A Double-Blind, Placebo-Controlled, Dose Escalation Study to Assess the Efficacy, Safety and Pharmacokinetics of Voclosporin in Adolescent and Pediatric Subjects With Lupus Nephritis
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 9
- 试验地点
- 6
- 主要终点
- Proportion of subjects with renal response
研究概览
简要总结
The purpose of this study is to assess the efficacy and safety of voclosporin compared to placebo in achieving renal response following 24 weeks of therapy in adolescent and pediatric subjects with active lupus nephritis (LN).
详细描述
This is a 24 week, dose escalation study of voclosporin in addition to standard of care with mycophenolate mofetil (MMF) and steroids, consisting of 3 treatment periods: Period 1 is double-blind, placebo controlled receiving 15.8 mg twice daily, Period 2 is open-label receiving 23.7 mg twice daily, Period 3 is open-label receiving 15.8 mg twice daily.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Double blind (participant, care provider, investigator and outcome assessor) for period 1 and Open-label for period 2 and period 3
入排标准
- 年龄范围
- 5 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Previous diagnosis of systemic lupus erythematosus (SLE) as per the 2019 EULAR/ ACR classification criteria.
- •Subjects with kidney biopsy confirmed active lupus nephritis.
排除标准
- •Estimated glomerular filtration rate (eGFR) <60 mL/minute/1.73 m2 at screening.
- •Current or medical history of:
- •Congenital or acquired immunodeficiency.
- •Clinically significant drug or alcohol abuse prior to screening.
- •Malignant neoplasm.
- •Lymphoproliferative disease or previous total lymphoid irradiation.
- •Known severe viral infections within 3 months of screening; or known human immunodeficiency virus infection, or hepatitis B or C virus infection at any time prior to screening.
- •Active tuberculosis (TB) or known history of TB/evidence of old TB if not taking prophylaxis with isoniazid.
- •Currently requiring renal dialysis (hemodialysis or peritoneal dialysis) or expected to require dialysis during the study period.
- •Other known clinically significant active medical conditions, for which the condition or the treatment of the condition may affect the study assessments or outcomes.
- •Currently taking or known need for any of the following medications:
- •Immunosuppression biologic agents within 12 weeks prior to randomization, cyclophosphamide, calcineurin inhibitors (CNIs) and live attenuated vaccines, initiation or dose change of ACE inhibitors/ARBs within 4 weeks prior to randomization, IV corticosteroids and IV immunoglobulin within 2 weeks prior to screening, strong CYP3A4/5 inhibitors and inducers within 2 weeks prior to randomization.
研究组 & 干预措施
Voclosporin treatment group 1
2 capsules (15.8 mg) BID of voclosporin
干预措施: Voclosporin (Drug)
Placebo treatment group 2
2 capsules BID of placebo
干预措施: Placebo Oral Capsule (Drug)
Voclosporin treatment group 3
3 capsules (23.7 mg) BID of voclosporin
干预措施: Voclosporin (Drug)
Voclosporin treatment group 4
Maximum dose of 2 capsules (15.8 mg) BID of voclosporin.
干预措施: Voclosporin (Drug)
结局指标
主要结局
Proportion of subjects with renal response
时间窗: Week 24
Renal response defined as UPCR ≤0.5 mg/mg, eGFR ≥60 mL/min/1.73 m2 or no decrease from baseline of \>20%, no rescue medication and no steroid use \>10 mg/day for ≥3 consecutive days or for ≥7 days in total between week 16 to 24
次要结局
- Time to UPCR of ≤0.5 mg/mg.(Baseline to Week 24)
- Proportion of subjects with partial renal response(Week 24)
- Number of treatment-emergent adverse events (TEAEs) will be summarized by treatment group(Baseline to Week 24)
- Time to 50% Reduction in UPCR(Baseline to Week 24)