Skip to main content
OpenTrials
Completed

NCT Number: NCT02165111

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

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Scleroderma Cardiovascular Diseases Connective Tissue Diseases Embolism and Thrombosis Livedoid Vasculopathy Peripheral Vascular Diseases Raynaud Disease Raynaud's Syndrome Scleroderma, Diffuse Scleroderma, Systemic Skin Diseases Skin Diseases, Vascular Skin and Connective Tissue Diseases Thrombosis Vascular Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Johns Hopkins Bayview Medical Center

Baltimore, Maryland, 21224, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Onabotulinumtoxin A

Drug

Other names: Botox, Botulinum toxin, Dysport

Sterile saline solution

Drug

Other names: Saline

Primary outcomes

  1. Change in Digital Blood Flow From Pre- to Post-injection.

    Time frame: Measured pre-injection and at one month post-injection.

    The primary outcome measure is change in blood flow to the fingers, from a pre-injection baseline to post-injection follow-up visit as measured by non-invasive laser Doppler imaging.

Secondary outcomes

  1. Rate of Change in Raynaud's Phenomenon Symptoms Measured With the Raynaud's Condition Score.

    Time frame: Weekly rate of change over the four-month study period.

    Raynaud's Condition Score is a patient-reported, validated outcomes scale that measures the severity of Raynaud's phenomenon on a scale of 0 ("No difficulty") to 10 ("Extreme difficulty"), where higher values represent a worse outcome.

    Data from each weekly report were combined using a statistical model (generalized linear population-average model) to calculate a weekly rate of change for each participant's hand, where a negative value represents a improvement over time and a positive value represents worsening over time.

  2. Number of Ulcers as Measure of Digital Ulcer Healing

    Time frame: Measured at one month post-injection.

    A secondary outcome of this study is the number of digital ulcers as determined by clinical examination. Mean number of ulcers was calculated as total number of ulcers / total number of hands in each group.

  3. Assessment of Raynaud's Symptoms Severity Using the Quick-DASH Score.

    Time frame: Measured at one month post-injection.

    A secondary outcome of this study is severity of Raynaud's symptoms as measured by the self-reported Quick-DASH score. The Quick-DASH scores measures the degree of hand and upper extremity function on a scale of 0 (not limited) to 100 (severely limited) where a higher value represents a worse outcome.

  4. Assessment of Raynaud's Symptom Severity Using the McCabe Cold Sensitivity Score.

    Time frame: Measured at one month post-injection.

    A secondary outcome of this study is the patient-reported sensitivity to coldness as measured by the McCabe Cold Sensitivity score. Patients' answers on this validated instrument are scored on a scale from 0 (no sensitivity) to 400 (extreme sensitivity), where a higher number represents a worse outcome.

  5. Assessment of Raynaud's Symptom Severity Using the VAS for Pain.

    Time frame: Measured at one month post-injection.

    A secondary outcome of this study is pain as measured by the validated visual-analog scale (VAS) for pain. The VAS pain instrument measures pain on a scale from 0 cm (no pain) to 10 cm (extreme pain)

Sponsors and collaborators

Lead sponsor

Johns Hopkins University

Other

Collaborators

  • Allergan

Registry information

Official study title

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

Important dates

Study start
2015
Primary completion
2015
Study completion
2015
First posted
Jun 17, 2014
Registry last updated
Dec 22, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.