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Phenylketonuria Clinical Trials

A listing of Phenylketonuria medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.


Found (7) clinical trials

The BMN 165 clinical development program has been designed to demonstrate the safety and efficacy of BMN 165 in reducing blood Phe concentrations in patients equal or greater than 18 years old with hyperphenylalaninemia due to PKU. Study BMN 165-301 is a Phase 3, open-label, randomized study designed to further ...


PKUDOS: Phenylketonuria (PKU) Demographic Outcomes and Safety Registry

The PKUDOS program is a voluntary, multicenter, strictly observational program for patients with PKU who have either received Kuvan therapy, or currently receive Kuvan, or intends to begin receiving Kuvan therapy within 90 days of entering the registry.

Phase N/A

Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response

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.

Phase N/A

Response to Phenylketonuria to Tetrahydrobiopterin (BH4)

Phenylketonuria(PKU) is an autosomal recessive disorder caused by a defect in the enzyme phenylalanine hydroxylase(PAH). this incidence of PKU in the US is about 1:15,000 births. The disease is pan ethnic with more prevalence among individuals of European ancestry. Recently, a number of patients with PKU showed a marked decrease ...


GMP Drink for PKU Study

This study will evaluate the safety, gastrointestinal (GI) tolerance, acceptability and compliance of a Glycomacropeptide-based protein substitute for patients with Phenylketonuria (PKU).

Phase N/A

Evaluation of PKU Explore

This is a prospective, acceptability study to evaluate the gastrointestinal tolerance, palatability and adherence of PKU explore in a four-week period for 15 participants aged between 6 months and 5 years for the dietary management of PKU. The outcome of this assessment will be used in a submission to the ...

Phase N/A

Observational Study on the Long Term Safety of Kuvan Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency

This is an observational, multicenter, drug registry Study. The study will have a total duration of 15 years, including a 10-year inclusion period. No diagnostic, therapeutic or experimental intervention is involved. Subjects will receive clinical assessments, medications and treatments solely as determined by their study physician.

Phase N/A