An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246)
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ClinicalTrials.gov Identifier: NCT02052791 |
Recruitment Status :
Completed
First Posted : February 3, 2014
Last Update Posted : February 16, 2021
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Spinal Muscular Atrophy | Drug: nusinersen | Phase 1 |
Expanded Access : An investigational treatment associated with this study is no longer available outside the clinical trial. More info ...
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 47 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | An Open-label Study to Assess the Safety and Tolerability of ISIS 396443 in Patients With Spinal Muscular Atrophy Who Previously Participated in 396443-CS2 or 396443-CS10 |
Actual Study Start Date : | January 31, 2014 |
Actual Primary Completion Date : | January 31, 2017 |
Actual Study Completion Date : | January 31, 2017 |

Arm | Intervention/treatment |
---|---|
Experimental: nusinersen |
Drug: nusinersen
Administered by intrathecal (IT) injection
Other Names:
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- Number of participants that experience Adverse Events (AEs) and Serious Adverse Events [ Time Frame: Up to 24 Months ]
- Number of participants with clinically significant neurological examination abnormalities [ Time Frame: Up to 24 Months ]
- Number of participants with clinically significant vital sign abnormalities [ Time Frame: Up to 24 Months ]
- Number of participants with clinically significant physical examination abnormalities [ Time Frame: Up to 24 Months ]
- Number of participants with clinically significant weight abnormalities [ Time Frame: Up to 24 Months ]
- Number of participants with clinically significant laboratory parameters [ Time Frame: Up to 24 Months ]
- Number or participants with clinically significant cerbrospinal fluid (CSF) laboratory parameters [ Time Frame: Up to Day 176 ]
- Number of participants with clinically significant electrocardiograms (ECGs) abnormalities [ Time Frame: Up to 24 Months ]
- Change from Baseline in concomitant medications [ Time Frame: Up to 24 Months ]
- PK parameters of nusinersen (ISIS 396443) in CSF levels: Maximum observed plasma drug concentration (Cmax) [ Time Frame: Pre-dose Day 176, Day 358 and Day 540 ]
- PK parameters of nusinersen in CSF levels: Time to reach maximum observed concentration (Tmax) [ Time Frame: Pre-Dose Day 176, Day 358 and Day 540 ]
- PK parameters of nusinersen in CSF levels: Area under the plasma concentrations time curve from the time of the intrathecal (IT) dose to the last collected sample (AUCinf) [ Time Frame: Pre-Dose Day 176, Day 358 and Day 540 ]

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Ages Eligible for Study: | Child, Adult, Older Adult |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Key Inclusion Criteria:
- Clinical signs attributable to Spinal Muscular Atrophy
- Satisfactory completion of dosing and all study visits in ISIS 396443-CS2 (NCT01703988) or ISIS 396443 CS10 (NCT01780246) with an acceptable safety profile, per Investigator judgement.
- Able to complete all study procedures, measurements and visits and parent/participant has adequately supportive psychosocial circumstances, in the opinion of the investigator
- Estimated life expectancy > 2 years from Screening
- Meets age-appropriate institutional criteria for use of anesthesia/sedation, if use is planned for study procedure
Key Exclusion Criteria:
- Have any new or worsening of existing condition which in the opinion of the Investigator would make the subject unsuitable for enrollment, or could interfere with the participant participating in or completing the study.
- Dosing in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246) within 180 days (6 months) of screening, or longer ago than 396 days (13 months) from screening
- Hospitalization for surgery (i.e. scoliosis surgery) or pulmonary event within 2 months of screening or planned during the duration of the study
- Presence of an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy
- Clinically significant abnormalities in hematology or clinical chemistry parameters
- Treatment with another investigational drug, biological agent, or device within 1-month of Screening or 5 half-lives of study agent, whichever is longer. Any history of gene therapy or cell transplantation.
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02052791
United States, Massachusetts | |
Boston Children's Hospital | |
Boston, Massachusetts, United States, 02115 | |
United States, New York | |
Columbia University Medical Center | |
New York, New York, United States, 10032 | |
United States, Texas | |
UT Southwestern Medical Center - Children's Medical Center Dallas | |
Dallas, Texas, United States, 75207 | |
United States, Utah | |
University of Utah School of Medicine | |
Salt Lake City, Utah, United States, 84132 |
Study Director: | Medical Director | Biogen |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Biogen |
ClinicalTrials.gov Identifier: | NCT02052791 |
Other Study ID Numbers: |
ISIS 396443-CS12 |
First Posted: | February 3, 2014 Key Record Dates |
Last Update Posted: | February 16, 2021 |
Last Verified: | February 2021 |
Spinal Muscular Atrophy SMA SMN SMNRx ISIS-SMNRx |
ISIS 396443 IONIS-SMNRx IONIS-SMN Rx Spinraza |
Muscular Atrophy Muscular Atrophy, Spinal Atrophy Pathological Conditions, Anatomical Neuromuscular Manifestations Neurologic Manifestations |
Nervous System Diseases Spinal Cord Diseases Central Nervous System Diseases Motor Neuron Disease Neurodegenerative Diseases Neuromuscular Diseases |