Trial · NCT02246218
A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders
NCT02246218, Phase 4, Completed, Amgen; results first posted 2019-03-05
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders. Trials, record NCT02246218. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02246218
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders
- NCT number
- NCT02246218
- Status
- Completed
- Phases
- Phase 4
- Enrollment
- 27 participants, actual
- Lead sponsor
- Amgen
- Conditions
- Urea Cycle Disorder
- Interventions
- RAVICTI
- First posted
- 2014-09-22
- Last updated
- 2024-07-01
- Other registries' IDs
- eudract:2016-003460-38
- Results first posted
- 2019-03-05
Source ClinicalTrials.gov
Registered outcomes54
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Successful Transition to RAVICTI With Controlled Ammonia (i.e. No Clinical Symptoms and Ammonia < 100 μmol/L): Cohort of 2 Months to <2 Years Participants | Up to Day 4 | The percentage of participants with successful transition is based on Investigator response to the question, "Has transition to 100% RAVICTI been successful with controlled ammonia?" For participants 2 months of age and older, after a minimum of 24 hours of ammonia monitoring following the first full dose of RAVICTI alone, the participant was effectively transitioned when following conditions were met: no signs and symptoms of hyperammonemia; ammonia level less than 100 μmol/L (without normalization of ammonia, ie, without conversion of values from local laboratories with varying normal ranges to standardized values); and eligible for discharge per Investigator judgment. |
| Primary | Percentage of Participants With Successful Transition to RAVICTI With Controlled Ammonia (i.e. No Clinical Symptoms and Ammonia < 100 μmol/L): Cohort of 0 Months to <2 Months Participants | Up to Day 4 | The percentage of participants with successful transition is based on Investigator response to the question, "Has transition to 100% RAVICTI been successful with controlled ammonia?" For participants \< 2 months of age, after a minimum of 24 hours of ammonia monitoring following the first full dose of RAVICTI alone, the participant was effectively transitioned when following conditions were met: no signs and symptoms of hyperammonemia; ammonia level less than 100 μmol/L (without normalization of ammonia, ie, without conversion of values from local laboratories with varying normal ranges to standardized values); and eligible for discharge per Investigator judgment. |
| Secondary | Rate of Hyperammonemic Crises (HACs): Cohort of 2 Months to <2 Years Participants | Day 8 through up to Month 6 | HAC is defined having signs and symptoms consistent with hyperammonemia (such as but not limited to frequent vomiting, nausea, headache, lethargy, irritability, combativeness, and/or somnolence) associated with high blood ammonia and requiring medical intervention. Rate of HACs per 6 months during the safety extension is calculated as sum of (number of HAC) / sum of (days during first 6 months starting on Day 8 or number days on RAVICTI, whichever is less) across all participants in the corresponding group. |
| Secondary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 2 Months to <2 Years Participants | From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months). | An adverse event (AE) is any untoward medical occurrence, whether or not the event is considered related to the study drug. A serious AE is any AE that: results in death; is life threatening; requires hospitalization or prolongation of existing hospitalization; results in disability/incapacity; is a congenital anomaly/birth defect; is an important medical event. TEAEs are defined as AEs with an onset date on or after the first dose of study medication until study discontinuation. The Investigator assessed the causal relationship of each TEAE to the study drug as not related, possibly related, or probably related. |
| Secondary | Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants | Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24 | |
| Secondary | Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 2 Months to <2 Years Participants | Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24 | |
