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CLINICAL TRIALS PROFILE FOR SPINRAZA

All Clinical Trials for Spinraza

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01494701 ↗	An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)	Completed	Biogen	Phase 1	2011-11-30	This objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics of a single dose of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA).
NCT01494701 ↗	An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)	Completed	Ionis Pharmaceuticals, Inc.	Phase 1	2011-11-30	This objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics of a single dose of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA).
NCT01703988 ↗	An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy	Completed	Biogen	Phase 1/Phase 2	2012-10-31	This study will test the safety, tolerability, and pharmacokinetics of escalating doses of nusinersen (ISIS 396443) administered into the spinal fluid either two or three times over the duration of the trial, in participants with spinal muscular atrophy (SMA). Four dose levels will be evaluated sequentially. Each dose level will be studied in a cohort of approximately 8 participants, where all participants will receive active drug.
NCT01703988 ↗	An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy	Completed	Ionis Pharmaceuticals, Inc.	Phase 1/Phase 2	2012-10-31	This study will test the safety, tolerability, and pharmacokinetics of escalating doses of nusinersen (ISIS 396443) administered into the spinal fluid either two or three times over the duration of the trial, in participants with spinal muscular atrophy (SMA). Four dose levels will be evaluated sequentially. Each dose level will be studied in a cohort of approximately 8 participants, where all participants will receive active drug.
NCT01839656 ↗	A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)	Completed	Biogen	Phase 2	2013-05-08	The primary objective is to examine the clinical efficacy of multiple doses of nusinersen (ISIS 396443) administered intrathecally to participants with Infantile-Onset Spinal Muscular Atrophy (SMA). The secondary objectives are to examine the safety and tolerability of multiple doses of nusinersen administered intrathecally to participants with infantile-onset SMA and to examine the cerebral spinal fluid (CSF) and plasma Pharmacokinetics (PK) of multiple doses of nusinersen administered intrathecally to participants with infantile-onset SMA.
NCT01839656 ↗	A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)	Completed	Ionis Pharmaceuticals, Inc.	Phase 2	2013-05-08	The primary objective is to examine the clinical efficacy of multiple doses of nusinersen (ISIS 396443) administered intrathecally to participants with Infantile-Onset Spinal Muscular Atrophy (SMA). The secondary objectives are to examine the safety and tolerability of multiple doses of nusinersen administered intrathecally to participants with infantile-onset SMA and to examine the cerebral spinal fluid (CSF) and plasma Pharmacokinetics (PK) of multiple doses of nusinersen administered intrathecally to participants with infantile-onset SMA.

Clinical Trial Conditions for Spinraza

Condition Name

Condition Name for Spinraza
Intervention	Trials
Spinal Muscular Atrophy	10
Muscular Atrophy, Spinal	2

Condition MeSH

Condition MeSH for Spinraza
Intervention	Trials
Muscular Atrophy, Spinal	12
Muscular Atrophy	12
Atrophy	12

Clinical Trial Locations for Spinraza

Trials by Country

Trials by Country for Spinraza
Location	Trials
United States	70
Canada	8
Japan	7
Australia	6
Italy	5

Trials by US State

Trials by US State for Spinraza
Location	Trials
New York	8
California	7
Utah	7
Massachusetts	7
Texas	6

Clinical Trial Progress for Spinraza

Clinical Trial Phase

Clinical Trial Phase for Spinraza
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	4
Phase 2	3

Clinical Trial Status

Clinical Trial Status for Spinraza
Clinical Trial Phase	Trials
Completed	4
Active, not recruiting	2
Terminated	2

Clinical Trial Sponsors for Spinraza

Sponsor Name

Sponsor Name for Spinraza
Sponsor	Trials
Biogen	11
Ionis Pharmaceuticals, Inc.	8
Winthrop University Hospital	1

Sponsor Type

Sponsor Type for Spinraza
Sponsor	Trials
Industry	19
Other	2