Trial · NCT03864029
Retrospective Observational Safety Effectiveness With Kuvan in hpA
NCT03864029, Completed, BioMarin Pharmaceutical
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Retrospective Observational Safety Effectiveness With Kuvan in hpA. Trials, record NCT03864029. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03864029
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Retrospective Observational Safety Effectiveness With Kuvan in hpA
- NCT number
- NCT03864029
- Status
- Completed
- Enrollment
- 26 participants, actual
- Lead sponsor
- BioMarin Pharmaceutical
- Conditions
- Tetrahydrobiopterin Deficiency
- Interventions
- KUVAN
- First posted
- 2019-03-06
- Last updated
- 2019-03-07
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Diagnosis on BH4 deficiency based on medical history - laboratory testing results indicating sub-type (% in each sub-type) • Incidents and severity of AE reported (% of incident / % of each severity) | 2010-2015 | |
| Primary | Retrospective KUVAN treatment history - dose (mg/kg/day), | 2010-2015 | |
| Primary | Retrospective KUVAN treatment history - length of exposure (days) | 2010-2015 | |
| Primary | Retrospective Baseline Phe concentration ≥ 450 μmol/L - laboratory testing results (μmol/L) | 2010-2015 | |
| Primary | Number of participants with AE reported (% of patient with AE reported) | 2010-2015 | |
| Primary | Incidents and severity of AE reported (% of incident / % of each severity) | 2010-2015 | |
| Primary | Retrospective blood Phe level as indicated in laboratory testing results (μmol/L) | 2010-2015 | |
| Primary | Retrospective body height as indicated in medical note (cm) | 2010-2015 | |
| Primary | Retrospective body weight as indicated in medical note (kg) | 2010-2015 | |
| Primary | Retrospective occipital / frontal circumferences as indicated in medical note (cm) | 2010-2015 |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
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/NCT03864029
GET https://conductscience.org/api/trials/v1/NCT03864029/registered