NCT01910168已完成不适用
A Single Center Study to Collect Samples From SMA Patients for Biomarker Analysis
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 36
- 主要终点
- SMN1/SMN2 detection in blood by mRNA assay
研究概览
简要总结
In this single center study blood samples for biomarker analysis will be collected from patients with spinal muscular atrophy. Up to 21 mL blood will be drawn from eligible patients at a single visit.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Self-identified as 5q-autosomal recessive spinal muscular atrophy (SMA) type I, II or III as judged by their neurologist upon diagnosis
- •Ability and willingness to provide blood samples
- •Willingness (by the patient or patient's parents or legal guardian) to complete to their best ability a questionnaire which requests specific clinical and genetic information
- •Able to participate and willing to give written informed consent or assent. Informed consent will be obtained from the patient, or the patient's parent or legal guardian.
排除标准
- •Any known genetic condition other than spinal muscular atrophy, unless it is not interfering with the purpose of this study based on the Sponsor's judgment
- •Participation in a clinical trial (except observational studies) within the previous 14 days
- •Donation of blood or significant blood loss within three months prior to screening
- •Concomitant disease or condition that could interfere with, or treatment of which might interfere with, the conduct of this study, or that would, in the opinion of the investigator, pose an unacceptable risk to the patient in this study
结局指标
主要结局
SMN1/SMN2 detection in blood by mRNA assay
时间窗: 1 day
次要结局
- SMN protein level in blood/lymphocytes(1 day)
研究者
相似试验
已完成
不适用
Collection of Blood Samples From SMART Study Participants for Future Genetic StudiesHIV InfectionsNCT00377169National Institute of Allergy and Infectious Diseases (NIAID)3,261
已完成
不适用
Parkinson's Disease Biomarker ProgramParkinson's DiseaseNCT01767818University of Texas Southwestern Medical Center238
已完成
4 期
Collection of Blood Samples From Patients With Relapsing MS Who Developed ITP After Receiving LemtradaMultiple SclerosisNCT03784898Genzyme, a Sanofi Company13
终止
不适用
Blood Sample Collection to Evaluate Biomarkers in Subjects With Untreated MelanomaMelanomaNCT03727087Exact Sciences Corporation348
终止
不适用
Blood Sample Collection to Evaluate Biomarkers in Subjects With Untreated Hematologic MalignanciesHematologic MalignancyNCT03727009Exact Sciences Corporation409