NCT03562065: MSC-SLE

Treatment of Refractory Systemic Lupus Erythematosus by Allogeneic Mesenchymal Stem Cells Derived From the Umbilical Cord

Status unknown Phase 1, PHASE2 Last updated 8 July 2021

What this trial tests

Phase 1, PHASE2 trial testing mesenchymal stem cells in Lupus Erythematosus in 10 participants. Status unknown.

Timeline

11 September 2019

Primary endpoint
1 June 2023

1 June 2024

Quick facts

Lead sponsor	Assistance Publique - Hôpitaux de Paris
Phase	Phase 1, PHASE2
Status	Status unknown
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	10
Start date	11 September 2019
Primary completion	1 June 2023
Estimated completion	1 June 2024
Sites	1 location across France

Drugs / interventions tested

• mesenchymal stem cells — full drug profile →

Conditions studied

• Lupus Erythematosus — all drugs for Lupus Erythematosus →
• Stem Cell Transplant — all drugs for Stem Cell Transplant →

Sponsor

Assistance Publique - Hôpitaux de Paris — full company profile →

Who can join

Adults 18 to 70, any sex, with Lupus Erythematosus or Stem Cell Transplant. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Systemic lupus erythematosus (SLE) is a rare (prevalence: 40- 50/100 000 persons) heterogeneous auto-immune and auto-inflammatory disease (AD), affecting both sexes and all races, with a peak incidence / prevalence among black people and a predilection for women in the 3rd-4th decade of life. SLE is characterized by successive periods of flares and remission, which may all vary in duration and quality. Prognosis of severe forms of SLE, which affect lung, heart or brain in addition to renal involvement, has improved, but still evolution remains pejorative in a subset of patients whose 10 years mortality remains 10-15%, even in tertiary referral centers. For 20 years, no new prospective clinical trial in the course of SLE has demonstrated its effectiveness. New biological therapies have not yet made the long awaited breakthrough in the treatment of severe SLE and only anti-Blys monoclonal antibody has gained indication in moderately active SLE. In addition, serious adverse side effects (progressive multifocal leukoencephalopathy) observed with several biologics in AD patients has dampened their expected benefits. For SLE subjects resistant to 1er or 2nd line conventional treatment, there is a need to develop more effective therapies with fewer long term side effects, based on new immunomodulatory and immunosuppressive strategies.

According to their in vitro immunomodulatory properties and ability to induce tissue repair mechanisms, mesenchymal stem cells (MSC) have been proposed as a new therapy for several AD, including SLE. The use of allogeneic umbilical cord-derived MSC is based on experimental and human clinical data, particularly produced by Nanjing team (Pr Sun) in China. It is also logical to select SLE patients with the same severity criteria as those used worldwide to validate the efficacy of anti-Blys therapies. Similarly, the analysis of the expected results should take into account criteria similar or comparable to those used for the pivotal clinical trials. This trial is a unique opportunity to set up collaboration between Saint-Louis APHP, clinical expert center for cell therapy in AD, and University College London for cell manufacturing.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Mesenchymal stem/stromal cells as a valuable source for the treatment of immune-mediated disorders.
   Markov A, Thangavelu L, Aravindhan S, Zekiy AO, et al · · 2021 · cited 174× · PMID 33736695 · DOI 10.1186/s13287-021-02265-1
2. Human umbilical cord-derived mesenchymal stem/stromal cells: a promising candidate for the development of advanced therapy medicinal products.
   Mebarki M, Abadie C, Larghero J, Cras A. · · 2021 · cited 116× · PMID 33637125 · DOI 10.1186/s13287-021-02222-y
3. Mesenchymal stem/stromal cell quality control: validation of mixed lymphocyte reaction assay using flow cytometry according to ICH Q2(R1).
   Nicotra T, Desnos A, Halimi J, Antonot H, et al · · 2020 · cited 23× · PMID 33004063 · DOI 10.1186/s13287-020-01947-6
4. Current cell therapies for systemic lupus erythematosus.
   Dao LTM, Vu TT, Nguyen QT, Hoang VT, et al · · 2024 · cited 16× · PMID 38920310 · DOI 10.1093/stcltm/szae044
5. Current advancements in cellular immunotherapy for autoimmune disease.
   Berry CT, Frazee CS, Herman PJ, Chen S, et al · · 2025 · cited 15× · PMID 39821376 · DOI 10.1007/s00281-024-01034-5
6. Adipose-derived mesenchymal stem cell therapy for connective tissue diseases and complications.
   Kotani T, Saito T, Suzuka T, Matsuda S. · · 2024 · cited 13× · PMID 39026275 · DOI 10.1186/s41232-024-00348-z
7. Allogeneic umbilical cord-derived mesenchymal stromal cells as treatment for systemic lupus erythematosus: a single-centre, open-label, dose-escalation, phase 1 study.
   Farge D, Biard L, Weil B, Girault V, et al · · 2025 · cited 5× · PMID 39706212 · DOI 10.1016/s2665-9913(24)00298-4
8. Stem Cell Therapy for Inflammatory Diseases: Progress, Challenges, and Future Directions.
   Wu C, Jin ZP, Weng SQ, Zhu JM, et al · · 2026 · PMID 41640457 · DOI 10.1002/mco2.70616