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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT00882323
Other study ID # KSPHO-SCT 0802
Secondary ID
Status Recruiting
Phase Phase 2
First received April 15, 2009
Last updated March 23, 2012
Start date November 2008
Est. completion date October 2012

Study information

Verified date March 2012
Source The Korean Society of Pediatric Hematology Oncology
Contact Hyoung Jin Kang, M.D, Ph.D
Phone 82 2 2072 3304
Email kanghj@snu.ac.kr
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 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.


Recruitment information / eligibility

Status Recruiting
Enrollment 33
Est. completion date October 2012
Est. primary completion date October 2012
Accepts healthy volunteers No
Gender Both
Age group 1 Year to 21 Years
Eligibility 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.

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment


Related Conditions & MeSH terms


Intervention

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)

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 engraftment potential of reduced toxicity fludarabine, cyclophosphamide plus thymoglobulin conditioning regimen for unrelated bone marrow transplantation in severe aplastic anemia. From Nov. 2008 to Oct. 2012 No
Secondary To evaluate toxicities of reduced toxicity fludarabine, cyclophosphamide plus thymoglobulin conditioning regimen for UBMT/PBSCT in SAA. From Nov. 2008 to Oct. 2012 No
Secondary To evaluate overall and EFS rate after UBMT/PBSCT. From Nov. 2008 to Oct. 2012 No
Secondary To evaluate GVHD and immunologic recovery after UBMT/PBSCT. From Nov. 2008 to Oct. 2012 No
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