Phase I/II Study of An Ex Vivo Immunotherapy for Treatment of Idiopathic Aplastic Anemia
Trial Snapshot
- Phase
- Phase 1
- Sponsor
- Enrollment
- 100
- Locations
- 1
- Primary Endpoint
- Survival
Study Overview
Brief Summary
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
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.
Study Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 10 Years to 80 Years (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Arms & Interventions
Hemathera
Patients will receive cell-based immunotherapy.
Intervention: Ex vivo immunotherapy (Drug)
Outcomes
Primary Outcomes
Survival
Time Frame: 2 years
Secondary Outcomes
- Hematological Parameters(2 years)
- Hematopoietic Activities in Bone Marrow(2 years)
- Quality of Life(2 years)