A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies (SHINE)
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| ClinicalTrials.gov Identifier: NCT02594124 |
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Recruitment Status :
Active, not recruiting
First Posted : November 1, 2015
Last Update Posted : August 9, 2023
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Sponsor:
Biogen
Information provided by (Responsible Party):
Biogen
- Study Details
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Brief Summary:
The primary objective is to evaluate the long-term safety and tolerability of nusinersen (ISIS 396443) administered by intrathecal (IT) injection to participants with Spinal Muscular Atrophy (SMA) who previously participated in investigational studies of nusinersen. The secondary objective is to examine the long-term efficacy of nusinersen administered by IT injection to participants with SMA who previously participated in investigational studies of nusinersen.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Spinal Muscular Atrophy | Drug: nusinersen | Phase 3 |
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 : | 292 participants |
| Allocation: | Non-Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | None (Open Label) |
| Masking Description: | During the blinded loading period, the following participants will be masked:
After the loading period has been completed, subsequent doses will be unblinded. |
| Primary Purpose: | Treatment |
| Official Title: | An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 |
| Actual Study Start Date : | November 4, 2015 |
| Estimated Primary Completion Date : | August 29, 2023 |
| Estimated Study Completion Date : | August 29, 2023 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Spinal muscular atrophy
MedlinePlus related topics:
Spinal Muscular Atrophy
Drug Information
available for:
Nusinersen
| Arm | Intervention/treatment |
|---|---|
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Experimental: Group 1
Participants transitioned from ISIS 396443-CS3B (NCT02193074)
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Drug: nusinersen
Administered by intrathecal (IT) injection
Other Names:
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Experimental: Group 2
Participants transitioned from ISIS 396443-CS4 (NCT02292537)
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Drug: nusinersen
Administered by intrathecal (IT) injection
Other Names:
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Experimental: Group 3
Participants transitioned from ISIS 396443-CS12 (NCT02052791)
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Drug: nusinersen
Administered by intrathecal (IT) injection
Other Names:
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Experimental: Group 4
Participants transitioned from ISIS 396443-CS3A (NCT01839656)
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Drug: nusinersen
Administered by intrathecal (IT) injection
Other Names:
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Experimental: Group 5
Participants transitioned from 232SM202 (NCT02462759)
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Drug: nusinersen
Administered by intrathecal (IT) injection
Other Names:
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Primary Outcome Measures :
- Number of participants experiencing Adverse events (AEs) and/or Serious Adverse Events (SAEs) [ Time Frame: Up to Day 1814 ]
- Number of participants with clinically significant vital sign abnormalities [ Time Frame: Up to Day 1814 ]
- Number of participants with clinically significant weight abnormalities [ Time Frame: Up to Day 1814 ]
- Number of participants with clinically significant neurological examination abnormalities [ Time Frame: Up to Day 1814 ]
- Number of participants with clinically significant laboratory assessment abnormalities [ Time Frame: Up to Day 1814 ]
- Number of participants with clinically significant coagulation parameter abnormalities [ Time Frame: Up to Day 1814 ]
- Number of participants with clinically significant 12-lead electrocardiograms (ECGs) abnormalities [ Time Frame: Up to Day 1814 ]
- Change from Baseline in concomitant medications [ Time Frame: Up to Day 1814 ]
Secondary Outcome Measures :
- Percentage of participants who attained motor milestones as assessed by World Health Organization (WHO) criteria [ Time Frame: Up to Day 1814 ]
- Percentage of participants who attained motor milestones as assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) [ Time Frame: Up to Day 1814 ]
- Time to death or permanent ventilation [ Time Frame: Up to Day 1814 ]
- Percentage of participants not requiring permanent ventilation [ Time Frame: Up to Day 1814 ]
- Change from Baseline in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) motor function scale [ Time Frame: Up to Day 1814 ]CHOP-INTEND tests includes 16 items structured to move from easiest to hardest with the grading including gravity eliminated (lower scores) to antigravity movements (higher scores). All item scores range from 0-4.
- Change from Baseline in Hammersmith Functional Motor Scale [ Time Frame: Up to Day 1814 ]The HFMSE tests motor function of participants with SMA. The original 20 item Hammersmith Functional Motor Scale was expanded to include 13 additional adapted items from the Gross Motor Function Measure to improve sensitivity for the higher functioning ambulant population.
