Trial · NCT03335488
Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)
NCT03335488, Phase 4, Completed, Amgen; results first posted 2023-07-10
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs). Trials, record NCT03335488. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03335488
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)
- NCT number
- NCT03335488
- Status
- Completed
- Phases
- Phase 4
- Enrollment
- 16 participants, actual
- Lead sponsor
- Amgen
- Conditions
- Urea Cycle Disorder
- Interventions
- RAVICTI, NaPBA
- First posted
- 2017-11-07
- Last updated
- 2024-07-01
- Other registries' IDs
- eudract:2015-000075-27
- Results first posted
- 2023-07-10
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Rate of Treatment Success (Percentage of Participants Defined as Treatment Success at Week 4) During the Initial Treatment Period | Week 4 | A participant was considered a Treatment Success for the assigned treatment arm if the participant had not experienced an unprovoked hyperammonemic crisis (HAC) (i.e., a HAC that cannot be attributed to one or more specific precipitating factors such as infection, intercurrent illness, diet noncompliance, treatment noncompliance, etc.) on the assigned treatment and had met at least 2 of the following 3 criteria: * Had absolute values at the 3 time points (pre-dose, after dose at 4 hours and 8 hours) of plasma ammonia levels which do not exceed ULN at the Week 4(End of Initial Treatment Period visit) * Had normal (≤ ULN) glutamine levels at the Week 4 (End of Initial Treatment Period visit at the time point Zero Hour. * Had normal (≤ ULN) essential amino acids including branched chain amino acid levels (threonine, phenylalanine, methionine, lysine, leucine, isoleucine, histidine, valine) at the End of Initial Treatment Period visit at time point Zero Hour. |
| Secondary | Rate of Drug Discontinuations (Percentage of Participants Who Discontinued Study Drug) Due to Any Reason in the Initial Treatment Period | Baseline through Week 4 | |
| Secondary | Change From Baseline in Fasting Plasma Ammonia Levels During the Initial Treatment Period | Baseline, Initial Treatment Period Week 1, Week 2, Week 3, Week 4 (0, 4, 8 hours post dose) | |
| Secondary | Plasma Ammonia Area Under the Curve (AUC) 0 to 8h at the End of the Initial Treatment Period | Week 4: hour 0 (predose), and hours 4 and 8 postdose | |
| Secondary | Peak Plasma Concentration (Cmax) of Ammonia at the End of the Initial Treatment Period | Week 4: hour 0 (predose), and hours 4 and 8 postdose |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Rate of Treatment Success (Percentage of Participants Defined as Treatment Success at Week 4) During the Initial Treatment Period | Week 4 | Modified Intent-to-Treat Population: all participants from the Safety population with no major eligibility violations and participants who had ammonia data post-randomization. | percentage of participants | NUMBER |
| SECONDARY | Rate of Drug Discontinuations (Percentage of Participants Who Discontinued Study Drug) Due to Any Reason in the Initial Treatment Period | Baseline through Week 4 | Modified Intent-to-Treat Population: all participants from the Safety population with no major eligibility violations and participants who had ammonia data post-randomization. | percentage of participants | NUMBER |
| SECONDARY | Change From Baseline in Fasting Plasma Ammonia Levels During the Initial Treatment Period | Baseline, Initial Treatment Period Week 1, Week 2, Week 3, Week 4 (0, 4, 8 hours post dose) | Modified Intent-to-Treat Population: all participants from the Safety population with no major eligibility violations and participants who had ammonia data post-randomization. Participants with an assessment at given time point. | µmol/L | MEAN |
| SECONDARY | Plasma Ammonia Area Under the Curve (AUC) 0 to 8h at the End of the Initial Treatment Period | Week 4: hour 0 (predose), and hours 4 and 8 postdose | Modified Intent-to-Treat Population: all participants from the Safety population with no major eligibility violations and participants who had ammonia data post-randomization. Participants with an assessment at given time point. | µmol*h /L | MEAN |
| SECONDARY | Peak Plasma Concentration (Cmax) of Ammonia at the End of the Initial Treatment Period | Week 4: hour 0 (predose), and hours 4 and 8 postdose | Modified Intent-to-Treat Population: all participants from the Safety population with no major eligibility violations and participants who had ammonia data post-randomization. Participants with an assessment at given time point. | µmol/L | MEAN |
