A Randomized, Double-Blinded, Placebo-Controlled Clinical Trial Assessing the Therapeutic Efficacy of Botulinum Toxin In Treating Scleroderma-Associated Raynaud's Syndrome
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Change in Digital Blood Flow From Pre- to Post-injection.
研究概览
简要总结
This is a randomized, double-blinded, clinical trial assessing the therapeutic efficacy of Botulinum toxin A (Onabotulinumtoxin A) in treating scleroderma-associated Raynaud's syndrome. Each patient will undergo injection with a treatment dose of Botulinum toxin A in one randomly-selected hand, and the contralateral hand will be injected with sterile saline (placebo) to serve as a control.
Study participants at the first study visit will complete study questionnaires, their hands will be assessed clinically for digital ulceration, and their hands will undergo non-invasive laser Doppler imaging to assess blood flow. After this initial assessment, the patients will undergo peri-arterial injection of Botulinum toxin A in one hand, and of sterile saline solution (placebo) in the other, in a randomized, blinded manner.
Patient will report the severity of their Raynaud's symptoms weekly over the four month study period. At one month post-injection, the patient will complete study questionnaires, their hands will be assessed clinically for digital ulceration, and their hands will undergo non-invasive laser Doppler imaging. At four months post-injection, the patient will again complete study questionnaires, their hands will be assessed clinically for digital ulceration, and their hands will undergo non-invasive laser Doppler imaging. In addition, patient will be given the option of one week post-injection visit, at which point the same assessment will be performed.
At the conclusion of the study, unblinding will occur.
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
- •Aged 18 years and older
- •Diagnosed with scleroderma.
- •Symptoms of Raynaud's syndrome affecting both hands (not necessarily to equal extents)
- •Ability to return/be available for follow-up evaluations
- •Able and willing to give informed consent
- •Able to speak and read in the English language.
排除标准
- •A history of Myasthenia gravis.
- •Reported allergy or hypersensitivity to any Botulinum toxin preparation.
- •Active infection in either hand.
- •Patients who have ever received Botulinum toxin vaccine.
- •Pregnant or lactating women.
- •Females unable or unwilling to maintain abstinence or use contraception for 28 days following the injections.
- •Patients who have previously undergone any vascular surgery on the upper extremity, including surgical sympathectomies.
- •Current use of any aminoglycoside antibiotic
研究组 & 干预措施
Onabotulinumtoxin A
One hand of each patient will be randomly selected for injection of Botulinum Toxin A (Onabotulinumtoxin A, 20 units/mL).
Injections are performed using a 30 gauge insulin syringe through the dorsal surface in seven locations: adjacent to the 2nd, 3rd, and 4th common digital arteries through the web spaces (10 units each=0.5mL each), and the radial side of the index finger metacarpal head, the ulnar side of the small finger metacarpal head, and each side of the thumb metacarpal head (5 units each= 0.25mL each). Total treatment dose of Botulinum Toxin A will not exceed 50 units per hand.
干预措施: Onabotulinumtoxin A (Drug)
Placebo
One hand of each patient will be randomly selected for injection of sterile saline solution (placebo).
Injections are performed using a 30 gauge insulin syringe through the dorsal surface in seven locations: adjacent to the 2nd, 3rd, and 4th common digital arteries through the web spaces (0.5 mL each), and the radial side of the index finger metacarpal head, the ulnar side of the small finger metacarpal head, and each side of the thumb metacarpal head (0.25 mL each).
干预措施: sterile saline solution (Drug)
结局指标
主要结局
Change in Digital Blood Flow From Pre- to Post-injection.
时间窗: Measured pre-injection and at one month post-injection.
The primary outcome measure is change in blood flow to the fingers, from a pre-injection baseline to post-injection follow-up visit as measured by non-invasive laser Doppler imaging.
次要结局
- Assessment of Raynaud's Symptom Severity Using the McCabe Cold Sensitivity Score.(Measured at one month post-injection.)
- Rate of Change in Raynaud's Phenomenon Symptoms Measured With the Raynaud's Condition Score.(Weekly rate of change over the four-month study period.)
- Number of Ulcers as Measure of Digital Ulcer Healing(Measured at one month post-injection.)
- Assessment of Raynaud's Symptoms Severity Using the Quick-DASH Score.(Measured at one month post-injection.)
- Assessment of Raynaud's Symptom Severity Using the VAS for Pain.(Measured at one month post-injection.)
研究者
Scott D. Lifchez
Associate Professor
Johns Hopkins University