Success Metrics

Clinical Success Rate
80.0%

Based on 4 completed trials

Completion Rate
80%(4/5)
Active Trials
2(29%)
Results Posted
125%(5 trials)
Terminated
1(14%)

Phase Distribution

Ph phase_1
1
14%
Ph phase_3
5
71%

Phase Distribution

1

Early Stage

0

Mid Stage

5

Late Stage

Phase Distribution6 total trials
Phase 1Safety & dosage
1(16.7%)
Phase 3Large-scale testing
5(83.3%)

Highest Phase Reached

Phase 3

Trial Status & Enrollment

Completion Rate

80.0%

4 of 5 finished

Non-Completion Rate

20.0%

1 ended early

Currently Active

2

trials recruiting

Total Trials

7

all time

Status Distribution
Active(2)
Completed(4)
Terminated(1)

Detailed Status

Completed4
Active, not recruiting2
Terminated1

Development Timeline

Analytics

Development Status

Total Trials
7
Active
2
Success Rate
80.0%
Most Advanced
Phase 3

Trials by Phase

Phase 11 (16.7%)
Phase 35 (83.3%)

Trials by Status

active_not_recruiting229%
completed457%
terminated114%

Recent Activity

2 active trials
Showing 5 of 7
terminatedphase_1

Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy

NCT03381729
completedphase_3

Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies

NCT03837184
completedphase_3

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

NCT03505099
completedphase_3

Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1

NCT03461289
completedphase_3

Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1

NCT03306277
View all 7 trials

Clinical Trials (7)

Showing 7 of 7 trials
NCT03381729Phase 1

Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy

Terminated
NCT03837184Phase 3

Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies

Completed
NCT03505099Phase 3

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

Completed
NCT03461289Phase 3

Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1

Completed
NCT03306277Phase 3

Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1

Completed
NCT04042025Phase 3

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

Active Not Recruiting
NCT03421977

Long-Term Follow-up Study for Patients From AVXS-101-CL-101

Active Not Recruiting

All 7 trials loaded

Drug Details

Intervention Type
BIOLOGICAL
Total Trials
7