Orofacial manifestations and microstomia are a frequent complication in systemic sclerosis (SSc) with a major impact on oral hygiene, dental care and quality of life. Peri-oral injection of allogeneic cultured adipose-derived stromal cells constitutes a promising approach to treat scleroderma-induced mouth fibrosis where no alternative therapy is validated. The aim of this phase 2 study is to compare efficacy and safety of perioral injection of allogeneic cultured adipose-derived stromal cells (AdMSC) versus placebo to improve oro-facial fibrosis in patients with systemic sclerosis.
Male or female patient ≥18 years of age,
Patient with systemic scleroderma according to the 2013 ACR/EULAR classification criteria,
Mouth Handicap in Systemic Sclerosis Scale (MHISS) score more than or equal to 20 (0-48),
Rodnan skin score on the face more than or equal to 1,
Patient participating in a clinical trial or having participated in a clinical trial within the previous 3 months,
Injection of botulinum toxin within 4 weeks prior to "inclusion visit",
Patient who underwent autologous hematopoietic stem cell transplantation (HSCT) within less than 1 year,
Local active labial herpes virus within 1 week prior to "inclusion visit",
Parallel
AdMSC
DrugAt day 0, patients will have AdMSC injections in perioral region. Patients will be followed-up for 24 weeks
Placebo
DrugAt day 0, patients will have placebo injections in perioral region. Patients will be followed-up for 24 weeks
Change in the Mouth Handicap in Systemic Sclerosis scale (MHISS)
the change in the Mouth Handicap in Systemic Sclerosis scale (MHISS) between baseline and week 12. An improvement of at least 5 points will be considered clinically significant
Safety of treatment
The safety throughout the course of the study (until week 24 since baseline) will be assessed by monitoring adverse events, serious adverse events and injection site reactions.
Efficacy on oral function by facial scanners
Efficacy on oral function is a composite measure derived from the change in maximum interincisal distance and mouth perimeter by facial scanners with digital acquisition of the patient's face in just a few seconds
Efficacy on orofacial handicap
Efficacy on orofacial handicap is a composite measure derived from change in Opening, Dryness and Aesthetic the 3 subscales of the MHISS
Patient satisfaction
the patients will be asked to fill in a simple questionnaire where their degree of satisfaction could be expressed by a composite measure derived by a semiquantitative score (unsatisfied, mildly/moderately satisfied, rather satisfied, and very satisfied), Scleroderma Health Assessment Questionnaire (sHAQ), Oral Health Assessment Tool (OHAT), Burden of Face Affected questionnaire (BOFA) and EQ-5D-5L
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