A Phase III: randomized, sham-controlled, double-blind trial to evaluate the efficacy and safety of intrathecal (IT) OAV101, single dose (1.2E4vg) in later onset type 2 SMA age > 2 to

Study Details	To compare the efficacy of OAV101 IT vs. sham control as measured by the change from baseline in HFMSE total score
Protocol Number	COAV101B12301
Phase	III
Therapeutic Area	Paediatrics with Type 2 SMA
Subject Types	With Medical Condition
Indication	Type 2 SMA
Principal Investigator	A/Prof Stacey Tay
Investigator Product / Device	OAV101
Sponsor	Novartis (Singapore) Pte Ltd

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A Phase III: randomized, sham-controlled, double-blind trial to evaluate the efficacy and safety of intrathecal (IT) OAV101, single dose (1.2E4vg) in later onset type 2 SMA age > 2 to <18 years of age, treatment naïve, sitting and never ambulatory (COAV101B12301)