Clinical trial NCT04523506: The Effects of Botulinum Toxin on Oral Aperture in Patients With Scleroderma
Official title: The Effects of Botulinum Toxin on Oral Aperture in Patients With Scleroderma
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: EARLY_PHASE1
Conditions: Scleroderma
Lead sponsor: University of Texas Southwestern Medical Center
Sponsor class: OTHER
First posted: 2020-08-21
Enrollment: 17
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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..

Primary outcomes:
measure: Changes in inter-labial distance
timeFrame: This will be measured at baseline before treatment, 2 weeks after treatment, and 3 months after treatment.
measure: Changes interincisal distance
timeFrame: This will be measured at baseline before treatment, 2 weeks after treatment, and 3 months after treatment.
measure: quality of life via a Skindex16 survey
timeFrame: This information will be collected at baseline before treatment, 2 weeks after treatment, and 3 months after treatment.
Secondary outcomes:
measure: Changes in the inter-commissural distance
timeFrame: This will be measured at baseline before treatment, 2 weeks after treatment, and 3 months after treatment.]
measure: Changes in the Mouth Handicap in Systemic Sclerosis Scale (MHISS)
timeFrame: This will be measured at baseline before treatment, 2 weeks after treatment, and 3 months after treatment.]
Other outcomes:
Locations:
city: Dallas
country: United States
facility: UT Southwestern Medical Center at Dallas - Dermatology Clinical Trials
state: Texas
Study officials:
affiliation: UT Southwestern Medical Center
name: Heather Goff, MD
role: PRINCIPAL_INVESTIGATOR
References: