Botulinum Toxin in Raynaud's Phenomenon

NCT05125029 | Recruiting Phase 4 DMC FDA Drug

• 1 other identifier: STUDY00002359
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 1

Brief Summary

Raynaud's Phenomenon (RP) is the most common vasospastic disorder encountered by hand surgeons with an estimated prevalence of 5% in the general population. It's manifestations are broad, and can range from minor to severe. Sometimes it can be refractory to treatments. The goal of this study is to evaluate the efficacy of Botulinum toxin (BT) in the treatment of treatment refractory RP by objective and subjective measures.

Conditions

Raynaud Phenomenon

Keywords

Botulinum toxin; Raynaud Phenomenon refractory to treatment

Outcome Measures

Primary Outcomes (2)

• Change in finger temperature measure

  Temperature of the each finger on each hand will be evaluated pre-treatment and at regular follow up intervals baseline, 4 weeks, 12 weeks, 24 weeks post injection

  Baseline, 4 weeks, 12 weeks, 24 weeks post injection

• Change in tissue Oximetry measure for each finger

  Tissue Oximetry of each finger on each hand will be recorded on each digit prior to treatment and at the allotted follow up intervals.

  Baseline, 4 weeks, 12 weeks, 24 weeks post injection

Secondary Outcomes (5)

• Changes in Raynaud Condition Score

  Performed at 4 weeks, 12 weeks, 16 weeks, 20 weeks, and 24 weeks. Week 16 and week 20 measurements will be performed via telephone interview.

• Changes in Visual Analog Scale (VAS) pain score

  Performed at 4 weeks, 12 weeks, 16 weeks, 20 weeks, and 24 weeks. Week 16 and week 20 measurements will be performed via telephone interview.

• Changes in PROMIS Pain Interference scale

  Performed at 4 weeks, 12 weeks, 16 weeks, 20 weeks, and 24 weeks. Week 16 and week 20 measurements will be performed via telephone interview.

• Changes in Hand subjectivity value

  Performed at 4 weeks, 12 weeks, 16 weeks, 20 weeks, and 24 weeks. Week 16 and week 20 measurements will be performed via telephone interview.

• Changes in QUICK Dash assessment

  Performed at 4 weeks, 12 weeks, 16 weeks, 20 weeks, and 24 weeks. Week 16 and week 20 measurements will be performed via telephone interview.

Study Arms (3)

Placebo

NO INTERVENTION

Prior to injection, patients will wait in a temperature controlled room for 30 minutes in order to allow time for normalization of baseline digital temperature. Once a patient is randomly selected via our randomization process, BT will be reconstituted by clinic nursing staff with sterile saline per manufacturer recommendations such that the investigating hand surgeon who will be performing the injection will be sufficiently blinded. After proper cleansing of the skin with alcohol swabs, BT will be sterilely administered percutaneously via a small-gauge needle and syringe into the base of each digit by the investigating hand surgeons within the Upper Extremity Division. The volar metacarpal head will be used as a standardized anatomic landmark for injection both to lessen the probability and magnitude of risk to deep structures of the hand as well as maximize probability of proper anatomic placement of the drug.

10 units of BT per digit

ACTIVE COMPARATOR

Prior to injection, patients will wait in a temperature controlled room for 30 minutes in order to allow time for normalization of baseline digital temperature. Once a patient is randomly selected via our randomization process, BT will be reconstituted by clinic nursing staff with sterile saline per manufacturer recommendations such that the investigating hand surgeon who will be performing the injection will be sufficiently blinded. After proper cleansing of the skin with alcohol swabs, BT will be sterilely administered percutaneously via a small-gauge needle and syringe into the base of each digit by the investigating hand surgeons within the Upper Extremity Division. The volar metacarpal head will be used as a standardized anatomic landmark for injection both to lessen the probability and magnitude of risk to deep structures of the hand as well as maximize probability of proper anatomic placement of the drug.

Drug: Botulinum toxin

20 units of BT per digit

ACTIVE COMPARATOR

Prior to injection, patients will wait in a temperature controlled room for 30 minutes in order to allow time for normalization of baseline digital temperature. Once a patient is randomly selected via our randomization process, BT will be reconstituted by clinic nursing staff with sterile saline per manufacturer recommendations such that the investigating hand surgeon who will be performing the injection will be sufficiently blinded. After proper cleansing of the skin with alcohol swabs, BT will be sterilely administered percutaneously via a small-gauge needle and syringe into the base of each digit by the investigating hand surgeons within the Upper Extremity Division. The volar metacarpal head will be used as a standardized anatomic landmark for injection both to lessen the probability and magnitude of risk to deep structures of the hand as well as maximize probability of proper anatomic placement of the drug.

Drug: Botulinum toxin

Interventions

Botulinum toxin DRUG

Botulinum toxin is a neurotoxic protein produced by the bacterium Clostridium botulinum that prevents the release of the neurotransmitter acetylcholine from the neuromuscular junction, thereby preventing muscular contraction. Eligible subjects will be randomized 1:1:1 into three different groups: 1) placebo 2) 10 Units of BT injected per digit and 3) 20 units of BT injected per digit.

10 units of BT per digit; 20 units of BT per digit

Eligibility Criteria

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

You may qualify if:

• Age 18 or older
• Patients with moderate to severe RP that is not controlled after 3 months of standard medical therapy as prescribed by a Rheumatologist, or severe RP with evidence of tissue loss.

You may not qualify if:

• Patients under the age of 18
• Patients who have previously undergone treatment with BT for RP within the past year
• Patients with active infection at the treatment site (active infection defined as cellulitis, purulence, fever, chills, or presence of elevated inflammatory markers, ie. WBC, ESR, CRP)
• Patients who have undergone prior digital sympathectomy surgery for RP
• Patients who have recently altered their medical regimen for RP within the past 4 weeks
• Patients with allergy or contraindication to BT injection

Sponsors & Collaborators

• Emory University: lead
• American Society for Surgery of the Hand: collaborator

Study Sites (1)

Emory University Hospital

Atlanta, Georgia, 30322, United States

RECRUITING

MeSH Terms

Conditions

Raynaud Disease

Interventions

Botulinum Toxins

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

Intervention Hierarchy (Ancestors)

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

Study Officials

• Paul A Ghareeb, MD

  Emory University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Paul A Ghareeb, MD

CONTACT

404-686-8143; paul.ghareeb@emory.edu

Anthony Karzon

CONTACT

akarzon@emory.edu

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor of Surgery

Study Record Dates

• First Submitted: October 26, 2021
• First Posted: November 18, 2021
• Study Start: February 10, 2022
• Primary Completion: December 1, 2025
• Study Completion: December 1, 2025
• Last Updated: February 12, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)