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

NCT04356755 | Recruiting Phase 2 DMC

• 1 other identifier: RC31/17/0447
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 7

Brief 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.

Conditions

Systemic Sclerosis

Outcome Measures

Primary Outcomes (2)

• Proportion of refractory active ischemic digital ulcers healed (complete or partial)

  Partial healing is defined as \> 50% reduction of the DU area or \> 50% re epidermisation of the DU.

  16 weeks

• Composite endpoint combining healing (complete or partial) without recurrence and without local or general complications

  Partial healing is defined as \> 50% reduction of the DU area or \> 50% re epidermisation of the DU. Local complications resulting from DU worsening: * Critical ischemic crisis necessitating hospitalization, * Gangrene, (auto) amputation, * Failure of conservative management: Surgical and chemical sympathectomy, vascular reconstructions, or any unplanned surgery in the management of hand SSc manifestation(s), * Use of parenteral prostanoids after treatment injection, * Requiring class II, III or IV narcotics or increase in existing dose of \> 50 % as compared to baseline, * Initiation of systemic antibiotics for the treatment of infection attributed to digital ulceration. General complications will be assessed by: * Clinical examination * Patient reported-outcomes * Laboratory assessments

  16 weeks

Secondary Outcomes (15)

• Percentage of participants with Digital Ulcer complete healing

  16 weeks

• Percentage of participants with Digital Ulcer partial healing

  16 weeks

• Percentage of participants with wound surface reduction <50%

  16 weeks

• Percentage of participants with new Digital Ulcer

  16 weeks

• Percentage of participants with Digital Ulcer complication

  16 weeks

Study Arms (2)

AdMSC

EXPERIMENTAL

Subcutaneous injections of cultured adipose-derived stroma/stem cells to heal refractory ischemic digital ulcers in patients with scleroderma

Drug: AdMSC

Placebo

PLACEBO COMPARATOR

Subcutaneous injections of placebo comparator to heal refractory ischemic digital ulcers in patients with scleroderma

Drug: Placebo

Interventions

AdMSC DRUG

At day 0, patients will have AdMSC injections in their ischemic DU. Patients will be followed-up for 16 weeks

AdMSC

Placebo DRUG

At day 0, patients will have placebo injections in their ischemic DU. Patients will be followed-up for 16 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 sclerosis according to the 2013 ACR/EULAR classification criteria9,
• Age \> 50 years and not treated with any kind of hormone replacement therapy for at least 2 years prior to screening, with amenorrhea for at least 24 consecutive months prior to screening. An assessment of serum follicle stimulating hormone showing a level of \> 40 TU/L at screening may be used to exclude childbearing potential, based on the discretion of the investigator,
• 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.
• Relative to each DU :
• Located beyond the proximal interphalangeal joint, on finger surface (included periungual ulcers),
• Of ischemic origin according to the physician,
• Not over subcutaneous calcifications or bone relief,
• Active DU,
• Refractory after 10±2 weeks of standard of care according to EULAR recommendations26 (that is either still active (chronic) or new occurrence despite standard of care)

You may not qualify if:

• Patients on statins, who have received treatment for less than 3 months prior to Screening or whose treatment has not been stable during this period,
• Treatment with disease modifying agents such as methotrexate, mycophenolate mofetil, azathioprine, tacrolimus, Interferons and cyclophosphamide, those drugs should be stop at least 1 month prior study entry.
• Treatment with oral corticosteroids (\> 10 mg/day of prednisone or equivalent),
• Use of topical growth factors, hyperbaric oxygen,
• Liposuction technically impossible,
• 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 RCP MATHEC),
• Positive HIV-1 or 2, HTLV-1 or 2, HBV or HCV,
• Patients with a history of stroke, myocardial infarction or severe arrhythmia in the last 6 months
• Patient who had severe cardiac failure in the last 6 months,
• Females who are pregnant or breastfeeding or plan to do so during the course of this study,
• Patient under judicial protection, - Refusal of the patient to participate in the study.
• Relative to each DU:
• Digital ulcer due to conditions other than scleroderma,
• Non ischemic digital ulcer,

Sponsors & Collaborators

• University Hospital, Toulouse: lead

Study Sites (7)

Grenoble Hospital

Grenoble, France

NOT YET RECRUITING

Lille Hopsital

Lille, France

NOT YET RECRUITING

Marseille Hospital

Marseille, France

NOT YET RECRUITING

Montpellier Hospital

Montpellier, France

NOT YET RECRUITING

Nantes Hospital

Nantes, France

NOT YET RECRUITING

Poitiers Hospital

Poitiers, France

NOT YET RECRUITING

CHU de Toulouse - Hôpital PURPAN-TSA

Toulouse, 31059, France

RECRUITING

MeSH Terms

Conditions

Scleroderma, Systemic

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Study Officials

• Grégory PUGNET, MD, PHD

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Grégory PUGNET, MD, PHD

CONTACT

05 61 77 71 26; pugnet.g@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: March 11, 2020
• First Posted: April 22, 2020
• Study Start: September 22, 2020
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): March 1, 2027
• Last Updated: December 31, 2025

Record last verified: 2025-12

Locations

FR (7)