The Effectiveness of Kuvan in Amish PKU Patients

PHASE4CompletedINTERVENTIONAL
Enrollment

7

Participants

Timeline

Start Date

January 22, 2018

Primary Completion Date

November 20, 2018

Study Completion Date

January 1, 2019

Conditions
Phenylketonuria
Interventions
DRUG

saproterin dihydrochloride

The drug will be given as described in the arm/group descriptions.

OTHER

Diet treatment

Patients will maintain a stable, Phe restricted diet (including formula) that is consistent with their diet at the time of enrollment. This will be monitored by food diaries kept for 3 days of each week.

Trial Locations (1)

44106

University Hospitals Cleveland Medical Center, Cleveland

Sponsors

Collaborators (1)

All Listed Sponsors
collaborator

BioMarin Pharmaceutical

INDUSTRY

lead

University Hospitals Cleveland Medical Center

OTHER