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

Anemia, Aplastic Clinical Trial

Official title:

Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Bone Marrow (or Mobilized Peripheral Blood) Transplantation in Severe Aplastic Anemia

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT00737685
• Other study ID #: KSPHO-SCT0401
• Status: Active, not recruiting
• Phase: Phase 2
• First received: August 18, 2008
• Last updated: March 23, 2012
• Start date: January 2006
• Est. completion date: December 2012

Study information

• Verified date: March 2012
• Source: The Korean Society of Pediatric Hematology Oncology
• Contact: n/a
• Is FDA regulated: No
• Health authority: Korea: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

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.

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)

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 30
• Est. completion date: December 2012
• Est. primary completion date: August 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 25 Years

Eligibility

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.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic

Intervention

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)

Locations

Country	Name	City	State
Korea, Republic of	Seoul National University Hospital	Seoul

Sponsors (1)

Lead Sponsor	Collaborator
The Korean Society of Pediatric Hematology Oncology

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To evaluate the engraftment potential, incidence and severity of acute graft versus host disease,toxicity of conditioning regimen for UBMT in SAA.		From Jan. 1. 2006 to Dec. 31. 2008. For 3 years.	No
Primary	To evaluate overall and EFS follow-up of 1 year after UBMT/PBSCT.		From Jan. 1. 2006 to Dec. 31. 2008. For 3 years	No
Secondary	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		From Jan. 1. 2006 to Dec. 31. 2008. For 3 years.	No