Safety and Efficacy of Patient's Own AD-MSC and AD-HSC Transplantation in Patients With Severe Aplastic Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 13 April 2015
• Main ID: NCT02407470
• Date of registration: 25/03/2015
• Prospective Registration: No
• Primary sponsor: Navy General Hospital, Beijing
• Public title: Safety and Efficacy of Patient's Own AD-MSC and AD-HSC Transplantation in Patients With Severe Aplastic Anemia
• Scientific title: A Multicenter, Randomized, Controlled Study of the Efficacy and Safety of the Combination of Adipose Tissue-derived Hematopoietic Stem Cells (AD-HSCs) and ATG in the Treatment of Severe Aplastic Anemia
• Date of first enrolment: January 2015
• Target sample size: 90
• Recruitment status: Recruiting
• URL: http://clinicaltrials.gov/show/NCT02407470
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1/Phase 2

Countries of recruitment

China

Contacts

• Name: james Q Yin, M.D.,Ph.D.
• Affiliation: The military general hospital of Beijing

• Name: Jianliang Shen, M.D.,Ph.D

• Name: James Q Yin, M.D.,Ph.D.

Key inclusion & exclusion criteria

Inclusion Criteria:

Male or female recipients must have histopathologically confirmed diagnosis of SAA-I
without or with more than 6 months after less than one treatment with ATG. Diagnostic
Criteria for Server Aplastic Anemia will be based on the definitions set forth by the
international Aplastic Anemia Study Group.

At least two of the following:

Absolute neutrophil count = 0.5 X 109/l, Platelet count = 20 X 109 /l, Anemia with
corrected reticulocyte count = 1%, and Bone marrow cellularity = 25%, or bone marrow
cellularity = 50% with fewer than 30% hematopoietic cell, Hepatic: alanine
aminotransferase (ALT)/ aspartate aminotransferase (AST) no greater than 4 times normal,
Bilirubin: no greater than 2 mg/dl, Renal: Creatinine clearance at least 50 ml/min,
Cardiovascular: Shortening fraction or ejection fraction at least 40% of normal for age by
echocardiogram or radionuclide scan.

No clinically significant comorbid illnesses (e.g., myocardial infarction or
cerebrovascular accident).

Exclusion Criteria:

Active and uncontrolled infection, Active bleeding, Severe allergic history of ATG, HIV-1
infection, Pregnancy or breastfeeding, Carbon monoxide lung diffusion capacity (DLCO) <40%
predicted, SAA-II, Patients with severe psychological disorders, Recipients of other
clinical trials.



Age minimum: 14 Years
Age maximum: 70 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Severe Aplastic Anemia

Intervention(s)

Drug: Rabbit antithymoglobulin (ATG)

Procedure: AD-MSC transdifferentiated HSCs (AD-HSCs)

Procedure: Adipose derived mesenchymal stem cells ( AD-MSCs)

Primary Outcome(s)

Engraftment at 42 days post AD-HSC transplantation for patients with severe aplastic anemia. [Time Frame: 42 days posttransplant]

Secondary Outcome(s)

Relapse [Time Frame: 1 year post transplant]

Evaluation of the occurrence of secondary malignancies [Time Frame: 6 months post transplant]

To estimate the overall survival (OS) at 1 year following AD-HSC transplantation for Patients with Severe Aplastic Anemia [Time Frame: 1 year]

Transfusional requirements [Time Frame: weekly untill 6 months]

Hematology labs [Time Frame: 12 weeks]

To assess treatment related mortality [Time Frame: 12 months]

Incidence of chronic graft-versus-host disease [Time Frame: 6 months]

Number of participants with adverse events as a measure of safety and tolerability of intravenous AD-HSC infusion in patients with severe aplastic anemia [Time Frame: weekly untill 12 months]

Secondary ID(s)

Ginkgocell-ADHSC-AA-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Peking Union Medical College Hospital

Chinese Academy of Medical Sciences

General Hospital of Beijing PLA Military Region