Virginia Commonwealth University — Biomedical Watch V2

Traitements14programmes

Essais7liés

Publications0liées

SourceDBlocale

Traitements

14

Molécule	Indication / population	Phase	Objectif	Pays	Résultat
ASPIRE-DM1 — DMCRN-02-001: Assessing Pediatric Endpoints in DM1The overall goal of the study is to establish valid clinical endpoint assessments for children with congenital myotonic dystrophy type 1 and develop biomarkers for the condition.	Myopathies	À vérifier	À vérifier	United States, Italy	À vérifier
END-EXT — Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) ExtensionMyotonic Dystrophy type 1 (DM1) is an autosomal dominant multisystemic disorder that causes progressive disability and shortened life expectancy. It is characterized by progressive weakness and myotonia, which preferentially affects the craniofacial, hand, and distal leg muscles. Many patients also experience difficulties with cognition, cardiac arrhythmias, respiratory failure, or cataracts. Currently there is no treatment to slow progression or reverse the symptoms.	Myopathies	À vérifier	Ralentissement de la progression + Traitement symptomatique	United States	À vérifier
Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)Building on previous work of the Myotonic Dystrophy Clinical Research Network (DMCRN), the present study seeks to overcome insufficient data on natural history; lack of reliable biomarkers; and incomplete characterization and limited biological understanding of the phenotypic heterogeneity of Myotonic Dystrophy 1 by examining strategies to improve the reliability by making further refinements in our sample collection and analysis procedures by developing strategies for managing patient heterogeneity going forward. Funding Source- FDA OOPD	Myopathies	À vérifier	À vérifier	United States, Canada, Germany, Italy, Netherlands, New Zealand, United Kingdom	À vérifier
GRASP-01-002 — Defining Endpoints in Becker Muscular DystrophyThis is a 24-month, observational study of 50 participants with Becker muscular dystrophy (BMD)	Myopathies	À vérifier	À vérifier	United States, New Zealand, United Kingdom	À vérifier
GRASP-01-003 — LGMD R1 Natural History StudyThis is a 24-month, observational study of 100 participants with Limb Girdle Muscular Dystrophy type R1, also known as CAPN3.	Myopathies	À vérifier	À vérifier	United States	À vérifier
PsyMINT — mHealth Intervention for Pain Self ManagementThis is a feasibility pilot test of a single-arm intervention to evaluate the beta version of an mHealth app-based behavioral intervention prior to scaling for a randomized controlled trial (RCT). This mHealth intervention is designed to enhance self-efficacy and support pain and symptom self-management among post-treatment cancer survivors.	Alzheimer	Non applicable	Traitement symptomatique	United States	À vérifier
TREAT-EXT — Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy ExtensionThis is a natural history study to improve the types of assessments and biological samples that will be used in clinical drug trials in both congenital myotonic dystrophy and childhood myotonic dystrophy.	Myopathies	À vérifier	À vérifier	United States, Brazil	À vérifier
TREAT-EXT — Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy ExtensionThis is a natural history study to improve the types of assessments and biological samples that will be used in clinical drug trials in both congenital myotonic dystrophy and childhood myotonic dystrophy.	Myopathies	À vérifier	À vérifier	United States, Brazil	À vérifier
END-EXT — Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) ExtensionMyotonic Dystrophy type 1 (DM1) is an autosomal dominant multisystemic disorder that causes progressive disability and shortened life expectancy. It is characterized by progressive weakness and myotonia, which preferentially affects the craniofacial, hand, and distal leg muscles. Many patients also experience difficulties with cognition, cardiac arrhythmias, respiratory failure, or cataracts. Currently there is no treatment to slow progression or reverse the symptoms.	Myopathies	À vérifier	Ralentissement de la progression + Traitement symptomatique	United States	À vérifier
GRASP-01-003 — LGMD R1 Natural History StudyThis is a 24-month, observational study of 100 participants with Limb Girdle Muscular Dystrophy type R1, also known as CAPN3.	Myopathies	À vérifier	À vérifier	United States	À vérifier
ASPIRE-DM1 — DMCRN-02-001: Assessing Pediatric Endpoints in DM1The overall goal of the study is to establish valid clinical endpoint assessments for children with congenital myotonic dystrophy type 1 and develop biomarkers for the condition.	Myopathies	À vérifier	À vérifier	United States, Italy	À vérifier
Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)Building on previous work of the Myotonic Dystrophy Clinical Research Network (DMCRN), the present study seeks to overcome insufficient data on natural history; lack of reliable biomarkers; and incomplete characterization and limited biological understanding of the phenotypic heterogeneity of Myotonic Dystrophy 1 by examining strategies to improve the reliability by making further refinements in our sample collection and analysis procedures by developing strategies for managing patient heterogeneity going forward. Funding Source- FDA OOPD	Myopathies	À vérifier	À vérifier	United States, Canada, Germany, Italy, Netherlands, New Zealand, United Kingdom	À vérifier
GRASP-01-002 — Defining Endpoints in Becker Muscular DystrophyThis is a 24-month, observational study of 50 participants with Becker muscular dystrophy (BMD)	Myopathies	À vérifier	À vérifier	United States, New Zealand, United Kingdom	À vérifier
PsyMINT — mHealth Intervention for Pain Self ManagementThis is a feasibility pilot test of a single-arm intervention to evaluate the beta version of an mHealth app-based behavioral intervention prior to scaling for a randomized controlled trial (RCT). This mHealth intervention is designed to enhance self-efficacy and support pain and symptom self-management among post-treatment cancer survivors.	Alzheimer	Non applicable	Traitement symptomatique	United States	À vérifier

Essais cliniques

7

Molécule	Indication / population	Phase	NCT	Titre	Statut
PsyMINT — mHealth Intervention for Pain Self Management	Alzheimer	Non applicable	NCT07332377	PsyMINT — mHealth Intervention for Pain Self Management	COMPLETED
GRASP-01-002 — Defining Endpoints in Becker Muscular Dystrophy	Myopathies	À vérifier	NCT05257473	GRASP-01-002 — Defining Endpoints in Becker Muscular Dystrophy	ACTIVE_NOT_RECRUITING
Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)	Myopathies	À vérifier	NCT03981575	Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)	RECRUITING
ASPIRE-DM1 — DMCRN-02-001: Assessing Pediatric Endpoints in DM1	Myopathies	À vérifier	NCT05224778	ASPIRE-DM1 — DMCRN-02-001: Assessing Pediatric Endpoints in DM1	RECRUITING
GRASP-01-003 — LGMD R1 Natural History Study	Myopathies	À vérifier	NCT05618080	GRASP-01-003 — LGMD R1 Natural History Study	ACTIVE_NOT_RECRUITING
END-EXT — Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension	Myopathies	À vérifier	NCT07700225	END-EXT — Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension	RECRUITING
TREAT-EXT — Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension	Myopathies	À vérifier	NCT06747884	TREAT-EXT — Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension	RECRUITING

Publications

0

Molécule	Indication / population	Titre	Journal	Date
Aucune publication.