Trial · NCT01780246
An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)
NCT01780246, Phase 1, Completed, Biogen
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701). Trials, record NCT01780246. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01780246
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)
- NCT number
- NCT01780246
- Status
- Completed
- Phases
- Phase 1
- Enrollment
- 18 participants, actual
- Lead sponsor
- Biogen
- Conditions
- Spinal Muscular Atrophy
- Interventions
- nusinersen
- First posted
- 2013-01-31
- Last updated
- 2021-02-16
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of participants that experience Adverse Events (AEs) and Serious Adverse Events | Up to 24 Weeks | |
| Primary | Number of participants with clinically significant neurological examination abnormalities | Up tp 24 Weeks | |
| Primary | Number of participants with clinically significant vital sign abnormalities | Up to 24 Weeks | |
| Primary | Number of participants with clinically significant physical examination abnormalities | Up to 24 Weeks | |
| Primary | Number of participants with clinically significant weight abnormalities | Up to 24 Weeks | |
| Primary | Number of participants with clinically significant laboratory parameters | Up to 24 Weeks | |
| Primary | Number of participants with clinically significant electrocardiograms (ECGs) abnormalities | Up to 24 Weeks | |
| Primary | Number of participants who use concomitant medications | Up to 24 Weeks | |
| Secondary | PK parameters of nusinersen (ISIS 396443): Maximum observed plasma drug concentration (Cmax) | Plasma at 1, 2, 4 and 6 hours after dosing | |
| Secondary | PK parameters of nusinersen: Time to reach maximum observed concentration (Tmax) | Plasma at 1, 2, 4 and 6 hours after dosing |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01780246
GET https://conductscience.org/api/trials/v1/NCT01780246/registered