NL-OMON50307招募中不适用
Outcome measures in Duchenne Muscular Dystrophy: A Natural History Study - Duchenne natural history study
适应症
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 20
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Observational
入排标准
- 年龄范围
- 2 至 99（—）
入选标准
- •NON AMBULANT DMD PATIENTS
- •1. Children, teenagers and adults from the age of 5 with DMD, who have lost the
- •ability to walk 10 meters with no support
- •2. The diagnosis of DMD must be documented by genetic testing.
- •3. Patients should preferably have deletions amenable of skipping of exons 51
- •or 53 or 45 or 44 or 46 or 50 or 52.
- •4. Patients should be capable of sitting upright in a wheelchair for at least
- •5. Patients should be stable from a respiratory point of view., AMBULANT DMD
- •1. Ambulant children from 5 years old and teenagers with DMD, and potential
- •candidates for future genetic therapies with antisense oligomer (AO) exon
- •2. The diagnosis of DMD must be documented by MLPA or a standard genetic test
- •for the disorder, genotypically confirmed to have an out-of-frame deletion(s)
- •that could be corrected by skipping exon 51 or 53 or 45 or 44 or 46 or 50 or 52
- •3. Ability to walk independently for at least 10 meters at recruitment.
- •4. Patients should receive the standard of care for DMD as recommended by the
- •NorthStar UK and TREAT-NMD (ie: on glucocorticoids treatment)
- •5. Sufficiently preserved pulmonary function (FVC >30%) and absence of symptoms
- •of cardiac failure.
排除标准
- •NON AMBULANT DMD PATIENTS:, 1. Patients who are currently involved in
- •interventional clinical trials aimed at restoring dystrophin will be excluded,
- •as their data could not be used to establish natural history of the disease
- •(participation in a previous interventional clinical trial prior to 6 months
- •from being recruited in the study is not an exclusion criterion)
- •2. Patients with severe intellectual impairment, who would be unable to
- •cooperate with examination
- •3. Patients/families we anticipate may have emotional/ psychological problems
- •if recruited in into a natural history study
- •4. Symptomatic cardiac failure
- •5. Recent (< 6 months) upper limb surgery or trauma
- •6. Anticipated surgery for anytime during the duration of the study
- •7. For the MRI substudy, patients with metal/metallic surgically inserted
- •equipment incompatible with MRI scan and patients suffering from claustrofobia.
- •, AMBULANT DMD PATIENTS:, 1. Patients who are currently involved in
- •interventional clinical trials aimed at restoring dystrophin will be excluded,
- •as their data could not be used to establish natural history of the disease
- •(participation in a previous interventional clinical trial prior to 6 months
- •from being recruited in the study is not an exclusion criterion)
- •2. Patients with severe intellectual impairment, who would be unable to
- •cooperate with examination
- •3. Patients/families we anticipate may have emotional/ psychological problems
- •if recruited in into a natural history study
- •4. Recent surgery or anticipated for anytime during the duration of the study
- •5. For the MRI substudy, patients with metal/metallic surgically inserted
- •equipment incompatible with MRI scan and patients suffering from claustrofobia.
研究者
相似试验
已完成
不适用
Study of outcome measures in muscular dystrophy clinical trialsDuchenne muscular dystrophyJPRN-UMIN000018235Muscular Dystrophy Clinical Trial Network50
已完成
2 期
utriceuticals in Duchenne muscular dystrophyNeurological - Other neurological disordersHuman Genetics and Inherited Disorders - Other human genetics and inherited disordersMusculoskeletal - Other muscular and skeletal disordersDuchenne muscular dystrophyACTRN12610000462088The Royal Children's Hospital48
进行中（未招募）
不适用
Duchenne muscular dystrophy: a clinical trial to find the optimum steroid regimen.Duchenne muscular dystrophy boys between 4 and 8 years of age, able to rise from the floor without support, not previously treated with steroids.MedDRA version: 14.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2010-023744-33-ITAZIENDA OSPEDALIERA DI PADOVA300
进行中（未招募）
不适用
Trial to find best steroid treatment for Duchenne muscular dystrophyDuchenne muscular dystrophy (DMD)MedDRA version: 14.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2010-023744-33-GBniversity of Rochester300
招募中
不适用
atural history study of Duchenne muscular dystrophy focusing on upper limb motor functioDuchenne muscular dystrophyJPRN-UMIN000046838ational Center of Neurology and Psychiatry100