Trial · NCT00661453
CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I
NCT00661453, Phase 1, Phase 2, Completed, University of Utah; results first posted 2015-06-15
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I. Trials, record NCT00661453. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00661453
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I
- NCT number
- NCT00661453
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 40 participants, actual
- Lead sponsor
- University of Utah
- Conditions
- Spinal Muscular Atrophy Type I
- Interventions
- Valproic Acid and Levocarnitine
- First posted
- 2008-04-18
- Last updated
- 2015-06-15
- Results first posted
- 2015-06-15
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Laboratory Safety Data | -2 weeks, + 2 weeks, 3 months, 6 months | |
| Primary | Anthropometric Measures of Nutritional Status (Body Mass Index [BMI] Z-scores, Weight for Length Ratios, Lean/Fat Mass Via DEXA, Growth Parameters, and Triceps Skinfold Measures) | -2 weeks, time 0, 3 months, 6 months | |
| Secondary | Time to Death or Ventilator Dependence (Defined as >16 Hours/Day) | monthly | |
| Secondary | Primary Caregiver Functional Rating Scale for SMA Type I Subjects (PCFRS) | time 0, and monthly for 12 months | |
| Secondary | Functional Motor Assessments: TIMPSI Scores | -2 weeks, time 0, 3 months, 6 months | |
| Secondary | Quantitative SMN mRNA and Protein Measures | -2 weeks, time 0 , 3 months, or 6 months | |
| Secondary | Maximum Ulnar CMAP Amplitude/Area and MUNE | -2 weeks, time 0, 3 months, 6 months | |
| Secondary | Whole Body DEXA Scanning for Lean Body Mass and Total Bone Mineral Density/ Content | -2 weeks or time 0, 3 months, 6 months |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Laboratory Safety Data | -2 weeks, + 2 weeks, 3 months, 6 months | |||
| PRIMARY | Anthropometric Measures of Nutritional Status (Body Mass Index [BMI] Z-scores, Weight for Length Ratios, Lean/Fat Mass Via DEXA, Growth Parameters, and Triceps Skinfold Measures) | -2 weeks, time 0, 3 months, 6 months | 20 participants have baseline values, only 12 have 3 and 6 month values | g | MEAN |
| SECONDARY | Time to Death or Ventilator Dependence (Defined as >16 Hours/Day) | monthly | |||
| SECONDARY | Primary Caregiver Functional Rating Scale for SMA Type I Subjects (PCFRS) | time 0, and monthly for 12 months | |||
| SECONDARY | Functional Motor Assessments: TIMPSI Scores | -2 weeks, time 0, 3 months, 6 months | |||
| SECONDARY | Quantitative SMN mRNA and Protein Measures | -2 weeks, time 0 , 3 months, or 6 months | |||
| SECONDARY | Maximum Ulnar CMAP Amplitude/Area and MUNE | -2 weeks, time 0, 3 months, 6 months | |||
| SECONDARY | Whole Body DEXA Scanning for Lean Body Mass and Total Bone Mineral Density/ Content | -2 weeks or time 0, 3 months, 6 months |
Source ClinicalTrials.gov
Results by group6
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Anthropometric Measures of Nutritional Status (Body Mass Index [BMI] Z-scores, Weight for Length Ratios, Lean/Fat Mass Via DEXA, Growth Parameters, and Triceps Skinfold Measures) | SMA Type 1 | Lean Mass Baseline | 4317.15 | Standard Deviation 1334.48 | g |
| Anthropometric Measures of Nutritional Status (Body Mass Index [BMI] Z-scores, Weight for Length Ratios, Lean/Fat Mass Via DEXA, Growth Parameters, and Triceps Skinfold Measures) | SMA Type 1 | Lean Mass 3 months | 4993.92 | Standard Deviation 1676.13 | g |
| Anthropometric Measures of Nutritional Status (Body Mass Index [BMI] Z-scores, Weight for Length Ratios, Lean/Fat Mass Via DEXA, Growth Parameters, and Triceps Skinfold Measures) | SMA Type 1 | Lean Mass 6 months | 5133.83 | Standard Deviation 2122.99 | g |
| Anthropometric Measures of Nutritional Status (Body Mass Index [BMI] Z-scores, Weight for Length Ratios, Lean/Fat Mass Via DEXA, Growth Parameters, and Triceps Skinfold Measures) | SMA Type 1 | Fat Mass Baseline | 3011.37 | Standard Deviation 1467.83 | g |
| Anthropometric Measures of Nutritional Status (Body Mass Index [BMI] Z-scores, Weight for Length Ratios, Lean/Fat Mass Via DEXA, Growth Parameters, and Triceps Skinfold Measures) | SMA Type 1 | Fat Mass 3 months | 3618.25 | Standard Deviation 1343.25 | g |
| Anthropometric Measures of Nutritional Status (Body Mass Index [BMI] Z-scores, Weight for Length Ratios, Lean/Fat Mass Via DEXA, Growth Parameters, and Triceps Skinfold Measures) | SMA Type 1 | Fat Mass 6 months | 4316.08 | Standard Deviation 1857.16 | g |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | SMA Type 1 | 38 | |
| Overall Study | COMPLETED | SMA Type 1 | 25 | |
| Overall Study | NOT COMPLETED | SMA Type 1 | 13 |
Source ClinicalTrials.gov
Adverse events72
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 | Anemia | Blood and lymphatic system disorders | SMA Type 1 | 1 | 38 | 1 |
| other | Middle Ear Inflammation | Ear and labyrinth disorders | SMA Type 1 | 1 | 38 | 1 |
| other | Ear and labyrinth disorders - Other, specify | Ear and labyrinth disorders | SMA Type 1 | 1 | 38 | 1 |
| other | Conjunctivitis | Eye disorders | SMA Type 1 | 1 | 38 | 1 |
| other | Abdominal Pain | Gastrointestinal disorders | SMA Type 1 | 1 | 38 | 1 |
| other | Diarrhea | Gastrointestinal disorders | SMA Type 1 | 2 | 38 | 2 |
| other | Flatulence | Gastrointestinal disorders | SMA Type 1 | 1 | 38 | 1 |
| other | Gastroesophageal reflux disease | Gastrointestinal disorders | SMA Type 1 | 1 | 38 | 1 |
| other | Vomiting | Gastrointestinal disorders | SMA Type 1 | 3 | 38 | 3 |
| other | Gastrointestinal disorders - Other, specify | Gastrointestinal disorders | SMA Type 1 | 3 | 38 | 3 |
1 to 10 of 72Source ClinicalTrials.gov
See all 72 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | SMA Type 1 | ||
| Total, other adverse events | SMA Type 1 | 36 | 38 |
| Total, serious adverse events | SMA Type 1 | 29 | 38 |
Source ClinicalTrials.gov
Papers39
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/NCT00661453
GET https://conductscience.org/api/trials/v1/NCT00661453/events?page=2