Phase 2
Completed N=21
Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response
Source: ClinicalTrials.gov NCT00841100 ↗Enrolled (actual)
21
Serious AEs
0.0%
Results posted
Oct 2019
Primary outcomePrimary: Percent Change in Blood Phenylalanine — -13.71; -8.13; 48.75 Percent change in blood phenylalanine
Summary
The investigators will investigate the effect of blood phenylalanine on Kuvan responsiveness in the same patients with PKU when their blood phenylalanine concentrations are altered by diet.
Lowering blood phenylalanine concentrations in Kuvan non-responsive patients with PKU will increase the frequency of Kuvan responsiveness.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change in Blood Phenylalanine |
-13.71; -8.13; 48.75 | — |
Eligibility Criteria
Inclusion Criteria
- all patients with a diagnosis of phenylketonuria
- patients who are over the age of 4 years,
- patients with a fasting blood phenylalanine ≥600µmol/l
Exclusion Criteria
- female patients who are pregnant or attempting to become pregnant
- children under four years of age
- Concurrent disease of condition that would interfere with study participation or safety such as history of neurological compromise or major medical, psychiatric or learning disorder unrelated to PKU
- Any condition that, in the view of the Investigators, places subject at high risk of poor treatment compliance or of not completing the study
- Use of L-Dopa, methotrexate, or other drugs that may inhibit folate metabolism
Data sourced from ClinicalTrials.gov (NCT00841100). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.