Overview

This trial is active, not recruiting.

Conditions scleroderma, raynaud's syndrome
Treatments onabotulinumtoxin a, sterile saline solution
Phase phase 3
Sponsor Johns Hopkins University
Collaborator Allergan
Start date January 2015
End date September 2016
Trial size 40 participants
Trial identifier NCT02165111, NA_00087346

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification efficacy study
Intervention model parallel assignment
Masking double blind (subject, caregiver, investigator, outcomes assessor)
Primary purpose treatment
Arm
(Experimental)
One hand of each patient will be randomly selected for injection of Botulinum Toxin A (Onabotulinumtoxin A, 20 units/mL). Injections are performed using a 30 gauge insulin syringe through the dorsal surface in seven locations: adjacent to the 2nd, 3rd, and 4th common digital arteries through the web spaces (10 units each=0.5mL each), and the radial side of the index finger metacarpal head, the ulnar side of the small finger metacarpal head, and each side of the thumb metacarpal head (5 units each= 0.25mL each). Total treatment dose of Botulinum Toxin A will not exceed 50 units per hand.
onabotulinumtoxin a Botox
(Placebo Comparator)
One hand of each patient will be randomly selected for injection of sterile saline solution (placebo). Injections are performed using a 30 gauge insulin syringe through the dorsal surface in seven locations: adjacent to the 2nd, 3rd, and 4th common digital arteries through the web spaces (0.5 mL each), and the radial side of the index finger metacarpal head, the ulnar side of the small finger metacarpal head, and each side of the thumb metacarpal head (0.25 mL each).
sterile saline solution Saline

Primary Outcomes

Measure
Change in Digital blood flow from pre- to post-injection.
time frame: Measured pre-injection and at one month, four months, and (in some patients) one week post-injection.

Secondary Outcomes

Measure
Assessment of Raynaud's symptoms severity using the Raynaud's Condition Score.
time frame: Measured periodically over the four month study period.
Digital ulcer healing measured by size of ulceration and time lapsed to full healing.
time frame: Measured at one month, four months, and (in some patients) one week post-injection.
Assessment of Raynaud's symptoms severity using the Quick-DASH score.
time frame: Measured periodically over the four month study period.
Assessment of Raynaud's symptom severity using the McCabe Cold Sensitivity score.
time frame: Measured periodically over the four month study period.
Assessment of Raynaud's symptom severity using the VAS for pain.
time frame: Measured periodically over the four month study period.

Eligibility Criteria

Male or female participants at least 18 years old.

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

Additional Information

Official title A Randomized, Double-Blinded, Placebo-Controlled Clinical Trial Assessing the Therapeutic Efficacy of Botulinum Toxin In Treating Scleroderma-Associated Raynaud's Syndrome
Principal investigator Scott D Lifchez, MD
Trial information was received from ClinicalTrials.gov and was last updated in June 2015.
Information provided to ClinicalTrials.gov by Johns Hopkins University.