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

Anemia, Aplastic Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT00399971
• Other study ID #: AAFocus
• Status: Recruiting
• Phase: Phase 1/Phase 2
• First received: November 14, 2006
• Last updated: November 7, 2008
• Start date: November 2002
• Est. completion date: December 2009

Study information

• Verified date: November 2008
• Source: Shenzhen Zhongxing Yangfan Biotech Co. Ltd.
• Contact: Demao Yang, PhD
• Phone: 86 755 2603 1580
• Email: dyang[@]yangfan-biotech.com
• Is FDA regulated: No
• Health authority: China: Ethics Committee
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 100
• Est. completion date: December 2009
• Est. primary completion date: June 2009
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 10 Years to 80 Years

Eligibility

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

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic

Intervention

Procedure:
• Ex Vivo Immunotherapy
Allogeneic and autologous ex vivo activated immune cells are given intravenously at least once a week

Drug:
• Ex vivo immunotherapy
i.v. infusions, once a week, at least 6 to 12 months

Locations

Country	Name	City	State
China	Department of Hematology, Liu Hua Qiao Hospital	Guangzhou	Guangdong

Sponsors (3)

Lead Sponsor	Collaborator
Shenzhen Zhongxing Yangfan Biotech Co. Ltd.	Liu Hua Qiao Hospital, The 12th Guangzhou Municipal Hospital

Country where clinical trial is conducted

China, 

References & Publications (2)

Chen J, Liu W, Wang X, Chen H, Wu J, Yang Y, Wu L, Yang D. Ex vivo immunotherapy for patients with benzene-induced aplastic anemia. J Hematother Stem Cell Res. 2003 Oct;12(5):505-14. — View Citation

Li G, Wang X, Wu L, Zhang W, Chen H, Xie Y, Yang D. Ex vivo activated immune cells promote survival and stimulate multilineage hematopoietic recovery in myelosuppressed mice. J Immunother. 2005 Jul-Aug;28(4):420-5. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Survival		2 years	No
Secondary	Hematological Parameters		2 years	No
Secondary	Hematopoietic Activities in Bone Marrow		2 years	No
Secondary	Quality of Life		2 years	No