Study identification

EU PAS number

EUPAS17360

Study ID

45633

Official title and acronym

Kuvan® Adult Maternal Paediatric European Registry (KAMPER)

DARWIN EU® study

No

Study countries

Austria
France
Germany
Italy
Netherlands
Portugal
Slovakia
Spain
Sweden

Study description

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.

Study status

Finalised
Research institutions and networks

Institutions

Multiple centres: 69 centres are involved in the study

Contact details

Kucuksayrac Ece

Primary lead investigator

Study timelines

Date when funding contract was signed

Actual:

Study start date

Actual:

Data analysis start date

Actual:

Date of interim report, if expected

Planned:
Actual:

Date of final study report

Planned:
Actual:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

BioMarin International Limited
Regulatory

Was the study required by a regulatory body?

Yes

Is the study required by a Risk Management Plan (RMP)?

EU RMP category 3 (required)