Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia

Aplastic Anemia Clinical Trial

Official title:

Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT01995331
• Other study ID #: YL20102601
• Status: Active, not recruiting
• Phase: Phase 4
• First received: November 21, 2013
• Last updated: November 21, 2013
• Start date: March 2012
• Est. completion date: March 2015

Study information

• Verified date: November 2013
• Source: Chinese Academy of Medical Sciences
• Contact: n/a
• Is FDA regulated: No
• Health authority: China: Ministry of Health
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 30
• Est. completion date: March 2015
• Est. primary completion date: March 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 1 Year to 18 Years

Eligibility

Inclusion Criteria:

• Acquired Childhood Severe Aplastic Anemia (SAA)

Exclusion Criteria:

• not Childhood and Acquired Severe Aplastic Anemia

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
• Aplastic Anemia

Intervention

Drug:
• cyclophosphamide,cyclosporine A
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.

Locations

Country	Name	City	State
China	Department of Pediatrics,Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College	Tianjin	Tianjin

Sponsors (1)

Lead Sponsor	Collaborator
Xiaofan Zhu

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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).	36 months	Yes
Secondary	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.	36 months	Yes