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Completed

NCT Number: NCT01016392

Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency

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.

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Key information

Conditions

Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency Amino Acid Metabolism, Inborn Errors Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Phenylketonurias

Age range

4 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Research Site, Graz, Austria

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Adult or pediatric subject (no age limit) of either gender with HPA due to PKU or BH4 deficiency.
  • Have been shown to be responsive to BH4 or Kuvan. (Note: For Spain only-Have been shown to be responsive to BH4 or for the newly diagnosed subjects to be responsive to Kuvan as defined in the Summary of Product Characteristics [SmPC]).
  • Currently being treated with Kuvan® at a participating centre.
  • Subject or parent/legal guardian willing and able to provide written signed informed consent and given before any data collection. If a child is old enough to read and write, a separate assent form will be given.

Exclusion criteria

  • Known hypersensitivity to Kuvan®
  • Legal incapacity or limited legal capacity without legal guardian representation
  • Breast-feeding

Treatment and study plan

Primary outcomes

  1. Incidence and description of Adverse Events and Serious Adverse Events (AEs/SAEs)

    Time frame: A maximum of 15 years treatment duration.

Secondary outcomes

  1. Incidence of AEs/SAEs in specific population (elderly, children, subjects with renal or hepatic insufficiency)

    Time frame: A maximum of 15 years treatment duration.

  2. Description on somatic growth (in BH4 deficient children < 3 years)

    Time frame: A maximum of 15 years treatment duration.

  3. Neurocognitive outcomes

    Time frame: A maximum of 15 years treatment duration.

  4. Neurological and psychiatric assessment

    Time frame: A maximum of 15 years treatment duration.

  5. Diet and Kuvan® treatment adherence

    Time frame: A maximum of 15 years treatment duration.

  6. Long-term sensitivity to Kuvan® treatment

    Time frame: A maximum of 15 years treatment duration.

  7. Blood Phe levels

    Time frame: A maximum of 15 years treatment duration.

  8. Tyrosine (Tyr) levels

    Time frame: A maximum of 15 years treatment duration.

  9. Pregnancy and delivery outcomes

    Time frame: A maximum of 15 years treatment duration.

Sponsors and collaborators

Lead sponsor

BioMarin Pharmaceutical

Industry

Registry information

Official study title

Kuvan® Adult Maternal Pediatric European Registry

Acronym: KAMPER

Important dates

Study start
2009
Primary completion
2021
Study completion
2021
First posted
Nov 19, 2009
Registry last updated
Apr 15, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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