- ICH GCP
- 미국 임상 시험 레지스트리
- 임상시험 NCT00731328
Donor Stem Cell Transplant After Busulfan, Fludarabine, Methylprednisolone, and Antithymocyte Globulin in Treating Patients With Bone Marrow Failure Syndrome
HLA-HAPLOIDENTICAL FAMILIAL DONOR HEMATOPOIETIC CELL TRANSPLANTATION AFTER REDUCED INTENSITY CONDITIONING OF BUSULFAN, FLUDARABINE, AND ANTI-THYMOCYTE GLOBULIN FOR PATIENTS WITH BONE MARROW FAILURE SYNDROME - A PHASE 2 STUDY
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.
연구 개요
상태
상세 설명
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.
연구 유형
등록 (실제)
단계
- 2 단계
연락처 및 위치
연구 장소
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Seoul, 대한민국, 138-736
- Asan Medical Center - University of Ulsan College of Medicine
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참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
DISEASE CHARACTERISTICS:
Diagnosis of any of the following bone marrow failure syndromes:
Severe aplastic anemia, meeting 1 of the following criteria:
- Not responsive to immunosuppressive therapy
- With recurrent cytopenia after immunosuppressive therapy or allogeneic hematopoietic cell transplantation
Low-risk myelodysplastic syndrome, including any of the following:
- Refractory anemia
- Refractory anemia with ringed sideroblasts
- Refractory cytopenia with multi-lineage dysplasia
Paroxysmal nocturnal hemoglobinuria, meeting 1 of the following criteria:
- With thrombotic episodes
- With severe cytopenia
No willing, suitable HLA-compatible donor in family or in donor registries
- Related donor with HLA-haploidentical mismatch at three or less of 6 loci
- Patients with very severe neutropenia (< 200/μL) or febrile episodes, who feel urgent need for allogeneic hematopoietic cell transplantation, are eligible without a search for HLA-matched unrelated donors
PATIENT CHARACTERISTICS:
- Karnofsky performance status 70-100%
- Bilirubin < 2.0 mg/dL
- AST < 3 times upper limit of normal
- Creatinine < 2.0 mg/dL
- Ejection fraction > 40% by MUGA scan
PRIOR CONCURRENT THERAPY:
- See Disease Characteristics
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 해당 없음
- 중재 모델: 단일 그룹 할당
- 마스킹: 없음(오픈 라벨)
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Donor cell engraftment
기간: 10-35 days after transplantation
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neutrophil count over 500/ul
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10-35 days after transplantation
|
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Regimen-related toxicities as assessed by NCI's Common Toxicity Criteria
기간: 0-60 months after transplantation
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various toxicities of treatment
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0-60 months after transplantation
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Acute and chronic GVHD
기간: 15-100 days; 100 days to 4 years
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ocurrence of acute or chronic GVHD after transplantation
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15-100 days; 100 days to 4 years
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overall survival
기간: 0-60 months
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patients surviving after transplantaion
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0-60 months
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event-free survival
기간: 0-60 months after transplatation
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patients undergoing transplantation and maintaining donor hematopoiesis
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0-60 months after transplatation
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공동 작업자 및 조사자
연구 기록 날짜
연구 주요 날짜
연구 시작
기본 완료 (실제)
연구 완료 (실제)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (추정)
연구 기록 업데이트
마지막 업데이트 게시됨 (추정)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
키워드
추가 관련 MeSH 약관
기타 연구 ID 번호
- CDR0000600351
- AMC-UUCM-2008-0038
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