Efficacy of Botulinum Toxin In Scleroderma-Associated Raynaud's Syndrome

Scleroderma Clinical Trial

Official title:

A Randomized, Double-Blinded, Placebo-Controlled Clinical Trial Assessing the Therapeutic Efficacy of Botulinum Toxin In Treating Scleroderma-Associated Raynaud's Syndrome

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02165111
• Other study ID #: NA_00087346
• Status: Active, not recruiting
• Phase: Phase 3
• First received: June 11, 2014
• Last updated: June 11, 2015
• Start date: January 2015
• Est. completion date: December 2016

Study information

• Verified date: June 2015
• Source: Johns Hopkins University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 40
• Est. completion date: December 2016
• Est. primary completion date: September 2016
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Raynaud Disease
• Raynaud's Syndrome
• Scleroderma
• Scleroderma, Diffuse
• Scleroderma, Localized
• Scleroderma, Systemic
• Syndrome

Intervention

Drug:
• Onabotulinumtoxin A

• sterile saline solution

Locations

Country	Name	City	State
United States	Johns Hopkins Bayview Medical Center	Baltimore	Maryland

Sponsors (2)

Lead Sponsor	Collaborator
Johns Hopkins University	Allergan

Country where clinical trial is conducted

United States, 

References & Publications (1)

Iorio ML, Masden DL, Higgins JP. Botulinum toxin A treatment of Raynaud's phenomenon: a review. Semin Arthritis Rheum. 2012 Feb;41(4):599-603. doi: 10.1016/j.semarthrit.2011.07.006. Epub 2011 Aug 24. Review. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Digital blood flow from pre- to post-injection.	The primary outcome measure is blood flow to the fingers, as measured by non-invasive laser Doppler imaging.	Measured pre-injection and at one month, four months, and (in some patients) one week post-injection.	No
Secondary	Assessment of Raynaud's symptoms severity using the Raynaud's Condition Score.	A secondary outcome of this study is severity of Raynaud's symptoms as measured by the self-reported Raynaud's Condition Score.	Measured periodically over the four month study period.	No
Secondary	Digital ulcer healing measured by size of ulceration and time lapsed to full healing.	A secondary outcome of this study is healing of existing digital ulcerations and/or prevention of new areas of digital ulceration.	Measured at one month, four months, and (in some patients) one week post-injection.	No
Secondary	Assessment of Raynaud's symptoms severity using the Quick-DASH score.	A secondary outcome of this study is severity of Raynaud's symptoms as measured by the self-reported Quick-DASH score.	Measured periodically over the four month study period.	No
Secondary	Assessment of Raynaud's symptom severity using the McCabe Cold Sensitivity score.	A secondary outcome of this study is severity of Raynaud's symptoms as measured by the self-reported McCabe Cold Sensitivity score.	Measured periodically over the four month study period.	No
Secondary	Assessment of Raynaud's symptom severity using the VAS for pain.	A secondary outcome of this study is severity of Raynaud's symptoms as measured by the visual-analog scale (VAS) for pain.	Measured periodically over the four month study period.	No