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

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ClinicalTrials.gov Identifier: NCT01016392
Recruitment Status : Active, not recruiting
First Posted : November 19, 2009
Last Update Posted : January 24, 2018
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

November 18, 2009
November 19, 2009
January 24, 2018
December 2009
December 2024   (Final data collection date for primary outcome measure)
Incidence and description of Adverse Events and Serious Adverse Events (AEs/SAEs) [ Time Frame: A maximum of 15 years treatment duration. ]
Same as current
Complete list of historical versions of study NCT01016392 on ClinicalTrials.gov Archive Site
  • Incidence of AEs/SAEs in specific population (elderly, children, subjects with renal or hepatic insufficiency) [ Time Frame: A maximum of 15 years treatment duration. ]
  • Description on somatic growth (in BH4 deficient children < 3 years) [ Time Frame: A maximum of 15 years treatment duration. ]
  • Neurocognitive outcomes [ Time Frame: A maximum of 15 years treatment duration. ]
  • Neurological and psychiatric assessment [ Time Frame: A maximum of 15 years treatment duration. ]
  • Diet and Kuvan® treatment adherence [ Time Frame: A maximum of 15 years treatment duration. ]
  • Long-term sensitivity to Kuvan® treatment [ Time Frame: A maximum of 15 years treatment duration. ]
  • Blood Phe levels [ Time Frame: A maximum of 15 years treatment duration. ]
  • Tyrosine (Tyr) levels [ Time Frame: A maximum of 15 years treatment duration. ]
  • Pregnancy and delivery outcomes [ Time Frame: A maximum of 15 years treatment duration. ]
Same as current
Not Provided
Not Provided
 
Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency
Kuvan® Adult Maternal Pediatric European Registry

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.
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.
Observational [Patient Registry]
Observational Model: Cohort
Time Perspective: Prospective
5 Years
Not Provided
Non-Probability Sample

All subjects with HPA due to PKU or BH4 deficiency followed at participating centres.

And specific groups (elderly, pediatric, pregnant women and subjects with renal or hepatic insufficiency).

Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency
Not Provided
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
627
625
December 2024
December 2024   (Final data collection date for primary outcome measure)

Inclusion Criteria:

• Adult or pediatric subject (4 years old or older) of either gender with HPA due to PKU.

Or

  • Adult or pediatric subject (no lower limit of age) of either gender with HPA due to 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
  • Have been shown to be responsive to BH4 or for the newly diagnosed subjects fo be responsive to Kuvan as defined in the SmPC

Exclusion Criteria:

  • Known hypersensitivity to Kuvan®
  • Legal incapacity or limited legal capacity without legal guardian representation
  • Breast-feeding
Sexes Eligible for Study: All
4 Years and older   (Child, Adult, Older Adult)
No
Contact information is only displayed when the study is recruiting subjects
Austria,   France,   Germany,   Italy,   Netherlands,   Portugal,   Slovakia,   Spain,   Sweden
Switzerland
 
NCT01016392
EMR700773-001
No
Not Provided
Not Provided
BioMarin Pharmaceutical
BioMarin Pharmaceutical
Not Provided
Study Director: Ignacio Alvarez, MD BioMarin Pharmaceutical
BioMarin Pharmaceutical
January 2018