Flu+CPM+rATG Conditioning Regimes for Unrelated Bone Marrow Transplantation (UBMT)(or Mobilized Peripheral Blood)in Severe Aplastic Anemia (SAA)

The recruitment status of this study is unknown because the information has not been verified recently.
Verified March 2012 by The Korean Society of Pediatric Hematology Oncology.
Recruitment status was  Active, not recruiting
Sponsor:
Information provided by:
The Korean Society of Pediatric Hematology Oncology
ClinicalTrials.gov Identifier:
NCT00737685
First received: August 18, 2008
Last updated: March 23, 2012
Last verified: March 2012
  Purpose

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 GVHD and rejection of organ transplants. As the fludarabine based conditioning regimens without total body irradiation have been reported to be promising for BMT/PBSCT from alternative donors in SAA, thymoglobulin was added to fludarabine and cyclophosphamide conditioning to reduce GVHD and to allow good engraftment in UBMT/UPBSCT.


Condition Intervention Phase
Anemia, Aplastic
Drug: cyclophosphamide, fludarabine , thymoglobulin
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Bone Marrow (or Mobilized Peripheral Blood) Transplantation in Severe Aplastic Anemia

Resource links provided by NLM:


Further study details as provided by The Korean Society of Pediatric Hematology Oncology:

Primary Outcome Measures:
  • To evaluate the engraftment potential, incidence and severity of acute graft versus host disease,toxicity of conditioning regimen for UBMT in SAA. [ Time Frame: From Jan. 1. 2006 to Dec. 31. 2008. For 3 years. ] [ Designated as safety issue: No ]
  • To evaluate overall and EFS follow-up of 1 year after UBMT/PBSCT. [ Time Frame: From Jan. 1. 2006 to Dec. 31. 2008. For 3 years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • To evaluate chronic GVHD and immunologic recovery after UBMT/PBSCT. and the efficacy of UBMT/PBSCT before immuno-suppressive therapy with anti-thymocyte globulin in severe aplastic anemia and long term toxicity of non-TBI based conditioning [ Time Frame: From Jan. 1. 2006 to Dec. 31. 2008. For 3 years. ] [ Designated as safety issue: No ]

Estimated Enrollment: 30
Study Start Date: January 2006
Estimated Study Completion Date: December 2012
Estimated Primary Completion Date: August 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Fludarabine Drug: cyclophosphamide, fludarabine , thymoglobulin
cyclophosphamide (50 mg/kg once daily i.v. on days -9, -8, -7 & -6) fludarabine (30 mg/m2 once daily i.v. on days -5, -4, -3 & -2) thymoglobulin (2.5 mg/kg once daily i.v. on days -3, -2 & -1)

Detailed Description:

GVHD prophylaxis recommendation tacrolimus (0.03 mg/kg/day i.v. by continuous infusion from day -2 and taper with an oral form until 1 year after BMT/PBSCT) methotrexate (15 mg/m2 i.v. on days 1 and 10 mg/m2 i.v. on days 3, 6, 11)

  Eligibility

Ages Eligible for Study:   up to 25 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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
  • No prior hematopoietic stem cell transplantation.
  • Age: no limits.
  • Performance status: ECOG 0-2.
  • Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases.

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

Exclusion Criteria:

  • Pregnant or nursing women.
  • Malignant or nonmalignant illness that is uncontrolled or whose control may be jeopardized by complications of study therapy.
  • Psychiatric disorder that would preclude compliance.
  • Congenital aplastic anemia including Fanconi anemia.
  • Manipulated bone marrow.
  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: NCT00737685

Locations
Korea, Republic of
Seoul National University Hospital
Seoul, Korea, Republic of, 110-744
Sponsors and Collaborators
The Korean Society of Pediatric Hematology Oncology
Investigators
Principal Investigator: Hyo Seop Ahn, M.D, Ph.D The Korean Society of Pediatric Hematology Oncology
  More Information

No publications provided by The Korean Society of Pediatric Hematology Oncology

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Hyo Seop Ahn, Seoul Natioanl University Hospital
ClinicalTrials.gov Identifier: NCT00737685     History of Changes
Other Study ID Numbers: KSPHO-SCT0401
Study First Received: August 18, 2008
Last Updated: March 23, 2012
Health Authority: Korea: Food and Drug Administration

Keywords provided by The Korean Society of Pediatric Hematology Oncology:
Pediatric
UBMT
Severe

Additional relevant MeSH terms:
Anemia
Anemia, Aplastic
Hematologic Diseases
Bone Marrow Diseases
Cyclophosphamide
Fludarabine monophosphate
Fludarabine
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
Antirheumatic Agents
Therapeutic Uses
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Antimetabolites, Antineoplastic
Antimetabolites

ClinicalTrials.gov processed this record on July 26, 2014