Nationwide Children's Hospital — Biomedical Watch V2

Traitements6programmes

Essais3liés

Publications1liées

SourceDBlocale

Traitements

6

Molécule	Indication / population	Phase	Objectif	Pays	Résultat
Gentamicin infusions twice a week for six monthsThe purpose of this study is to determine the safety of giving intravenous (IV) gentamicin to boys with Duchenne muscular dystrophy who have stop codon mutations.	Myopathies	Phase 1	À vérifier	United States	À vérifier
Limb Girdle Muscular Dystrophy Type 2E Recruitment StudyThis study is to recruit and establish baseline measurements for potential subjects that may be eligible for a gene therapy trial. Specifically, this trial is recruiting individuals who are suspected or have been confirmed to have Limb Girdle Muscular Dystrophy type 2E (LGMD2E).	Myopathies	À vérifier	Thérapie génique	United States	À vérifier
Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular DystrophyThe goal of this observational study is to understand how young children with LAMA2-related dystrophy move and change over time. We will also learn about how this condition impacts other body systems. Participants will undergo: * Neuromuscular assessments * Blood collections * Swallowing and breathing assessments * Questionnaires	Myopathies	À vérifier	À vérifier	United States	À vérifier
Gentamicin infusions twice a week for six monthsThe purpose of this study is to determine the safety of giving intravenous (IV) gentamicin to boys with Duchenne muscular dystrophy who have stop codon mutations.	Myopathies	Phase 1	À vérifier	United States	À vérifier
Limb Girdle Muscular Dystrophy Type 2E Recruitment StudyThis study is to recruit and establish baseline measurements for potential subjects that may be eligible for a gene therapy trial. Specifically, this trial is recruiting individuals who are suspected or have been confirmed to have Limb Girdle Muscular Dystrophy type 2E (LGMD2E).	Myopathies	À vérifier	Thérapie génique	United States	À vérifier
Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular DystrophyThe goal of this observational study is to understand how young children with LAMA2-related dystrophy move and change over time. We will also learn about how this condition impacts other body systems. Participants will undergo: * Neuromuscular assessments * Blood collections * Swallowing and breathing assessments * Questionnaires	Myopathies	À vérifier	À vérifier	United States	À vérifier

Essais cliniques

3

Molécule	Indication / population	Phase	NCT	Titre	Statut
Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy	Myopathies	À vérifier	NCT06503367	Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy	RECRUITING
Limb Girdle Muscular Dystrophy Type 2E Recruitment Study	Myopathies	À vérifier	NCT03492346	Limb Girdle Muscular Dystrophy Type 2E Recruitment Study	UNKNOWN
Gentamicin infusions twice a week for six months	Myopathies	Phase 1	NCT00451074	Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons	COMPLETED

Publications

1

Molécule	Indication / population	Titre	Journal	Date
Gentamicin infusions twice a week for six months		[Efficacy of electroacupuncture nerve stimulation therapy for interstitial cystitis/bladder pain syndrome].	Zhongguo zhen jiu = Chinese acupuncture & moxibustion