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

April 14, 2022 updated by: BioMarin Pharmaceutical

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.

Study Overview

Status

Completed

Conditions

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 Type

Observational

Enrollment (Actual)

627

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Austria
      • Graz, Austria
        • Research Site
      • Innsbruck, Austria
        • Research Site
      • Salzburg, Austria
        • Research Site
      • Vienna, Austria
        • Research Site
  • France
      • Amiens, France
        • Research Site
      • Angers, France
        • Research Site
      • Bordeaux, France
        • Research Site
      • Brest, France
        • Research Site
      • Bron, France
        • Research Site
      • Caen, France
        • Research Site
      • Dijon, France
        • Research Site
      • Lille, France
        • Research Site
      • Limoges, France
        • Research Site
      • Marseille, France
        • Research Site
      • Nantes, France
        • Research Site
      • Nice, France
        • Research Site
      • Paris, France
        • Research Site - Armand Trousseau
      • Paris, France
        • Research Site - Necker
      • Reims, France
        • Research Site
      • Rouen, France
        • Research Site
      • Strasbourg, France
        • Research Site
      • Tarbes, France
        • Research Site
      • Toulouse, France
        • Research Site
      • Tours, France
        • Research Site
      • Vandoeuvre les Nancy, France
        • Research Site
  • Germany
      • Berlin, Germany
        • Research Site
      • Cottbus, Germany
        • Research Site
      • Duesseldorf, Germany
        • Research Site
      • Frankfurt, Germany
        • Research Site
      • Hamburg, Germany
        • Research Site
      • Heidelberg, Germany
        • Research Site
      • Jena, Germany
        • Research Site
      • Leipzig, Germany
        • Research Site
      • Magdeburg, Germany
        • Research Site
      • Muenchen, Germany
        • Research Site
      • Muenster, Germany
        • Research Site
      • Reutlingen, Germany
        • Research Site
  • Italy
      • Bologna, Italy
        • Research Site
      • Catania, Italy
        • Research Site
      • Catanzaro, Italy
        • Research Site
      • Genova, Italy
        • Research Site
      • Milano, Italy
        • Research Site
      • Napoli, Italy
        • Research Site - Ospedale Annunziata
      • Napoli, Italy
        • Research Site - Policlinico Federico
      • Padova, Italy
        • Research Site
      • Roma, Italy
        • Research Site
  • Netherlands
      • Amsterdam, Netherlands
        • Research Site
      • Groningen, Netherlands
        • Research Site
      • Maastricht, Netherlands
        • Research Site
      • Rotterdam, Netherlands
        • Research Site
      • Utrecht, Netherlands
        • Research Site
  • Portugal
      • Porto, Portugal
        • Research Site
  • Slovakia
      • Banska Bystrica, Slovakia
        • Research Site
      • Kosice, Slovakia
        • Research Site
  • Spain
      • Bilbao, Spain
        • Research Site
      • Esplugues De Llobregat, Spain
        • Research Site
      • Madrid, Spain
        • Research Site
      • Malaga, Spain
        • Research Site
      • Murcia, Spain
        • Research Site
      • Palma De Mallorca, Spain
        • Research Site
      • Pamplona, Spain
        • Research Site
      • Santa Cruz de Tenerife, Spain
        • Research Site
      • Santander, Spain
        • Research Site
      • Santiago de Compostela, Spain
        • Research Site
      • Sevilla, Spain
        • Research Site
      • Zaragoza, Spain
        • Research Site
  • Sweden
      • Gothenburg, Sweden
        • Research Site
      • Stockholm, Sweden
        • Research Site
      • Uppsala, Sweden
        • Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 months and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

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).

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence and description of Adverse Events and Serious Adverse Events (AEs/SAEs)
Time Frame: A maximum of 15 years treatment duration.
A maximum of 15 years treatment duration.

Secondary Outcome Measures

Outcome Measure
Time Frame
Incidence of AEs/SAEs in specific population (elderly, children, subjects with renal or hepatic insufficiency)
Time Frame: A maximum of 15 years treatment duration.
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.
A maximum of 15 years treatment duration.
Neurocognitive outcomes
Time Frame: A maximum of 15 years treatment duration.
A maximum of 15 years treatment duration.
Neurological and psychiatric assessment
Time Frame: A maximum of 15 years treatment duration.
A maximum of 15 years treatment duration.
Diet and Kuvan® treatment adherence
Time Frame: A maximum of 15 years treatment duration.
A maximum of 15 years treatment duration.
Long-term sensitivity to Kuvan® treatment
Time Frame: A maximum of 15 years treatment duration.
A maximum of 15 years treatment duration.
Blood Phe levels
Time Frame: A maximum of 15 years treatment duration.
A maximum of 15 years treatment duration.
Tyrosine (Tyr) levels
Time Frame: A maximum of 15 years treatment duration.
A maximum of 15 years treatment duration.
Pregnancy and delivery outcomes
Time Frame: A maximum of 15 years treatment duration.
A maximum of 15 years treatment duration.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

BioMarin Pharmaceutical

Investigators

  • Study Director: Ece Kucuksayrac, MD, BioMarin Pharmaceutical

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

December 1, 2009

Primary Completion (Actual)

May 31, 2021

Study Completion (Actual)

May 31, 2021

Study Registration Dates

First Submitted

November 18, 2009

First Submitted That Met QC Criteria

November 18, 2009

First Posted (Estimate)

November 19, 2009

Study Record Updates

Last Update Posted (Actual)

April 15, 2022

Last Update Submitted That Met QC Criteria

April 14, 2022

Last Verified

April 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • EMR700773-001

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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