MicrostomiaEarly Phase 1NCT04656704
Hyaluronidase in Treating Oral Microstomia in Patients With Sclerosing Skin Disease
A Early Phase 1 study testing hyaluronidase injected intradermally in people with Microstomia and Scleroderma, run by Brigham and Women's Hospital.
Trial summary
The principal objective is to measure the degree to which oral microstomia caused by sclerosing skin disease improves after treating patients with local hyaluronidase injections. Investigator will determine improvements in oral aperture by measuring the centimeters of the height of oral opening. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Microstomia, Scleroderma, CREST Syndrome
- What is being tested?
- hyaluronidase injected intradermally
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 1 Jan 2020to 1 Jan 2022
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- At least 18 years of age
- Established diagnosis of a sclerosing skin disease (i.e., systemic sclerosis, CREST, scleromyxedema, morphea) by a dermatologist
- Ability and willingness to provide informed consent, participate in study visits, and undergo visit procedures
- Symptoms are not resolved by current treatment
Who cannot join
- Prior treatment with hyaluronidase for oral microstomia
- Treatment with hyaluronic acid soft tissue filler in perioral region within the last 1.5 years
- Treatment with any semi-permanent or permanent soft tissue filler in the perioral region in the last 3 years
- New treatment initiation (within 3 months) with immune-regulating medication, including methotrexate, azathioprine, mycophenolate mofetil, cyclosporine, intravenous immunoglobulins, systemic steroids, or rituximab
- New initiation of physical/occupational therapy for muscles of the face within the last 3 months
- Known hypersensitivity to hyaluronidase
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- Women of reproductive potential who are or plan to become pregnant or breastfeed during study participation or are unable or not willing to use birth control during the study. Options for birth control include abstinence, double barrier (i.e. male condom and female diaphragm), vasectomy, intrauterine device, and hormonal contraception. Females who have not had menses within 1 year of the baseline, bilateral tubal ligation, hysterectomy, and/or bilateral oophorectomy do not require additional methods of contraception during study participation
- Unstable condition or status, as per study investigator's judgment, that may lead to more likely discontinuation from the study including, but are not limited to, major, recurrent medical illnesses that may require hospital admission, and/or inability to participate in all study visits
- Concurrent topical steroid use during the study (Note: topical steroid use up until the start of the study enrollment is allowed and no washout period will be required)
- Concurrent ingestion of high dose salicylates, cortisone, ACTH, estrogens, or antihistamines
About this study
Official title: Investigating Hyaluronidase in Treating Oral Microstomia in Patients With Sclerosing Skin Disease
The principal objective is to measure the degree to which oral microstomia caused by sclerosing skin disease improves after treating patients with local hyaluronidase injections. Investigator will determine improvements in oral aperture by measuring the centimeters of the height of oral opening.
The secondary objectives are: Investigator will aim to assess changes in quality of life and functionality, by serial calculations of the Mouth Handicap in Systemic Sclerosis (MHISS). In addition, investigator would like to investigate how many treatments are required prior to treatment efficacy plateauing. Since there is minimal data on the use of hyaluronidase for oral microstomia, it is not yet clear how many treatments are ideally required for maximal effect.
Read more
Patients will be brought in monthly for photographs, examination, assessment, and treatment.
Our hypothesis is that hyaluronidase injections will significantly improve patients' ability to open their mouths and oral functionality. It remains unclear how many treatments will be required for maximal effect.
Read the detailed description
Investigator will recruit 8 patients with oral microstomia caused by a sclerotic skin disorder. Patients must be 18 years of age or older with an established diagnosis of a sclerotic skin disorder by a dermatologist. Sclerotic skin disorders may include scleroderma, CREST, scleromyxedema, and morphea involving the face.
At the initial screening visit, demographic information will be obtained, inclusion and exclusion criteria will be reviewed, and informed consent will be obtained for those deemed eligible for enrollment. Patients who qualify for the study, and who have never been exposed to hyaluronidase before, will have 0.1 cc hyaluronidase injected to assess whether they have an allergic reaction.
