Kuvan® Adult Maternal Paediatric European Registry (KAMPER)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/45633
• 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

BioMarin Pharmaceuticals

Multiple centres: 69 centres are involved in the study

Study timelines

Date when funding contract was signed

Actual: 30/04/2009

Study start date

Actual: 08/12/2009

Data analysis start date

Actual: 28/02/2011

Date of interim report, if expected

Planned: 29/06/2020

Actual: 29/06/2020

Date of final study report

Planned: 30/11/2021

Actual: 10/12/2021

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)