| Secondary | Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants | Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24 | |
| Secondary | Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 2 Months to <2 Years Participants | Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24 | |
| Secondary | Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 2 Months to <2 Years Participants | Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24 | |
| Secondary | Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 2 Months to <2 Years Participants | Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24 |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 registered outcomesOutcomes54
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With Successful Transition to RAVICTI With Controlled Ammonia (i.e. No Clinical Symptoms and Ammonia < 100 μmol/L): Cohort of 2 Months to <2 Years Participants | Up to Day 4 | Safety Population: all enrolled participants who received any amount of study medication. | percentage of participants | NUMBER |
| PRIMARY | Percentage of Participants With Successful Transition to RAVICTI With Controlled Ammonia (i.e. No Clinical Symptoms and Ammonia < 100 μmol/L): Cohort of 0 Months to <2 Months Participants | Up to Day 4 | Safety Population: all enrolled participants who received any amount of study medication. | percentage of participants | NUMBER |
| SECONDARY | Rate of Hyperammonemic Crises (HACs): Cohort of 2 Months to <2 Years Participants | Day 8 through up to Month 6 | Safety Population: all enrolled participants who received any amount of study medication and had an assessment. | HACs per half-year of patient exposure | NUMBER |
| SECONDARY | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 2 Months to <2 Years Participants | From the first dose of study treatment through 30 days after the final dose (mean [SD] duration of treatment was 9.13 [6.838] months). | Safety Population: all enrolled participants who received any amount of study medication. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants | Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24 | Safety Population: all enrolled participants who received any amount of study medication and had an assessment at given time point. | µmol/L | MEAN |
| SECONDARY | Amino Acid Assessment: Baseline and Change From Baseline in Glutamine Up to Month 24: Cohort of 2 Months to <2 Years Participants | Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24 | Safety Population: all enrolled participants who received any amount of study medication and had an assessment at given time point. | µmol/L | MEAN |
| SECONDARY | Amino Acid Assessment: Baseline and Change From Baseline in Sum of Glutamine and Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants | Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24 | Safety Population: all enrolled participants who received any amount of study medication and had an assessment at given time point. | µmol/L | MEAN |
| SECONDARY | Amino Acid Assessment: Baseline and Change From Baseline in Isoleucine Up to Month 24: Cohort of 2 Months to <2 Years Participants | Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24 | Safety Population: all enrolled participants who received any amount of study medication and had an assessment at given time point. | µmol/L | MEAN |
| SECONDARY | Amino Acid Assessment: Baseline and Change From Baseline in Leucine Up to Month 24: Cohort of 2 Months to <2 Years Participants | Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24 | Safety Population: all enrolled participants who received any amount of study medication and had an assessment at given time point. | µmol/L | MEAN |
| SECONDARY | Amino Acid Assessment: Baseline and Change From Baseline in Valine Up to Month 24: Cohort of 2 Months to <2 Years Participants | Baseline, Day 7, Month 2, Month 3, Month 4, Month 5, Month 6, Month 9, Month 12, Month 15, Month 24 | Safety Population: all enrolled participants who received any amount of study medication and had an assessment at given time point. | µmol/L | MEAN |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 outcomesResults by group295
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Successful Transition to RAVICTI With Controlled Ammonia (i.e. No Clinical Symptoms and Ammonia < 100 μmol/L): Cohort of 2 Months to <2 Years Participants | RAVICTI: Age 2 Months to < 2 Years | 100 | percentage of participants | ||
