Long-term Follow-up Study of Patients Receiving OnasemnogeneAbeparvovec-xioi
A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101 A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving ...
Spinal Muscular Atrophy Type I;Spinal Muscular Atrophy Type II;Spinal Muscular Atrophy Type III;SMA
Biological: OnasemnogeneAbeparvovec-xioi
Novartis Gene Therapies
NULL
Active, not recruiting
N/A
N/A
All
85
Phase 3
United States;Australia;Belgium;Canada;France;Italy;Japan;Taiwan;United Kingdom;Korea, Republic of;Spain United States;Australia;Belgium;Canada;France;Italy;Japan;Taiwan;United Kingdom;Korea, Republic of;S ...
Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One ...
Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients Wi ...
Pre-Symptomatic Study of Intravenous OnasemnogeneAbeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 Pre-Symptomatic Study of Intravenous OnasemnogeneAbeparvovec-xioiin Spinal Muscular Atrophy (SMA) f ...
A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants With Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy With Multiple Copies of SMN2 A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants With Genetically Diagnose ...
Spinal Muscular Atrophy
Biological: onasemnogeneabeparvovec-xioi
Novartis Gene Therapies
PRA Health Sciences
Completed
N/A
42 Days
All
30
Phase 3
United States;Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Republic of;Spain;Taiwan;United Kingdom United States;Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Republic of;Spain;Taiwan;Un ...
Study of Intrathecal Administration of OnasemnogeneAbeparvovec-xioi for Spinal Muscular Atrophy
Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting But Non-ambulatory Patients With Spinal Muscular Atrophy Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting But Non-ambulatory Patients With ...
Long-Term Follow-up Study for Patients From AVXS-101-CL-101
A Long Term Follow up Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS 101 A Long Term Follow up Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clini ...