allogenic adipose-tissue-derived mesenchymal stem cells for Systemic sclerosis (systemic scleroderma)

Inclusion criteria

• {"criterion_text":"- Male or female patient ≥18 years of age\n- Patient with systemic scleroderma according to the 2013 ACR/EULAR classification criteria\n- Mouth Handicap in Systemic Sclerosis Scale (MHISS) score more than or equal to 20 (0–48)\n- Rodnan skin score on the face more than or equal to 1\n- Maximal mouth opening of less than 40 mm (distance between the dental arches)\n- Patient must have provided written informed consent prior to enrolment\n- Patient must be able to understand the requirements of participating in the protocol\n- Patient affiliated to a social security system"}

Exclusion criteria

• {"criterion_text":"- Patient participating in a clinical trial or having participated in a clinical trial within the previous 3 months\n- Vulnerable patients (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)\n- Injection of botulinum toxin within 4 weeks prior to “inclusion visit”\n- Patient who underwent autologous hematopoietic stem cell transplantation (HSCT) within less than 1 year\n- Local active labial herpes virus within 1 week prior to “inclusion visit\",\n- Patients with an indication for intensification by autologous HSCT (according to EBMT guidelines and national MATHEC-SFGMTC guidelines),\n- History of cancer in the last five years, except for successful excised basal cell/squamous cell carcinoma, or successfully excised early melanoma of the skin. Subjects, who had successfully tumor resection or radiation or chemotherapy more than 5 years prior to inclusion and no recurrence, may be enrolled in the study,\n- Radio- or chemotherapy in progress\n- Females who are pregnant or breastfeeding or plan to be or do so during the course of this study,\n- Women of childbearing potential (WOCBP) who are sexually active and unwilling to use an adequate birth control method"}

Primary endpoints

• {"endpoint_text":"- 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","definition_or_measurement_approach":"Change in MHISS score from baseline to week 12; an improvement of ≥5 points considered clinically significant."}

Secondary endpoints

• {"endpoint_text":"- 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\n- Change in maximum interincisal distance and mouth perimeter\n- Change in Opening, Dryness and Aesthetic of the 3 subscales of the MHISS;\n- Change in patient-reported outcomes (patient satisfaction assessment (the patients will be asked to fill in a simple questionnaire where their degree of satisfaction could be expressed by a semiquantitative score (unsatisfied, mildly/moderately satisfied, rather satisfied, and very satisfied), sHAQ, OHAT, BOFA questionnaire and EQ-5D-5L at W4, W12 and W24;\n- Change in oral habits and hygiene measured by oral health and hygiene questionnaire at W24\n- Change in oral microbiota (only at Toulouse) at W12 and 24\n- Change in Plaque index (reflecting the ability to maintain oral hygiene) at W12 an W24 (only at Toulouse),\n- Change in DMFT (decayed missing filled teeth) and Panoramic x-ray at W24;\n- Change in mandibular tracking (only at Toulouse at W4, W12 and W24 and articular and neuro-muscular activity (only at Toulouse) at W12 and W24\n- Change posture by stabilometry (only at Toulouse) at W12 and W24\n- Change in psycho-social aspects and oro-facial pains by EDAS21, Epworth at W4, W12 and W24 and Combadazou-Destruhaut questionnaire (only at Toulouse) at W12 and W24 and drawings of consciousness (only at Toulouse) at W24\n- Change in modified Rodnan skin score at W12 and W24;\n- Change in dry mouth syndrome at W4, W12 and W24 (Change in Xerostomia Inventory questionnaire; Change in Salivary flow; Change in the salivary pH\n- Change in Standardised two-dimensional photographs or facial scan at Toulouse at W4,12 and 24\n- Change in immunomonitoring of vascular and antifibrotic biomarkers expression at W12;\n- Not an evaluation criterion but the creation of a serum, plasma biobank at baseline, W12 and W24).\n- Not an evaluation criterion but the creation of oral microbiota biobank (only at Toulouse at W12 and W24)","definition_or_measurement_approach":"Safety: monitoring of AEs, SAEs and injection site reactions through week 24. Interincisal distance and mouth perimeter: measured at scheduled visits (W4, W12, W24). MHISS subscales: change in Opening, Dryness and Aesthetic subscales. Patient-reported outcomes: satisfaction questionnaire, sHAQ, OHAT, BOFA, EQ-5D-5L at W4/W12/W24. Oral habits/hygiene: oral health questionnaire at W24. Oral microbiota: microbiota analysis at W12 and W24 (Toulouse only). Plaque index: measured at W12 and W24 (Toulouse only). DMFT and panoramic x-ray: assessed at W24. Mandibular tracking and neuromuscular activity: measurements at specified visits (Toulouse only). Stabilometry posture assessment: W12 and W24 (Toulouse only). Psycho-social and pain instruments: EDAS21, Epworth, Combadazou-Destruhaut, drawings at specified visits. mRSS: measured at W12 and W24. Dry mouth: Xerostomia Inventory, salivary flow and pH at W4/W12/W24. Standardised photos/face scan at W4/W12/W24 (Toulouse). Immunomonitoring biomarkers at W12. Creation of serum/plasma and oral microbiota biobanks at baseline/W12/W24 as described."}

France

Earliest CTIS Part Ii Submission Date

11-07-2025

Latest Decision Or Authorization Date

01-08-2025

Processing Time Days

21

Number Of Sites

5

Number Of Participants

50

Primary sponsor

Full Name

Centre Hospitalier Universitaire De Toulouse

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

France