| Percentage of Participants With Successful Transition to RAVICTI With Controlled Ammonia (i.e. No Clinical Symptoms and Ammonia < 100 μmol/L): Cohort of 0 Months to <2 Months Participants | RAVICTI: Age 0 to < 2 Months | 100 | percentage of participants | ||
| Rate of Hyperammonemic Crises (HACs): Cohort of 2 Months to <2 Years Participants | RAVICTI: Age 2 Months to < 2 Years | 0.005 | HACs per half-year of patient exposure | ||
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 2 Months to <2 Years Participants | RAVICTI: Age 2 Months to < 2 Years | ≥ 1 TEAE | 10 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 2 Months to <2 Years Participants | RAVICTI: Age 2 Months to < 2 Years | ≥ 1 Related TEAE | 4 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 2 Months to <2 Years Participants | RAVICTI: Age 2 Months to < 2 Years | ≥ 1 Serious TEAE | 6 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 2 Months to <2 Years Participants | RAVICTI: Age 2 Months to < 2 Years | ≥ 1 Serious Related TEAE | 0 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 2 Months to <2 Years Participants | RAVICTI: Age 2 Months to < 2 Years | Fatal Outcome TEAE | 1 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, Deaths, and Discontinuations Due to TEAEs: Cohort of 2 Months to <2 Years Participants | RAVICTI: Age 2 Months to < 2 Years | ≥ 1 TEAE Leading to Study Discontinuation | 1 | Participants | |
| Amino Acid Assessment: Baseline and Change From Baseline in Glutamate Up to Month 24: Cohort of 2 Months to <2 Years Participants | RAVICTI: Age 2 Months to < 2 Years | Baseline | 122.43 | Standard Deviation 118.620 | µmol/L |
1 to 10 of 295Source ClinicalTrials.gov
See all 295 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | RAVICTI: Age 2 Months to < 2 Years | 10 | |
| Overall Study | STARTED | RAVICTI: Age 0 to < 2 Months | 16 | |
| Overall Study | COMPLETED | RAVICTI: Age 2 Months to < 2 Years | 6 | |
| Overall Study | COMPLETED | RAVICTI: Age 0 to < 2 Months | 10 | |
| Overall Study | NOT COMPLETED | RAVICTI: Age 2 Months to < 2 Years | 4 | |
| Overall Study | NOT COMPLETED | RAVICTI: Age 0 to < 2 Months | 6 |
Source ClinicalTrials.gov
Adverse events254
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 | Venous thrombosis limb | Vascular disorders | RAVICTI: Age 2 Months to < 2 Years | 1 | 10 | |
| other | Venous thrombosis limb | Vascular disorders | RAVICTI: Age 0 to <2 Months | 0 | 16 | |
| other | Seasonal allergy | Immune system disorders | RAVICTI: Age 2 Months to < 2 Years | 1 | 10 | |
| other | Seasonal allergy | Immune system disorders | RAVICTI: Age 0 to <2 Months | 0 | 16 | |
| other | Pyrexia | General disorders | RAVICTI: Age 2 Months to < 2 Years | 2 | 10 | |
| other | Pyrexia | General disorders | RAVICTI: Age 0 to <2 Months | 2 | 16 | |
| other | Catheter site rash | General disorders | RAVICTI: Age 2 Months to < 2 Years | 0 | 10 | |
| other | Catheter site rash | General disorders | RAVICTI: Age 0 to <2 Months | 1 | 16 | |
| other | Device occlusion | General disorders | RAVICTI: Age 2 Months to < 2 Years | 0 | 10 | |
| other | Device occlusion | General disorders | RAVICTI: Age 0 to <2 Months | 1 | 16 |
1 to 10 of 254Source ClinicalTrials.gov
See all 254 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | RAVICTI: Age 2 Months to < 2 Years | 1 | 10 |
| Total, all-cause mortality | RAVICTI: Age 0 to <2 Months | 0 | 16 |
| Total, other adverse events | RAVICTI: Age 2 Months to < 2 Years | 8 | 10 |
| Total, other adverse events | RAVICTI: Age 0 to <2 Months | 16 | 16 |
| Total, serious adverse events | RAVICTI: Age 2 Months to < 2 Years | 6 | 10 |
| Total, serious adverse events | RAVICTI: Age 0 to <2 Months | 11 | 16 |
Source ClinicalTrials.gov
Papers2
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| RAVICTI: Age 2 Months to < 2 Years vs RAVICTI: Age 0 to <2 Months: deaths from any cause | Trial comparisons |
| RAVICTI: Age 2 Months to < 2 Years vs RAVICTI: Age 0 to <2 Months: serious adverse events | 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/NCT02246218
GET https://conductscience.org/api/trials/v1/NCT02246218/registered?page=2