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Developmental Phase

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The expected start time interval of the test

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  • Hospital/Foundation

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  • Asia Regional PI

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Investigator-initiated trial(IIT)

  • Yes

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Recruiting

List

5Cases

2020-08-15 - 2035-12-31

Phase III

Active
A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101

  • Condition/Disease

    Spinal Muscular Atrophy

  • Test Drug

    AVXS-101

Participate Sites
1Sites

Recruiting1Sites

2021-09-30 - 2023-08-01

Phase III

Completed
A Phase IIIb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA)

  • Condition/Disease

    Pediatric patients with spinal muscular atrophy (SMA)

  • Test Drug

    OAV101

Participate Sites
2Sites

Recruiting1Sites

Study ended1Sites

2023-01-10 - 2031-02-26

Phase IV

Active

  • Condition/Disease

  • Test Drug

Participate Sites
2Sites

Recruiting2Sites

2019-06-01 - 2021-08-01

Phase III

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

  • Condition/Disease

    Spinal Muscular Atrophy Type 1

  • Test Drug

    AVXS-101

Participate Sites
1Sites

Recruiting1Sites

2023-01-10 - 2035-06-15

Phase IV

  • Condition/Disease

  • Test Drug

Participate Sites
2Sites

Recruiting2Sites

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