NCT04356755

Subcutaneous Injections of ASC to Heal Digital Ulcers in Patients With Scleroderma.

Subcutaneous Injections of Cultured Adipose-derived Stroma/ Stem Cells to Heal Refractory Ischemic Digital Ulcers in Patients With Scleroderma

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 32 (estimated)
• Sponsor: University Hospital, Toulouse · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Ischemic digital ulcers (DUs) are a frequent complication in systemic sclerosis with a major impact on hand function and quality of life. Digital injection of cultured adipose-derived stromal cell (AdMSC) constitutes a promising approach to treat scleroderma-induced refractory ischemic DUs where no alternative therapy is validated. The aim of this phase 2 study is to compare efficacy and safety of digital injection of AdMSC versus placebo for healing refractory active ischemic digital ulcers in patients with systemic sclerosis.

Detailed description

Systemic sclerosis (SSc) is a systemic autoimmune disease characterized by an autoimmune-mediated microangiopathy and progressive fibrosis. Ischemic digital ulcers (DUs) are frequent in the disease course. DUs are an expression of the severity of the microangiopathy. DUs lead to pain, infection, gangrene, autoamputation, impaired hand use and impaired quality of life. The management of DUs is often based on optimal wound care to promote healing and and repeated hospitalizations to perform onerous prostacyclin infusions to reduce pain and accelerate healing. With optimal standard of care, only 60% of DUs are healed after 3 months and 46.2% experiences recurrence during that time among them 11.2% experiences a chronic evolution. No drug has demonstrated a positive effect on refractory DUs healing. The rational underlying the use of cultured adipose-derived stromal cell (AdMSC) in this indication is based on the finding of AdMSC, in vitro and in vivo, angiogenic and anti-inflammatory potential in other ischemic pathologies, with an excellent safety profile. The pilot phase of the ACellDREAM trial demonstrated the feasibility and safety of AdMSC transplantation in patients with non- revascularizable critical limb ischemia and showed improvement in ulcer evolution and wound healing. The EFS-O culture procedure safety is validated and is already in use in ongoing French and European clinical trials. Two pilot studies showed the safety of adipose tissue grafting for scleroderma-Induced DU. The SCLERADEC pilot study outlines the safety, in 12 SSc patients, of the digital injection of adipose- derived stromal vascular fraction, which is a heterogeneous population of cells including only 36% of uncultured AdMSC. An improvement in hand disability, quality of life and DUs was observed, the phase II is ongoing. The hypothesis of the study is that digital injection of AdMSC could be efficacious for scleroderma-induced refractory ischemic DUs healing by digital vascular regeneration in a clinical situation where no alternative therapy is validated.

Conditions

• Systemic Sclerosis

Interventions

Type	Name	Description
DRUG	AdMSC	At day 0, patients will have AdMSC injections in their ischemic DU. Patients will be followed-up for 16 weeks
DRUG	Placebo	At day 0, patients will have placebo injections in their ischemic DU. Patients will be followed-up for 16 weeks

Timeline

• Start date: 2020-09-22
• Primary completion: 2027-03-01
• Completion: 2027-03-01
• First posted: 2020-04-22
• Last updated: 2025-12-31

Locations

7 sites across 1 country: France