Source ClinicalTrials.gov
Results by group20
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Rate of Treatment Success (Percentage of Participants Defined as Treatment Success at Week 4) During the Initial Treatment Period | RAVICTI | 81.8 | percentage of participants | ||
| Rate of Treatment Success (Percentage of Participants Defined as Treatment Success at Week 4) During the Initial Treatment Period | NaPBA | 80.0 | percentage of participants | ||
| Rate of Drug Discontinuations (Percentage of Participants Who Discontinued Study Drug) Due to Any Reason in the Initial Treatment Period | RAVICTI | 0 | percentage of participants | ||
| Rate of Drug Discontinuations (Percentage of Participants Who Discontinued Study Drug) Due to Any Reason in the Initial Treatment Period | NaPBA | 0 | percentage of participants | ||
| Change From Baseline in Fasting Plasma Ammonia Levels During the Initial Treatment Period | RAVICTI | Week 1 | 6.5 | Standard Deviation 21.16 | µmol/L |
| Change From Baseline in Fasting Plasma Ammonia Levels During the Initial Treatment Period | NaPBA | Week 1 | 0.0 | Standard Deviation 10.12 | µmol/L |
| Change From Baseline in Fasting Plasma Ammonia Levels During the Initial Treatment Period | RAVICTI | Week 2 | 25.5 | Standard Deviation 59.88 | µmol/L |
| Change From Baseline in Fasting Plasma Ammonia Levels During the Initial Treatment Period | NaPBA | Week 2 | -10.4 | Standard Deviation 10.17 | µmol/L |
| Change From Baseline in Fasting Plasma Ammonia Levels During the Initial Treatment Period | RAVICTI | Week 3 | 7.4 | Standard Deviation 35.91 | µmol/L |
| Change From Baseline in Fasting Plasma Ammonia Levels During the Initial Treatment Period | NaPBA | Week 3 | -10.9 | Standard Deviation 6.40 | µmol/L |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 results by groupParticipant flow24
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Initial Treatment Period | STARTED | RAVICTI -> RAVICTI | 11 | |
| Initial Treatment Period | STARTED | NaPBA -> RAVICTI | 5 | |
| Initial Treatment Period | COMPLETED | RAVICTI -> RAVICTI | 11 | |
| Initial Treatment Period | COMPLETED | NaPBA -> RAVICTI | 5 | |
| Initial Treatment Period | NOT COMPLETED | RAVICTI -> RAVICTI | 0 | |
| Initial Treatment Period | NOT COMPLETED | NaPBA -> RAVICTI | 0 | |
| Transition Period | STARTED | RAVICTI -> RAVICTI | 0 | |
| Transition Period | STARTED | NaPBA -> RAVICTI | 5 | |
| Transition Period | COMPLETED | RAVICTI -> RAVICTI | 0 | |
| Transition Period | COMPLETED | NaPBA -> RAVICTI | 5 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 participant flowAdverse events172
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 | Anaemia | Blood and lymphatic system disorders | Initial Treatment Period: RAVICTI | 1 | 11 | |
| other | Anaemia | Blood and lymphatic system disorders | Initial Treatment Period: NaPBA | 0 | 5 | |
| other | Anaemia | Blood and lymphatic system disorders | Transition Period: RAVICTI | 0 | 5 | |
| other | Anaemia | Blood and lymphatic system disorders | Maintenance and Safety Periods Combined | 1 | 16 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Initial Treatment Period: RAVICTI | 0 | 11 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Initial Treatment Period: NaPBA | 0 | 5 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Transition Period: RAVICTI | 0 | 5 | |
| other | Lymphadenopathy | Blood and lymphatic system disorders | Maintenance and Safety Periods Combined | 1 | 16 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Initial Treatment Period: RAVICTI | 0 | 11 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Initial Treatment Period: NaPBA | 1 | 5 |
1 to 10 of 172Source ClinicalTrials.gov
See all 172 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Initial Treatment Period: RAVICTI | 0 | 11 |
| Total, all-cause mortality | Initial Treatment Period: NaPBA | 0 | 5 |
| Total, all-cause mortality | Transition Period: RAVICTI | 0 | 5 |
| Total, all-cause mortality | Maintenance and Safety Periods Combined | 0 | 16 |
| Total, other adverse events | Initial Treatment Period: RAVICTI | 6 | 11 |
| Total, other adverse events | Initial Treatment Period: NaPBA | 2 | 5 |
| Total, other adverse events | Transition Period: RAVICTI | 1 | 5 |
| Total, other adverse events | Maintenance and Safety Periods Combined | 8 | 16 |
| Total, serious adverse events | Initial Treatment Period: RAVICTI | 2 | 11 |
| Total, serious adverse events | Initial Treatment Period: NaPBA | 0 | 5 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT03335488
GET https://conductscience.org/api/trials/v1/NCT03335488/flow?page=2