Clonal Fetal Mesenchymal Stem Cells (cfMSCs) for the Control of Immune-related Disorders

Study Overview
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Brief Summary
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The primary objectives are to evaluate the safety and efficacy of infusion of the third party fully-characterized clonally derived fetal MSCs (cfMSCs) for the control of severe symptoms associated with acute and chronic immune-related disorders and tissue damage.

Detailed Description
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MSCs have been extensively studied and clinically evaluated for the treatment of autoimmune diseases and graft versus host disease (GVHD) after hematopoietic stem cell transplantation (HSCT). The variable source of MSCs and the lack of consistency of primary tissue-derived MSCs are major obstacles to reliable translational applications of such therapeutic cell products. Fetal tissue-derived clonal MSCs (cfMSCs) have extended expansion potential and express rich levels of various growth factors, and thus can achieve quality consistency. Careful evaluation of fMSCs in clinical studies has not been conducted. Autoimmune diseases involve aberrant immune responses that harm tissues and organs. GVHD is a serious and often fatal problem associated with HSCT. MSCs have immunomodulatory and immunosuppressive effects. In many studies, MSCs have demonstrated promising beneficial effects that reduce severe autoimmune reactions, diminish symptoms of chronic GvHD and therapy-resistant acute GvHD including steroid-resistant GVHD. The safety and therapeutic effects of phenotype and functionally characterized fMSCs still require extensive clinical evaluation. This study aims to assess the safety and the potential beneficial effects of infusion of various dosages of third party fMSCs for the control of severe symptoms associated with acute and chronic immune-related disorders.

Official Title
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Clonal Fetal Mesenchymal Stem Cells (cfMSCs) for the Control of Immune-related Disorders

Conditions
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Immune Related Disorder, Tissue Damage

Intervention / Treatment
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* Biological: clonal fetal MSCs


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Informed consent. No available alternative treatment that can reduce the symptoms Patients are required to meet the following inclusion criteria: Any patient that has clinically documented abnormal immune or age-related disorders including acute and chronic GVHD. Patients may receive best available treatment for the control of disease symptoms. Patients with symptoms associated with genetic defects or infectious diseases are not eligible. Exclusion Criteria: Inability to give informed consent. Patients with ongoing infection or history of cancer. Patients with poor clinical conditions with the life expectancy of less than 14 days. Pregnancy.

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Single arm, cfMSC to treat immune disorders<br>cfMSCs treatment | Biological: clonal fetal MSCs<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 | Safety of cfMSC infusion acute and prolonged | up to one month | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of participants with reduced symptoms or stabilized conditions after treatment | Short term clinical effects measured by physiological and serological parameters related to the disease condition and symptoms using prepared study assessment table. | after 1 month from fMSC infusion | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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MSC, GVHDs, transplantation