Safety and Efficacy Study of Ex Vivo Immunotherapy for Treatment of Aplastic Anemia

Study Overview
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Brief Summary
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Our hypothesis is that ex vivo activated immune cells would produce multiple known and unknown potent hematopoietic cytokines, working in concert, these cytokines help stem cell growth and differentiation. Additionally, these cells travel and home to bone marrow as well as spleen and liver involved in hematopoietic activities, where direct cell-cell contact may be beneficial.

Detailed Description
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Patients will be required to stay in our hospitals to receive the immunotherapy. Allogeneic peripheral blood mononuclear cells from healthy donors and the autologous peripheral mononuclear cells will alternately used for the treatment. Patients are expected to spend at least 6 to 12 months in the hospital and won't be released until they have improved significantly.

Official Title
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Phase I/II Study of An Ex Vivo Immunotherapy for Treatment of Idiopathic Aplastic Anemia

Conditions
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Anemia, Aplastic

Intervention / Treatment
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* Procedure: Ex Vivo Immunotherapy
* Drug: Ex vivo immunotherapy


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Clinical Diagnosis of Idiopathic Aplastic Anemia Not Eligible for Bone Marrow Transplantation Not Responsive to Conventional Immunosuppressive Therapy Exclusion Criteria: Leukemia or MDS or PNH or Fanconi's Disease pregnancy Allergic to Blood Product Severe Hypertension or Heart Disease Liver or Kidney Disease

Ages Eligible for Study
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Minimum Age: 10 Years
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
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Hemathera<br>Patients will receive cell-based immunotherapy. | Procedure: Ex Vivo Immunotherapy<br>* Allogeneic and autologous ex vivo activated immune cells are given intravenously at least once a week<br>* Other names: Aplastic Anemia, ex vivo Immunotherapy;Drug: Ex vivo immunotherapy<br>* i.v. infusions, once a week, at least 6 to 12 months<br>* Other names: Aplastic Anemia, ex vivo Immunotherapy;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Survival |  | 2 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hematological Parameters |  | 2 years | 
| Hematopoietic Activities in Bone Marrow |  | 2 years | 
| Quality of Life |  | 2 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Idiopathic Aplastic Anemia, Has Failed Conventional Immunosuppressive Therapy, Not Eligible For Bone Marrow Transplantation, C15.378.071.085