Raynaud's Syndrome
Conditions
Keywords
Scleroderma, Raynaud's Syndrome
Brief summary
The investigators are studying a medical condition known as Raynaud's Phenomenon (RP). RP is a painful condition in which cold surroundings or stressful events trigger pain, a cold sensation, and in severe cases, sores and ulcers on one's fingers. The investigators think that RP results from problems with some of the components of the blood vessels, surrounding nerves, and some of the circulating messengers in the bloodstream. Although there are many different medicines that are currently used to treat RP, the investigators have not yet found a cure for severe cases of this condition. Furthermore, the investigators have found few medicines that show consistent healing of the ulcers that may be associated with RP. Some of the treatments include medicines that are used to treat high blood pressure known as calcium channel blockers. Medications that thin the blood (anticoagulants) such as aspirin have also been used to treat RP. In this study, the investigators will investigate a new treatment for RP known as Botulinum toxin A. Botulinum toxin is a chemical that is temporarily toxic to nerves, but has been approved by the government for the treatment of several other conditions including but not limited to: excessive sweating, neck pain associated with a condition known as cervical dystonia, uncontrollable forcible closure of the eyelids known as blepharospasm, and a condition known as strabismus in which the eyes are not in alignment with one another. This study will aim to greatly improve the quality of life and pain associated with RP. Previous studies also support the chance that Botulinum toxin will help to heal some of the ulcers associated with RP. Although there is no current standard of care, many of the patients in the study will have already failed or are unable to tolerate commonly used treatments such as lifestyle modifications, calcium channel blockers, nitroglycerin, and anticoagulants. If the patient chooses to participate in the study, he/she will be randomly assigned to receive an injection with Botulinum toxin in either the left or right hand. The other hand will be injected with saline solution. Neither the patient nor the physician or research personnel seen in follow-up will know which hand is injected with Botulinum toxin.
Interventions
DRUGbotulinum toxin A
Botulinum toxin A will be injected into participants hand total 40units
Sponsors
Emory University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 89 Years
Healthy volunteers
No
Inclusion criteria
Adult (\>18 years) patients with severe RP of any etiology will be invited to participate. Inclusion criteria include a) history of digital infarcts or ulcerations, b) unresponsive to standard therapies, or c) impending potential for digital amputation. Patients on other standard therapies such as antiplatelet agents, vasodilators, and calcium channel blockers will be included. -
Exclusion criteria
Pregnancy, mild disease, patients with myasthenia gravis, botulinum toxin allergy, and phobia of needles are
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Mean Digital Temperature Difference From Baseline | 6 weeks | Each digit temperature was measured and recorded at baseline, and then measured and re-recorded after 3 minute intervals following a 20 second 4 degree Celsius ice bath immersion. |
Countries
United States
Participant flow
Recruitment details
10 subjects recruited from Emory Dept of Dermatology
Pre-assignment details
all subjects were assigned to group
Participants by arm
| Arm | Count |
|---|---|
| Botox single-drug dosage comparison cross-over study | 10 |
| Total | 10 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Lost to Follow-up | 1 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Botox |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 10 Participants |
| Age, Continuous | 30 years STANDARD_DEVIATION 1 |
| Region of Enrollment United States | 10 participants |
| Sex: Female, Male Female | 8 Participants |
| Sex: Female, Male Male | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 0 / 9 |
| serious Total, serious adverse events | 0 / 9 |
Outcome results
Primary
Mean Digital Temperature Difference From Baseline
Each digit temperature was measured and recorded at baseline, and then measured and re-recorded after 3 minute intervals following a 20 second 4 degree Celsius ice bath immersion.
Time frame: 6 weeks
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Botox | Mean Digital Temperature Difference From Baseline | 1.30 Celsius |