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

NCT ID: NCT00737685

Last Updated: 2012-03-26

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2006-01-31
• Study Completion Date: 2012-12-31

Brief 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.

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)

Conditions

Anemia, Aplastic

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Fludarabine

Group Type: EXPERIMENTAL

cyclophosphamide, fludarabine , thymoglobulin

Intervention Type: DRUG

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)

Interventions

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)

Intervention Type: DRUG

Eligibility 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.

• Maximum Eligible Age: 25 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

The Korean Society of Pediatric Hematology Oncology

NETWORK

Sponsor Role: lead

Responsible Party

Seoul Natioanl University Hospital

Principal Investigators

Hyo Seop Ahn, M.D, Ph.D

Role: PRINCIPAL_INVESTIGATOR

The Korean Society of Pediatric Hematology Oncology

Locations

Seoul National University Hospital

Seoul, , South Korea

Countries

South Korea

References

Kang HJ, Shin HY, Park JE, Chung NG, Cho B, Kim HK, Kim SY, Lee YH, Lim YT, Yoo KH, Sung KW, Koo HH, Im HJ, Seo JJ, Park SK, Ahn HS; Korean Society of Pediatric Hematology-Oncology. Successful engraftment with fludarabine, cyclophosphamide, and thymoglobulin conditioning regimen in unrelated transplantation for severe aplastic anemia: A phase II prospective multicenter study. Biol Blood Marrow Transplant. 2010 Nov;16(11):1582-8. doi: 10.1016/j.bbmt.2010.05.010. Epub 2010 May 26.

Reference Type: DERIVED

PMID: 20685256 (View on PubMed)

Other Identifiers

KSPHO-SCT0401

Identifier Type: -

Identifier Source: org_study_id