CompletedPhase 3

Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2

United States30 participantsStarted 2018-04-02

Plain-language summary

To evaluate the safety and efficacy of intravenous onasemnogene abeparvovec-xioi in pre-symptomatic patients with SMA and 2 or 3 copies SMN2

Age range42 Days

SexALL

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Cohort 1: Number of Participants Who Achieved Sitting Alone for at Least 30 Seconds

Timeframe: From Day 1 up to 18 months of age visit

Cohort 2: Number of Participants Who Achieved Standing Alone for at Least 3 Seconds

Timeframe: From Day 1 up to 24 months of age visit

NCT IDNCT03505099

SponsorNovartis Gene Therapies

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2021-06-15

Results submitted2021-12-14