Solid Biosciences Inc. — Biomedical Watch V2

Traitements5programmes

Essais3liés

Publications1liées

SourceDBlocale

Traitements

5

Molécule	Indication / population	Phase	Objectif	Pays	Résultat
SGT-001This is a controlled, open-label, single-ascending dose study to evaluate the safety and tolerability of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Participants will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 5 years. The protocol was amended to drop the control arm after 4 participants were dosed.	Myopathies	Phase 1/2	À vérifier	United States	À vérifier
SGT-003This is a Phase 3, double-blind, placebo-controlled study with the primary objective of evaluating the efficacy of a single IV infusion of SGT-003 in pediatric ambulant male participants with DMD. The secondary objectives include the evaluation of additional efficacy and safety outcomes. The study will be divided into 2 parts. Participants will be randomized 1:1 to either SGT-003 in Part 1 followed by placebo in Part 2 or to placebo in Part 1 followed by SGT-003 in Part 2. Participants will continue to be monitored in long term follow up (LTFU) for at least 5 years from their SGT-003 dosing date.	Myopathies	Phase 3	À vérifier	United States, Australia, Canada	À vérifier
SGT-003This is a Phase 3, double-blind, placebo-controlled study with the primary objective of evaluating the efficacy of a single IV infusion of SGT-003 in pediatric ambulant male participants with DMD. The secondary objectives include the evaluation of additional efficacy and safety outcomes. The study will be divided into 2 parts. Participants will be randomized 1:1 to either SGT-003 in Part 1 followed by placebo in Part 2 or to placebo in Part 1 followed by SGT-003 in Part 2. Participants will continue to be monitored in long term follow up (LTFU) for at least 5 years from their SGT-003 dosing date.	Myopathies	Phase 3	Thérapie génique	United States, Australia, Canada	À vérifier
SGT-001This is a controlled, open-label, single-ascending dose study to evaluate the safety and tolerability of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Participants will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 5 years. The protocol was amended to drop the control arm after 4 participants were dosed.	Myopathies	Phase 1/2	À vérifier	United States	À vérifier
SGT-003This is a Phase 3, double-blind, placebo-controlled study with the primary objective of evaluating the efficacy of a single IV infusion of SGT-003 in pediatric ambulant male participants with DMD. The secondary objectives include the evaluation of additional efficacy and safety outcomes. The study will be divided into 2 parts. Participants will be randomized 1:1 to either SGT-003 in Part 1 followed by placebo in Part 2 or to placebo in Part 1 followed by SGT-003 in Part 2. Participants will continue to be monitored in long term follow up (LTFU) for at least 5 years from their SGT-003 dosing date.	Myopathies	Phase 1/2	Thérapie génique	United States, Canada, Italy, United Kingdom	À vérifier

Essais cliniques

3

Molécule	Indication / population	Phase	NCT	Titre	Statut
SGT-003	Myopathies	Phase 1/2	NCT06138639	A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)	RECRUITING
SGT-001	Myopathies	Phase 1/2	NCT03368742	IGNITE DMD — Microdystrophin Gene Transfer Study in Adolescents and Children With DMD	ACTIVE_NOT_RECRUITING
SGT-003	Myopathies	Phase 3	NCT07160634	A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)	RECRUITING

Publications

1

Molécule	Indication / population	Titre	Journal	Date
SGT-003		Gene therapy in Duchenne muscular dystrophy.	Archives de pediatrie : organe officiel de la Societe francaise de pediatrie