Sham Procedure ( DrugBank: - )


1 disease
IDDisease name (Link within this page)Number of trials
3Spinal muscular atrophy4

3. Spinal muscular atrophy


Clinical trials : 217 Drugs : 149 - (DrugBank : 33) / Drug target genes : 54 - Drug target pathways : 80
No.TrialIDDate_
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agemin
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PhaseCountries
1JPRN-jRCT2061200040
03/08/202125/12/2020Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyEscalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Spinal Muscular Atrophy- Part A: Participants with later-onset SMA will receive loading doses of 28 mg of nusinersen intrathecally on Days 1, 15 and 29 followed by maintenance doses of 28 mg on Days 149 and 269.

- Active Comparator
Part B: Participants with infantile- or later-onset SMA will receive loading doses of 12 mg of nusinersen intrathecally on Days 1, 15, 29, and 64 followed by maintenance doses of 12 mg on Days 183 and 279. Sham procedure will be administered on Day 135.

- 50/28 mg Group
Part B: Participants with infantile- or later-onset SMA will receive loading doses of 50 mg of nusinersen intrathecally on Days 1 and 15 followed by maintenance doses of 28 mg on Days 135 and 279. Sham procedure will be administered on Days 29, 64 and 183.

- Part C: Participants who have been receiving the approved dose for at least 1 year prior to entry in this study, will receive a single bolus dose of 50 mg of nusinersen intrathecally on Day 1 (4 months after their most recent maintenance dose) followed by maintenance doses of 28 mg on Days 121 and 241.
Berger ZdenekNULLRecruitingNot applicableNot applicableBoth152Phase 2-3Spain;Taiwan;United States;Japan;Estonia;Ireland;Latvia;Colombia
2NCT02462759
(ClinicalTrials.gov)
August 19, 201514/5/2015A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).A Phase 2, Randomized, Double-blind, Sham-procedure Controlled Study to Assess the Safety and Tolerability and Explore the Efficacy of ISIS 396443 (BIIB058) Administered Intrathecally in Subjects With Spinal Muscular Atrophy Who Are Not Eligible to Participate in the Clinical Studies ISIS 396443-CS3B or ISIS 396443-CS4Spinal Muscular AtrophyDrug: Nusinersen;Procedure: Sham ProcedureBiogenNULLTerminatedN/AN/AAll21Phase 2United States;Germany
3NCT02292537
(ClinicalTrials.gov)
November 24, 201412/11/2014A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Later-onset Spinal Muscular AtrophySpinal Muscular AtrophyDrug: Nusinersen;Procedure: Sham procedureBiogenNULLCompleted2 Years12 YearsAll126Phase 3United States;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom
4NCT02193074
(ClinicalTrials.gov)
August 19, 201414/7/2014A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular AtrophyA Phase 3, Randomized, Double-Blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Infantile-onset Spinal Muscular AtrophySpinal Muscular AtrophyDrug: nusinersen;Procedure: Sham procedureBiogenNULLTerminatedN/A210 DaysAll122Phase 3United States;Australia;Belgium;Canada;France;Germany;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;Hong Kong;Taiwan