NCT00907478已完成4 期
A Prospective, Phase IV, Open-Label, Multi-Center Study Evaluating Changes in Bone Marrow Morphology in Adult Subjects Receiving Romiplostim for the Treatment of Thrombocytopenia Associated With Immune (Idiopathic) Thrombocytopenia Purpura (ITP)
适应症
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- Amgen
- 入组人数
- 169
- 主要终点
- Percentage of Participants With Collagen Fibrosis
研究概览
简要总结
The purpose of this study is to evaluate changes in bone marrow morphology (structure) after long-term exposure to romiplostim.
详细描述
Participants diagnosed with ITP according to the American Society of Hematology (ASH) Guidelines were sequentially enrolled into the following groups:
- Bone marrow biopsy at Baseline and Year 1
- Bone marrow biopsy at Baseline and Year 2
- Bone marrow biopsy at Baseline and Year 3.
All participants received romiplostim for 3 years, unless withdrawn from the study early. Participants returned for one visit for End of Study (EOS) procedures 4 weeks after romiplostim discontinuation, or, for participants who were withdrawn from the study due to the presence of collagen fibrosis, or had a change to grade 3 reticulin, at 12 weeks after discontinuation of romiplostim.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of ITP according to American Society of Hematology (ASH) guidelines
- •Subject must have had a bone marrow biopsy within one year prior to planned first dose of romiplostim (with available bone marrow tissue block or unstained histological slides to send to a central laboratory for interpretation) or must consent to a pre-treatment bone marrow biopsy within 3 weeks prior to planned first dose of romiplostim. Central laboratory interpretation is required prior to first dose of romiplostim
- •Subject must agree to a scheduled bone marrow biopsy at Year 1, Year 2, or Year 3 following romiplostim treatment and any unscheduled biopsies if clinically indicated
- •Subject ≥18 years of age
- •Baseline bone marrow reticulin grade of 0, 1, 2, or 3 according to the modified Bauermeister grading scheme as assessed by central laboratory interpretation
- •Platelet count < 50 x 10^9/L
- •Must have received at least 1 prior ITP therapy (examples of ITP therapy include corticosteroids, intravenous immunoglobulin [IVIG], splenectomy)
- •Subject (or legally-acceptable representative) is willing and able to provide written informed consent
排除标准
- •Baseline bone marrow biopsy positive for collagen fibrosis
- •Any known history of or currently active bone marrow stem cell disorder, hematological malignancy, myeloproliferative disorder, myelodysplastic syndrome
- •Any current active malignancy
- •Any prior exposure to cytostatic chemotherapy or radiotherapy for malignancy
- •Subject has undergone pacemaker placement, cardiac ablation of arrhythmia, and/or any current treatment with Vaughan Williams Class IA - IC and Class III agents (Vaughan Williams, 1970)
- •Subject has participated in any study evaluating pegylated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF), recombinant human thrombopoietin (rHuTPO), or thrombopoietin receptor agonists (ie romiplostim or eltrombopag)
- •Subject has a known hypersensitivity to any recombinant E coli-derived product
- •Subject is currently enrolled in or has not yet completed (at least 4 weeks since ending) other investigational device or drug trial(s) or subject is receiving other investigational agent(s)
- •Other investigational procedures are excluded
- •Subject of child-bearing potential is evidently pregnant (eg positive pregnancy test) or is breast feeding
- •Subject is not using adequate contraceptive precautions
- •Subject has any kind of disorder that compromises the ability of the subject to give written informed consent and does not have a legally acceptable representative and/or is unable to comply with study procedures.
结局指标
主要结局
Percentage of Participants With Collagen Fibrosis
时间窗: At Years 1, 2 or 3 after initial exposure of romiplostim
The percentage of participants who developed collagen fibrosis as evidenced by trichrome staining. Bone marrow biopsy samples were assessed using the modified Bauermeister grading scale by a central laboratory.
次要结局
- Percentage of Participants Who Developed an Increased Modified Bauermeister Grade(At Year 1, Year 2, or Year 3 post romiplostim exposure)
- Number of Participants With Improvement of Reticulin to a Grade of ≤ 2 for Participants Who Developed Grade 3 Reticulin(12 weeks after romiplostim discontinuation)
- Number of Participants With Collagen Fibrosis 12 Weeks After Romiplostim Discontinuation in Participants Who Developed Collagen Fibrosis at Years 1, 2, or 3(12 weeks after romiplostim discontinuation)
- Percentage of Participants With Clinically Relevant Changes in Total Cardiac Output Corrected (QTc) Intervals(Baseline, Week 3 and Week 12)
- Number of Participants With Adverse Events (AEs)(From the first dose of study drug until 4 weeks after treatment discontinuation or 12 weeks after treatment discontinuation for patients who developed collagen fibrosis or a change to grade 3 reticulin; the overall median treatment duration was 154 weeks.)
- Percentage of Participants With CTCAE Grade ≥ 2 Shift in Anemia or Neutropenia(From the first dose of study drug until 4 weeks after treatment discontinuation or 12 weeks after treatment discontinuation for patients who developed collagen fibrosis or a change to grade 3 reticulin; the overall median treatment duration was 154 weeks.)
- Number of Participants Who Developed Antibodies or Neutralizing Antibodies to Romiplostim or to Endogenous Thrombopoietin(Every 24 weeks and at the end of study visit (4 weeks or 12 weeks after study drug discontinuation).)
研究者
相似试验
已完成
不适用
Follow-up of Bone Quality in Long-Term Bone Marrow Transplant SurvivorsOsteoporotic FracturesNCT00594295University of Wisconsin, Madison15
已完成
不适用
Study Investigating the Interactions of Bone and Hematopoiesis in the ElderlyMyelodysplastic SyndromesNCT02867085Technische Universität Dresden297
已完成
不适用
Combination of Bone Marrow Concentrate and Iliac Cancellous Bone Graft in Alveolar Cleft GraftingAlveolar Cleft GraftingNCT04414423Fayoum University18
已完成
不适用
Musculoskeletal Outcomes After Bone Marrow TransplantSurvivors of Bone Marrow TransplantAutologous and AllogeneicNCT00558948Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins48
进行中（未招募）
不适用
A longitudinal 2-year bone marrow study of eltrombopag olamine (SB-497115-GR) in previously treated adults, with chronic immune (idiopathic) thrombocytopenic purpura (ITP).Chronic Idiopathic Thrombocytopenic PurpuraMedDRA version: 15.1Level: PTClassification code 10021245Term: Idiopathic thrombocytopenic purpuraSystem Organ Class: 10005329 - Blood and lymphatic system disordersEUCTR2009-010421-39-DEGlaxoSmithKline200