Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia
Primary Purpose
Aplastic Anemia
Status
Unknown status
Phase
Phase 4
Locations
China
Study Type
Interventional
Intervention
cyclophosphamide,cyclosporine A
Sponsored by
About this trial
This is an interventional treatment trial for Aplastic Anemia focused on measuring aplastic anemia;, cyclophosphamide;, immunosuppressive therapy;, cyclosporine A
Eligibility Criteria
Inclusion Criteria:
- Acquired Childhood Severe Aplastic Anemia (SAA)
Exclusion Criteria:
- not Childhood and Acquired Severe Aplastic Anemia
Sites / Locations
- Department of Pediatrics,Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
moderate-dose cyclophosphomide
Arm Description
Outcomes
Primary Outcome Measures
The reponse of Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia
Complete response (CR) was defined as achieving normal levels of hemoglobin adjusted for age, platelet count >100×109/L, and ANC>1.5×109/L. Partial response (PR) was defined as transfusion independence, reticulocyte count >30×109/L, platelet count >30×109/L, and ANC >0.5×109/L above the baseline. Persistence of transfusion requirement or death was evidence of no response (NR).
Secondary Outcome Measures
The side effect of Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia
To report the death rate and the death cause , the infection rate and the pathogenic bacteria, especialy the fungal infection rate.
To report other toxicity.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01995331
Brief Title
Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia
Official Title
Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia
Study Type
Interventional
2. Study Status
Record Verification Date
November 2013
Overall Recruitment Status
Unknown status
Study Start Date
March 2012 (undefined)
Primary Completion Date
March 2015 (Anticipated)
Study Completion Date
March 2015 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Xiaofan Zhu
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
Severe aplastic anemia (SAA)is characterized by the depletion of hematopoietic precursors associated with life-threatening complications. High-dose cyclophosphamide has been found to yield a complete response (CR) in adults and children with SAA.However, the optimal dosage of cyclophosphamide for patients in childhood remains unclear. So we explore the ideal dosage of cyclophosphamide for the treatment of children with SAA.
Detailed Description
Tisdale et al. (2000,2002) attempted to compare immunosuppression using ATG/CSA with high-dose cyclophosphamide (50 mg/kg/d for 4 consecutive days) plus CSA in a randomized trial of newly diagnosed adults with SAA. Both groups received CSA as part of the treatment regimen. However, the trial was terminated prematurely due to excessive morbidity among the patients treated in the cyclophosphamide arm. They documented that invasive fungal infections were severe among the cyclophosphamide group. Between January 2008 through May 2009, in our department, nine pediatric patients with a diagnosis of SAA were enrolled a study with lower dose of cyclophosphamide with 30mg/kg/day for 4 consecutive days and combination with CSA, this study shows promise for children with severe aplastic anemia.Now we want explore the dosage of cyclophosphamide.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Aplastic Anemia
Keywords
aplastic anemia;, cyclophosphamide;, immunosuppressive therapy;, cyclosporine A
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
30 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
moderate-dose cyclophosphomide
Arm Type
Other
Intervention Type
Drug
Intervention Name(s)
cyclophosphamide,cyclosporine A
Other Intervention Name(s)
Drug,Mesnaum
Intervention Description
Drug,cyclophosphamide,cyclophosphamide (30 mg/kg/day) administered intravenously (IV) over 1 hr for 4 consecutive days Drug,cyclosporine A,5mg-12mg/kg.d,CSA was administered orally 40 days after the fourth dose of cyclophosphamide and maintained for 3 years. The dose of CSA was adjusted to maintain trough drug concentration above 150 μg/L and peak drug concentration above 300 μg/L.
Drug, human granulocyte colony-stimulating factor (rhG-CSF), 5 μg/kg/day subcutaneously starting 24 hrs after the fourth dose of cyclophosphamide, and it was withdrawed when ANC was >1×109/L.
Primary Outcome Measure Information:
Title
The reponse of Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia
Description
Complete response (CR) was defined as achieving normal levels of hemoglobin adjusted for age, platelet count >100×109/L, and ANC>1.5×109/L. Partial response (PR) was defined as transfusion independence, reticulocyte count >30×109/L, platelet count >30×109/L, and ANC >0.5×109/L above the baseline. Persistence of transfusion requirement or death was evidence of no response (NR).
Time Frame
36 months
Secondary Outcome Measure Information:
Title
The side effect of Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia
Description
To report the death rate and the death cause , the infection rate and the pathogenic bacteria, especialy the fungal infection rate.
To report other toxicity.
Time Frame
36 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Acquired Childhood Severe Aplastic Anemia (SAA)
Exclusion Criteria:
not Childhood and Acquired Severe Aplastic Anemia
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Xiaifan Zhu, MD
Organizational Affiliation
Department of Pediatrics, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences
Official's Role
Principal Investigator
Facility Information:
Facility Name
Department of Pediatrics,Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
City
Tianjin
State/Province
Tianjin
ZIP/Postal Code
300020
Country
China
12. IPD Sharing Statement
Learn more about this trial
Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia
We'll reach out to this number within 24 hrs