Mesenchymal Stem Cells Infusion in Patients With Autoimmune Diseases

NCT06888973 | Enrolling By Invitation Phase 1 DMC

• 1 other identifier: NIBMT-MSC-autoimmune disease
• Study Type: interventional
• Target: 200
• Locations: 1 country
• Sites: 2

Brief Summary

The goal of this study is to learn if mesenchymal stem cell therapy (treatment group) can effectively treat autoimmune diseases, when compared to normal saline (given to placebo group). The primary outcome measures will be clinical improvement based on the respective disease specific clinical scores, normalization of T-lymphocyte subsets and \> 50% reduction in disease specific antibody titres. The study will also document the type and frequency of any adverse event or side effects, reported by or seen in any of the trial participants. Patients in treatment group will receive single session of MSC therapy and placebo group will receive 0.9% saline solution. The participants will be followed at 3 and 6 months.

Conditions

Rheumatoid Arthritis; Systemic Lupus Erythematosus (SLE); Systemic Sclerosis; Ankylosing Spondylitis (AS); Juvenile Idiopathic Arthritis; Polymyositis/Dermatomyositis

Keywords

Mesenchymal stem cells; Autoimmune diseases; Rheumatoid arthritis; Systemic Lupus Erythematosus (SLE); Systemic Sclerosis; Ankylosing Spondylitis (AS); Juvenile Idiopathic Arthritis; Polymyositis/dermatomyositis

Outcome Measures

Primary Outcomes (11)

• Primary Outcome Measures for RA

  Change From Baseline in the Disease Activity Score- Erythrocyte Sedimentation Rate (DAS28-ESR) at Week 48 The DAS28-ESR is a score on a scale (0 to 10) that is a measure of the subject's disease activity. It is based on the tender joint count (28 joints), swollen joint count (28 joints), patient's global assessment of disease activity (mm), and ESR (mm/hour). Lower score indicates less disease activity. Flares in disease activity are defined as an increase in this score of greater than 1.2 and remission is defined as achieving a DAS28-ESR score of less than 2.6.

  Day 0, Weeks 12, 24, 48

• Primary Outcome Measures for RA

  Change From Baseline in Tender Joint Count Score at Week 48 Tender Joint Count (TJC) is calculated based on tenderness response of 28 joints. TJC possible values range from 0 to 28. A lower TJC indicates less joint tenderness. Change from baseline is computed as Week 48 value minus baseline value. A negative value in change from baseline indicates an improvement.

  Day 0, Weeks 12, 24, 48

• Primary Outcome Measures for RA

  Change From Baseline in Swollen Joint Count at Week 48 Swollen Joint Count (SJC) is calculated based on swelling response of 28 joints. SJC possible values range from 0 to 28. A lower SJC indicates less joint swelling. Change from baseline is computed as Week 48 value minus baseline value. A negative value in change from baseline indicates an improvement

  Day 0, Weeks 12, 24, 48

• Primary Outcome Measures for RA

  Change From Baseline in Patient's Assessment of Arthritis Pain-Visual Analog Scale (PAAP-VAS) at Week 48 Change from Baseline in PAAP-VAS (0 to 100 millimeters visual analog scale, 0 being no pain and 100 being most severe pain) is computed as the value at Week 48 minus the Baseline value. A negative value in change from Baseline indicates an improvement.

  Day 0, Weeks 12, 24, 48

• Primary Outcome Measures for RA

  Change From Baseline in Patient's Global Assessment of Disease Activity- Visual Analog Scale (PtGADA-VAS) at Week 48 Change from Baseline in PtGADA-VAS (0 to 100 mm visual analog scale, 0 being no symptoms and 100 being severe symptoms) is computed as the value at Week 48 minus the Baseline value. A negative value in change from Baseline indicates an improvement.

  Day 0, Weeks 12, 24, 48

• Primary Outcome Measures for RA

  Change From Baseline in Physician's Global Assessment of Patient's Disease Activity- Visual Analog Scale (PhGADA-VAS) at Week 48

  Day 0, Weeks 12, 24, 48

• Primary Outcome Measures for Systemic lupus erythematosus

  Proportion of patients achieving BILAG-based Composite Lupus Assessment (BICLA)

  Day 0, Weeks 12, 24, 48

• Primary Outcome Measures for systemic sclerosis

  Change From Baseline in Modified Rodnan Skin Score (mRSS) at Week 48 Skin thickness was assessed by the mRSS. The mRSS was rated with scores ranging from 0 (normal) to 3 (severe skin thickening) across 17 different sites. The total score was the sum of the individual skin scores in the 17 body areas (e.g., face, hands, fingers; proximal area of the arms, distal area of the arms, thorax, abdomen; proximal area of the legs, and distal area of the legs, feet), giving a range of 0-51 units and had been validated for participants with systemic sclerosis (SSc). A negative change from baseline showed improvement.

