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Reduced Toxicity Fludarabine (Flu) + Cyclophosphamide (CPM) + Rabbit Antithymocyte Globulin (rATG) Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia (SAA)

This study is currently recruiting participants.
Verified March 2012 by The Korean Society of Pediatric Hematology Oncology
Sponsor:
Information provided by:
The Korean Society of Pediatric Hematology Oncology
ClinicalTrials.gov Identifier:
NCT00882323
First received: April 15, 2009
Last updated: March 23, 2012
Last verified: March 2012
History of Changes

April 15, 2009
March 23, 2012
November 2008
October 2012   (final data collection date for primary outcome measure)
To evaluate engraftment potential of reduced toxicity fludarabine, cyclophosphamide plus thymoglobulin conditioning regimen for unrelated bone marrow transplantation in severe aplastic anemia. [ Time Frame: From Nov. 2008 to Oct. 2012 ] [ Designated as safety issue: No ]
Same as current
Complete list of historical versions of study NCT00882323 on ClinicalTrials.gov Archive Site
  • To evaluate toxicities of reduced toxicity fludarabine, cyclophosphamide plus thymoglobulin conditioning regimen for UBMT/PBSCT in SAA. [ Time Frame: From Nov. 2008 to Oct. 2012 ] [ Designated as safety issue: No ]
  • To evaluate overall and EFS rate after UBMT/PBSCT. [ Time Frame: From Nov. 2008 to Oct. 2012 ] [ Designated as safety issue: No ]
  • To evaluate GVHD and immunologic recovery after UBMT/PBSCT. [ Time Frame: From Nov. 2008 to Oct. 2012 ] [ Designated as safety issue: No ]
Same as current
Not Provided
Not Provided
 
Reduced Toxicity Fludarabine (Flu) + Cyclophosphamide (CPM) + Rabbit Antithymocyte Globulin (rATG) Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia (SAA)
Reduced Toxicity Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia

Anti-thymocyte globulin (ATG) has been used in severe aplastic anemia as a part of the conditioning regimen. Among the many kinds of ATG preparations, thymoglobulin had been found to be more effective in preventing graft versus host disease (GVHD) and rejection of organ transplants. As the fludarabine based conditioning regimens without total body irradiation have been reported to be promising for transplantation from alternative donors in SAA, thymoglobulin was added to fludarabine and cyclophosphamide conditioning to reduce GVHD and to allow good engraftment in unrelated donor transplantation. Our previous phase II study of fludarabine, cyclophosphamide plus thymoglobulin conditioning resulted in good engraftment (100%) and survival rate (74%). But grade III/IV toxicities occurred in 25% of patients and all events were treatment related mortalities. As cyclophosphamide is more toxic agent than fludarabine, we plan a new phase II study re; 'reduced toxicity fludarabine, cyclophosphamide plus thymoglobulin conditioning regimen for unrelated donor transplantation in severe aplastic anemia' by reducing dosage of cyclophosphamide and increasing dosage of fludarabine.
Not Provided
Interventional
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Aplastic Anemia
Drug: Cyclophosphamide, Fludarabine, Thymoglobulin

cyclophosphamide (60 mg/kg once daily i.v. on days -8, -7)

fludarabine (40 mg/m2 once daily i.v. on days -6, -5, -4, -3, -2)

thymoglobulin (2.5 mg/kg once daily i.v. on days -4, -3, -2)
Experimental: Fludarabine
Intervention: Drug: Cyclophosphamide, Fludarabine, Thymoglobulin
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
33
October 2012
October 2012   (final data collection date for primary outcome measure)

Inclusion Criteria:

  1. Diagnosis of severe aplastic anemia defined by any two or three peripheral blood criteria and either marrow criterion.

    • Peripheral blood

      1. Neutrophils < 0.5 x 109/l
      2. Platelets < 20 x 109/l
      3. Corrected reticulocytes < 1%

    • Bone marrow

      1. Severe hypocellularity (< 25%)
      2. Moderate hypocellularity (25-30%) with hematopoietic cells representing < 30% of residual cells
  2. No prior hematopoietic stem cell transplantation.
  3. Age: no limits.
  4. Performance status: ECOG 0-2.

  5. Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases:

    • Heart: a shortening fraction > 30% and ejection fraction > 45%.
    • Liver: total bilirubin < 2 × upper limit of normal; ALT < 3 × upper
    • Kidney: creatinine <2 × normal or a creatinine clearance (GFR) > 60 ml/min/1.73m2.
  6. Patients must lack any active viral infections or active fungal infection.
  7. Appropriate donor is available: Matched in 6/6 of A, B, DR loci.
  8. Patients (or one of parents if patients age < 19) should sign informed consent.

Exclusion Criteria:

  1. Pregnant or nursing women.
  2. Malignant or nonmalignant illness that is uncontrolled or whose control may be jeopardized by complications of study therapy.
  3. Psychiatric disorder that would preclude compliance.
  4. Congenital aplastic anemia including Fanconi anemia.
  5. Manipulated bone marrow.
Both
1 Year to 21 Years
No
Contact: Hyoung Jin Kang, M.D, Ph.D 82 2 2072 3304 kanghj@snu.ac.kr
Contact: Ji Won Lee, M.D 82 2 2072 0177 agnesjw@hanmil.net
Korea, Republic of
 
NCT00882323
KSPHO-SCT 0802
Yes
The Korean Society of Hematology, The Korean Society of Pediatric of Hematology Oncology
The Korean Society of Pediatric Hematology Oncology
Not Provided
Principal Investigator: Hyo seop Ahn, M.D, Ph. D The Korean Society of Pediatric Hematology Oncology
The Korean Society of Pediatric Hematology Oncology
March 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP