NCT01380964已完成不适用
Research of Biomarkers for Disease Diagnosis, Disease Monitoring and Therapeutic Treatment Response in Duchenne Muscular Dystrophy Patients
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Genethon
- 入组人数
- 220
- 试验地点
- 1
- 主要终点
- IBiSD aims to identify and validate new and disease-specific biomarkers.
研究概览
简要总结
The purpose of this study is to identify potential biomarkers for the diagnosis, disease progression assessment and response to treatment in patients with Duchenne Muscular Dystrophy.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 3 Years 至 20 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 是
入选标准
- •FOR PATIENTS:
- •Diagnosis of DMD confirmed by genetic testing
- •Age over 3 years
- •Weight over 15 kg
- •Informed consent signed
- •FOR CONTROLS:
- •Age over 3 years
- •Male gender
- •Weight over 15 kg
- •Subjects with national health insurance coverage
- •Informed consent signed
- •Nonacute or chronic muscular, allergic, infectious, endocrine or inflammatory disorder in the 3 weeks preceding inclusion
排除标准
- •FOR PATIENTS:
- •Concomitant chronic or acute muscular, endocrine, infectious, allergic or inflammatory disorder in the three weeks preceding the blood test
- •Intake of medicines other than angiotensin-converting enzyme inhibitors, beta blockers, dietary supplements, vitamins, alendronate and methylphenidate. Steroids (and medicines prescribed with them such as calcium supplements and proton pump inhibitors) will be discussed
- •Mental retardation or autism
- •Vaccination or treatment with immunoglobulins within the three months preceding inclusion
- •FOR CONTROLS:
- •Concomitant chronic or acute muscular, neurological (including mental retardation and autism), infectious or inflammatory disorder in the three weeks preceding the blood test
- •Vaccination or treatment with immunoglobulins within the three months preceding inclusion
结局指标
主要结局
IBiSD aims to identify and validate new and disease-specific biomarkers.
时间窗: End of study
This study will establish the relevance of urinary and blood biomarkers for the diagnosis, follow-up and assessment of treatment response in patients with DMD (IBiSD1, 2 and 4). IBiSD will also attempt to establish the seroprevalence to the different strains of AAV in patients with DMD (IBiSD3).
次要结局
未报告次要终点
研究者
研究点 (1)
Loading locations...
相似试验
招募中
不适用
Investigation of biomarkers for diagnosis and treatment of pulmonary hypertension.Pulmonary hypertensionJPRN-UMIN000032888Juntendo University50
招募中
不适用
Identification of biomarkers for diagnosis and prognosis in the patients with obstructive sleep apneaobstructive sleep apneaJPRN-UMIN000021503Osaka university graduate School of medicine The department of geriatric and general medicine200
招募中
不适用
Research to find new indicators for diagnosis and treatment of bladder cancer using exosomes in urinebladder cancerJPRN-UMIN000045726St.Marianna University School of Medicine420
尚未招募
Unknown
Research to develop diagnostic and therapeutic methods for cancer of the renal pelvis and ureterCancer of the renal pelvis and ureterrenal pelvis cancer, ureteral cancerD014516, D007682JPRN-jRCT1030220502Hayakawa Nozomi250
已完成
不适用
Brain Connectivity in Neurodevelopmental Disorders in Response to TreatmentAutism Spectrum DisordersPsychosisFragile X SyndromeNCT01364818University of North Carolina, Chapel Hill25