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

NCT ID: NCT02165111

Last Updated: 2016-12-22

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 40 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-01-31
• Study Completion Date: 2015-09-30

Brief 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.

Conditions

Scleroderma; Raynaud's Syndrome

Keywords

Scleroderma, Systemic; Raynaud Disease; Botulinum Toxins, Type A

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Onabotulinumtoxin A

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.

Group Type: EXPERIMENTAL

Onabotulinumtoxin A

Intervention Type: DRUG

Placebo

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).

Group Type: PLACEBO_COMPARATOR

sterile saline solution

Intervention Type: DRUG

Interventions

Onabotulinumtoxin A

Intervention Type: DRUG

sterile saline solution

Intervention Type: DRUG

Other Intervention Names

Botox; Botulinum toxin; Dysport; Saline

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Allergan

INDUSTRY

Sponsor Role: collaborator

Johns Hopkins University

OTHER

Sponsor Role: lead

Responsible Party

Scott D. Lifchez

Associate Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Scott D Lifchez, MD

Role: PRINCIPAL_INVESTIGATOR

Johns Hopkins University

Locations

Johns Hopkins Bayview Medical Center

Baltimore, Maryland, United States

Countries

United States

References

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.

Reference Type: BACKGROUND

PMID: 21868066 (View on PubMed)

Bello RJ, Cooney CM, Melamed E, Follmar K, Yenokyan G, Leatherman G, Shah AA, Wigley FM, Hummers LK, Lifchez SD. The Therapeutic Efficacy of Botulinum Toxin in Treating Scleroderma-Associated Raynaud's Phenomenon: A Randomized, Double-Blind, Placebo-Controlled Clinical Trial. Arthritis Rheumatol. 2017 Aug;69(8):1661-1669. doi: 10.1002/art.40123. Epub 2017 Jun 26.

Reference Type: DERIVED

PMID: 28426903 (View on PubMed)

Other Identifiers

NA_00087346

Identifier Type: -

Identifier Source: org_study_id