Trial · NCT01082328
Response to Kuvan® in Subjects With Phenylketonuria (PKU) in a 4 Weeks Testing Period
NCT01082328, Phase 4, Completed, Merck KGaA, Darmstadt, Germany; results first posted 2013-08-01
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Response to Kuvan® in Subjects With Phenylketonuria (PKU) in a 4 Weeks Testing Period. Trials, record NCT01082328. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01082328
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Response to Kuvan® in Subjects With Phenylketonuria (PKU) in a 4 Weeks Testing Period
- NCT number
- NCT01082328
- Status
- Completed
- Phases
- Phase 4
- Enrollment
- 59 participants, actual
- Lead sponsor
- Merck KGaA, Darmstadt, Germany
- Conditions
- Phenylketonuria
- Interventions
- Kuvan®
- First posted
- 2010-03-08
- Last updated
- 2014-02-27
- Results first posted
- 2013-08-01
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With at Least 30 Percent Reduction From Baseline in Blood Phenylalanine (Phe) Level | Baseline up to Day 28 +/- 1 | Response to treatment was defined as 30 percent reduction from Baseline in blood phenylalanine (Phe) Level during the 28 +/- 1 days. |
| Secondary | Number of Participants With Adverse Events (AEs), Treatment Emergent Adverse Events, Treatment Related Adverse Events and AEs Leading to Withdrawal | Baseline up to Day 42 +/- 3 | An Adverse Event (AE) is defined as any untoward medical occurrence in the form of signs, symptoms, abnormal laboratory findings, or diseases that emerges or worsens relative to Baseline during a clinical study with an investigational medicinal product (IMP), regardless of causal relationship and even if no IMP has been administered. |
| Secondary | Percentage of Early-, Late-, Partial-Responders and Non-responders to Treatment With Kuvan® | Baseline up to Day 28 +/- 1 | Early responders defined as percentage of participants with at least 30 percent reduction in Phe levels within the first seven days of treatment. Late responders defined as percentage of participants with less than 30 percent reduction in Phe levels within first seven days of treatment, but at least 30 percent reduction in Phe levels within 28 +/- 1 days of treatment. Partial responders defined as percentage of participants with Phe levels reduction between 10 and 30 percent at any blood measurement within the 28 +/- 1 days of treatment. Non-responders defined as percentage of participants with a Phe level reduction of less than 10 percent within 28 +/- 1 days. |
| Secondary | Percentage of Participants With Greater Than or Equal to (>=) 30 Percent, 20 to 30 Percent, 10 to 20 Percent and Less Than (<) 10 Percent Reduction in Blood Phe Levels According to Phenylketonuria (PKU) Phenotypes | Baseline up to Day 28 +/- 1 | The Phenylketonuria (PKU) is categorized as per phenotype into classical PKU: (blood Phe levels greater than \[\>\] 1200 micromole per liter \[mcmol/l\]), mild PKU (blood Phe levels 600 to 1200 mcmol/l), mild Hyperphenylalaninaemia (HPA) (blood Phe levels 300 to 600 mcmol/l). |
| Secondary | Percentage of Early-, Late- and Partial-Responders According to Phenotype | Baseline up to Day 28 +/- 1 | The PKU is categorized as per phenotype into classical PKU: (blood Phe levels \> 1200 mcmol/l), mild PKU (blood Phe levels 600 to 1200 mcmol/l), mild HPA (blood Phe levels 300 to 600 mcmol/l). Early responders defined as percentage of participants with at least 30 percent reduction in Phe levels within the first seven days of treatment. Late responders defined as percentage of participants with less than 30 percent reduction in Phe levels within first seven days of treatment, but at least 30 percent reduction in Phe levels within 28 +/- 1 days of treatment. Partial responders defined as percentage of participants with Phe levels reduction between 10 and 30 percent at any blood measurement within the 28 +/- 1 days of treatment. |
| Secondary | Mean Change From Baseline in Blood Phenylalanine-to-tyrosine Ratio | Baseline, Day 28 | Phenylalanine-to-tyrosine ratio is the best indicator of dopamine availability in PKU. The change in blood phenylalanine-to-tyrosine ratio at Day 28 was calculated as blood phenylalanine-to-tyrosine ratio at Day 28 minus blood phenylalanine-to-tyrosine ratio at Baseline. |
Source ClinicalTrials.gov
Outcomes6
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 at Least 30 Percent Reduction From Baseline in Blood Phenylalanine (Phe) Level | Baseline up to Day 28 +/- 1 | Full analysis set (FAS) population included all the participants with a valid Baseline blood Phe level measure and who received at least one dose of Kuvan®. | Percentage of participants | NUMBER |
| SECONDARY | Number of Participants With Adverse Events (AEs), Treatment Emergent Adverse Events, Treatment Related Adverse Events and AEs Leading to Withdrawal | Baseline up to Day 42 +/- 3 | Safety population included all the participants with a valid Baseline blood Phe level measure and who received at least one dose of Kuvan®. | Participants | NUMBER |
