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

NCT01309802 | Completed Phase 2 Results DMC

• 1 other identifier: NEU-SIUSOM-11-001
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Raynaud's Disease

Keywords

botox; raynaud's; botulinum toxin; botulinum toxin A; raynaud's disease; raynaud's syndrome; raynaud's phenomenon; ischemic digits; ischemia; botox treatment; onabotulinum; onabotulinum toxin; onabotulinum toxin type A

Outcome Measures

Primary Outcomes (1)

• Percentage of Patient Reported Pain-free Days

  Subjective pain scales \[visual analogue scale (VAS) and faces pain assessment\]. Subjects reporting total number of pain free days within the time period of 0-28 days.

  baseline to 28 days

Secondary Outcomes (5)

• Pain Related Quality of Life

  change from baseline to 28 days

• Hand Function

  baseline to 28 days

• Patient Satisfaction

  baseline to 28 days

• Tissue Perfusion

  baseline to 28 days

• EQ-5D A Standardised Patient Reported Measure of Health Status for Clinical and Economic Appraisal

  baseline to 28 days

Study Arms (2)

placebo

NO INTERVENTION

no intervention

onabotulinum toxin type-A

ACTIVE COMPARATOR

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

Drug: onabotulinum toxin type-A

Interventions

onabotulinum toxin type-A DRUG

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

Also known as: BOTOX®, botulinum toxin type A

onabotulinum toxin type-A

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Southern Illinois University: lead

Study Sites (1)

Southern Illinois University School of Medicine

Springfield, Illinois, 62702, United States

Location

Related Publications (5)

• Neumeister MW. Botulinum toxin type A in the treatment of Raynaud's phenomenon. J Hand Surg Am. 2010 Dec;35(12):2085-92. doi: 10.1016/j.jhsa.2010.09.019.

  PMID: 21134617 BACKGROUND

• Neumeister MW, Chambers CB, Herron MS, Webb K, Wietfeldt J, Gillespie JN, Bueno RA Jr, Cooney CM. Botox therapy for ischemic digits. Plast Reconstr Surg. 2009 Jul;124(1):191-201. doi: 10.1097/PRS.0b013e3181a80576.

  PMID: 19568080 BACKGROUND

• Sycha T, Graninger M, Auff E, Schnider P. Botulinum toxin in the treatment of Raynaud's phenomenon: a pilot study. Eur J Clin Invest. 2004 Apr;34(4):312-3. doi: 10.1111/j.1365-2362.2004.01324.x. No abstract available.

  PMID: 15086364 BACKGROUND

• Van Beek AL, Lim PK, Gear AJL, Pritzker MR. Management of vasospastic disorders with botulinum toxin A. Plast Reconstr Surg. 2007 Jan;119(1):217-226. doi: 10.1097/01.prs.0000244860.00674.57.

  PMID: 17255677 BACKGROUND

• Fregene A, Ditmars D, Siddiqui A. Botulinum toxin type A: a treatment option for digital ischemia in patients with Raynaud's phenomenon. J Hand Surg Am. 2009 Mar;34(3):446-52. doi: 10.1016/j.jhsa.2008.11.026.

  PMID: 19258141 BACKGROUND

Related Links

• U.S. Food and Drug Administration
• Drugs@FDA; U.S. Food and Drug Administration

MeSH Terms

Conditions

Raynaud Disease; Ischemia

Interventions

Botulinum Toxins, Type A

Condition Hierarchy (Ancestors)

Livedoid Vasculopathy; Thrombosis; Embolism and Thrombosis; Vascular Diseases; Cardiovascular Diseases; Peripheral Vascular Diseases; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Botulinum Toxins; Metalloendopeptidases; Endopeptidases; Peptide Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes; Metalloproteases; Bacterial Proteins; Proteins; Amino Acids, Peptides, and Proteins; Bacterial Toxins; Toxins, Biological; Biological Factors

Results Point of Contact

• Title: Research Administrator
• Organization: Southern Illinois University School of Medicine

surgeryresearch@siumed.edu

217-545-2531

Study Officials

• Michael W Neumeister, MD

  Southern Illinois University School of Medicine

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 4, 2011
• First Posted: March 7, 2011
• Study Start: May 1, 2011
• Primary Completion: May 19, 2016
• Study Completion: July 13, 2016
• Last Updated: January 8, 2019
• Results First Posted: June 2, 2017

Record last verified: 2018-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)