  Day 0, Weeks 12, 24, 48

• Primary Outcome Measures for systemic sclerosis

  Percentage of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

  Day 0, Weeks 12, 24, 48

• Primary outcome measures in ankylosing spondlitis

  The Proportion of Participants Who Achieve an ASAS 20 Response (Assessment of SpondyloArthritis International Society Criteria) ASAS20 response is defined as an improvement of ≥20% and ≥1 units on a scale of 10 in at least three of the four ASAS main domains and no worsening of ≥20% and ≥1 unit on a scale of 10 in the remaining domain

  Week 16

• Primary Outcome Measure for dermatomyositis

  Comparison Between the Time to Improvement Between the Two Groups of Idiopathic Inflammatory Myopathy Patients at week 48 ASAS20 response is defined as an improvement of ≥20% and ≥1 units on a scale of 10 in at least three of the four ASAS main domains and no worsening of ≥20% and ≥1 unit on a scale of 10 in the remaining domain

  Day 0, Weeks 12, 24, 48

Secondary Outcomes (25)

• Secondary Outcome Measures for RA

  Day 0, Weeks 12, 24, 48

• Secondary Outcome Measures for RA

  Day 0, Weeks 12, 24, 48

• Secondary Outcome Measures for RA

  Day 0, Week 12, week 24, week48

• Secondary Outcome Measures for SLE

  Day 0, Weeks 12, 24, 48

• Secondary Outcome Measures for SLE

  Day 0, Weeks 12, 24, 48

Study Arms (2)

Treatment group

EXPERIMENTAL

MSC therapy group

Biological: Mesenchymal Stem Cells

Placebo group

PLACEBO COMPARATOR

Placebo (0.9% saline)

Other: Placebo

Interventions

Mesenchymal Stem Cells BIOLOGICAL

allogeneic bone marrow-derived mesenchymal stem cells therapy

Treatment group

Placebo OTHER

0.9% Saline as placebo

Placebo group

Eligibility Criteria

• Age: 18 Years - 50 Years
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• A- RA
• Years and older
• Diagnosis of RA, as defined by fulfilling 2010 American College of Rheumatology (ACR) criteria
• Positive for rheumatoid factor (RF) and/or anticyclic citrullinated peptide (CCP)
• The presence of arthritis symptoms for more than 6 weeks but less than 5 year
• Active RA, as defined as DAS ESR\>3.1 (as defined by moderate and sever RA )
• Willing to adhere to the study requirements
• Willing to use acceptable effective forms of contraception

You may not qualify if:

• Allergy to methotrexate (MTX)
• Previous exposure to anti-CD20 monoclonal antibody (mAb) or other type(s) of mAb therapy
• Receipt of intra-articular injections within 4 weeks prior to study entry
• Unwilling to stop drinking alcohol (ETOH)
• History of alcohol or substance abuse
• Active infection, or chronic or persistent infection that might worsen with immunosuppressive treatment (e.g., Human Immunodeficiency Virus \[HIV\], hepatitis B virus \[HBV\], hepatitis C virus \[HCV\], tuberculosis \[TB\])
• Interstitial lung disease observed by chest x-ray \[chest radiograph\]
• Known coronary artery disease or significant cardiac arrhythmias or severe congestive heart failure (New York Heart Association \[NYHA\] classes III or IV)
• Definitive diagnosis of another autoimmune rheumatologic disease (e.g., systemic lupus erythematosus \[SLE\], scleroderma, primary Sjögren's syndrome, primary vasculitis)
• History of immunoglobulin E (IgE)-mediated or non-IgE-mediated hypersensitivity or known anaphylaxis to mouse proteins
• History of cancer. Exception: participants with previous resected basal or squamous cell carcinoma, treated cervical dysplasia, or treated in situ Grade I cervical cancer within 5 years prior to study entry are not excluded from study eligibility
• History of positive purified protein derivative (PPD) test (i.e., positive tuberculosis \[TB\] test or mantoux test) without treatment for TB infection or chemoprophylaxis for TB exposure
• Live vaccine within 3 months of study entry
• Any psychiatric disorder that would prevent a participant from providing informed consent
• Pregnancy or breastfeeding women.

Sponsors & Collaborators

• National Institute of Blood and Marrow Transplant (NIBMT), Pakistan: lead
• Pak Emirates Military Hospital: collaborator

Study Sites (2)

Armed Forces Bone Marrow Transplant Centre/ National Institute of Blood and Marrow Transplant (AFBMTC/NIBMT)

Rawalpindi, Pakistan

Location

Pak Emirates Military Hospital

Rawalpindi, Pakistan

Location

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MeSH Terms

Conditions

Arthritis, Rheumatoid; Lupus Erythematosus, Systemic; Scleroderma, Systemic; Spondylitis, Ankylosing; Arthritis, Juvenile; Dermatomyositis; Autoimmune Diseases

Condition Hierarchy (Ancestors)

Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Immune System Diseases; Skin Diseases; Axial Spondyloarthritis; Spondylarthropathies; Spondylarthritis; Spondylitis; Spinal Diseases; Bone Diseases; Ankylosis; Polymyositis; Myositis; Muscular Diseases; Neuromuscular Diseases; Nervous System Diseases

Study Officials

• Memoona Haider, MBBS, FCPS

  National University of Medical Sciences

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Model Details: Double-Blind, Randomized, Placebo-Controlled Trial

Study Record Dates

• First Submitted: January 14, 2025
• First Posted: March 21, 2025
• Study Start: February 1, 2025
• Primary Completion: February 1, 2025
• Study Completion: February 1, 2026
• Last Updated: March 21, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

PA (2)