Trial record 1 of 2 for:
Embrace | SMA
A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA). (EMBRACE)
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT02462759 |
Recruitment Status :
Terminated
(The study was terminated early to roll over participants to open label extension study NCT02594124.)
First Posted : June 4, 2015
Results First Posted : January 27, 2020
Last Update Posted : February 17, 2021
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Sponsor:
Biogen
Information provided by (Responsible Party):
Biogen
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Study Type | Interventional |
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Study Design | Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment |
Condition |
Spinal Muscular Atrophy |
Interventions |
Drug: Nusinersen Procedure: Sham Procedure |
Enrollment | 21 |
Participant Flow
Recruitment Details | Participants were recruited from sites in the US and Germany. Part 1 was terminated early as positive efficacy results were observed in interim analysis of study NCT02193074 and it was considered unethical to continue this part of study. Part 2 was also terminated early to rollover and continue to follow participants in study NCT02594124. |
Pre-assignment Details | Total of 21 participants with SMA were randomized in Part 1 of the study(7 participants in sham procedure group,14 participants in ISIS 396443 group).1 participant died in sham procedure group of Part 1.Total of 20 participants were enrolled to receive ISIS 396443 in open-label phase of Part 2.Integrated analysis was performed for Part 1 and 2. |
Arm/Group Title | Sham Procedure (Part 1) | ISIS 396443 (Part 1) | ISIS 396443 (Part 2) |
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Sham procedure on Day 1, 15, 29, 64, 183 and 302. | Single dose of 9.6 milligrams (mg) to 12.0 mg ISIS 396443, based on participant's age, intrathecal bolus injection loading doses on Day 1, 15, 29, 64 and maintenance doses, every 4 months, on Day 183 and 302. | Participants who were in Sham procedure group in Part 1, received single dose of 12.0 mg ISIS 396443 intrathecal bolus injection loading doses on Day 1, 15, 29, 64 and maintenance doses, every 4 months, on Day 183, 302, 421, 540, 659 and 778 in Part 2; participants who were in ISIS 396443 group in Part 1 continued to receive a single dose of 9.6 mg to 12.0 mg ISIS 396443 intrathecal bolus injection maintenance doses on Day 1, 120, 239, 358, 477, 596 and 715 in Part 2. |
Period Title: Part 1: Double Blind | |||
Started | 7 | 14 | 0 |
Completed | 6 | 14 | 0 |
Not Completed | 1 | 0 | 0 |
Reason Not Completed | |||
Death | 1 | 0 | 0 |
Period Title: Part 2: Open-Label Phase | |||
Started | 0 | 0 | 20 |
Completed | 0 | 0 | 20 |
Not Completed | 0 | 0 | 0 |
Baseline Characteristics
Arm/Group Title | Sham Procedure (Part 1) | ISIS 396443 (Part 1) | Total | |
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Sham procedure on Day 1, 15, 29, 64, 183 and 302. | Single dose of 9.6 milligrams (mg) to 12.0 mg ISIS 396443, based on participant's age, intrathecal bolus injection loading doses on Day 1, 15, 29, 64 and maintenance doses, every 4 months, on Day 183 and 302. | Total of all reporting groups | |
Overall Number of Baseline Participants | 7 | 14 | 21 | |
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The Intent-to-treat (ITT) population include all participants who were randomized, receive at least 1 dose of ISIS 396443 or sham procedure.
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Age, Continuous
Mean (Standard Deviation) Unit of measure: Months |
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Number Analyzed | 7 participants | 14 participants | 21 participants | |
24.4 (13.83) | 19.4 (10.12) | 21.1 (11.39) | ||
Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 7 participants | 14 participants | 21 participants | |
Female |
5 71.4%
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5 35.7%
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10 47.6%
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Male |
2 28.6%
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9 64.3%
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11 52.4%
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Race/Ethnicity, Customized
Measure Type: Count of Participants Unit of measure: Participants |
Number Analyzed | 7 participants | 14 participants | 21 participants |
Ethnicity: Hispanic or Latino |
2 28.6%
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1 7.1%
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3 14.3%
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Ethnicity: Not Hispanic or Latino |
4 57.1%
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9 64.3%
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13 61.9%
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Ethnicity: Not reported due to confidentiality |
1 14.3%
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4 28.6%
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5 23.8%
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Race: Asian |
3 42.9%
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2 14.3%
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5 23.8%
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Race: White |
2 28.6%
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7 50.0%
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9 42.9%
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Race: Other |
1 14.3%
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1 7.1%
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2 9.5%
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Race: Not reported due to confidentiality |
1 14.3%
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4 28.6%
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5 23.8%
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Outcome Measures
Adverse Events
Limitations and Caveats
Despite the early termination of both Parts of the study, the data from this study is of quality and reliable.
More Information
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts
the PI's rights to discuss or publish trial results after the trial is completed.
Our agreement is subject to confidentiality but generally the PI can publish, for noncommercial purposes only, results and methods of the trial, but no other Sponsor Confidential Information. PI must give Sponsor no less than 60 days to review any manuscript for a proposed publication and must delay publication for up to an additional 90 days thereafter if Sponsor needs to file any patent application to protect any of Sponsor's intellectual property contained in the proposed publication.
Results Point of Contact
Name/Title: | Biogen Study Medical Director |
Organization: | Biogen |
Phone: | 866-633-4636 |
EMail: | clinicaltrials@biogen.com |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Biogen |
ClinicalTrials.gov Identifier: | NCT02462759 |
Other Study ID Numbers: |
232SM202 2014-003657-33 ( EudraCT Number ) |
First Submitted: | May 14, 2015 |
First Posted: | June 4, 2015 |
Results First Submitted: | September 24, 2019 |
Results First Posted: | January 27, 2020 |
Last Update Posted: | February 17, 2021 |