Clinical trial NCT05125029: Botulinum Toxin in Raynaud's Phenomenon
Official title: Double Blind RCT to Evaluate the Effect of Botulinum Toxin in Raynaud Phenomenon
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Raynaud Phenomenon
Keywords: Botulinum toxin
Raynaud Phenomenon refractory to treatment
Lead sponsor: Emory University
Sponsor class: OTHER
Collaborators: name: American Society for Surgery of the Hand
First posted: 2021-11-18
Enrollment: 36
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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

Primary outcomes:
measure: Change in finger temperature measure
timeFrame: Baseline, 4 weeks, 12 weeks, 24 weeks post injection
measure: Change in tissue Oximetry measure for each finger
timeFrame: Baseline, 4 weeks, 12 weeks, 24 weeks post injection
Secondary outcomes:
measure: Changes in Raynaud Condition Score
timeFrame: 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.
measure: Changes in Visual Analog Scale (VAS) pain score
timeFrame: 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.
measure: Changes in PROMIS Pain Interference scale
timeFrame: 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.
measure: Changes in Hand subjectivity value
timeFrame: 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.
measure: Changes in QUICK Dash assessment
timeFrame: 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.
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Emory University Hospital
state: Georgia
Study officials:
affiliation: Emory University
name: Paul A Ghareeb, MD
role: PRINCIPAL_INVESTIGATOR
References: