Trial · NCT01881984
Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation
NCT01881984, Phase 1, Completed, University of Pittsburgh; results first posted 2017-07-11
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation. Trials, record NCT01881984. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01881984
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Use of Ravicti™ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation
- NCT number
- NCT01881984
- Status
- Completed
- Phases
- Phase 1
- Enrollment
- 4 participants, actual
- Lead sponsor
- University of Pittsburgh
- Conditions
- Medium-chain Acyl-CoA Dehydrogenase (MCAD) Deficiency
- Interventions
- Ravicti
- First posted
- 2013-06-20
- Last updated
- 2017-09-25
- Results first posted
- 2017-07-11
- Limitations and caveats
The purpose of this phase 1 trial was to establish a safe dose for treatment of patients with MCAD deficiency due to the common mutations, and to evaluate biomarkers of fatty acid metabolism in treated patients vs their pretreatment values.
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Metabolic Stress | 7 weeks | Changes in the assessments of metabolic stress pre- and post-dosing with Ravicti will be the main outcome variable. |
| Secondary | Pharmacokinetic (pK)Analysis | 7 weeks | Results from the pharmacokinetic (pK)analysis (the rate of conversion of the phenylbutyrate to phenylacetate) will also be reviewed to assess for changes pre- and post-dosing with Ravicti as well as changes in these levels at the different doses of Ravicti. |
Source ClinicalTrials.gov
Outcomes2
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Metabolic Stress | 7 weeks | Due to the small sample size in this Phase I study, details on the analysis cannot be provided due to concerns with subject confidentiality. | ||
| SECONDARY | Pharmacokinetic (pK)Analysis | 7 weeks | Due to the small sample size in this Phase I study, details on the analysis cannot be provided due to concerns with subject confidentiality. |
Source ClinicalTrials.gov
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Ravicti | 4 | |
| Overall Study | COMPLETED | Ravicti | 4 | |
| Overall Study | NOT COMPLETED | Ravicti | 0 |
Source ClinicalTrials.gov
Adverse events7
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 | Dry mouth | Gastrointestinal disorders | Ravicti | 1 | 4 | 1 |
| other | Elevated PBA level | Investigations | Ravicti | 2 | 4 | 2 |
| other | Nausea | Gastrointestinal disorders | Ravicti | 2 | 4 | 2 |
| other | Vomiting | Gastrointestinal disorders | Ravicti | 1 | 4 | 1 |
| other | Neck pain | Musculoskeletal and connective tissue disorders | Ravicti | 1 | 4 | 1 |
| other | Thromboembolic event | Vascular disorders | Ravicti | 1 | 4 | 1 |
| other | Decreased reflexes | Nervous system disorders | Ravicti | 1 | 4 | 1 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ravicti | 0 | 4 |
| Total, other adverse events | Ravicti | 3 | 4 |
| Total, serious adverse events | Ravicti | 0 | 4 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 23141465 | BACKGROUND |
Source PubMed
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/NCT01881984
GET https://conductscience.org/api/trials/v1/NCT01881984/registered