Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency Clinical Trial
— KAMPEROfficial title:
Kuvan® Adult Maternal Pediatric European Registry
| NCT number | NCT01016392 |
| Other study ID # | EMR700773-001 |
| Secondary ID | |
| Status | Completed |
| Phase | |
| First received | |
| Last updated | |
| Start date | December 2009 |
| Est. completion date | May 31, 2021 |
| Verified date | April 2022 |
| Source | BioMarin Pharmaceutical |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Observational [Patient 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.
| Status | Completed |
| Enrollment | 627 |
| Est. completion date | May 31, 2021 |
| Est. primary completion date | May 31, 2021 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 4 Years and older |
| Eligibility | 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 |
| Country | Name | City | State |
|---|---|---|---|
| Austria | Research Site | Graz | |
| Austria | Research Site | Innsbruck | |
| Austria | Research Site | Salzburg | |
| Austria | Research Site | Vienna | |
| France | Research Site | 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 - Armand Trousseau | Paris | |
| France | Research Site - Necker | Paris | |
| France | Research Site | 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 | |
| Germany | Research Site | 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 | |
| Italy | Research Site | Bologna | |
| Italy | Research Site | Catania | |
| Italy | Research Site | Catanzaro | |
| Italy | Research Site | Genova | |
| Italy | Research Site | Milano | |
| Italy | Research Site - Ospedale Annunziata | Napoli | |
| Italy | Research Site - Policlinico Federico | Napoli | |
| Italy | Research Site | Padova | |
| Italy | Research Site | Roma | |
| Netherlands | Research Site | Amsterdam | |
| Netherlands | Research Site | Groningen | |
| Netherlands | Research Site | Maastricht | |
| Netherlands | Research Site | Rotterdam | |
| Netherlands | Research Site | Utrecht | |
| Portugal | Research Site | Porto | |
| Slovakia | Research Site | Banska Bystrica | |
| Slovakia | Research Site | Kosice | |
| Spain | Research Site | 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 | |
| Sweden | Research Site | Gothenburg | |
| Sweden | Research Site | Stockholm | |
| Sweden | Research Site | Uppsala |
| Lead Sponsor | Collaborator |
|---|---|
| BioMarin Pharmaceutical |
Austria, France, Germany, Italy, Netherlands, Portugal, Slovakia, Spain, Sweden,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Incidence and description of Adverse Events and Serious Adverse Events (AEs/SAEs) | A maximum of 15 years treatment duration. | ||
| Secondary | Incidence of AEs/SAEs in specific population (elderly, children, subjects with renal or hepatic insufficiency) | A maximum of 15 years treatment duration. | ||
| Secondary | Description on somatic growth (in BH4 deficient children < 3 years) | A maximum of 15 years treatment duration. | ||
| Secondary | Neurocognitive outcomes | A maximum of 15 years treatment duration. | ||
| Secondary | Neurological and psychiatric assessment | A maximum of 15 years treatment duration. | ||
| Secondary | Diet and Kuvan® treatment adherence | A maximum of 15 years treatment duration. | ||
| Secondary | Long-term sensitivity to Kuvan® treatment | A maximum of 15 years treatment duration. | ||
| Secondary | Blood Phe levels | A maximum of 15 years treatment duration. | ||
| Secondary | Tyrosine (Tyr) levels | A maximum of 15 years treatment duration. | ||
| Secondary | Pregnancy and delivery outcomes | A maximum of 15 years treatment duration. |