Hyaluronidase in Treating Oral Microstomia in Patients With Sclerosing Skin Disease

NCT04656704 | Withdrawn Early Phase 1 DMC FDA Drug

• 1 other identifier: 2020P003096
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief 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. 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.

Conditions

Microstomia; Scleroderma; CREST Syndrome; Scleromyxedema; Morphea

Outcome Measures

Primary Outcomes (1)

• 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.

  Weeks 0, 4, 8, 12, 16, 20, and 24

Study Arms (1)

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

Drug: hyaluronidase injected intradermally

Interventions

hyaluronidase injected intradermally DRUG

Patients will receive 200 units of hyaluronidase injected intradermally to the cutaneous lips on Weeks 0, 4, 8, 12, and 16, and 20

Hyaluronidase 200mg monthly

Eligibility Criteria

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

You may qualify if:

• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Brigham and Women's Hospital: lead

MeSH Terms

Conditions

Microstomia; Scleroderma, Diffuse; CREST Syndrome; Scleromyxedema; Scleroderma, Localized

Condition Hierarchy (Ancestors)

Mouth Abnormalities; Mouth Diseases; Stomatognathic Diseases; Stomatognathic System Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Scleroderma, Systemic; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Esophageal Motility Disorders; Deglutition Disorders; Esophageal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Raynaud Disease; Livedoid Vasculopathy; Thrombosis; Embolism and Thrombosis; Vascular Diseases; Cardiovascular Diseases; Peripheral Vascular Diseases; Telangiectasis; Scleroderma, Limited; Skin Diseases, Vascular; Calcinosis; Calcium Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Mucinoses

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Attending Physician

Study Record Dates

• First Submitted: November 30, 2020
• First Posted: December 7, 2020
• Study Start: January 1, 2020
• Primary Completion: January 1, 2022
• Study Completion: January 1, 2022
• Last Updated: January 14, 2021

Record last verified: 2021-01