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

December 23, 2025 updated by: University Hospital, Toulouse
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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Not yet recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
50

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

  • France
      • Bordeaux, France
      • Dijon, France
        • Dijon Hospital
        • Contact:
        • Principal Investigator:
          • Martin NIVET
      • Montpellier, France
        • Montpellier Hospital
        • Contact:
        • Principal Investigator:
          • Alexandre MARIA
      • Nantes, France
      • Toulouse, France
        • Toulouse Hospital
        • Principal Investigator:
          • Gregory Pugnet
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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",
  • Patients with an indication for intensification by autologous HSCT (according to EBMT guidelines and national MATHEC-SFGMTC guidelines),
  • History of cancer in the last five years, except for successfully excised basal cell/squamous cell carcinoma, or successfully excised early melanoma of the skin. Subjects, who had a successful tumor resection or radiation or chemotherapy more than 5 years prior to inclusion and no recurrence, may be enrolled in the study,
  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: AdMSC
Patients will be standard of care (physiotherapy and daily self-administered exercises) and receive allogeneic AdMSC injection in the perioral (lips) region.
Drug: AdMSC
At day 0, patients will have AdMSC injections in perioral region. Patients will be followed-up for 24 weeks
Placebo Comparator: Placebo
Patients will be standard of care (physiotherapy and daily self-administered exercises) and receive placebo injections in the perioral (lips) region
Drug: Placebo
At day 0, patients will have placebo injections in perioral region. Patients will be followed-up for 24 weeks

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Change in the Mouth Handicap in Systemic Sclerosis scale (MHISS)
Time Frame: Day 0, 12 weeks after injection
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 Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Safety of treatment
Time Frame: Day 0, 4, 12 and 24 weeks after injection
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.
Day 0, 4, 12 and 24 weeks after injection
Efficacy on oral function by facial scanners
Time Frame: Day 0, 4, 12 and 24 weeks after injection
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
Day 0, 4, 12 and 24 weeks after injection
Efficacy on orofacial handicap
Time Frame: Day 0, 4, 12 and 24 weeks after injection
Efficacy on orofacial handicap is a composite measure derived from change in Opening, Dryness and Aesthetic the 3 subscales of the MHISS
Day 0, 4, 12 and 24 weeks after injection
Patient satisfaction
Time Frame: Day 0, 4, 12 and 24 weeks after injection
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
Day 0, 4, 12 and 24 weeks after injection
Oral habits and hygiene
Time Frame: Day 0 and 24 weeks after injection
Change in oral habits and hygiene is a composite measure derived from oral health and hygiene questionnaire
Day 0 and 24 weeks after injection
oral microbiota
Time Frame: Day 0, 12 and 24 weeks after injection
Change in oral microbiota mesuared in unstimulated saliva
Day 0, 12 and 24 weeks after injection
Plaque index
Time Frame: Day 0, 12 and 24 weeks after injection
Change in Plaque index (reflecting the ability to maintain oral hygiene)
Day 0, 12 and 24 weeks after injection
Change in decayed missing filled teeth
Time Frame: Day 0 and 24 weeks after injection
Change in decayed missing filled teeth (DMFT) index : a commonly used index validated by the WHO to assess oral status and the examinator simply counts the number of decayed, missing (due to caries or periodontal disease) and restored/filled teeth.
Day 0 and 24 weeks after injection
Change in mandibular tracking
Time Frame: Day 0, 4, 12 and 24 weeks after injection
Change Mandibular tracking is a composite measure derived from mandibular tracking and articular and neuro-muscular activity. Mandibular tracking and neuro-muscular activity will be measured with electrodes positioned in order to assess muscular activity at rest, muscle activity during mouth closure as well as during extreme movement of mouth opening and closing, mandibular propulsion and deduction. Muscle contraction synchronism and contraction efficacy will also be measured as well as the 3-dimensional innoclusion space and mandibular movement during swallowing
Day 0, 4, 12 and 24 weeks after injection
Posture by stabilometry
Time Frame: Day 0, 12 and 24 weeks after injection
Change posture by stabilometry
Day 0, 12 and 24 weeks after injection
psycho-social aspects and oro-facial pains
Time Frame: Day 0, 4, 12 and 24 weeks after injection
Change in psycho-social aspects and oro-facial pains is a composite measure derived from EDAS21, Epworth and Combadazou-Destruhaut questionnaire
Day 0, 4, 12 and 24 weeks after injection
Rodnan skin score
Time Frame: Day 0, 12 and 24 weekds after injection
Change in modified Rodnan skin score
Day 0, 12 and 24 weekds after injection
Dry mouth syndrome
Time Frame: Day 0, 4, 12 and 24 weeks after injection
Change in dry mouth syndrome is a composite measure derived from the change in Xerostomia Inventory questionnaire , in salivary flow, in the salivary pH and Salivary pH
Day 0, 4, 12 and 24 weeks after injection
Efficacy on aesthetics
Time Frame: Day 0, 4 , 12 and 24 weeks after injection
the efficacy on aesthetics assessed by Standardized two-dimensional photographs or face scan
Day 0, 4 , 12 and 24 weeks after injection
Immunomonitoring of vascular and antifibrotic biomarkers
Time Frame: Day 0 and 12 weekds after injection
Immunomonitoring of vascular and antifibrotic biomarkers (V5. Endothelin-1, Endostatin, Endogline, Angiotensin I and II, Tie 1 and 2, VEGF, sICAM-1, sVCAM, E-selectin, CXCL4) measured in blood samples
Day 0 and 12 weekds after injection

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
University Hospital, Toulouse

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 1, 2026

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 30, 2028

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
July 31, 2028

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 4, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 23, 2025

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 8, 2026

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
January 8, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 23, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
December 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • RC31/22/0258
  • 2024-518516-39-00 (Ctis)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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