A Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Males with Duchenne Muscular Dystrophy

Conditions

Duchenne muscular dystrophy

Brief summary

Incidence of treatment-emergent adverse events (AEs) through Day 360, Change from baseline of microdystrophin protein levels at Day 90

Detailed description

Change from baseline of microdystrophin tissue distribution at Day 90, Change from baseline of microdystrophin tissue distribution at Day 360, Change from baseline of microdystrophin protein levels at Day 360, Change from baseline in time to rise velocity at Day 360 and Day 540, Change from baseline in stride velocity 95th centile (SV95C) at Day 360 and Day 540, Change from baseline in 10-meter walk/run velocity at Day 360 and Day 540, Change from baseline in 4-stair climb velocity at Day 360 and Day 540, Change from baseline in the North Star Ambulatory Assessment (NSAA) score at Day 360 and Day 540, Change from baseline in 6-minute walk test (6MWT) distance at Day 360 and Day 540, Incidence of clinically significant laboratory abnormalities through Day 360 and Day 540, Incidence of clinically significant abnormalities in vital signs through Day 360 and Day 540, Incidence of clinically significant abnormalities in physical examinations through Day 360 and Day 540, Incidence of cardiac abnormalities by electrocardiogram (ECG) or echocardiography (ECHO) through Day 360 and Day 540

Related papers

No related papers found yet.

Interventions

None listed

Eligibility

Sex/Gender

Male

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Incidence of treatment-emergent adverse events (AEs) through Day 360, Change from baseline of microdystrophin protein levels at Day 90	—

Secondary

Measure	Time frame
Change from baseline of microdystrophin tissue distribution at Day 90, Change from baseline of microdystrophin tissue distribution at Day 360, Change from baseline of microdystrophin protein levels at Day 360, Change from baseline in time to rise velocity at Day 360 and Day 540, Change from baseline in stride velocity 95th centile (SV95C) at Day 360 and Day 540, Change from baseline in 10-meter walk/run velocity at Day 360 and Day 540, Change from baseline in 4-stair climb velocity at Day 360 and Day 540, Change from baseline in the North Star Ambulatory Assessment (NSAA) score at Day 360 and Day 540, Change from baseline in 6-minute walk test (6MWT) distance at Day 360 and Day 540, Incidence of clinically significant laboratory abnormalities through Day 360 and Day 540, Incidence of clinically significant abnormalities in vital signs through Day 360 and Day 540, Incidence of clinically significant abnormalities in physical examinations through Day 360 and Day 540, Incidence of cardiac	—

Measure

Time frame

Change from baseline of microdystrophin tissue distribution at Day 90, Change from baseline of microdystrophin tissue distribution at Day 360, Change from baseline of microdystrophin protein levels at Day 360, Change from baseline in time to rise velocity at Day 360 and Day 540, Change from baseline in stride velocity 95th centile (SV95C) at Day 360 and Day 540, Change from baseline in 10-meter walk/run velocity at Day 360 and Day 540, Change from baseline in 4-stair climb velocity at Day 360 and Day 540, Change from baseline in the North Star Ambulatory Assessment (NSAA) score at Day 360 and Day 540, Change from baseline in 6-minute walk test (6MWT) distance at Day 360 and Day 540, Incidence of clinically significant laboratory abnormalities through Day 360 and Day 540, Incidence of clinically significant abnormalities in vital signs through Day 360 and Day 540, Incidence of clinically significant abnormalities in physical examinations through Day 360 and Day 540, Incidence of cardiac

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Countries

Italy

Outcome results