Trial · NCT00227266
Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy
NCT00227266, Phase 2, Completed, University of Utah; results first posted 2011-05-03
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy. Trials, record NCT00227266. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00227266
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy
- NCT number
- NCT00227266
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 94 participants, actual
- Lead sponsor
- University of Utah
- Conditions
- Spinal Muscular Atrophy
- Interventions
- Valproic Acid and Levocarnitine, Placebo
- First posted
- 2005-09-27
- Last updated
- 2025-03-19
- Results first posted
- 2011-05-03
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Safety Labs | -4 wks, 0, 2 wks, 3 mo, 6 mo, 9 mo, 12 mo for safety labs; throughout for AEs | Participants will have labs drawn regularly to maintain appropriate dosing and monitor liver function |
| Primary | Efficacy, Measured Through Motor Function Assessments | -4wks, 0, 3 mo, 6 mo, 12 mo | |
| Primary | Modified Hammersmith Change From Baseline to 6 Months | 0 months, 6 months | Comparison of Modified Hammersmith Change from baseline to 6 months. Scores range from 0 to 40. A higher score indicates a better outcome. This scale is used to assess gross motor abilities of non-ambulant children with SMA in multiple research trials as well as in clinical settings. |
| Secondary | Quantitative Assessment of SMN mRNA From Blood Samples | -4wks or 0, 3 mo, 6 mo, 12 mo | |
| Secondary | Peds QL™ Assessment: Parental Version (All), Child Versions (> 5yrs) | -4wks, 0, 3mo, 6mo, 12mo | |
| Secondary | Max CMAP Amplitude (Mean) | 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available) | The maximum Compound Motor Action Potential (CMAP) is a measurement obtained through EMG testing that is associated with disease progression. In this study, we measure the maximum CMAP by stimulating one nerve in the hand and measuring the response of the muscle. This is done multiple times, the outcome used is the highest peak, or response observed. |
| Secondary | Max CMAP Amplitude Median | 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available) | The maximum Compound Motor Action Potential (CMAP) is a measurement obtained through EMG testing that is associated with disease progression. In this study, we measure the maximum CMAP by stimulating one nerve in the hand and measuring the response of the muscle. This is done multiple times, the outcome used is the highest peak, or response observed. |
| Secondary | Ulnar MUNE | -4 wks, 0, 3 mo, 6 mo, 12 mo | |
| Secondary | Growth and Vital Sign Parameters | -4 wks, 0, 3mo, 6mo, 12mo | |
| Secondary | Nutritional Status | -4 wks, 0, 3mo, 6mo, 12mo |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes14
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Safety Labs | -4 wks, 0, 2 wks, 3 mo, 6 mo, 9 mo, 12 mo for safety labs; throughout for AEs | |||
| PRIMARY | Efficacy, Measured Through Motor Function Assessments | -4wks, 0, 3 mo, 6 mo, 12 mo | |||
| SECONDARY | Quantitative Assessment of SMN mRNA From Blood Samples | -4wks or 0, 3 mo, 6 mo, 12 mo | |||
| SECONDARY | Peds QL™ Assessment: Parental Version (All), Child Versions (> 5yrs) | -4wks, 0, 3mo, 6mo, 12mo | |||
| SECONDARY | Max CMAP Amplitude (Mean) | 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available) | mV | MEAN | |
| SECONDARY | Max CMAP Amplitude Median | 1 month prior to official enrollment, beginning of study (0 months), 6 months, 12 months (data point not available) | mV | MEDIAN | |
| SECONDARY | Ulnar MUNE | -4 wks, 0, 3 mo, 6 mo, 12 mo | |||
| SECONDARY | Growth and Vital Sign Parameters | -4 wks, 0, 3mo, 6mo, 12mo | |||
| SECONDARY | Nutritional Status | -4 wks, 0, 3mo, 6mo, 12mo | |||
| POST_HOC | Modified Hammersmith Extend Baseline | 1 month prior to enrollment, at enrollment (0 months) | Analysis was determined per protocol | Score | MEAN |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group32
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Max CMAP Amplitude (Mean) | Cohort 1a Sitters Placebo Then Treatment | Baseline | 2.28 | Standard Deviation 1.55 | mV |
| Max CMAP Amplitude (Mean) | Cohort 1b Sitters Treatment | Baseline | 2.93 | Standard Deviation 1.56 | mV |
| Max CMAP Amplitude (Mean) | Cohort 2 Standers and Walkers - Treatment | Baseline | 5.52 | Standard Deviation 2.56 | mV |
| Max CMAP Amplitude (Mean) | Cohort 1a Sitters Placebo Then Treatment | 6 months | 2.32 | Standard Deviation 1.75 | mV |
| Max CMAP Amplitude (Mean) | Cohort 1b Sitters Treatment | 6 months | 2.37 | Standard Deviation 1.82 | mV |
| Max CMAP Amplitude (Mean) | Cohort 2 Standers and Walkers - Treatment | 6 months | 6.56 | Standard Deviation 2.99 | mV |
| Max CMAP Amplitude Median | Cohort 1a Sitters Placebo Then Treatment | Baseline | 1.91 | Full Range 0.5 to 7.66 | mV |
| Max CMAP Amplitude Median | Cohort 1b Sitters Treatment | Baseline | 2.2 | Full Range 0.5 to 6.66 | mV |
| Max CMAP Amplitude Median | Cohort 2 Standers and Walkers - Treatment | Baseline | 5.3 | Full Range 1.2 to 10.42 | mV |
| Max CMAP Amplitude Median | Cohort 1a Sitters Placebo Then Treatment | 6 months | 1.44 | Full Range 0.5 to 6.14 | mV |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1a Sitters Placebo Then Treatment | 31 | |
| Overall Study | STARTED | Cohort 1b Sitters Treatment | 30 | |
| Overall Study | STARTED | Cohort 2 Standers and Walkers - Treatment | 33 | |
| Overall Study | COMPLETED | Cohort 1a Sitters Placebo Then Treatment | 30 | |
| Overall Study | COMPLETED | Cohort 1b Sitters Treatment | 30 | |
| Overall Study | COMPLETED | Cohort 2 Standers and Walkers - Treatment | 29 | |
| Overall Study | NOT COMPLETED | Cohort 1a Sitters Placebo Then Treatment | 1 | |
| Overall Study | NOT COMPLETED | Cohort 1b Sitters Treatment | 0 | |
| Overall Study | NOT COMPLETED | Cohort 2 Standers and Walkers - Treatment | 4 |
Source ClinicalTrials.gov
Adverse events198
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 | Abdominal Pain Upper | Gastrointestinal disorders | Cohort 1a Sitters Placebo Then Treatment | 2 | 31 | |
| other | Abdominal Pain Upper | Gastrointestinal disorders | Cohort 1b Sitters Treatment | 3 | 30 | |
| other | Abdominal Pain Upper | Gastrointestinal disorders | Cohort 2 Standers and Walkers - Treatment | 7 | 33 | |
| other | Agitation | Nervous system disorders | Cohort 1a Sitters Placebo Then Treatment | 1 | 31 | |
| other | Agitation | Nervous system disorders | Cohort 1b Sitters Treatment | 0 | 30 | |
| other | Agitation | Nervous system disorders | Cohort 2 Standers and Walkers - Treatment | 0 | 33 | |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Cohort 1a Sitters Placebo Then Treatment | 1 | 31 | |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Cohort 1b Sitters Treatment | 0 | 30 | |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Cohort 2 Standers and Walkers - Treatment | 0 | 33 | |
| other | Bronchitis | Infections and infestations | Cohort 1a Sitters Placebo Then Treatment | 0 | 31 |
1 to 10 of 198Source ClinicalTrials.gov
See all 198 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1a Sitters Placebo Then Treatment | ||
| Total, all-cause mortality | Cohort 1b Sitters Treatment | ||
| Total, all-cause mortality | Cohort 2 Standers and Walkers - Treatment | ||
| Total, other adverse events | Cohort 1a Sitters Placebo Then Treatment | 18 | 31 |
| Total, other adverse events | Cohort 1b Sitters Treatment | 23 | 30 |
| Total, other adverse events | Cohort 2 Standers and Walkers - Treatment | 28 | 33 |
| Total, serious adverse events | Cohort 1a Sitters Placebo Then Treatment | 1 | 31 |
| Total, serious adverse events | Cohort 1b Sitters Treatment | 4 | 30 |
| Total, serious adverse events | Cohort 2 Standers and Walkers - Treatment | 4 | 33 |
Source ClinicalTrials.gov
Papers41
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/NCT00227266
GET https://conductscience.org/api/trials/v1/NCT00227266/results?page=2