Qualifying patients will undergo monthly intradermal injections of 200 units of hyaluronidase into the cutaneous lips by the study staff into through 6 treatments. Subjects will be evaluated for improvement in primary and secondary endpoints using clinical examination, photographs, and questionnaires during initial and follow-up visits.
Since this is a pilot study, there are only 2 case reports published wherein hyaluronidase was used for similar indications. One study injected approximately between 120 and 150 units for morphea-induced microstomia; this study listed locations of injection sites (ie, right jaw line, left jawline, and chin). Another used between 20 and 200 units of hyaluronidase for scleroderma-induced microstomia; investigators injected in 2 rows of the cutaneous lips and approximated number of injections in a schematic. Investigator elected to inject the higher end of dosage used, 200 units (1 full vial of hyaluronidase), as this was effectively used in a publication. Additionally, there has not been any dose-related side effect concerns with hyaluronidase to our knowledge. It is common practice to use multiple vials to treat hyaluronic acid complications, which is safely done.
Subjects will be evaluated for improvement in primary and secondary endpoints using clinical examination, photographs, and questionnaires during initial and follow-up visits.
- Conditions
- Microstomia, Scleroderma, CREST Syndrome, Scleromyxedema, Morphea
- Being tested
- hyaluronidase injected intradermally
- Medical subject headings
- Microstomia, Scleroderma, Diffuse, CREST Syndrome, Scleromyxedema, Scleroderma, Localized
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hyaluronidase injected intradermallyDrug
Patients will receive 200 units of hyaluronidase injected intradermally to the cutaneous lips on Weeks 0, 4, 8, 12, and 16, and 20
| Study group | Role | What they receive |
|---|---|---|
| Hyaluronidase 200mg monthly | Experimental | Hyaluronidase 200mg monthly (Weeks 0, 4, 8, 12, 16, 20); for six doses; a 6-month treatment course with follow-up 1 month after Receives: Drug: hyaluronidase injected intradermally |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change of oral aperture at the largest diameter
Change of oral aperture at the largest diameter, in centimeters, with the patient's mouth opened as widely as possible. Measurements will be completed by study personnel.
Measured: Weeks 0, 4, 8, 12, 16, 20, and 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Dec 2020
- Study start1 Jan 2020 (estimated)
- Primary completion1 Jan 2022 (estimated)
- Study completion1 Jan 2022 (estimated)
Why it stopped: PI no longer pursuing clinical trial
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MicrostomiaStudies of the same condition, open and under way.
- More trials run by Brigham and Women's HospitalThe 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Brigham and Women's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Avery LaChance, Brigham and Women's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04656704
- Sponsor's protocol ID
- 2020P003096
- Status last verified
- Jan 2021
- First submitted
- 30 Nov 2020
- Last updated
- 14 Jan 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Investigating Hyaluronidase in Treating Oral Microstomia in Patients With Sclerosing Skin Disease
- ClinicalTrials.gov ID
- NCT04656704
- Acronym / sponsor ID
- 2020P003096
- Conditions
- Microstomia, Scleroderma, CREST Syndrome, Scleromyxedema, Morphea
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: PI no longer pursuing clinical trial
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Brigham and Women's HospitalUniversity, hospital or other
- Study start
- 1 Jan 2020 (estimated)
- Primary completion
- 1 Jan 2022 (estimated)
- Study completion
- 1 Jan 2022 (estimated)
- First posted
- 7 Dec 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.
The principal objective is to measure the degree to which oral microstomia caused by sclerosing skin disease improves after treating patients with local hyaluronidase injections. Investigator will determine improvements in oral aperture by measuring the centimeters of the height of oral opening.
The secondary objectives are: Investigator will aim to assess changes in quality of life and functionality, by serial calculations of the Mouth Handicap in Systemic Sclerosis (MHISS). In addition, investigator would like to investigate how many treatments are required prior to treatment efficacy plateauing. Since there is minimal data on the use of hyaluronidase for oral microstomia, it is not yet clear how many treatments are ideally required for maximal effect.
