A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)
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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: NCT02628743 |
Recruitment Status :
Completed
First Posted : December 11, 2015
Results First Posted : August 9, 2019
Last Update Posted : August 9, 2019
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Sponsor:
Hoffmann-La Roche
Information provided by (Responsible Party):
Hoffmann-La Roche
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Study Type | Interventional |
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Study Design | Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment |
Condition |
Muscular Atrophy, Spinal |
Intervention |
Drug: Olesoxime |
Enrollment | 131 |
Participant Flow
Recruitment Details | |
Pre-assignment Details |
Arm/Group Title | Olesoxime |
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Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube. |
Period Title: Overall Study | |
Started | 131 |
Completed | 0 |
Not Completed | 131 |
Reason Not Completed | |
Other | 3 |
Withdrawal by Subject | 50 |
Study Terminated by Sponsor | 76 |
Physician Decision | 2 |
Baseline Characteristics
Arm/Group Title | Olesoxime | |
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Participants received a dose of 10 mg/kg suspension once a day (QD) orally or via a naso-gastric or gastronomy tube. Participants who consented to dose increase received 10 milligrams per kilogram (mg/kg) suspension twice a day (BID) either orally or via a naso-gastric or gastrostomy tube. | |
Overall Number of Baseline Participants | 131 | |
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[Not Specified]
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Age, Continuous
Mean (Standard Deviation) Unit of measure: Years |
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Number Analyzed | 131 participants | |
14.7 (5.9) | ||
Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 131 participants | |
Female |
66 50.4%
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Male |
65 49.6%
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Race (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 131 participants | |
American Indian or Alaska Native |
0 0.0%
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Asian |
3 2.3%
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Native Hawaiian or Other Pacific Islander |
0 0.0%
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Black or African American |
1 0.8%
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White |
94 71.8%
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More than one race |
0 0.0%
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Unknown or Not Reported |
33 25.2%
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Race/Ethnicity, Customized
Measure Type: Number Unit of measure: Participants |
Number Analyzed | 131 participants |
Hispanic or Latino | 1 | |
Not Hispanic or Latino | 89 | |
Not Stated | 29 | |
Unknown | 12 |
Outcome Measures
Adverse Events
Limitations and Caveats
[Not Specified]
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.
The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
Results Point of Contact
Name/Title: | Medical Communications |
Organization: | Hoffmann-La Roche |
Phone: | 800 821-8590 |
EMail: | genentech@druginfo.com |
Responsible Party: | Hoffmann-La Roche |
ClinicalTrials.gov Identifier: | NCT02628743 |
Other Study ID Numbers: |
BN29854 2015-001589-25 ( EudraCT Number ) |
First Submitted: | December 1, 2015 |
First Posted: | December 11, 2015 |
Results First Submitted: | June 12, 2019 |
Results First Posted: | August 9, 2019 |
Last Update Posted: | August 9, 2019 |