Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02165111
Recruitment Status : Completed
First Posted : June 17, 2014
Results First Posted : December 22, 2016
Last Update Posted : December 22, 2016
Sponsor:
Collaborator:
Allergan
Information provided by (Responsible Party):
Scott D. Lifchez, Johns Hopkins University

Tracking Information
First Submitted Date  ICMJE June 11, 2014
First Posted Date  ICMJE June 17, 2014
Results First Submitted Date  ICMJE September 1, 2016
Results First Posted Date  ICMJE December 22, 2016
Last Update Posted Date December 22, 2016
Study Start Date  ICMJE January 2015
Actual Primary Completion Date September 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 28, 2016)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: June 13, 2014)
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. ]
The primary outcome measure is blood flow to the fingers, as measured by non-invasive laser Doppler imaging.
Change History Complete list of historical versions of study NCT02165111 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: October 28, 2016)
  • 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.
  • 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.
  • 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.
  • 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.
  • 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)
Original Secondary Outcome Measures  ICMJE
 (submitted: June 13, 2014)
  • Assessment of Raynaud's symptoms severity using the Raynaud's Condition Score. [ Time Frame: Measured periodically over the four month study period. ]
    A secondary outcome of this study is severity of Raynaud's symptoms as measured by the self-reported Raynaud's Condition Score.
  • 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. ]
    A secondary outcome of this study is healing of existing digital ulcerations and/or prevention of new areas of digital ulceration.
  • Assessment of Raynaud's Symptoms Severity Using the Quick-DASH Score. [ Time Frame: Measured periodically over the four month study period. ]
    A secondary outcome of this study is severity of Raynaud's symptoms as measured by the self-reported Quick-DASH score.
  • Assessment of Raynaud's Symptom Severity Using the McCabe Cold Sensitivity Score. [ Time Frame: Measured periodically over the four month study period. ]
    A secondary outcome of this study is severity of Raynaud's symptoms as measured by the self-reported McCabe Cold Sensitivity score.
  • Assessment of Raynaud's Symptom Severity Using the VAS for Pain. [ Time Frame: Measured periodically over the four month study period. ]
    A secondary outcome of this study is severity of Raynaud's symptoms as measured by the visual-analog scale (VAS) for pain.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy of Botulinum Toxin In Scleroderma-Associated Raynaud's Syndrome
Official Title  ICMJE A Randomized, Double-Blinded, Placebo-Controlled Clinical Trial Assessing the Therapeutic Efficacy of Botulinum Toxin In Treating Scleroderma-Associated Raynaud's Syndrome
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.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Scleroderma
  • Raynaud's Syndrome
Intervention  ICMJE
  • Drug: Onabotulinumtoxin A
    Other Names:
    • Botox
    • Botulinum toxin
    • Dysport
  • Drug: sterile saline solution
    Other Name: Saline
Study Arms  ICMJE
  • Experimental: 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.

    Intervention: Drug: Onabotulinumtoxin A
  • Placebo Comparator: 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).

    Intervention: Drug: sterile saline solution
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: June 13, 2014)
40
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE September 2015
Actual Primary Completion Date September 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02165111
Other Study ID Numbers  ICMJE NA_00087346
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Scott D. Lifchez, Johns Hopkins University
Study Sponsor  ICMJE Johns Hopkins University
Collaborators  ICMJE Allergan
Investigators  ICMJE
Principal Investigator: Scott D Lifchez, MD Johns Hopkins University
PRS Account Johns Hopkins University
Verification Date October 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP