Trial · NCT00528268
Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy
NCT00528268, Phase 1, Phase 2, Completed, University of Utah; results first posted 2015-07-03
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy. Trials, record NCT00528268. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00528268
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy
- NCT number
- NCT00528268
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 22 participants, actual
- Lead sponsor
- University of Utah
- Conditions
- Spinal Muscular Atrophy
- Interventions
- Sodium phenylbutyrate
- First posted
- 2007-09-12
- Last updated
- 2025-02-28
- Results first posted
- 2015-07-03
Source ClinicalTrials.gov
Registered outcomes1
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Safety and Tolerability of Sodium Phenylbutyrate in Neonates and Infants With SMA | 24 months | The number of participants able to achieve specific developmental milestones, such as sitting unsupported or walking independently, during the study period. |
Source ClinicalTrials.gov
Outcomes1
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Safety and Tolerability of Sodium Phenylbutyrate in Neonates and Infants With SMA | 24 months | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group4
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Safety and Tolerability of Sodium Phenylbutyrate in Neonates and Infants With SMA | Cohort 1 | Able to sit unsupported | 1 | Participants | |
| Safety and Tolerability of Sodium Phenylbutyrate in Neonates and Infants With SMA | Cohort 2 | Able to sit unsupported | 8 | Participants | |
| Safety and Tolerability of Sodium Phenylbutyrate in Neonates and Infants With SMA | Cohort 1 | Able to walk independently | 0 | Participants | |
| Safety and Tolerability of Sodium Phenylbutyrate in Neonates and Infants With SMA | Cohort 2 | Able to walk independently | 1 | Participants |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1 | 6 | |
| Overall Study | STARTED | Cohort 2 | 8 | |
| Overall Study | COMPLETED | Cohort 1 | 5 | |
| Overall Study | COMPLETED | Cohort 2 | 7 | |
| Overall Study | NOT COMPLETED | Cohort 1 | 1 | |
| Overall Study | NOT COMPLETED | Cohort 2 | 1 |
Source ClinicalTrials.gov
Adverse events106
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Serous Otitis Media Bilateral | Infections and infestations | Cohort 1 | 4 | 6 | 6 |
| other | Serous Otitis Media Bilateral | Infections and infestations | Cohort 2 | 3 | 8 | 4 |
| other | Eye Infection | Infections and infestations | Cohort 1 | 1 | 6 | 1 |
| other | Eye Infection | Infections and infestations | Cohort 2 | 1 | 8 | 2 |
| other | Upper Respiratory Infection | Infections and infestations | Cohort 1 | 2 | 6 | 2 |
| other | Upper Respiratory Infection | Infections and infestations | Cohort 2 | 3 | 8 | 4 |
| other | Flu symptoms | Infections and infestations | Cohort 1 | 2 | 6 | 2 |
| other | Flu symptoms | Infections and infestations | Cohort 2 | 5 | 8 | 10 |
| other | Rhinitis | Infections and infestations | Cohort 1 | 2 | 6 | 2 |
| other | Rhinitis | Infections and infestations | Cohort 2 | 1 | 8 | 1 |
1 to 10 of 106Source ClinicalTrials.gov
See all 106 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1 | ||
| Total, all-cause mortality | Cohort 2 | ||
| Total, other adverse events | Cohort 1 | 6 | 6 |
| Total, other adverse events | Cohort 2 | 8 | 8 |
| Total, serious adverse events | Cohort 1 | 6 | 6 |
| Total, serious adverse events | Cohort 2 | 7 | 8 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants1
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.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| R01HD054599 | Therapeutic Opportunities in Spinal Muscular Atrophy | SWOBODA, KATHRYN J. | UNIVERSITY OF UTAH | EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH & HUMAN DEVELOPMENT | 2007 to 2011 |
Source NIH RePORTER
Trial comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Cohort 1 vs Cohort 2: serious adverse events |
Source Trial comparisons
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/NCT00528268
GET https://conductscience.org/api/trials/v1/NCT00528268/events?page=2