The Effects of Botulinum Toxin on Oral Aperture in Patients With Scleroderma

NCT ID: NCT04523506

Last Updated: 2021-04-28

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: EARLY_PHASE1
• Total Enrollment: 17 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-05-08
• Study Completion Date: 2021-04-22

Brief Summary

This study will evaluate the use of botulinum toxin for microstomia (also known as reduced oral aperture) in scleroderma patients. Botox is a neurotoxin that functions as a paralytic by preventing the release of acetylcholine to inhibit muscle contracture and decrease fibrosis by decreasing differentiation of fibroblasts to myofibroblasts, decreasing expression of collagen, and increasing expression of matrix metalloproteinase1-3. The study will include three arms: the temporomandibular joint (TMJ) group who will receive injections of Botox to the masseter, the perioral group who will receive injections of Botox around the lips, and a control group who will receive no treatment for ROA. Outcome measurements will include measurement of oral aperture size through measurement inter-labial distance and between the upper and lower lips and the inter-incisal distance, patient satisfaction via a Skindex16 survey, mouth disability via the Mouth Handicap in Systemic Sclerosis Scale (MHISS), and patient and physician satisfaction using the Visual Analogic Scale (VAS). The maximum number of subjects to be consented for this study is 30. The study is expected to last four months per subject from time of consent to last clinical evaluation. Conditions that may result in a subject exiting the study prior to completion date include non-compliance, withdrawal of consent, or safety concerns such as adverse events as a result of treatment.

Conditions

Scleroderma

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

the temporomandibular joint (TMJ) group

This study will evaluate the use of botulinum toxin for microstomia (reduced oral aperture) in scleroderma patients. The TMJ group will be injected with two units of Botox into four different injection points to each masseter (16 units of Botox total) at the initial visit. No additional Botox will be injected in subsequent visits. Botox is a neurotoxin that functions as a paralytic by preventing the release of acetylcholine to inhibit muscle contracture and decrease fibrosis by decreasing differentiation of fibroblasts to myofibroblasts, decreasing expression of collagen, and increasing expression of matrix metalloproteinase1

Group Type: EXPERIMENTAL

Botulinum toxin(Botox)

Intervention Type: BIOLOGICAL

This study will evaluate the use of botulinum toxin for reduced oral aperture in scleroderma patients. Botox is a neurotoxin that functions as a paralytic by preventing the release of acetylcholine to inhibit muscle contracture and decrease fibrosis by decreasing differentiation of fibroblasts to myofibroblasts, decreasing expression of collagen, and increasing expression of matrix metalloproteinase.

the perioral group

Biological/Vaccine: Botulinum toxin(Botox) This study will evaluate the use of botulinum toxin for microstomia (reduced oral aperture) in scleroderma patients. The perioral group will be injected with two units of Botox into eight different injection points (16 units of Botox total) around the lips (in the orbicularis oris). Botox is a neurotoxin that functions as a paralytic by preventing the release of acetylcholine to inhibit muscle contracture and decrease fibrosis by decreasing differentiation of fibroblasts to myofibroblasts, decreasing expression of collagen, and increasing expression of matrix metalloproteinase1-3.

Group Type: EXPERIMENTAL

Botulinum toxin(Botox)

Intervention Type: BIOLOGICAL

This study will evaluate the use of botulinum toxin for reduced oral aperture in scleroderma patients. Botox is a neurotoxin that functions as a paralytic by preventing the release of acetylcholine to inhibit muscle contracture and decrease fibrosis by decreasing differentiation of fibroblasts to myofibroblasts, decreasing expression of collagen, and increasing expression of matrix metalloproteinase.

Interventions

Botulinum toxin(Botox)

This study will evaluate the use of botulinum toxin for reduced oral aperture in scleroderma patients. Botox is a neurotoxin that functions as a paralytic by preventing the release of acetylcholine to inhibit muscle contracture and decrease fibrosis by decreasing differentiation of fibroblasts to myofibroblasts, decreasing expression of collagen, and increasing expression of matrix metalloproteinase.

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

1. Ddiagnosis of scleroderma (defined by the 2013 Classification Criteria for Systemic Sclerosis4,5)23,24 ) who also have microstomia (reduced oral aperture), defined as an inter-incisal distance less than 50 mm 6-7)m13,14 .

2. Male and female subjects.

3. English and non-English speakers.

4. Subjects aged 18 years old to 65 years old will be considered

Exclusion Criteria

1. Patients under 18 years old will be excluded.

2. Patients with a known history of a hypersensitivity to any Botox formulation or to any of the components in the formulation,

3. Active skin infection at the proposed injection site.

4. Concomitant neuromuscular disorder.

5. Pregnant or lactating.

6. Missing incisors.

7. treatment with: cyclophosphamide, Ultraviolet A-1 therapy, or topical calcitriol..

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

University of Texas Southwestern Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Heather Woodworth Goff

Associate Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Heather Goff, MD

Role: PRINCIPAL_INVESTIGATOR

UT Southwestern Medical Center

Locations

UT Southwestern Medical Center at Dallas - Dermatology Clinical Trials

Dallas, Texas, United States

Countries

United States

Other Identifiers

STU-2020-0169

Identifier Type: -

Identifier Source: org_study_id