SclerodermaEarly Phase 1NCT04523506
The Effects of Botulinum Toxin on Oral Aperture in Patients With Scleroderma
A Early Phase 1 study testing Botulinum toxin(Botox) in people with Scleroderma, run by University of Texas Southwestern Medical Center at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Scleroderma
- What is being tested?
- Botulinum toxin(Botox)
- Type of treatment
- Vaccines, Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 1 siteUnited States
- Study dates
- 8 May 2020to 22 Apr 2021
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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 .
- Male and female subjects.
- English and non-English speakers.
- Subjects aged 18 years old to 65 years old will be considered
Who cannot join
- Patients under 18 years old will be excluded.
- Patients with a known history of a hypersensitivity to any Botox formulation or to any of the components in the formulation,
- Active skin infection at the proposed injection site.
- Concomitant neuromuscular disorder.
- Pregnant or lactating.
- Missing incisors.
- treatment with: cyclophosphamide, Ultraviolet A-1 therapy, or topical calcitriol..
Trial locations and contacts
1 site in 1 country.
United States1 site
UT Southwestern Medical Center at Dallas - Dermatology Clinical Trials
Dallas, Texas, 75390-8802
About this study
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
- Being tested
- Botulinum toxin(Botox)
- Medical subject headings
- Scleroderma, Diffuse
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Botulinum toxin(Botox)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.
| Study group | Role | What they receive |
|---|---|---|
| the temporomandibular joint (TMJ) group | Experimental | 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 Receives: Biological: Botulinum toxin(Botox) |
| the perioral group | Experimental | 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. Receives: Biological: Botulinum toxin(Botox) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in inter-labial distance
Inter-labial distance is defined as the distance between the upper and lower lip at maximum mouth opening. Distance will be measured using digital calipers by the same operator
Measured: This will be measured at baseline before treatment, 2 weeks after treatment, and 3 months after treatment.
Changes interincisal distance
Interincisal distance is defined by the didistance between upper and lower incisor at maximum mouth opening. Distance will be measured using digital calipers by the same operator
Measured: This will be measured at baseline before treatment, 2 weeks after treatment, and 3 months after treatment.
quality of life via a Skindex16 survey
This is a patient quality of life survey using the skindex 16 survey form.
Measured: This information will be collected at baseline before treatment, 2 weeks after treatment, and 3 months after treatment.
Secondary outcomes (2)
Changes in the inter-commissural distance
Inter-commissural distance is defined by the distance from corner to corner at the angle of the lip (also known as the oral commissures). Distance will be measured using digital calipers by the same operator.
Measured: This will be measured at baseline before treatment, 2 weeks after treatment, and 3 months after treatment.]
Changes in the Mouth Handicap in Systemic Sclerosis Scale (MHISS)
Mouth disability will be assessed via the Mouth Handicap in Systemic Sclerosis Scale (MHISS). MHISS range from 0 to 48 with 48 being worse disability
Measured: This will be measured at baseline before treatment, 2 weeks after treatment, and 3 months after treatment.]
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Aug 2020
- Study start8 May 2020
- Primary completion22 Apr 2021
- Study completion22 Apr 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for SclerodermaStudies of the same condition, open and under way.
- More trials run by University of Texas Southwestern Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Texas Southwestern Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Heather Woodworth Goff, University of Texas Southwestern Medical Center
- Investigators
- Heather Goff, MD, Principal investigator, University of Texas Southwestern Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04523506
- Sponsor's protocol ID
- STU-2020-0169
- Status last verified
- Apr 2021
- First submitted
- 19 Jul 2020
- Last updated
- 28 Apr 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided: To be decided by research team
Study at a glance
From the registry record, last updated by the sponsor on 28 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effects of Botulinum Toxin on Oral Aperture in Patients With Scleroderma
- ClinicalTrials.gov ID
- NCT04523506
- Acronym / sponsor ID
- STU-2020-0169
- Conditions
- Scleroderma
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- University of Texas Southwestern Medical CenterUniversity, hospital or other
- Study start
- 8 May 2020 (actual)
- Primary completion
- 22 Apr 2021 (actual)
- Study completion
- 22 Apr 2021 (actual)
- First posted
- 21 Aug 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| the temporomandibular joint (TMJ) group | Experimental | Biological: Botulinum toxin(Botox)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 |
| the perioral group | Experimental | Biological: Botulinum toxin(Botox)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. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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 .
- Male and female subjects.
- English and non-English speakers.
- Subjects aged 18 years old to 65 years old will be considered
Key exclusion criteria (7 listed)
- Patients under 18 years old will be excluded.
- Patients with a known history of a hypersensitivity to any Botox formulation or to any of the components in the formulation,
- Active skin infection at the proposed injection site.
- Concomitant neuromuscular disorder.
- Pregnant or lactating.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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. | Biological | the perioral group, the temporomandibular joint (TMJ) group |
Primary outcome
Changes in inter-labial distance
Time frame: This will be measured at baseline before treatment, 2 weeks after treatment, and 3 months after treatment.
Inter-labial distance is defined as the distance between the upper and lower lip at maximum mouth opening. Distance will be measured using digital calipers by the same operator
Changes interincisal distance
Time frame: This will be measured at baseline before treatment, 2 weeks after treatment, and 3 months after treatment.
Interincisal distance is defined by the didistance between upper and lower incisor at maximum mouth opening. Distance will be measured using digital calipers by the same operator
quality of life via a Skindex16 survey
Time frame: This information will be collected at baseline before treatment, 2 weeks after treatment, and 3 months after treatment.
This is a patient quality of life survey using the skindex 16 survey form.
2 secondary outcomes
Changes in the inter-commissural distance
Time frame: This will be measured at baseline before treatment, 2 weeks after treatment, and 3 months after treatment.]
Changes in the Mouth Handicap in Systemic Sclerosis Scale (MHISS)
Time frame: This will be measured at baseline before treatment, 2 weeks after treatment, and 3 months after treatment.]
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Apr 2021.
- Individual participant dataSharing planned: Undecided. To be decided by research team
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Heather Goff, MD principal investigator
University of Texas Southwestern Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Botulinum toxin(Botox) in people with Scleroderma, run by University of Texas Southwestern Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Apr 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes in inter-labial distance (time frame: This will be measured at baseline before treatment, 2 weeks after treatment, and 3 months after treatment.). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Apr 2021.