Alemtuzumab and Low-Dose Cyclosporine in Treating Patients With Severe Aplastic Anemia or Acquired Marrow Failure
Alemtuzumab and Low-Dose Cyclosporine-A as Alternative Immunosuppressive Treatment for Severe Aplastic Anemia (SAA) and Single-Lineage Aplastic Patients
RATIONALE: Immunosuppressive therapies, such as alemtuzumab and cyclosporine, may improve bone marrow function and increase blood cell counts. Giving alemtuzumab together with cyclosporine may be an effective treatment for severe aplastic anemia or acquired marrow failure.
PURPOSE: This phase II trial is studying the side effects of giving alemtuzumab together with cyclosporine and to see how well it works in treating patients with severe aplastic anemia or acquired marrow failure.
연구 개요
상세 설명
OBJECTIVES:
Primary
- Determine the safety of alemtuzumab and low-dose cyclosporine, as defined by occurrence of adverse effects, in patients with severe aplastic anemia or single lineage acquired marrow failure.
- Determine the efficacy of this regimen, in terms of overall survival, hematological response (partial and complete response, including time to response) and failure-free survival (failure is defined as no response, chronic treatment-maintained response, or relapse), in these patients.
Secondary
- Evaluate the incidence of adverse effects after treatment.
- Evaluate the long-term safety of alemtuzumab treatment.
- Determine the time to achieve a complete hematological response.
- Determine the proportion of patients maintaining hematological response free of any treatment.
- Determine the incidence of relapse in responding patients.
- Determine the incidence of severe infections.
- Determine the requirement for IV antibiotics and antifungal therapy.
- Determine the requirement for red cell and platelet transfusion.
- Determine the incidence of CMV reactivation.
- Determine the kinetics of immune reconstitution.
- Determine the incidence of paroxysmal nocturnal hemoglobinuria clone (lymphoid or myeloid) development.
- Determine the incidence of clonal evolution (i.e., karyotypic abnormalities or secondary myelodysplasia/leukemia).
OUTLINE: Patients receive alemtuzumab subcutaneously on days 1-5*. Patients also receive oral cyclosporine beginning on day 7 and continuing for ≥ 180 days, followed by a taper according to clinical condition.
NOTE: *Patients with single lineage aquired marrow failure receive alemtuzumab on days 1-4.
After completion of study therapy, patients will be followed up every 3 months for up to 2 years.
연구 유형
등록 (예상)
단계
- 2 단계
연락처 및 위치
연구 장소
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Naples, 이탈리아, 80131
- 모병
- Federico II University Medical School
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연락하다:
- Bruno Rotoli, MD
- 전화번호: 39-081-746-2068
- 이메일: rotoli@unina.it
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참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
DISEASE CHARACTERISTICS:
Diagnosis of 1 of the following:
Severe or very severe aplastic anemia, as defined by the following criteria:
Meets ≥ 2 of the following criteria:
- Absolute neutrophil count < 0.5 x 10^9/L (severe) or < 0.2 x 10^9/L (very severe)
- Platelet count < 20 x 10^9/L
- Reticulocyte count < 20 x 10^9/L
- Hypocellular bone marrow (< 30% cellularity) without evidence of fibrosis or malignant cells
- Single lineage acquired marrow failure (e.g., pure red cell aplasia, agranulocytosis, amegakaryocytic thrombocytopenia)
- Paroxysmal nocturnal hemoglobinuria clone allowed
Failed first-line therapy with antithymocyte globulin (ATG) and cyclosporine OR not eligible for ATG-based studies
- Failure is defined as lack of hematological response, requirement for chronic immunosuppressive treatment to sustain response, or relapse
- Not eligible for a low-risk stem cell transplantation
- No evidence of risky myelodysplastic syndromes (i.e., IPSS 3-4), as defined by the presence of marrow blast excess or karyotypic abnormalities, or other primitive marrow disease
- No history of constitutional aplastic anemia (e.g., Fanconi anemia or dyskeratosis congenita)
PATIENT CHARACTERISTICS:
- WHO performance status 0-2
- Not pregnant or nursing
- No active malignant tumor within the past 5 years
- Transaminases ≤ 3 times upper limit of normal (ULN)
- Albumin ≥ 1.5 g/L
- Creatinine ≤ 3 times ULN
- No CMV viremia, as defined by positive PCR or pp65 test
- No cardiac failure (i.e., ejection fraction < 35%)
- No other concurrent life-threatening disease (including HIV infection)
PRIOR CONCURRENT THERAPY:
- No prior allogeneic stem cell transplantation
- At least 2 weeks since prior cyclosporine or filgrastim (G-CSF)
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 무작위화되지 않음
- 마스킹: 없음(오픈 라벨)
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
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전반적인 생존
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Safety, as defined by occurrence of adverse effects
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Hematologic response (partial and complete response, including time to response)
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Failure-free survival (failure is defined as no response, chronic treatment-maintained response, or relapse)
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2차 결과 측정
결과 측정 |
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Incidence of adverse effects after treatment
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Long-term safety of alemtuzumab treatment
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Time to achieve a complete hematological response
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Proportion of patients maintaining hematological response free of any treatment
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Incidence of relapse in responding patients
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Incidence of severe infections
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Requirement for IV antibiotics and antifungal therapy
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Requirement for red cell and platelet transfusion
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Incidence of CMV reactivation
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Kinetics of immune reconstitution
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Incidence of paroxysmal nocturnal hemoglobinuria (PNH) clone (lymphoid or myeloid) development
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Incidence of clonal evolution (i.e., karyotypic abnormalities or secondary myelodysplasia/leukemia)
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공동 작업자 및 조사자
수사관
- 수석 연구원: Bruno Rotoli, MD, Federico II University
연구 기록 날짜
연구 주요 날짜
연구 시작
기본 완료
연구 완료
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
- UNMS-ALESAA
- CDR0000639649 (레지스트리 식별자: PDQ (Physician Data Query))
- EU-20927
- EUDRACT-2008-001151-22
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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알렘투주맙에 대한 임상 시험
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Genzyme, a Sanofi Company완전한
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Milton S. Hershey Medical Center종료됨