Double Blind RCT to Evaluate the Effect of Botulinum Toxin in Raynaud Phenomenon
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 24
- 试验地点
- 1
- 主要终点
- Change in tissue Oximetry measure for each finger
研究概览
简要总结
Raynaud's Phenomenon (RP) is the most common vasospastic disorder encountered by hand surgeons with an estimated prevalence of 5% in the general population. It's manifestations are broad, and can range from minor to severe. Sometimes it can be refractory to treatments. The goal of this study is to evaluate the efficacy of Botulinum toxin (BT) in the treatment of treatment refractory RP by objective and subjective measures.
详细描述
Raynaud's Phenomenon (RP) is the most common vasospastic (i.e. conditions where small blood vessels near the surface of the skin have spasms that limit blood flow) disorder encountered by hand surgeons with an estimated prevalence of 5% in the general population. The clinical manifestations are broad, and can range from minor pain, cold sensitivity and color changes to impaired hand function, intolerable pain and tissue loss/digital ulceration. Treatment algorithms include activity modification and medical therapies. However, refractory RP can warrant surgical intervention, such as peri-arterial sympathectomy, of which has significant associated morbidity. While the exact mechanism is still being elucidated, several retrospective reviews have demonstrated efficacy of BT in the treatment of RP. Specifically, increases in post treatment perfusion have been demonstrated and clinical evidence of improvement in pain scores and tissue loss are reported. However, the literature varies in regard to injection technique (location and dose) and duration of action.
The goal of this study is to evaluate the efficacy of BT in the treatment of treatment refractory RP by objective and subjective measures. This study will perform a single-site double-blinded, randomized, placebo-controlled trial evaluating two different doses of BT (10 units and 20 units) injected into each patient's fingers of both hands versus injection of a normal saline control, given that there is currently no widely accepted standard of care for the treatment of BT. Following injection, patients will be evaluated in regular intervals to assess for impact on finger temperature, oxygenation, patient reported outcome measures (PROMs), and effect on tissue loss.
The study will take place under Emory University School of Medicine, specifically the Upper Extremity Division. Patients may also be originally seen under the Rheumatology Division and subsequently referred to the Upper Extremity Division for study participation. In total, the study aims to recruit 36 patients. There will be no outside recruitment or advertisement materials. With this research study, the study team seeks to establish a standardized injection technique and dosage for this disease and to determine duration of symptom alleviation.
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
- Double (Participant, Investigator)
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 or older
- •Patients with moderate to severe RP that is not controlled after 3 months of standard medical therapy as prescribed by a Rheumatologist, or severe RP with evidence of tissue loss.
排除标准
- •Patients under the age of 18
- •Patients who have previously undergone treatment with BT for RP within the past year
- •Patients with active infection at the treatment site (active infection defined as cellulitis, purulence, fever, chills, or presence of elevated inflammatory markers, ie. WBC, ESR, CRP)
- •Patients who have undergone prior digital sympathectomy surgery for RP
- •Patients who have recently altered their medical regimen for RP within the past 4 weeks
- •Patients with allergy or contraindication to BT injection
研究组 & 干预措施
10 units of BT per digit
Prior to injection, patients will wait in a temperature controlled room for 30 minutes in order to allow time for normalization of baseline digital temperature. Once a patient is randomly selected via our randomization process, BT will be reconstituted by clinic nursing staff with sterile saline per manufacturer recommendations such that the investigating hand surgeon who will be performing the injection will be sufficiently blinded. After proper cleansing of the skin with alcohol swabs, BT will be sterilely administered percutaneously via a small-gauge needle and syringe into the base of each digit by the investigating hand surgeons within the Upper Extremity Division. The volar metacarpal head will be used as a standardized anatomic landmark for injection both to lessen the probability and magnitude of risk to deep structures of the hand as well as maximize probability of proper anatomic placement of the drug.
干预措施: Botulinum toxin (Drug)
20 units of BT per digit
Prior to injection, patients will wait in a temperature controlled room for 30 minutes in order to allow time for normalization of baseline digital temperature. Once a patient is randomly selected via our randomization process, BT will be reconstituted by clinic nursing staff with sterile saline per manufacturer recommendations such that the investigating hand surgeon who will be performing the injection will be sufficiently blinded. After proper cleansing of the skin with alcohol swabs, BT will be sterilely administered percutaneously via a small-gauge needle and syringe into the base of each digit by the investigating hand surgeons within the Upper Extremity Division. The volar metacarpal head will be used as a standardized anatomic landmark for injection both to lessen the probability and magnitude of risk to deep structures of the hand as well as maximize probability of proper anatomic placement of the drug.
干预措施: Botulinum toxin (Drug)
结局指标
主要结局
Change in tissue Oximetry measure for each finger
时间窗: Baseline, 4 weeks, 12 weeks, 24 weeks post injection
Tissue Oximetry of each finger on each hand will be recorded on each digit prior to treatment and at the allotted follow up intervals.
Change in finger temperature measure
时间窗: Baseline, 4 weeks, 12 weeks, 24 weeks post injection
Temperature of the each finger on each hand will be evaluated pre-treatment and at regular follow up intervals baseline, 4 weeks, 12 weeks, 24 weeks post injection
Finger Temperature Measure
时间窗: Baseline, 4 weeks, 12 weeks, 24 weeks post injection
Finger temperature measure of each finger on each hand was recorded on each digit before treatment and at the allotted follow-up intervals; the average of all digits on both hands was combined and recorded as the value for each point in data collection.
Tissue Oximetry Measure for Each Finger
时间窗: Baseline, 4 weeks, 12 weeks, 24 weeks post injection
Tissue Oximetry of each finger on each hand was recorded on each digit before treatment and at the allotted follow-up intervals. The average of all digits on both hands was combined and reported.
次要结局
- Changes in Visual Analog Scale (VAS) pain score(Performed at 4 weeks, 12 weeks, 16 weeks, 20 weeks, and 24 weeks. Week 16 and week 20 measurements will be performed via telephone interview.)
- Changes in Hand subjectivity value(Performed at 4 weeks, 12 weeks, 16 weeks, 20 weeks, and 24 weeks. Week 16 and week 20 measurements will be performed via telephone interview.)
- Changes in Raynaud Condition Score(Performed at 4 weeks, 12 weeks, 16 weeks, 20 weeks, and 24 weeks. Week 16 and week 20 measurements will be performed via telephone interview.)
- Changes in PROMIS Pain Interference scale(Performed at 4 weeks, 12 weeks, 16 weeks, 20 weeks, and 24 weeks. Week 16 and week 20 measurements will be performed via telephone interview.)
- Changes in QUICK Dash assessment(Performed at 4 weeks, 12 weeks, 16 weeks, 20 weeks, and 24 weeks. Week 16 and week 20 measurements will be performed via telephone interview.)
- Raynaud Condition Score(Performed at 4 weeks, 12 weeks, 16 weeks, 20 weeks, and 24 weeks.)
- Visual Analog Scale (VAS) Pain Score(Performed at 4 weeks, 12 weeks, 16 weeks, 20 weeks, and 24 weeks.)
- PROMIS Pain Interference Scale(4 weeks, 12 weeks, 16 weeks, 20 weeks, and 24 weeks.)
- Hand Subjectivity Value(Performed at 4 weeks, 12 weeks, 16 weeks, 20 weeks, and 24 weeks.)
- QUICK Dash Assessment(Performed at 4 weeks, 12 weeks, 16 weeks, 20 weeks, and 24 weeks.)
研究者
Paul A Ghareeb, MD
Assistant Professor of Surgery
Emory University