Patients will be brought in monthly for photographs, examination, assessment, and treatment.
Our hypothesis is that hyaluronidase injections will significantly improve patients' ability to open their mouths and oral functionality. It remains unclear how many treatments will be required for maximal effect.
Detailed description
Investigator will recruit 8 patients with oral microstomia caused by a sclerotic skin disorder. Patients must be 18 years of age or older with an established diagnosis of a sclerotic skin disorder by a dermatologist. Sclerotic skin disorders may include scleroderma, CREST, scleromyxedema, and morphea involving the face.
At the initial screening visit, demographic information will be obtained, inclusion and exclusion criteria will be reviewed, and informed consent will be obtained for those deemed eligible for enrollment. Patients who qualify for the study, and who have never been exposed to hyaluronidase before, will have 0.1 cc hyaluronidase injected to assess whether they have an allergic reaction.
Qualifying patients will undergo monthly intradermal injections of 200 units of hyaluronidase into the cutaneous lips by the study staff into through 6 treatments. Subjects will be evaluated for improvement in primary and secondary endpoints using clinical examination, photographs, and questionnaires during initial and follow-up visits.
Since this is a pilot study, there are only 2 case reports published wherein hyaluronidase was used for similar indications. One study injected approximately between 120 and 150 units for morphea-induced microstomia; this study listed locations of injection sites (ie, right jaw line, left jawline, and chin). Another used between 20 and 200 units of hyaluronidase for scleroderma-induced microstomia; investigators injected in 2 rows of the cutaneous lips and approximated number of injections in a schematic. Investigator elected to inject the higher end of dosage used, 200 units (1 full vial of hyaluronidase), as this was effectively used in a publication. Additionally, there has not been any dose-related side effect concerns with hyaluronidase to our knowledge. It is common practice to use multiple vials to treat hyaluronic acid complications, which is safely done.
Subjects will be evaluated for improvement in primary and secondary endpoints using clinical examination, photographs, and questionnaires during initial and follow-up visits.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hyaluronidase 200mg monthly | Experimental | Drug: hyaluronidase injected intradermallyHyaluronidase 200mg monthly (Weeks 0, 4, 8, 12, 16, 20); for six doses; a 6-month treatment course with follow-up 1 month after |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- At least 18 years of age
- Established diagnosis of a sclerosing skin disease (i.e., systemic sclerosis, CREST, scleromyxedema, morphea) by a dermatologist
- Ability and willingness to provide informed consent, participate in study visits, and undergo visit procedures
- Symptoms are not resolved by current treatment
Key exclusion criteria (10 listed)
- Prior treatment with hyaluronidase for oral microstomia
- Treatment with hyaluronic acid soft tissue filler in perioral region within the last 1.5 years
- Treatment with any semi-permanent or permanent soft tissue filler in the perioral region in the last 3 years
- New treatment initiation (within 3 months) with immune-regulating medication, including methotrexate, azathioprine, mycophenolate mofetil, cyclosporine, intravenous immunoglobulins, systemic steroids, or rituximab
- New initiation of physical/occupational therapy for muscles of the face within the last 3 months
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 |
|---|---|---|
| hyaluronidase injected intradermallyPatients will receive 200 units of hyaluronidase injected intradermally to the cutaneous lips on Weeks 0, 4, 8, 12, and 16, and 20 | Drug | Hyaluronidase 200mg monthly |
Primary outcome
Change of oral aperture at the largest diameter
Time frame: Weeks 0, 4, 8, 12, 16, 20, and 24
Change of oral aperture at the largest diameter, in centimeters, with the patient's mouth opened as widely as possible. Measurements will be completed by study personnel.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jan 2021.
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing hyaluronidase injected intradermally in people with Microstomia and Scleroderma, run by Brigham and Women's Hospital.
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 10 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: withdrawn, as of the sponsor's last update on 14 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
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 was withdrawn before enrolling participants, so there are no results.
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?
Change of oral aperture at the largest diameter (time frame: Weeks 0, 4, 8, 12, 16, 20, and 24). 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 14 Jan 2021.