Donor Stem Cell Transplant After Busulfan, Fludarabine, Methylprednisolone, and Antithymocyte Globulin in Treating Patients With Bone Marrow Failure Syndrome

Phase 2

Completed

• Conditions: Leukemia; Myelodysplastic Syndromes; Nonmalignant Neoplasm; Paroxysmal Nocturnal Hemoglobinuria

• Registration Number: NCT00731328
• Lead Sponsor: Asan Medical Center

Brief Summary

RATIONALE: Giving low doses of chemotherapy and antithymocyte globulin before a donor stem cell transplant helps stop the growth of abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining abnormal cells (graft-versus-tumor effect).

PURPOSE: This phase II trial is studying how well a donor stem cell transplant works after busulfan, fludarabine, methylprednisolone, and antithymocyte globulin in treating patients with bone marrow failure syndrome.

Detailed Description

OBJECTIVES:

* To evaluate the efficacy of HLA-haploidentical familial donor hematopoietic stem cell transplantation after reduced-intensity conditioning regimen comprising busulfan, fludarabine phosphate, and anti-thymocyte globulin in patients with bone marrow failure syndromes.

OUTLINE:

* Reduced-intensity conditioning regimen: Patients receive busulfan IV daily on days -7 and -6, fludarabine phosphate IV over 30 minutes on days -7 to -2, anti-thymocyte globulin (ATG) IV over 4 hours on days -4 to -1, and methylprednisolone IV over 30 minutes starting 30 minutes before ATG on days -4 to -1.

* HLA-haploidentical donor hematopoietic stem cell transplantation: Patients receive donor hematopoietic stem cells via Hickman catheter over 1 hour on days 0 or 1.

* Graft-versus-host-disease prophylaxis (GVHD): Patients receive cyclosporine IV over 2-4 hours every 12 hours starting on day -1 (cyclosporine can be given orally once oral medication can be tolerated) and methotrexate IV on days 2, 4 , 7, and 12. In the absence of GVHD, cyclosporine is tapered starting between days 30 to 60.

After completion of study treatment, patients are followed periodically for 1 year.

Study Timeline

• Study Start: 2008-04
• Study First Submitted: 2008-08-08
• Study First Submitted QC: 2008-08-08
• Study First Posted: 2008-08-11
• Primary Completion: 2013-12
• Study Completion: 2013-12
• Status Verified: 2015-12
• Last Update Submitted: 2015-12-29
• Last Update Posted: 2015-12-31

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Donor cell engraftment	10-35 days after transplantation	neutrophil count over 500/ul

Secondary Outcome Measures

Name	Time	Method
overall survival	0-60 months	patients surviving after transplantaion
event-free survival	0-60 months after transplatation	patients undergoing transplantation and maintaining donor hematopoiesis
Regimen-related toxicities as assessed by NCI's Common Toxicity Criteria	0-60 months after transplantation	various toxicities of treatment
Acute and chronic GVHD	15-100 days; 100 days to 4 years	ocurrence of acute or chronic GVHD after transplantation

Trial Locations

Locations (1)

Asan Medical Center - University of Ulsan College of Medicine; 🇰🇷 Seoul, Korea, Republic of