Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia

NCT01995331 | Unknown Phase 4 DMC

• 1 other identifier: YL20102601
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

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.

Conditions

Aplastic Anemia

Keywords

aplastic anemia;; cyclophosphamide;; immunosuppressive therapy;; cyclosporine A

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (1)

• The side effect of Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia

  36 months

Study Arms (1)

moderate-dose cyclophosphomide

OTHER

Drug: cyclophosphamide,cyclosporine A

Interventions

cyclophosphamide,cyclosporine A DRUG

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.

Also known as: Drug,Mesnaum

moderate-dose cyclophosphomide

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Acquired Childhood Severe Aplastic Anemia (SAA)

You may not qualify if:

• not Childhood and Acquired Severe Aplastic Anemia

Sponsors & Collaborators

• Xiaofan Zhu: lead

Study Sites (1)

Department of Pediatrics,Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College

Tianjin, Tianjin Municipality, 300020, China

Location

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

Cyclophosphamide; Cyclosporine

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins

Study Officials

• Xiaifan Zhu, MD

  Department of Pediatrics, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: UNKNOWN
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: chief physician

Study Record Dates

• First Submitted: November 21, 2013
• First Posted: November 26, 2013
• Study Start: March 1, 2012
• Primary Completion: March 1, 2015
• Study Completion: March 1, 2015
• Last Updated: November 26, 2013

Record last verified: 2013-11

Locations

CN (1)