Phase 3
N=80
Efficacy of Botulinum Toxin In Scleroderma-Associated Raynaud's Syndrome
Scleroderma · Raynaud's Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT02165111 ↗Enrolled (actual)
80
Serious AEs
0.0%
Results posted
Dec 2016
Primary outcome: Primary: Change in Digital Blood Flow From Pre- to Post-injection. — -36.19; -6.10 Blood flow, measured in LDI flux units,
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Onabotulinumtoxin A (Drug); sterile saline solution (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Johns Hopkins University
- Primary completion
- Sep 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Digital Blood Flow From Pre- to Post-injection. |
-36.19; -6.10 | — |
| SECONDARY Rate of Change in Raynaud's Phenomenon Symptoms Measured With the Raynaud's Condition Score. |
-0.18; -0.14 | — |
| SECONDARY Number of Ulcers as Measure of Digital Ulcer Healing |
0.45; 0.53 | — |
| SECONDARY Assessment of Raynaud's Symptoms Severity Using the Quick-DASH Score. |
26.96; 29.11 | — |
| SECONDARY Assessment of Raynaud's Symptom Severity Using the McCabe Cold Sensitivity Score. |
185.31; 187.19 | — |
| SECONDARY Assessment of Raynaud's Symptom Severity Using the VAS for Pain. |
2.68; 3.05 | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria
- 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.
Exclusion Criteria
- 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
Data sourced from ClinicalTrials.gov (NCT02165111). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.