- Change from Baseline in Revised Upper Limb Module (RULM) [ Time Frame: Up to Day 1814 ]
- Change from Baseline in 6-Minute Walk Test (6MWT) [ Time Frame: Up to Day 1814 ]6MWT: walking up and down a 25 meter track without aids or orthotics as fast as possible for 6 minutes. Lap splits, minute splits and total distance are recorded, in addition to any rests and falls.
- Change from Baseline in Compound Muscular Action Potential (CMAP) [ Time Frame: Up to Day 1814 ]CMAP is an electrophysiological technique that can be used to determine the approximate number of motor neurons in a muscle or group of muscles.
- Change from Baseline in body length and/or height (for all participants) [ Time Frame: Up to Day 1814 ]
- Change from Baseline in head circumference (for participants up to 36 months of age) [ Time Frame: Up to Day 1814 ]
- Change from Baseline in chest circumference (for participants up to 36 months of age) [ Time Frame: Up to Day 1814 ]
- Change from Baseline in arm circumference (for participants up to 36 months of age) [ Time Frame: Up to Day 1814 ]
- Proportion of CMAP responders [ Time Frame: Up to Day 1814 ]
- Number of participants with motor milestones achieved [ Time Frame: Up to Day 1814 ]
- Proportion of participants who achieved standing alone [ Time Frame: Up to Day 1814 ]
- Proportion of participants who achieved walking with assistance [ Time Frame: Up to Day 1814 ]
- Number of participants with serious respiratory events [ Time Frame: Up to Day 1814 ]
- Number of participants hospitalized [ Time Frame: Up to Day 1814 ]
- Duration of hospitalizations [ Time Frame: Up to Day 1814 ]
- Change from Baseline in Cobb-Angle on X-Ray of the thoracolumbar spine [ Time Frame: Up to Day 1814 ]
- Change from Baseline in Quality of Life (QOL) Questionnaires [ Time Frame: Up to Day 1814 ]
- Number of Disease-related hospitalizations and AEs [ Time Frame: Up to Day 1814 ]
- Overall survival rate [ Time Frame: Up to Day 1814 ]
Information from the National Library of Medicine
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | Child, Adult, Older Adult |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Key Inclusion Criteria:
- Signed informed consent of parent or guardian and signed informed assent of participant, if indicated per participant's age and institutional guidelines.
- Completion of the index study in accordance with the study protocol or as a result of Sponsor decision (e.g., early termination of the index study) within the preceding 16 weeks
Key Exclusion Criteria:
- Have any condition or worsening condition which in the opinion of the Investigator would make the participant unsuitable for enrollment, or could interfere with the participant participating in or completing the study
- Clinically significant abnormalities in hematology or clinical chemistry parameters or electrocardiogram (ECG), as assessed by the Site Investigator, at the Screening visit that would render the participant unsuitable for participation in the study
- Participant's parent or legal guardian is not willing or able to meet standard of care guidelines (including vaccinations and respiratory syncytial virus prophylaxis if available), nor provide nutritional and respiratory support throughout the study
- Treatment with another investigational agent, biological agent, or device within one month of Screening, or 5 half-lives of study agent, whichever is longer
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Information from the National Library of Medicine
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): NCT02594124
Locations
Show 47 study locations
Sponsors and Collaborators
Biogen
Investigators
| Study Director: | Medical Director | Biogen |
Additional Information:
| Responsible Party: | Biogen |
| ClinicalTrials.gov Identifier: | NCT02594124 |
| Other Study ID Numbers: |
ISIS 396443-CS11 2015-001870-16 ( EudraCT Number ) |
| First Posted: | November 1, 2015 Key Record Dates |
| Last Update Posted: | August 9, 2023 |
| Last Verified: | August 2023 |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
| Product Manufactured in and Exported from the U.S.: | No |
Keywords provided by Biogen:
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SMA SMN SMNRx ISIS-SMNRx ISIS 396443 |
SHINE IONIS-SMNRx IONIS-SMN Rx Spinraza nusinersen |
Additional relevant MeSH terms:
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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 |