Clinical Application of Stem Cell Educator Therapy in Alopecia Areata

Study Overview
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Brief Summary
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Alopecia areata (AA) is a common autoimmune disease that results in loss of body hair in varying degrees. The condition is estimated to affect more than 6.8 million people in the United States alone (naaf.org), with a worldwide prevalence of 0.1% to 0.2% and calculated lifetime risk of 2%. AA is the most common form of the disease, in which areas of complete hair loss arise within normal hair-bearing skin. Other forms include alopecia totalis (AT), characterized by total loss of scalp hair, and alopecia universalis (AU), characterized by complete loss of body hair. AA and its variants can have devastating effects on patients' quality of life and social functioning. At present, curative therapy for AA does not exist. Therapeutic options are currently very limited, such as intralesional injections of glucocorticoids and induction of allergic contact dermatitis. These therapies are not effective for many patients and are generally impractical for patients with diffuse AA, AT or AU. Recently, Janus kinase (JAK) inhibitors were effective for the treatment of severe AA. However, for those patients who do respond, relapses are common after discontinuation of treatment, due to the existing of autoimmune memory T cells. Stem Cell Educator (SCE) therapy, which uses only autologous mononuclear cells that are externally exposed to cord blood stem cells, has previously been proven safe and effective in subjects for the improvement of type 1 diabetes (T1D), T2D and other autoimmune diseases such as alopecia areata. Minoxidil is the FDA approved drug for the treatment of androgenetic alopecia (AGA) in 1988. This trial will explore the therapeutic potential of Stem Cell Educator therapy for the treatment of AA by using topical minoxidil as control.

Official Title
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Clinical Treatment of Alopecia Areata With Stem Cell Educator Therapy

Conditions
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Alopecia Areata, Alopecia Totalis, Alopecia Universalis

Intervention / Treatment
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* Combination Product: Stem Cell Educator therapy


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Adult patients ( 18 years) Must have a clinical diagnosis of AA, at least 50% hair loss involving the scalp For cases in which there is 80% or more scalp hair loss, the duration of the severity of hair loss must be 10 years or less Stable or worsening hair loss for at least 6 months without evidence of hair regrowth Patients must not have received any treatments known to affect AA within 2 months of screening Patients must agree that they are not permitted to use any other treatment besides topical minoxidil known to affect AA during a period of 6 months after undergoing SCE therapy Adequate venous access for apheresis Ability to provide informed consent For female patients only, willingness to use FDA-recommended birth control (http://www.fda.gov/downloads/ForConsumers/ByAudience/ForWomen/FreePublications/UCM356451.pdf) until 6 months post treatment. Must agree to comply with all study requirements and be willing to complete all study visits Exclusion Criteria: AST or ALT 2 > x upper limit of normal. Abnormal bilirubin (total bilirubin > 1.2 mg/dL, direct bilirubin > 0.4 mg/dL) Creatinine > 2.0 mg/dl. Known coronary artery disease or EKG suggestive of coronary artery disease unless cardiac clearance for apheresis is obtained from a cardiologist. Known active infection such as Hepatitis B, Hepatitis C, or Human Immunodeficiency Virus (HIV) Pregnancy assessed by a positive serum pregnancy test or breastfeeding mothers Use of immunosuppressive medication within one month of enrollment including but not limited to prednisone, cyclosporine, tacrolimus, sirolimus, and chemotherapy. Presence of any other autoimmune diseases (lupus, rheumatoid arthritis, scleroderma, etc.) Anticoagulation other than ASA. Hemoglobin < 10 g/dl or platelets < 100 k/ml Is unable or unwilling to provide informed consent Presence of any other physical or psychological medical condition that, in the opinion of the investigator, would preclude participation Significant cardiovascular diseases that would make use of oral minoxidil inappropriate.

Ages Eligible for Study
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Minimum Age: 18 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
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: AA subjects will receive the treatment with Stem Cell Educator therapy
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Hair regrowth by SCE<br>AA subjects will receive Stem Cell Educator therapy. Hair regrowth will be evaluated during six-month follow-up studies. | Combination Product: Stem Cell Educator therapy<br>* AA subjects will be recruited and followed by the treatment with SCE therapy.<br>|
| Experimental: Minoxidil therapy<br>Control subjects will receive treatment with topical 5% minoxidil | Combination Product: Stem Cell Educator therapy<br>* AA subjects will be recruited and followed by the treatment with SCE therapy.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The percentage change in scalp hair growth. | The primary endpoint was the percentage change in scalp hair growth, measured with the Severity of Alopecia Tool (SALT) score. | Hair regrowth will be evaluated at different time points post receiving Stem Cell Educator therapy in 1, 3, and 6 months. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Feasibility of SCE therapy | The feasibility will be determined by the number of patients who were unable to complete SCE Therapy. | 6 months | 
| Preliminary efficacy of SCE therapy | This will be determined by the duration of maintaining hair growth following SCE therapy. | 6 months | 
| Efficacy of modulation of autoimmune-related memory T-cell markers | Measurements of immune markers' changes will be preformed by flow cytometry such as CD8+CD45RO+CCR7- effector memory T cells. Peripheral blood mononuclear cells (PBMC) will be collected at 1, 3, 6 month post the SCE therapy. | 6 months |