Trial · NCT01732471
Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria
NCT01732471, Phase 3, Completed, Merck KGaA, Darmstadt, Germany; results first posted 2014-07-28
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria. Trials, record NCT01732471. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01732471
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria
- NCT number
- NCT01732471
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 90 participants, actual
- Lead sponsor
- Merck KGaA, Darmstadt, Germany
- Conditions
- Phenylketonuria
- Interventions
- Kuvan®
- First posted
- 2012-11-22
- Last updated
- 2014-07-28
- Results first posted
- 2014-07-28
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Response to Kuvan® (Sapropterin Dihydrochloride) Treatment | Day 8 | Response to Kuvan® (sapropterin dihydrochloride) treatment was defined as a reduction in blood phenylalanine levels of greater than or equal to 30% at Day 8 as compared to baseline. |
| Secondary | Percent Change From Baseline in Blood Phenylalanine Levels at Day 8 in Overall Population | Baseline, Day 8 | Percent change in blood phenylalanine levels after 8-day Kuvan® therapy (response test period) was calculated as (blood phenylalanine level at Day 8 minus blood phenylalanine level at baseline)\*100/ blood phenylalanine level at baseline. |
| Secondary | Percent Change From Baseline in Blood Phenylalanine Levels at Day 8 in Sub-population of Responders | Baseline, Day 8 | Percent change in blood phenylalanine levels after 8-day Kuvan® therapy (response test period) was calculated as (blood phenylalanine level at Day 8 minus blood phenylalanine level at baseline)\*100/ blood phenylalanine level at baseline. |
| Secondary | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) in Overall Safety Population | Baseline up to Week 11 | An adverse event (AE) was defined as any new untoward medical occurrences/worsening of pre-existing medical condition without regard to possibility of causal relationship. A serious adverse event (SAE) was an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect. |
Source ClinicalTrials.gov
Outcomes4
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 Response to Kuvan® (Sapropterin Dihydrochloride) Treatment | Day 8 | Overall (ITT) population included all participants who had efficacy assessment result from at least 1 visit except for the inclusion visit. | percentage of participants | NUMBER |
| SECONDARY | Percent Change From Baseline in Blood Phenylalanine Levels at Day 8 in Overall Population | Baseline, Day 8 | Overall (ITT) population included all participants who had efficacy assessment result from at least 1 visit except for the inclusion visit. | percent change | MEAN |
| SECONDARY | Percent Change From Baseline in Blood Phenylalanine Levels at Day 8 in Sub-population of Responders | Baseline, Day 8 | Sub-population of responders included participants with reduction in blood phenylalanine levels of greater than or equal to 30% at Day 8 as compared to baseline. | percent change | MEAN |
| SECONDARY | Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) in Overall Safety Population | Baseline up to Week 11 | Overall safety population included all participants who received at least 1 dose of investigational medicinal product. | participants | NUMBER |
Source ClinicalTrials.gov
Results by group5
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 Response to Kuvan® (Sapropterin Dihydrochloride) Treatment | Kuvan® | 33.3 | 95% Confidence Interval 23.7 to 44.1 | percentage of participants | |
| Percent Change From Baseline in Blood Phenylalanine Levels at Day 8 in Overall Population | Kuvan® | -14.14 | Standard Deviation 28.35 | percent change | |
| Percent Change From Baseline in Blood Phenylalanine Levels at Day 8 in Sub-population of Responders | Kuvan® | -44.25 | Standard Deviation 15.13 | percent change | |
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) in Overall Safety Population | Kuvan® | AEs | 24 | participants | |
| Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) in Overall Safety Population | Kuvan® | SAEs | 1 | participants |
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 | Kuvan® | 90 | |
| Overall Study | COMPLETED | Kuvan® | 89 | |
| Overall Study | NOT COMPLETED | Kuvan® | 1 |
Source ClinicalTrials.gov
Adverse events20
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 | Kuvan® | 1 | 90 | |
| other | Bronchitis | Respiratory, thoracic and mediastinal disorders | Kuvan® | 1 | 90 | |
| other | Diarrhoea | Gastrointestinal disorders | Kuvan® | 1 | 90 | |
| other | Dysmenorrhoea | Reproductive system and breast disorders | Kuvan® | 1 | 90 | |
| other | Faeces pale | Gastrointestinal disorders | Kuvan® | 1 | 90 | |
| other | Gastroduodenitis | Gastrointestinal disorders | Kuvan® | 1 | 90 | |
| other | Genital labial adhesions | Reproductive system and breast disorders | Kuvan® | 1 | 90 | |
| other | Haematuria | Renal and urinary disorders | Kuvan® | 1 | 90 | |
| other | Heat stroke | Injury, poisoning and procedural complications | Kuvan® | 1 | 90 | |
| other | Iodine deficiency | Metabolism and nutrition disorders | Kuvan® | 1 | 90 |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 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® | 23 | 90 |
| Total, serious adverse events | Kuvan® | 1 | 90 |
Source ClinicalTrials.gov
Papers0
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/NCT01732471
GET https://conductscience.org/api/trials/v1/NCT01732471/registered