Phase 3

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

September 10, 2020

https://clinicaltrials.gov/ct2/show/NCT03837184?type=Intr&cond=spinal+muscular+atrophy&phase=0124&lupd_s=08%2F27%2F2020&lupd_d=14&sort=nwst

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

DESCRIPTION:
Condition: Spinal Muscular Atrophy Type I
Intervention: Biological: Onasemnogene Abeparvovec-xioi
Sponsors: AveXis, Inc.; PRA Health Sciences
Active, not recruiting

CLINICALTRIALS.GOV IDENTIFIER:
NCT03837184

FIRST POSTED:
Tue, 12 Feb 2019 12:00:00 EST

LAST UPDATE POSTED:
09/10/20 07:19AM

STUDY LINK / URL:
https://clinicaltrials.gov/ct2/show/NCT03837184?type=Intr&cond=spinal+muscular+atrophy&phase=0124&lupd_s=08%2F27%2F2020&lupd_d=14&sort=nwst

A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants

July 22, 2020

https://clinicaltrials.gov/ct2/show/NCT02908685?type=Intr

https://clinicaltrials.gov/ct2/show/NCT02908685?type=Intr&cond=Spinal+Muscular+Atrophy&lupd_s=06%2F19%2F2020&lupd_d=14&sort=nwst

STUDY TITLE:
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants

DESCRIPTION:
Condition: Muscular Atrophy, Spinal
Interventions: Drug: Placebo; Drug: Risdiplam
Sponsor: Hoffmann-La Roche
Active, not recruiting

CLINICALTRIALS.GOV IDENTIFIER:
NCT02908685

FIRST POSTED:
Wed, 21 Sep 2016 12:00:00 EDT

LAST UPDATE POSTED:
07/03/20 07:39PM

STUDY LINK / URL:
https://clinicaltrials.gov/ct2/show/NCT02908685?type=Intr&cond=Spinal+Muscular+Atrophy&lupd_s=06%2F19%2F2020&lupd_d=14&sort=nwst

Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

June 29, 2020

https://clinicaltrials.gov/ct2/show/NCT04089566?type=Intr

https://clinicaltrials.gov/ct2/show/NCT04089566?type=Intr&cond=spinal+muscular+atrophy&phase=0124&lupd_s=06%2F24%2F2020&lupd_d=14&sort=nwst

STUDY TITLE:
Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

DESCRIPTION:
Condition: Muscular Atrophy, Spinal
Intervention: Drug: Nusinersen
Sponsor: Biogen
Recruiting

CLINICALTRIALS.GOV IDENTIFIER:
NCT04089566

FIRST POSTED:
Fri, 13 Sep 2019 12:00:00 EDT

LAST UPDATE POSTED:
07/08/20 02:33PM

STUDY LINK / URL:
https://clinicaltrials.gov/ct2/show/NCT04089566?type=Intr&cond=spinal+muscular+atrophy&phase=0124&lupd_s=06%2F24%2F2020&lupd_d=14&sort=nwst

Single-Dose Gene Replacement Therapy for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion

April 28, 2020

https://clinicaltrials.gov/ct2/show/NCT03837184

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

Study Type : Interventional (Clinical Trial)
Estimated Enrollment : 6 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : May 15, 2019
Estimated Primary Completion Date : April 1, 2021
Estimated Study Completion Date : April 1, 2021