Description Module
The Description Module contains narrative descriptions of the clinical trial, including a brief summary and detailed description. These descriptions provide important information about the study's purpose, methodology, and key details in language accessible to both researchers and the general public.
Description Module path is as follows:
Study -> Protocol Section -> Description Module
Description Module
Ignite Creation Date:
2025-12-24 @ 3:50 PM
Ignite Modification Date:
2025-12-24 @ 3:50 PM
NCT ID:
NCT01016392
Brief Summary:
Kuvan® is a synthetic copy of a body's own substance called tetrahydrobiopterin (BH4). BH4 is required by the body to use an amino acid called phenylalanine in order to build another substance called tyrosine.
Kuvan® received marketed authorisation in Europe in December 2008 and is now available in several European countries for the treatment of Hyperphenylalaninemia (HPA).
The primary objective is to assess the long-term safety in subjects treated with Kuvan®.
Secondary objectives are to provide additional information regarding:
* Safety in specific subject groups (elderly, pediatric, pregnant women and subjects with renal or hepatic insufficiency).
* Growth and neurocognitive outcomes for subjects with hyperphenylalaninemia (HPA) who are receiving treatment with Kuvan®.
* Progress and outcome of pregnancy for women with HPA who become pregnant while receiving treatment with Kuvan® (these women will be enrolled in a dedicated sub-registry).
* Assessment of adherence to diet and to Kuvan®.
* Assessment of long-term sensitivity to Kuvan®treatment.
Detailed Description:
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.
Study:
NCT01016392
Study Brief:
Protocol Section:
NCT01016392