AO GENERIUM — Biomedical Watch V2

Traitements2programmes

Essais1liés

Publications0liées

SourceDBlocale

Traitements

2

Molécule	Indication / population	Phase	Objectif	Pays	Résultat
GNR-097The study will evaluate the tolerability, safety and efficacy of gene therapy product in boys with Duchenne muscular dystrophy (DMD). In Phase I the participants will be included in two sequential dose cohorts with increasing doses of the investigational product. Based on the results of Phase I, the dose of the investigational product for use in Phase II will be determined. Phase II is a randomized, single-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene therapy at the beginning of the second year.	Myopathies	Phase 1/2	Thérapie génique	Belarus, Russia	À vérifier
GNR-097The study will evaluate the tolerability, safety and efficacy of gene therapy product in boys with Duchenne muscular dystrophy (DMD). In Phase I the participants will be included in two sequential dose cohorts with increasing doses of the investigational product. Based on the results of Phase I, the dose of the investigational product for use in Phase II will be determined. Phase II is a randomized, single-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene therapy at the beginning of the second year.	Myopathies	Phase 1/2	Thérapie génique	Belarus, Russia	À vérifier

Essais cliniques

1

Molécule	Indication / population	Phase	NCT	Titre	Statut
GNR-097	Myopathies	Phase 1/2	NCT07673809	A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy	RECRUITING

Publications

0

Molécule	Indication / population	Titre	Journal	Date
Aucune publication.