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

Sponsor
BioMarin Pharmaceutical (Industry)
Overall Status
Completed
CT.gov ID
NCT01016392
Collaborator
(none)
627
Enrollment
69
Locations
138
Duration (Months)
9.1
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease Intervention/Treatment Phase

    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 Design

    Study Type:
    Observational [Patient Registry]
    Actual Enrollment :
    627 participants
    Observational Model:
    Cohort
    Time Perspective:
    Prospective
    Official Title:
    Kuvan® Adult Maternal Pediatric European Registry
    Study Start Date :
    Dec 1, 2009
    Actual Primary Completion Date :
    May 31, 2021
    Actual Study Completion Date :
    May 31, 2021

    Outcome Measures

    Primary Outcome Measures

    1. Incidence and description of Adverse Events and Serious Adverse Events (AEs/SAEs) [A maximum of 15 years treatment duration.]

    Secondary Outcome Measures

    1. Incidence of AEs/SAEs in specific population (elderly, children, subjects with renal or hepatic insufficiency) [A maximum of 15 years treatment duration.]

    2. Description on somatic growth (in BH4 deficient children < 3 years) [A maximum of 15 years treatment duration.]

    3. Neurocognitive outcomes [A maximum of 15 years treatment duration.]

    4. Neurological and psychiatric assessment [A maximum of 15 years treatment duration.]

    5. Diet and Kuvan® treatment adherence [A maximum of 15 years treatment duration.]

    6. Long-term sensitivity to Kuvan® treatment [A maximum of 15 years treatment duration.]

    7. Blood Phe levels [A maximum of 15 years treatment duration.]

    8. Tyrosine (Tyr) levels [A maximum of 15 years treatment duration.]

    9. Pregnancy and delivery outcomes [A maximum of 15 years treatment duration.]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    4 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Research Site Graz Austria
    2 Research Site Innsbruck Austria
    3 Research Site Salzburg Austria
    4 Research Site Vienna Austria
    5 Research Site Amiens France
    6 Research Site Angers France
    7 Research Site Bordeaux France
    8 Research Site Brest France
    9 Research Site Bron France
    10 Research Site Caen France
    11 Research Site Dijon France
    12 Research Site Lille France
    13 Research Site Limoges France
    14 Research Site Marseille France
    15 Research Site Nantes France
    16 Research Site Nice France
    17 Research Site - Armand Trousseau Paris France
    18 Research Site - Necker Paris France
    19 Research Site Reims France
    20 Research Site Rouen France
    21 Research Site Strasbourg France
    22 Research Site Tarbes France
    23 Research Site Toulouse France
    24 Research Site Tours France
    25 Research Site Vandoeuvre les Nancy France
    26 Research Site Berlin Germany
    27 Research Site Cottbus Germany
    28 Research Site Duesseldorf Germany
    29 Research Site Frankfurt Germany
    30 Research Site Hamburg Germany
    31 Research Site Heidelberg Germany
    32 Research Site Jena Germany
    33 Research Site Leipzig Germany
    34 Research Site Magdeburg Germany
    35 Research Site Muenchen Germany
    36 Research Site Muenster Germany
    37 Research Site Reutlingen Germany
    38 Research Site Bologna Italy
    39 Research Site Catania Italy
    40 Research Site Catanzaro Italy
    41 Research Site Genova Italy
    42 Research Site Milano Italy
    43 Research Site - Ospedale Annunziata Napoli Italy
    44 Research Site - Policlinico Federico Napoli Italy
    45 Research Site Padova Italy
    46 Research Site Roma Italy
    47 Research Site Amsterdam Netherlands
    48 Research Site Groningen Netherlands
    49 Research Site Maastricht Netherlands
    50 Research Site Rotterdam Netherlands
    51 Research Site Utrecht Netherlands
    52 Research Site Porto Portugal
    53 Research Site Banska Bystrica Slovakia
    54 Research Site Kosice Slovakia
    55 Research Site Bilbao Spain
    56 Research Site Esplugues De Llobregat Spain
    57 Research Site Madrid Spain
    58 Research Site Malaga Spain
    59 Research Site Murcia Spain
    60 Research Site Palma De Mallorca Spain
    61 Research Site Pamplona Spain
    62 Research Site Santa Cruz de Tenerife Spain
    63 Research Site Santander Spain
    64 Research Site Santiago de Compostela Spain
    65 Research Site Sevilla Spain
    66 Research Site Zaragoza Spain
    67 Research Site Gothenburg Sweden
    68 Research Site Stockholm Sweden
    69 Research Site Uppsala Sweden

    Sponsors and Collaborators

    • BioMarin Pharmaceutical

    Investigators

    • Study Director: Ece Kucuksayrac, MD, BioMarin Pharmaceutical

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    BioMarin Pharmaceutical
    ClinicalTrials.gov Identifier:
    NCT01016392
    Other Study ID Numbers:
    • EMR700773-001
    First Posted:
    Nov 19, 2009
    Last Update Posted:
    Apr 15, 2022
    Last Verified:
    Apr 1, 2022
    Keywords provided by BioMarin Pharmaceutical
    Observational post-authorization safety study on Kuvan®.
    Additional relevant MeSH terms:
    Phenylketonurias

    Study Results

    No Results Posted as of Apr 15, 2022