Clinical trial NCT06737380: UC-MSC Cell Therapy Study for SLE Patients
Official title: A Phase I, Open-Label Study to Evaluate the Safety and Tolerability of Subcutaneous Administration of Umbilical Cord Derived - Mesenchymal Stromal Cell Therapy in Addition to Standard of Care as A Treatment For Active Systemic Lupus Erythematosus
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: SLE
Lupus
Systemic Lupus Erthematosus
Systemic Lupus Erythematosus
Systemic Lupus Erythematosus (SLE)
Lead sponsor: LiveKidney.Bio
Sponsor class: INDUSTRY
Collaborators: name: Medical University of South Carolina
name: Galilee CBR
First posted: 2024-12-17
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The goal of this clinical trial is to evaluate the safety and effectiveness of UC-MSCs in adults with systemic lupus erythematosus (SLE).

The main questions this study aims to answer are:

1. Can UC-MSCs improve kidney function and reduce SLE disease activity?
2. Are UC-MSCs safe and well-tolerated in this patient population?

Participants in this study will:

* Receive UC-MSCs in a single dose in addition to standard of care treatment.
* Provide blood and urine samples for laboratory assessments, including biomarkers and immune profiling (e.g., cytokines, complement proteins, and autoantibodies).
* Attend regular clinic visits for physical exams, disease activity scoring, and imaging tests to monitor kidney health.
* Complete assessments for safety, such as monitoring for adverse events and changes in laboratory values.

This study aims to provide new insights into treatment options for SLE and lupus nephritis, addressing an unmet medical need in this population.


Eligibility criteria: Inclusion Criteria:

1. Age 18-75 years at the time of screening
2. Adults participants with the diagnosis of systemic lupus erythematosus (SLE) by meeting at least 4 of the11 criteria included in the American College of Rheumatology (ACR) classification and/or the Systemic Lupus International Collaborating Clinics (SLICC) criteria, at the screening visit.
3. Must have a positive ANA (≥1:160 titer) or positive dsDNA antibody test within 6 months of screening
4. An eGFR of ≥ 30 mL/min/1.73 m2 at screening
5. At least one SLE background therapy (e.g. immunosuppressant and/or antimalarial) is required for ≥ 12 weeks before the screening visit, must be at a stable dose for ≥ 8 weeks before the screening visit, and must remain stable until randomization and throughout study participation or have taken at least one background therapy and have discontinued due to intolerance.
6. Participants on corticosteroids must be at a stable dose for ≥ 4 weeks before the screening visit; the prednisone-equivalent dose cannot exceed 0.5mg/kg body weight and will be tapered during the study to 10mg or less by week 20 at the discretion of the investigators.
7. SLEDAI-2K ≥6 at the time of screening
8. Non-pregnant (via negative pregnancy test)/non-breast-feeding women and women with no intention to become pregnant/to breast-feed during the term of the trial, both women and men should commit to use a proper contraceptive
9. Participant able to provide written informed consent
10. Must be able to adhere to the study visit schedule and other protocol requirements.

Exclusion Criteria:

1. History of any non-systemic lupus erythematosus (non-SLE) disease that required treatment with oral or parenteral corticosteroids for more than a total of 2 weeks within the 3 months preceding informed consent to participate in the study.
2. History of dialysis within 12 months prior to signing the ICF or expected need for renal replacement therapy (dialysis or renal transplant) within a six-month period after enrollment
3. Concurrent enrollment in another interventional lupus clinical study within the predicted washout time of the investigational product prior to signing ICF
4. Receipt of any commercially available biologic agent within the washout period described above prior to signing the ICF
5. Receipt of IV pulse corticosteroid within 6 months prior to signing the ICF
6. Previous treatment with any type of cellular therapy e.g., Tregs or CAR-T cells
7. Major surgery within 3 months prior to signing the ICF or major surgery planned during the study period
8. Has other inflammatory diseases that might confound the evaluations of safety, including but not limited to rheumatoid arthritis (RA), psoriatic arthritis (PsA), RA/lupus overlap, psoriasis, Crohn's disease, or active Lyme disease
9. Confirmed positive test for active hepatitis B, hepatitis C, HIV or TB.
10. History of cancer, apart from squamous or basal cell carcinoma of the skin and cervical carcinoma in situ
11. Any other comorbidity which may render the participant unfit for study participation according to the investigator's judgement.

Primary outcomes:
measure: Incidence of Treatment-Emergent Adverse Events
timeFrame: day 1 to day 168
Secondary outcomes:
measure: Change from baseline in the SLEDAI 2K following administration
timeFrame: day 1 to day 168
measure: Change from baseline in the BILAG Index score following administration
timeFrame: day 1 to day 168
measure: Efficacy by prednisone-equivalent corticosteroid doses change at each visit
timeFrame: day 1 to day 168
Other outcomes:
measure: A comparative analysis of pre-treatment versus post-treatment levels of Anti-ENA (Extractable Nuclear Antigens)
timeFrame: day 1 to day 168
measure: A comparative analysis of pre-treatment versus post-treatment levels of Anti-dsDNA Antibodies
timeFrame: day 1 to day 168
measure: A comparative analysis of pre-treatment versus post-treatment levels of Total Serum IgG Levels
timeFrame: day 1 to day 168
measure: A comparative analysis of pre-treatment versus post-treatment results of CBC
timeFrame: day 1 to day 168
measure: A comparative analysis of pre-treatment versus post-treatment levels of Anti-nucleosome antibodies
timeFrame: day 1 to day 168
measure: A comparative analysis of pre-treatment versus post-treatment levels of Erythrocyte sedimentation rate
timeFrame: day 1 to day 168
measure: A comparative analysis of pre-treatment versus post-treatment levels of CRP
timeFrame: day 1 to day 168
measure: A comparative analysis of pre-treatment versus post-treatment levels of Cytokines
timeFrame: day 1 to day 168
measure: A comparative analysis of pre-treatment versus post-treatment Protein/Creatinine ratio in spot urine
timeFrame: day 1 to day 168
measure: A comparative analysis of pre-treatment versus post-treatment 24-hour urine protein levels
timeFrame: day 1 to day 168
Locations:
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
Study officials:
References: