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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00882323
• Date of registration: 15/04/2009
• Prospective Registration: No
• Primary sponsor: The Korean Society of Pediatric Hematology Oncology
• Public title: Reduced Toxicity Fludarabine (Flu) + Cyclophosphamide (CPM) + Rabbit Antithymocyte Globulin (rATG) Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia (SAA)
• Scientific title: Reduced Toxicity Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia
• Date of first enrolment: November 2008
• Target sample size: 33
• Recruitment status: Recruiting
• URL: http://clinicaltrials.gov/show/NCT00882323
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

Korea, Republic of

Contacts

• Name: Hyoung Jin Kang, M.D, Ph.D

• Name: Hyo seop Ahn, M.D, Ph. D
• Affiliation: The Korean Society of Pediatric Hematology Oncology

• Name: Hyo Seop Ahn, M.D, Ph.D

Key inclusion & exclusion criteria

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.



Age minimum: 1 Year
Age maximum: 21 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Aplastic Anemia

Intervention(s)

Drug: Cyclophosphamide, Fludarabine, Thymoglobulin

Primary Outcome(s)

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]

Secondary Outcome(s)

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]

To evaluate GVHD and immunologic recovery after UBMT/PBSCT. [Time Frame: From Nov. 2008 to Oct. 2012]

To evaluate overall and EFS rate after UBMT/PBSCT. [Time Frame: From Nov. 2008 to Oct. 2012]

Secondary ID(s)

KSPHO-SCT 0802

Source(s) of Monetary Support

Please refer to primary and secondary sponsors