit of normal
研究组 & 干预措施
Placebo
Placebo Comparator
0.5% human albumin solution stabilized with 250 mmol/L L-proline
干预措施: Placebo (Biological)
IgPro10
Experimental
10% liquid formulation of human immunoglobulin for intravenous use
干预措施: IgPro10 (Biological)
结局指标
主要结局
Response on American College of Rheumatology Combined Response Index in Diffuse Systemic Sclerosis (ACR CRISS) score in IgPro10 vs Placebo
时间窗: Over 48 weeks
次要结局
- Proportion of responders (ACR CRISS > 0.6)(Over 72 weeks)
- Proportion of subjects meeting cardiopulmonary or renal failure criteria in ACR CRISS Step 1 events(Over 48 weeks)
- Mean change from Baseline in Modified Rodnan Skin Score (mRSS)(Baseline and over48 weeks)
- Mean change from Baseline in Health Assessment Questionnaire - Disability Index (HAQ-DI)(Baseline and over 72 weeks)
- Mean change from Baseline in Forced Vital Capacity (FVC)% predicted(Baseline and over 72 weeks)
- Mean change from Baseline in diffusing capacity of lung for carbon monoxide (DLCO)% predicted(Baseline and over 72 weeks)
- Mean change from Baseline in Physician Global Assessment (MDGA)(Baseline and over 72 weeks)
- Mean change from Baseline in Patient Global Assessment (PGA)(Baseline and over 48 weeks)
- Mean change from Baseline in UCLA Scleroderma Clinical Trial Consortium Gastrointestinal Tract 2.0 (UCLA SCTC GIT 2.0) total score and subscale(Baseline and over 48 weeks)
- Mean change from Baseline in Scleroderma Skin Patient Reported Outcome (SSPRO) score in IgPro10 vs Placebo(Baseline and up to 48 weeks)
- Proportion of responders in mRSS(Up to 48 weeks)
- Time to treatment failure (time from first infusion to time of first event) in IgPro10 vs Placebo(Over 48 weeks)
- Proportion of subjects with events at Week 48 in IgPro10 vs Placebo(Over 48 weeks)
- Mean change from Baseline in Cochin Hand Function Scale in IgPro10 vs Placebo(Baseline and over 48 weeks)
- Mean change from Baseline in Scleroderma Health Assessment Questionnaire (SHAQ) score in IgPro10 vs Placebo(Baseline and over 48 weeks)
- Mean change from baseline in muscle strength as measured by Manual Muscle Testing 8 (MMT) in IgPro10 vs Placebo(Baseline and over 48 weeks)
- Number of subjects with adverse events (AEs) including any AEs, treatment-emergent AEs (TEAEs), serious AEs (SAEs), and AEs of special interest (AESIs)(Over 72 weeks)
- Percentage of subjects with AEs, TEAEs, SAEs, AESIs(Over 72 weeks)
- Concentration of serum trough IgG levels at Baseline and prior to first infusion(Baseline and up to 72 weeks)
- Mean change from Baseline in Modified Rodnan skin score (mRSS)(Baseline and over 72 weeks)
- Mean change from Baseline in Patient global assessment (PGA)(Baseline and over 72 weeks)
- Mean change from Baseline in diffusing capacity of lung for carbon monoxide (DLCO)% predicted(Baseline and over 48 weeks)
- Proportion of responders (ACR CRISS > 0.6)(Over 48 weeks)
- Mean change from Baseline in Health Assessment Questionnaire - Disability Index (HAQ-DI)(Baseline and over 48 weeks)
- Mean change from Baseline in Forced Vital Capacity (FVC)% predicted(Baseline and over 48 weeks)
- Mean change from Baseline in Physician Global Assessment (MDGA)(Baseline and over 48 weeks)
- Number of subjects with adverse events (AEs) including any AEs, treatment-emergent AEs (TEAEs), serious AEs (SAEs), and AEs of special interest (AESIs)(Over 48 weeks)
- Percentage of subjects with AEs, TEAEs, SAEs, AESIs(Over 48 weeks)
研究者
研究点 (77)
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