| SECONDARY | Percentage of Early-, Late-, Partial-Responders and Non-responders to Treatment With Kuvan® | Baseline up to Day 28 +/- 1 | FAS population included all the participants with a valid Baseline blood Phe level measure and who received at least one dose of Kuvan®. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Greater Than or Equal to (>=) 30 Percent, 20 to 30 Percent, 10 to 20 Percent and Less Than (<) 10 Percent Reduction in Blood Phe Levels According to Phenylketonuria (PKU) Phenotypes | Baseline up to Day 28 +/- 1 | FAS population included all the participants with a valid Baseline blood Phe level measure and who received at least one dose of Kuvan®. 'N' (number of participants analyzed) signifies participants who were evaluable for this measure. 'n' signifies number of participants who were evaluable for specified categories at different time points. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Early-, Late- and Partial-Responders According to Phenotype | Baseline up to Day 28 +/- 1 | FAS population included all the participants with a valid Baseline blood Phe level measure and who received at least one dose of Kuvan®. 'N' (number of participants analyzed) signifies participants who were evaluable for this measure. 'n' signifies number of participants who were evaluable for specified categories at different time points. | Percentage of participants | NUMBER |
| SECONDARY | Mean Change From Baseline in Blood Phenylalanine-to-tyrosine Ratio | Baseline, Day 28 | FAS population included all the participants with a valid Baseline blood Phe level measure and who received at least one dose of Kuvan®. 'n' signifies number of participants who were evaluable for specified categories at different time points. | Ratio | MEAN |
Source ClinicalTrials.gov
Results by group32
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 at Least 30 Percent Reduction From Baseline in Blood Phenylalanine (Phe) Level | Kuvan® | 75 | 95% Confidence Interval 62 to 85 | Percentage of participants | |
| Number of Participants With Adverse Events (AEs), Treatment Emergent Adverse Events, Treatment Related Adverse Events and AEs Leading to Withdrawal | Kuvan® | AEs | 58 | Participants | |
| Number of Participants With Adverse Events (AEs), Treatment Emergent Adverse Events, Treatment Related Adverse Events and AEs Leading to Withdrawal | Kuvan® | Treatment Emergent AEs | 57 | Participants | |
| Number of Participants With Adverse Events (AEs), Treatment Emergent Adverse Events, Treatment Related Adverse Events and AEs Leading to Withdrawal | Kuvan® | Treatment Related AEs | 36 | Participants | |
| Number of Participants With Adverse Events (AEs), Treatment Emergent Adverse Events, Treatment Related Adverse Events and AEs Leading to Withdrawal | Kuvan® | AEs leading to withdrawal | 1 | Participants | |
| Percentage of Early-, Late-, Partial-Responders and Non-responders to Treatment With Kuvan® | Kuvan® | Early Responders | 64.4 | 95% Confidence Interval 50.9 to 76.4 | Percentage of participants |
| Percentage of Early-, Late-, Partial-Responders and Non-responders to Treatment With Kuvan® | Kuvan® | Late Responders | 10.2 | 95% Confidence Interval 3.8 to 20.8 | Percentage of participants |
| Percentage of Early-, Late-, Partial-Responders and Non-responders to Treatment With Kuvan® | Kuvan® | Partial Responders | 25.4 | 95% Confidence Interval 15.0 to 38.4 | Percentage of participants |
| Percentage of Early-, Late-, Partial-Responders and Non-responders to Treatment With Kuvan® | Kuvan® | Non-responders | 0 | 95% Confidence Interval 0 to 0 | Percentage of participants |
Percentage of Participants With Greater Than or Equal to (>=) 30 Percent, 20 to 30 Percent, 10 to 20 Percent and Less Than (<) 10 Percent Reduction in Blood Phe Levels According to Phenylketonuria (PKU) Phenotypes | Kuvan® | Mild HPA: >= 30 percent (n=7) | 57 | Percentage of participants |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Kuvan® | 59 | |
| Overall Study | COMPLETED | Kuvan® | 58 | |
| Overall Study | NOT COMPLETED | Kuvan® | 1 |
Source ClinicalTrials.gov
Adverse events73
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 | Dizziness | Cardiac disorders | Kuvan® | 2 | 59 | |
| other | Ear pain | Ear and labyrinth disorders | Kuvan® | 1 | 59 | |
| other | Dry eye | Eye disorders | Kuvan® | 2 | 59 | |
| other | Eye pruritus | Eye disorders | Kuvan® | 1 | 59 | |
| other | Abdominal pain | Gastrointestinal disorders | Kuvan® | 4 | 59 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Kuvan® | 6 | 59 | |
| other | Aphthous stomatitis | Gastrointestinal disorders | Kuvan® | 1 | 59 | |
| other | Diarrhoea | Gastrointestinal disorders | Kuvan® | 14 | 59 | |
| other | Dyspepsia | Gastrointestinal disorders | Kuvan® | 3 | 59 | |
| other | Flatulence | Gastrointestinal disorders | Kuvan® | 3 | 59 |
1 to 10 of 73Source ClinicalTrials.gov
See all 73 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Kuvan® | ||
| Total, other adverse events | Kuvan® | 58 | 59 |
| Total, serious adverse events | Kuvan® | 0 | 59 |
Source ClinicalTrials.gov
Papers22
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/NCT01082328
GET https://conductscience.org/api/trials/v1/NCT01082328/results?page=2