A Phase IIIb, Multicentre, Open-Label, Randomized,Controlled Study of the Efficacy, Safety, andPopulation Pharmacokinetics of SapropterinDihydrochloride (Kuvan®) in Phenylketonuria (PKU)Patients

Recruiting

• Conditions: PKU, KUVA

• Registration Number: NL-OMON26957
• Lead Sponsor: In The Netherlands1. University Medical Center Amsterdam 2. Academisch Ziekenhuis Maastricht

Brief Summary

The first publication will be a publication of the results of the analysis of the primary endpoint(s) that will include data from all trial sites. The Investigator will inform the Sponsor in advance about any plans to publish or present data from the trial. Any publications and presentations of the results (abstracts in journals or newspapers, oral presentations, etc.), either in whole or in part, by Investigators or their representatives, will require pre-submission review by the Sponsor. The Sponsor will not suppress or veto publications but maintains the right to delay publication in order to protect intellectual property rights.

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Last Update Posted: 28 February 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: Not specified
• Target Recruitment: 50

Inclusion Criteria

1. Male or female PKU infants and young children
<4 years of age at the scheduled Day 1 visit of the
26-week Study Period (taking into consideration
the maximum of 21 days in the Screening Period);

Exclusion Criteria

The exclusion criteria consist of:

1. Use of Kuvan®, Biopten®, or any unregistered
preparation of tetrahydrobiopterin within the
previous 30 days, unless for the purposes of a BH4
responsiveness test;

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Dietary Phe tolerance after 26 weeks (6 months) of<br>treatment with Kuvan® + a Phe-restricted diet, as<br>compared to just a Phe-restricted diet alone.