Efficacy and Safety of Allogenic Cultured Adipose-derived Mesenchymal Stromal Cell Injections on MoUth Fibrosis and Handicap in Patients With Systemic sclEroderma

NCT07326033 | Not Yet Recruiting Phase 2 DMC

• 2 other identifiers: RC31/22/02582024-518516-39-00
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 5

Brief Summary

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.

Conditions

Systemic Scleroderma

Keywords

Systemic Scleroderma; microstomia; oral health-related quality of life; oral pathology; regenerative medicine; allogenic cultured adipose-derived stromal cells

Outcome Measures

Primary Outcomes (1)

• 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

  Day 0, 12 weeks after injection

Secondary Outcomes (15)

• Safety of treatment

  Day 0, 4, 12 and 24 weeks after injection

• Efficacy on oral function by facial scanners

  Day 0, 4, 12 and 24 weeks after injection

• Efficacy on orofacial handicap

  Day 0, 4, 12 and 24 weeks after injection

• Patient satisfaction

  Day 0, 4, 12 and 24 weeks after injection

• Oral habits and hygiene

  Day 0 and 24 weeks after injection

Study Arms (2)

AdMSC

EXPERIMENTAL

Patients will be standard of care (physiotherapy and daily self-administered exercises) and receive allogeneic AdMSC injection in the perioral (lips) region.

Drug: AdMSC

Placebo

PLACEBO COMPARATOR

Patients will be standard of care (physiotherapy and daily self-administered exercises) and receive placebo injections in the perioral (lips) region

Drug: Placebo

Interventions

AdMSC DRUG

At day 0, patients will have AdMSC injections in perioral region. Patients will be followed-up for 24 weeks

AdMSC

Placebo DRUG

At day 0, patients will have placebo injections in perioral region. Patients will be followed-up for 24 weeks

Placebo

Eligibility Criteria

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

You may qualify if:

• 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,
• Maximal mouth opening of less than 40 mm (distance between the dental arches)
• Patient must have provided written informed consent prior to enrolment,
• Patient must be able to understand their requirements of participating in the protocol.
• Patient affiliated to a social security system.

You may not qualify if:

• Patient participating in a clinical trial or having participated in a clinical trial within the previous 3 months,
• Patient who underwent autologous hematopoietic stem cell transplantation (HSCT) within less than 1 year,
• Patients with an indication for intensification by autologous HSCT (according to EBMT guidelines and national MATHEC-SFGMTC guidelines),
• Radio- or chemotherapy in progress,
• Females who are pregnant or breastfeeding or plan to be or do so during the course of this study,
• Women of childbearing potential (WOCBP) who are sexually active and unwilling to use an adequate birth control method,
• Vulnerable patient (persons deprived of their liberty by judicial or administrative decision, persons undergoing psychiatric treatment, persons admitted to a health or social establishment for purposes other than research) according to article L1121-6 of the Public Health Code

Sponsors & Collaborators

• University Hospital, Toulouse: lead

Study Sites (5)

Bordeaux Hospital

Bordeaux, France

Location

Dijon Hospital

Dijon, France

Location

Montpellier Hospital

Montpellier, France

Location

Nantes Hospital

Nantes, France

Location

Toulouse Hospital

Toulouse, France

Location

MeSH Terms

Conditions

Scleroderma, Systemic; Microstomia

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Mouth Abnormalities; Mouth Diseases; Stomatognathic Diseases; Stomatognathic System Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Gregory PUGNET, MD, PHD

CONTACT

0561323954; pugnet.g@chu-toulouse.fr

Charline DAGUZAN

CONTACT

0561778490; daguzan.c@chu-toulouse.fr

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 4, 2025
• First Posted: January 8, 2026
• Study Start: January 1, 2026
• Primary Completion (Estimated): April 30, 2028
• Study Completion (Estimated): July 31, 2028
• Last Updated: January 8, 2026

Record last verified: 2025-12

Locations

FR (5)