A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of SNF472 When Added to Background Care for the Treatment of Calciphylaxis
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 71
- 试验地点
- 37
- 主要终点
- Absolute Change in the BWAT - CUA Score for the Primary Lesion
研究概览
简要总结
The primary objectives are to assess the efficacy, safety, and tolerability of SNF472 compared to placebo when added to background care for the treatment of calciphylaxis (CUA).
详细描述
The formation and growth of calcified deposits in arterioles and other small blood vessels appears to be fundamental to the development of CUA especially in end stage renal disease patients. This phase 3 double-blind, randomized, placebo-controlled study is designed to assess the effect of SNF472 when added to background care to improve wound healing, as evaluated using Bates-Jensen Wound Assessment Tool (BWAT) scoring and pain as reported by the subject using a VAS scale. The study consists of a double-blind, randomized, placebo controlled period of 12 weeks followed by an open-label period of 12 weeks.. .
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
- •Female or male subjects, 18 years of age or older
- •Receiving maintenance HD in a clinical setting for at least 2 weeks prior to screening
- •Clinical diagnosis of CUA by the Investigator including ≥1 CUA lesion with ulceration of the epithelial surface
- •CUA wound-related pain shown by a Pain VAS score ≥50 out of 100
- •Primary lesion that can be clearly photographed for the purpose of protocol-specified wound healing assessments.
- •Willing and able to understand and sign the informed consent form and willing to comply with all aspects of the protocol
排除标准
- •History of treatment with bisphosphonates within 3 months of baseline
- •Severely ill subjects without a reasonable expectation of survival for at least 6 months
- •Subjects with a scheduled parathyroidectomy during the study period
- •Expectation for kidney transplant within the next 6 months based on Investigator assessment or identification of a known living donor
- •Pregnant or trying to become pregnant, currently breastfeeding, or of childbearing potential (including perimenopausal women who have had a menstrual period within one year) and not willing to comply with protocol required contraception criteria
- •Significant noncompliance with dialysis
- •History of active malignancy within the last year with the exception of localized basal cell or squamous cell carcinoma
- •Clinically significant illness other than CUA within 30 days
- •Participation in an investigational study and receipt of an investigational drug or investigational use of a licensed drug within 30 days prior to screening.
- •History or presence of active alcoholism or drug abuse as determined by the Investigator within 6 months
- •Mental impairment, current significant psychiatric disease, or other conditions or circumstances that would make the subject unlikely to complete the study or comply with the study procedures.
- •Subjects whose CUA lesions exhibit significant improvement, in the opinion of the Investigator, between the first and second screening visit
研究组 & 干预措施
SNF472 (Open-Label)
Dose: 7 mg/kg SNF472 diluted in 100 mL physiological saline.
干预措施: Experimatenl SNF472 (Open-label) (Drug)
结局指标
主要结局
Absolute Change in the BWAT - CUA Score for the Primary Lesion
时间窗: from Baseline to Week 12
The Bates Jensen Wound Assessment Tool (BWAT) CUA score ranges from a minimum score of 8 (best) to a maximum score of 40 (worst). BWAT-CUA= Bates-Jensen Wound Assessment Tool-Calcific Uremic Arteriolopathy
Absolute Change in Pain Visual Analog Score
时间窗: from Baseline to Week 12
The Pain Visual Analog Scale (VAS) score ranges from a minimum score of 0 (no pain) to 100 (worst possible pain).
次要结局
- Absolute Change in the BWAT Total Score for the Primary Lesion(from Baseline to Week 12)
- Absolute Change in the Wound-Quality of Life Score(from Baseline to Week 12)
- Rate of Change in Opioid Use as Measured in Morphine Milligram Equivalents (MME)(from Baseline to Week 12)
- Qualitative Wound Image Evaluation for the Primary Lesion(at Week 12)