A Two-Part Study of BOTOX® Therapy for Ischemic Digits

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Michael Neumeister, MD, Southern Illinois University
ClinicalTrials.gov Identifier:
NCT01309802
First received: March 4, 2011
Last updated: April 3, 2014
Last verified: April 2014

March 4, 2011
April 3, 2014
May 2011
December 2014   (final data collection date for primary outcome measure)
number of pain-free days [ Time Frame: change from baseline to 28 days ] [ Designated as safety issue: No ]
Subjective pain scales [visual analogue scale (VAS) and faces pain assessment]
Same as current
Complete list of historical versions of study NCT01309802 on ClinicalTrials.gov Archive Site
  • quality of life [ Time Frame: change from baseline to 28 days ] [ Designated as safety issue: No ]
    SF-12v2® Health Survey - Pain Enhanced
  • hand function [ Time Frame: change from baseline to 28 days ] [ Designated as safety issue: No ]
    Quick-DASH (Disabilities of the Arm, Shoulder, and Hand) Outcome Measure
  • patient satisfaction [ Time Frame: change from baseline to 28 days ] [ Designated as safety issue: No ]
    SF-12v2® Health Survey - Pain Enhanced
  • tissue perfusion [ Time Frame: change from baseline to 28 days ] [ Designated as safety issue: No ]
    Doppler perfusion imager and Periscan image analysis software
  • quality-adjusted life-years [ Time Frame: change from baseline to 28 days ] [ Designated as safety issue: No ]
    EuroQol (EQ-5D)
Same as current
Not Provided
Not Provided
 
A Two-Part Study of BOTOX® Therapy for Ischemic Digits
A 28-Day Randomized, Double-Blind, Placebo-Controlled Clinical Trial and 5-Year Prospective Outcomes Study: A Two-Part Study of BOTOX® Therapy for Ischemic Digits

Treating patients with Raynaud's phenomenon who have chronic pain and ulcerations is extremely challenging. Published reports and our previous work support our hypothesis that symptomatic patients experience relief of pain and healing of ulcerations with minimal adverse effects when treated with botulinum toxin type A (Btx-A) injections for Raynaud's phenomenon. The proposed study is the first clinical trial and prospective study designed to document whether or not 1) Btx-A injection relieves pain in a patient's hand affected with Raynaud's disease better than a placebo within 28 days of injection, and 2) Btx-A injection relieves pain associated with Raynaud's disease for longer than 28 days, improving patients' quality of life. Through this study we intend to further determine the effect of injected Btx-A on relieving chronic pain and ulcerations to the ischemic hand while characterizing the patients for whom this treatment is most effective.

PROJECT SUMMARY OVERVIEW: Treating patients with Raynaud's phenomenon who have chronic pain and ulcerations is extremely challenging. Pharmacologic vasodilators and surgical sympathectomies offer variable benefits. Case reports, small retrospective outcomes studies, and our previous work documenting symptomatic patients treated with botulinum toxin type A (Btx-A) injections for Raynaud's phenomenon have demonstrated relief of pain and healing of ulcerations with minimal adverse effects. We propose to conduct the first clinical trial and prospective study documenting the efficacy of this novel treatment modality.

STUDY AIMS: The aims of this proposal are to 1) examine the short-term efficacy of Btx-A injection compared to placebo in treating pain associated with digit ischemia due to Raynaud's disease, and 2) describe the long-term efficacy of Btx-A injection in treating pain associated with digit ischemia due to Raynaud's disease by measuring patient satisfaction and quality of life changes over time.

APPROACH: Two groups of patients will be enrolled: Group 1 will consist of patients with primary Raynaud's disease (n=20) and Group 2 of patients with secondary Raynaud's (n=20). Comparisons between treatment (Btx) and placebo (saline) will occur during the first 28 days to determine Btx-A's short-term efficacy. Follow-up visits will occur at Days 7 and 28. Post-assessment on Day 28 marks the beginning of the longitudinal observational study of patient outcomes. Placebo will no longer be used and patients still suffering from pain will be eligible for additional Btx-A injections. Patients may receive up to 4 injections of Btx-A during the 1-year study period if pain or ulcerations recur. During the study period participants will be followed to collect data on pain-free intervals, ulcer healing, subsequent treatment choices, patient satisfaction, and changes in quality of life and hand function. Group comparisons will be made to analyze results. Further stratifications for data analysis will be made as enrollment numbers allow to control for additional demographic and disease variables. Quality-adjusted life-years will be calculated to help determine the societal and individual cost of this treatment.

HYPOTHESIS: We hypothesize that 1) Btx-A injection relieves ischemic pain associated with Raynaud's disease better than a placebo within 28 days of injection, and 2) Btx-A injection relieves ischemic pain associated with Raynaud's disease for longer than 28 days, improving patients' quality of life. Through this study we intend to further elucidate the efficacy of injected Btx-A on relieving chronic pain and ulcerations to the ischemic hand while characterizing the patients for whom this treatment is most effective. This data will help us to apply for national funding to become the coordinating center for a multi-center clinical trial. The results of this research have enormous potential to impact millions of patients who suffer with Raynaud's phenomenon.

Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Outcomes Assessor)
Primary Purpose: Treatment
Raynaud's Disease
Drug: onabotulinum toxin type-A
up to 4 injections per hand dosage per injection: 100 units diluted in 2.0 mL normal saline; dosing will not exceed 360 units in a 3 month interval frequency: no less than 28 days between injections duration: during Study Year 1
Other Names:
  • BOTOX®
  • botulinum toxin type A
  • No Intervention: placebo
    no intervention
  • Active Comparator: onabotulinum toxin type-A
    up to 4 injections per hand dosage per injection: 100 units diluted in 2.0 mL normal saline; dosing will not exceed 360 units in a 3 month interval frequency: no less than 28 days between injections duration: during Study Year 1
    Intervention: Drug: onabotulinum toxin type-A

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
40
May 2018
December 2014   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • aged 18-75 years
  • diagnosed with Raynaud's disease/phenomenon
  • ischemia not due to peripheral artery disease or other vascular disease
  • otherwise healthy individual
  • up-to-date tetanus immunization
  • ability to return/be available for follow-up evaluations
  • ability/willingness to give informed consent

Exclusion Criteria:

  • HIV/AIDS positive or otherwise immunocompromised
  • history of neuromuscular disease
  • reported allergy to BOTOX®; reported allergy to lidocaine or other local anesthetic agent
  • ever received botulinum toxin vaccine
  • ultrasound or angiogram showing digital ischemia due to blocked vessel and not Raynaud's disease
  • history or symptoms of any significant medical problem in the last year (i.e., bradycardia, impaired cardiovascular function, liver disease)
  • symptoms of infection or illness during initial enrollment
  • pregnant or lactating women
  • unable or unwilling to maintain abstinence or use contraception for 28 days following all injections
  • cognitive impairment
Both
18 Years to 75 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT01309802
NEU-SIUSOM-11-001
Yes
Michael Neumeister, MD, Southern Illinois University
Southern Illinois University
Not Provided
Principal Investigator: Michael W Neumeister, MD Southern Illinois University School of Medicine
Southern Illinois University
April 2014

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