Subcutaneous Injections of Autologous Cultured Adipose-Derived Stroma/ Stem Cells to Heal Refractory Ischemic Digital Ulcers in Patients with Scleroderma

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Centre Hospitalier Universitaire De Toulouse

Participant type

Patients

Age range

18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

22 Sep 2020 → ongoing

Decision date (initial)

2023-10-27

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

External identifiers

EU CT number

2023-508432-53-00

EudraCT number

2019-003906-28

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To compare efficacy and safety of digital injection of allogeneic adipose-derived stromal cell (CellReady®) versus placebo for healing refractory (chronic and/or recurrent in the 3 months following a DU occurrence) active ischemic digital ulcers in patients with systemic sclerosis in a 16 weeks placebo- controlled trial.

Conditions and MedDRA coding

Systemic sclerosis

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Over 18 years old systemic sclerosis according to the 2013 ACR/EULAR classification criteria,
2. at least one digital ulcer at baseline showing all the following characteristics: a) located beyond the proximal interphalangeal joint, on finger surface (included periungueal ulcers), b) of ischemic origin according to the physician, c) not over subcutaneous calcifications or bone relief, d) active DU, e) refractory after 10±2 weeks of standard of care (that is either still active or new occurrence despite standard of care)- Patient must have provided written informed consent prior to enrolment,
3. Patient must be able to understand their requirements of participating in the protocol,
4. Patient affiliated to a social security system.

Exclusion criteria 24

1. Patient participating in a clinical trial or having participated in a clinical trial within the previous 3 months,
2. 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,
3. 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,
4. Systemic antibiotics (oral and TV) to treat infected DU(s) within 4 weeks prior to “inclusion visit”,
5. Use of topical growth factors, hyperbaric oxygen,
6. Local injection of botulinum toxin in an affected finger within 4 weeks prior to “inclusion visit”,
7. Treatment with disease modifying agents such as methotrexate, mycophenolate mofetil, azathioprine, tacrolimus, Interferons and cyclophosphamide, within one month prior injection
8. Treatment with oral corticosteroids (> 10 mg/day of prednisone or equivalent),
9. Surgical sympathectomy of the upper limbs or surgical wound debridement within 1 month prior to “inclusion visit”,
10. Patient who underwent autologous hematopoietic stem cell transplantation (HSCT) within less than 1 year,
11. Patients with an indication for intensification by autologous HSCT (according to EBMT guidelines),
12. 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 successfully tumor resection or radiation or chemotherapy more than 5 years from inclusion and no recurrence, may be enrolled in the study,
13. Subjects who have active proliferative retinopathy,
14. Patients with a history of stroke, myocardial infarction or severe arrhythmia in the last 6 months
15. Patient who had severe cardiac failure in the last 6 months
16. Females who are pregnant or breastfeeding or plan to do so during the course of this study,
17. Patient under judicial protection or unable to express consent
18. Vulnerable patient (pregnancy or lactating women, parturients, 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
19. Refusal of the patient to participate in the study.
20. Digital ulcer due to conditions other than scleroderma
21. Non ischemic digital ulcer,
22. Ulcers with osteomyelitis, or clinically uncontrolled infection,
23. Infected digital ulcer requiring systemic antibiotherapy,
24. Digital ulcer requiring urgent surgery.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Proportion of refractory active ischemic digital ulcers healed (complete or partial) without recurrence at 16 weeks. The main evaluation criterion is a composite endpoint combining healing (complete or partial) without recurrence at 16 weeks and without local or general complications.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Centre Hospitalier Universitaire De Toulouse

Sponsor organisation

Centre Hospitalier Universitaire De Toulouse

Address

2 Rue Viguerie

City

Toulouse

Postcode

31300

Country

France

Scientific contact point

Organisation

Centre Hospitalier Universitaire De Toulouse

Contact name

clinical research assistant

Public contact point

Organisation

Centre Hospitalier Universitaire De Toulouse

Contact name

clinical research assistant

Locations

1 EU/EEA country · 7 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 5 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications