NCT02074631已完成2 期
Prevention of Bone Loss After Pediatric Hematopoietic Cell Transplantation
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 63
- 试验地点
- 2
- 主要终点
- Lumbar Spine Bone Mineral Content
研究概览
简要总结
This is a Phase 2, open-label, randomized, controlled clinical study of pediatric subjects treated with pamidronate with calcium and vitamin D versus calcium and vitamin D alone following hematopoietic cell transplantation (HCT). The purpose of this study is to test the hypothesis that subjects receiving pamidronate with calcium and vitamin D will have higher lumbar spine bone mineral content (LBMC) measured by dual-energy X-ray tomography (DXA) at 1 year post-HCT than subjects receiving calcium and vitamin D alone (Control Group).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 20 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Allogeneic hematopoietic cell transplant for hematologic malignancy (i.e. leukemia, lymphoma including ALL, AML, CML, NHL, HL) in complete remission; myelodysplastic syndrome (active dysplasia and/or blasts are permitted, but must not have active leukemia) or idiopathic severe aplastic anemia (SAA)
- •Non-malignant diseases including idiopathic severe aplastic anemia (SAA) and other bone marrow failure disorders, hemoglobinopathies, adrenoleukodystrophy, immune deficiencies/dysregulation disorders who will be receiving myeloablative or reduced toxicity preparative regimens that meet the following criteria:
- •Regimens include those that are TBI based if the TBI dose is > 500cGy single dose or > 800cGy fractionated, or doses <500 cGy if combined with busulfan or treosulfan. These also include chemotherapy only based regimens that contain myeloablative doses of busulfan (>8mg/kg) or treosulfan without TBI.
- •Patients with severe aplastic anemia are eligible regardless of conditioning regimen
- •Myeloablative preparative regimen (for SAA any conditioning therapy allowed)
- •Male or female ≥1 but ≤ 20 years of age at time of study enrollment
- •Patient or parent(s)/legal guardian(s) is able and willing to provide informed consent. Assent will be obtained per local institutional policy. Subjects who turn 18 during the course of the study will be consented at that time of their next visit by a member of the research staff.
排除标准
- •History of a primary bone malignancy involving the lumbar spine
- •Prior and/or planned concomitant medical therapy during the study period (through Day 360 post-HCT) with other bisphosphonates, Denosumab, or Teriparatide
- •Pregnancy or breastfeeding - menstruating females must have a negative pregnancy test prior to study enrollment and agree to repeat pregnancy testing and contraception use per protocol as pamidronate is Pregnancy Category D - positive evidence of human fetal risk based on adverse reaction data
- •Renal insufficiency, defined as creatinine level greater than the upper limit of normal for age
- •Hereditary metabolic bone disease or skeletal dysplasia (e.g., osteopetrosis or OI) or primary hyperparathyroidism
- •Other indications for HCT, including Fanconi anemia, other form of inherited bone marrow failure diseases, metabolic disorder, hemoglobinopathy, or immune deficiency
- •Clinically significant fractures as defined by ISCD (a long bone fracture of the lower extremities, vertebral compression fracture, or two or more long bone fractures of the upper extremities) (88,89) indicated by a cast or a spine x-ray within the last 2 weeks
- •Known or suspected allergy to pamidronate or related products
- •Planned administration of an investigational study drug or agent that either can interact with pamidronate or have an independent effect on bone mineral density within the 4 weeks prior to randomization (Day 90) or planned use during study participation (Day 90 through Day 360)
- •Impending invasive dental procedure that would be expected to occur during study participation (through Day 360)
研究组 & 干预措施
Control group
Active Comparator
Subjects will receive a standard recommended dose of calcium and vitamin D.
干预措施: Calcium and vitamin D (Drug)
Pamidronate Group
Experimental
Subjects randomized to pamidronate treatment will receive infusions approximately 100, 180, and 270 days after HCT along with calcium and vitamin D.
干预措施: Pamidronate (Drug)
Pamidronate Group
Experimental
Subjects randomized to pamidronate treatment will receive infusions approximately 100, 180, and 270 days after HCT along with calcium and vitamin D.
干预措施: Calcium and vitamin D (Drug)
结局指标
主要结局
Lumbar Spine Bone Mineral Content
时间窗: 1 year after HCT
次要结局
- Cytokine Levels (Interleukin IL-6, IL-7, and TNF-α)(7 days, 14 days, 21 days, 90 days after HCT)
- Ratio of Receptor Activator of the Nuclear Factor-κB Ligand [RANKL] and Osteoprotegerin [OPG](7 days, 14 days, 21 days, and 90 days after HCT)
- Markers of Bone Formation (Procollagen Type 1 N-terminal Propeptide [P1NP])(7, 14, 21, 90, 180, 360 days after HCT)
- Total Bone Mineral Density (BMD), Cortical BMD, Trabecular BMD, and Estimated Bone Strength Measured by pQCT(1 year after HCT)
- Marker of Bone Resorption (Carboxy-terminal Collagen Crosslinks [CTX](7, 14, 21, 90, 180, 360 days after HCT)
- Receptor Activator of the Nuclear Factor-κB Ligand [RANKL], Osteoprotegerin [OPG](7 days, 14 days, 21 days, and 90 days after HCT)
- Marker of Bone Resorption Deoxypyridinoline [DPD])(7, 14, 21, 90, 180, 360 days after HCT)
- Total Body Bone Mineral Content (TBMC; Excluding Head; Adjusted for Height, Age, Sex, Tanner Stage, and Race)(1 year after HCT)
- Marker of Bone Formation Osteocalcin [OCN])(7, 14, 21, 90, 180, 360 days after HCT)
研究者
研究点 (2)
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