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Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Xiaofan Zhu, Chinese Academy of Medical Sciences
ClinicalTrials.gov Identifier:
NCT01995331
First received: November 21, 2013
Last updated: NA
Last verified: November 2013
History: No changes posted
  Purpose

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.


Condition Intervention Phase
Aplastic Anemia
Drug: cyclophosphamide,cyclosporine A
Phase 4

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia

Resource links provided by NLM:


Further study details as provided by Chinese Academy of Medical Sciences:

Primary Outcome Measures:
  • The reponse of Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia [ Time Frame: 36 months ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: 36 months ] [ Designated as safety issue: Yes ]

    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.



Estimated Enrollment: 30
Study Start Date: March 2012
Estimated Study Completion Date: March 2015
Estimated Primary Completion Date: March 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
moderate-dose cyclophosphomide 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.

Other Name: Drug,Mesnaum

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.

  Eligibility

Ages Eligible for Study:   1 Year to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Acquired Childhood Severe Aplastic Anemia (SAA)

Exclusion Criteria:

  • not Childhood and Acquired Severe Aplastic Anemia
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01995331

Locations
China, Tianjin
Department of Pediatrics,Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
Tianjin, Tianjin, China, 300020
Sponsors and Collaborators
Xiaofan Zhu
Investigators
Principal Investigator: Xiaifan Zhu, MD Department of Pediatrics, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences
  More Information

No publications provided

Responsible Party: Xiaofan Zhu, chief physician, Chinese Academy of Medical Sciences
ClinicalTrials.gov Identifier: NCT01995331     History of Changes
Other Study ID Numbers: YL20102601
Study First Received: November 21, 2013
Last Updated: November 21, 2013
Health Authority: China: Ministry of Health

Keywords provided by Chinese Academy of Medical Sciences:
aplastic anemia;
cyclophosphamide;
immunosuppressive therapy;
cyclosporine A

Additional relevant MeSH terms:
Anemia
Anemia, Aplastic
Bone Marrow Diseases
Hematologic Diseases
Cyclophosphamide
Cyclosporine
Cyclosporins
Alkylating Agents
Anti-Infective Agents
Antifungal Agents
Antineoplastic Agents
Antineoplastic Agents, Alkylating
Antirheumatic Agents
Dermatologic Agents
Enzyme Inhibitors
Immunologic Factors
Immunosuppressive Agents
Molecular Mechanisms of Pharmacological Action
Myeloablative Agonists
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses

ClinicalTrials.gov processed this record on November 20, 2014