Trial · NCT01977820
Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria
NCT01977820, Phase 2, Terminated, BioMarin Pharmaceutical; results first posted 2016-02-12
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria. Trials, record NCT01977820. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01977820
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria
- NCT number
- NCT01977820
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 2 participants, actual
- Lead sponsor
- BioMarin Pharmaceutical
- Conditions
- Phenylketonuria
- Interventions
- Sapropterin, Placebo
- First posted
- 2013-11-07
- Last updated
- 2018-02-22
- Other registries' IDs
- eudract:2010-024311-13
- Results first posted
- 2016-02-12
- Limitations and caveats
The study was prematurely discontinued by the Sponsor due to severe enrolment difficulties as a consequence of the limited availability of treatment naïve patients with a diagnosis of phenyl ketonuria.
Source ClinicalTrials.gov
Registered outcomes1
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Subjects With Adverse Events (AEs), Serious AEs (SAEs), AEs Leading to Death and AEs Leading to Discontinuation | Screening up to 24 weeks + 4-week follow-up | An AE was defined as any new untoward medical occurrences/worsening of pre-existing medical condition without regard to possibility of causal relationship. A 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
Outcomes1
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Subjects With Adverse Events (AEs), Serious AEs (SAEs), AEs Leading to Death and AEs Leading to Discontinuation | Screening up to 24 weeks + 4-week follow-up | The safety analysis population included all the randomized subjects who received at least one dose of study treatment. | Subjects | NUMBER |
Source ClinicalTrials.gov
Results by group8
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Subjects With Adverse Events (AEs), Serious AEs (SAEs), AEs Leading to Death and AEs Leading to Discontinuation | Sapropterin | AEs | 1 | Subjects | |
| Number of Subjects With Adverse Events (AEs), Serious AEs (SAEs), AEs Leading to Death and AEs Leading to Discontinuation | Placebo | AEs | 0 | Subjects | |
| Number of Subjects With Adverse Events (AEs), Serious AEs (SAEs), AEs Leading to Death and AEs Leading to Discontinuation | Sapropterin | SAEs | 0 | Subjects | |
| Number of Subjects With Adverse Events (AEs), Serious AEs (SAEs), AEs Leading to Death and AEs Leading to Discontinuation | Placebo | SAEs | 0 | Subjects | |
| Number of Subjects With Adverse Events (AEs), Serious AEs (SAEs), AEs Leading to Death and AEs Leading to Discontinuation | Sapropterin | AEs leading to death | 0 | Subjects | |
| Number of Subjects With Adverse Events (AEs), Serious AEs (SAEs), AEs Leading to Death and AEs Leading to Discontinuation | Placebo | AEs leading to death | 0 | Subjects | |
| Number of Subjects With Adverse Events (AEs), Serious AEs (SAEs), AEs Leading to Death and AEs Leading to Discontinuation | Sapropterin | AEs leading to discontinuation | 0 | Subjects | |
| Number of Subjects With Adverse Events (AEs), Serious AEs (SAEs), AEs Leading to Death and AEs Leading to Discontinuation | Placebo | AEs leading to discontinuation | 0 | Subjects |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Sapropterin | 1 | |
| Overall Study | STARTED | Placebo | 1 | |
| Overall Study | COMPLETED | Sapropterin | 0 | |
| Overall Study | COMPLETED | Placebo | 0 | |
| Overall Study | NOT COMPLETED | Sapropterin | 1 | |
| Overall Study | NOT COMPLETED | Placebo | 1 |
Source ClinicalTrials.gov
Adverse events8
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 | Headache | Nervous system disorders | Sapropterin | 1 | 1 | |
| other | Headache | Nervous system disorders | Placebo | 0 | 1 | |
| other | Abdominal Pain | Gastrointestinal disorders | Sapropterin | 1 | 1 | |
| other | Abdominal Pain | Gastrointestinal disorders | Placebo | 0 | 1 | |
| other | Aphthous stomatitis | Gastrointestinal disorders | Sapropterin | 1 | 1 | |
| other | Aphthous stomatitis | Gastrointestinal disorders | Placebo | 0 | 1 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Sapropterin | 1 | 1 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Placebo | 0 | 1 |
Source ClinicalTrials.gov
Adverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Sapropterin | ||
| Total, all-cause mortality | Placebo | ||
| Total, other adverse events | Sapropterin | 1 | 1 |
| Total, other adverse events | Placebo | 0 | 1 |
| Total, serious adverse events | Sapropterin | 0 | 1 |
| Total, serious adverse events | Placebo | 0 | 1 |
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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Sapropterin vs Placebo: serious adverse events |
Source 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/NCT01977820
GET https://conductscience.org/api/trials/v1/NCT01977820/registered