NCT00003336已完成2 期
A Pilot Study of Unrelated Umbilical Cord Blood Transplantation in Patients With Severe Aplastic Anemia, Inborn Errors in Metabolism, or Inherited Hematologic Stem Cell Disorders
适应症
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Event-free survival by disease assessment
研究概览
简要总结
RATIONALE: Umbilical cord blood transplantation may allow doctors to give higher doses of chemotherapy or radiation therapy and kill more cancer cells.
PURPOSE: This phase II trial is studying how well umbilical cord blood transplantation works in treating patients with severe aplastic anemia, malignant thymoma, or myelodysplasia.
详细描述
OBJECTIVES:
- Determine the rates of durable engraftment in patients with severe aplastic anemia, myelodysplastic syndrome, inborn errors of metabolism, or inherited hematopoietic disorders, refractory to medical management, who are undergoing high-dose chemoradiotherapy followed by unrelated cord blood (UCB) transplantation.
- Evaluate the rate and quality of immunologic reconstitution in this patient population.
OUTLINE: Patients are stratified according to weight (under 45 kg vs over 45 kg).
Patients receive high-dose chemotherapy and/or radiotherapy as a conditioning regimen beginning 6-9 days before the umbilical cord blood transplant (UCBT). The regimen varies according to the underlying cause of the anemia, but could include busulfan, cyclophosphamide or melphalan, anti-thymocyte globulin or methylprednisolone, and/or radiation therapy. One day after the conditioning regimen is completed, patients receive the UCBT.
Patients are followed weekly for 3 months, at 6 months, then every 6 months for 2.5 years, then annually thereafter.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 55 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •DISEASE CHARACTERISTICS:
- •Histologically confirmed diagnosis of severe aplastic anemia based on bone marrow cellularity of less than 20%
- •Must meet at least two of the following criteria:
- •Granulocyte count less than 500/mm^3
- •Platelet count less than 20,000/mm^3
- •Reticulocyte count less than 50,000/mm^3
- •Following etiologies eligible:
- •Fanconi's anemia
- •Hypoplastic leukemia
- •Monosomy 7
- •Drug exposure (chloramphenicol, NSAIDS)
- •Viral exposure (EBV, hepatitis, parvovirus, HIV)
- •Nutritional deficiencies
- •Paroxysmal nocturnal hemoglobinuria
- •Amegakaryocytic thrombocytopenia OR
- •Histologically confirmed myelodysplastic syndrome (MDS) that is refractory to medical management or with cytogenic abnormalities predictive of transformation into acute leukemia, including 5q-, 7q-, monosomy 7, or trisomy 8
- •The following etiologies only are eligible:
- •Refractory anemia
- •Refractory anemia with ringed sideroblasts
- •De novo primary MDS
- •Therapy-related secondary MDS OR
- •Confirmed diagnosis of inherited hematopoietic disorder that is refractory to medical management
- •Following etiologies eligible:
- •Severe combined immunodeficiency
- •Familial erythrophagocytic lymphohistiocytosis
- •Wiskott-Aldrich syndrome
- •Kostmann's syndrome (infantile histiocytosis)
- •Chronic granulomatous disease
- •Leukocytic adhesion deficiency
- •Chediak-Higashi syndrome
- •Paroxysmal nocturnal hemoglobinuria
- •Fanconi's anemia
- •Dyskeratosis congenita
- •Diamond-Blackfan anemia
- •Amegakaryocytic thrombocytopenia
- •Osteopetrosis
- •Gaucher's disease
- •Lesch-Nyhan syndrome
- •Mucopolysaccharidoses
- •Lipodoses
- •Autologous or haploidentical related peripheral blood stem cells available as backup
- •Serologically matched umbilical cord blood unit available in the New York Blood Center's Placental Blood Project, or other acceptable umbilical cord blood registry
- •PATIENT CHARACTERISTICS:
- •55 and under
- •Performance status:
- •Zubrod 0-1
- •Karnofsky 80-100%
- •Life expectancy:
- •At least 3 months
- •Hematopoietic:
- 另有 26 项未显示
排除标准
- 未提供
结局指标
主要结局
Event-free survival by disease assessment
时间窗: at 100 days and at 6, 9, 12, 18, and 24 months
次要结局
- Umbilical cord blood donor engraftment by chimerism and complete blood count (CBC) at time of myeloid recovery.(100 days and at 6, 9, 12, 18, and 24 months)
研究者
研究点 (1)
Loading locations...
相似试验
已完成
2 期
Umbilical Cord Blood Transplantation in Treating Patients With High-Risk Hematologic CancerGraft Versus Host DiseaseMyelodysplastic SyndromesMyelodysplastic/Myeloproliferative DiseasesLymphomaLeukemiaNCT00003335Case Comprehensive Cancer Center44
已完成
2 期
Umbilical Cord Blood Transplantation in Treating Patients With Hematologic Cancer or Other Hematologic or Metabolic DiseasesThymic CarcinomaGraft Versus Host DiseaseMyelodysplastic SyndromesLeukemiaNCT00003662Roswell Park Cancer Institute90
终止
2 期
Myloablative Cord Blood Transplant in Haematological Malignancies (MAC UCBT)Other Haematological DiseasesLeukaemiaLymphomaNCT02310997University College, London11
终止
2 期
Umbilical Cord Blood (UCB) Transplant, Fludarabine, Melphalan, and Anti-thymocyte Globulin (ATG) in Treating Patients With Hematologic CancerMyeloproliferative DisordersMultiple Myeloma and Plasma Cell NeoplasmMyelodysplastic SyndromesLymphomaLeukemiaNCT00827099Northside Hospital, Inc.5
已完成
1 期
Combination Chemotherapy and Total-Body Irradiation Before Donor Umbilical Cord Blood Transplant in Treating Patients With Advanced Hematologic Cancer, Metastatic Breast Cancer, or Kidney CancerMyelodysplastic SyndromesKidney CancerMultiple Myeloma and Plasma Cell NeoplasmBreast CancerLymphomaLeukemiaNCT00365287Masonic Cancer Center, University of Minnesota148