Ignite Creation Date:
2025-12-24 @ 3:50 PM
Ignite Modification Date:
2025-12-27 @ 10:40 AM
Study NCT ID:
NCT01016392
Status:
COMPLETED
Last Update Posted:
2022-04-15
First Post:
2009-11-18
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Switzerland']}, 'conditionBrowseModule': {'meshes': [{'id': 'D010661', 'term': 'Phenylketonurias'}], 'ancestors': [{'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D000592', 'term': 'Amino Acid Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 627}, 'targetDuration': '5 Years', 'patientRegistry': True}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2009-12'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-04', 'completionDateStruct': {'date': '2021-05-31', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-04-14', 'studyFirstSubmitDate': '2009-11-18', 'studyFirstSubmitQcDate': '2009-11-18', 'lastUpdatePostDateStruct': {'date': '2022-04-15', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2009-11-19', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2021-05-31', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence and description of Adverse Events and Serious Adverse Events (AEs/SAEs)', 'timeFrame': 'A maximum of 15 years treatment duration.'}], 'secondaryOutcomes': [{'measure': 'Incidence of AEs/SAEs in specific population (elderly, children, subjects with renal or hepatic insufficiency)', 'timeFrame': 'A maximum of 15 years treatment duration.'}, {'measure': 'Description on somatic growth (in BH4 deficient children < 3 years)', 'timeFrame': 'A maximum of 15 years treatment duration.'}, {'measure': 'Neurocognitive outcomes', 'timeFrame': 'A maximum of 15 years treatment duration.'}, {'measure': 'Neurological and psychiatric assessment', 'timeFrame': 'A maximum of 15 years treatment duration.'}, {'measure': 'Diet and Kuvan® treatment adherence', 'timeFrame': 'A maximum of 15 years treatment duration.'}, {'measure': 'Long-term sensitivity to Kuvan® treatment', 'timeFrame': 'A maximum of 15 years treatment duration.'}, {'measure': 'Blood Phe levels', 'timeFrame': 'A maximum of 15 years treatment duration.'}, {'measure': 'Tyrosine (Tyr) levels', 'timeFrame': 'A maximum of 15 years treatment duration.'}, {'measure': 'Pregnancy and delivery outcomes', 'timeFrame': 'A maximum of 15 years treatment duration.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Observational post-authorization safety study on Kuvan®.'], 'conditions': ['Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency']}, 'descriptionModule': {'briefSummary': "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.\n\nKuvan® received marketed authorisation in Europe in December 2008 and is now available in several European countries for the treatment of Hyperphenylalaninemia (HPA).\n\nThe primary objective is to assess the long-term safety in subjects treated with Kuvan®.\n\nSecondary objectives are to provide additional information regarding:\n\n* Safety in specific subject groups (elderly, pediatric, pregnant women and subjects with renal or hepatic insufficiency).\n* Growth and neurocognitive outcomes for subjects with hyperphenylalaninemia (HPA) who are receiving treatment with Kuvan®.\n* 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).\n* Assessment of adherence to diet and to Kuvan®.\n* Assessment of long-term sensitivity to Kuvan®treatment.", 'detailedDescription': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '4 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'All subjects with HPA due to PKU or BH4 deficiency followed at participating centres.\n\nAnd specific groups (elderly, pediatric, pregnant women and subjects with renal or hepatic insufficiency).', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Adult or pediatric subject (no age limit) of either gender with HPA due to PKU or BH4 deficiency.\n* 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\\]).\n* Currently being treated with Kuvan® at a participating centre.\n* 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.\n\nExclusion Criteria:\n\n* Known hypersensitivity to Kuvan®\n* Legal incapacity or limited legal capacity without legal guardian representation\n* Breast-feeding'}, 'identificationModule': {'nctId': 'NCT01016392', 'acronym': 'KAMPER', 'briefTitle': 'Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency', 'organization': {'class': 'INDUSTRY', 'fullName': 'BioMarin Pharmaceutical'}, 'officialTitle': 'Kuvan® Adult Maternal Pediatric European Registry', 'orgStudyIdInfo': {'id': 'EMR700773-001'}}, 'contactsLocationsModule': {'locations': [{'city': 'Graz', 'country': 'Austria', 'facility': 'Research Site', 'geoPoint': {'lat': 47.06733, 'lon': 15.44197}}, {'city': 'Innsbruck', 'country': 'Austria', 'facility': 'Research Site', 'geoPoint': {'lat': 47.26266, 'lon': 11.39454}}, {'city': 'Salzburg', 'country': 'Austria', 'facility': 'Research Site', 'geoPoint': {'lat': 47.79941, 'lon': 13.04399}}, {'city': 'Vienna', 'country': 'Austria', 'facility': 'Research Site', 'geoPoint': {'lat': 48.20849, 'lon': 16.37208}}, {'city': 'Amiens', 'country': 'France', 'facility': 'Research Site', 'geoPoint': {'lat': 49.9, 'lon': 2.3}}, {'city': 'Angers', 'country': 'France', 'facility': 'Research Site', 'geoPoint': {'lat': 47.47156, 'lon': -0.55202}}, {'city': 'Bordeaux', 'country': 'France', 'facility': 'Research Site', 'geoPoint': {'lat': 44.84124, 'lon': -0.58046}}, {'city': 'Brest', 'country': 'France', 'facility': 'Research Site', 'geoPoint': {'lat': 48.39029, 'lon': -4.48628}}, {'city': 'Bron', 'country': 'France', 'facility': 'Research Site', 'geoPoint': {'lat': 45.73865, 'lon': 4.91303}}, {'city': 'Caen', 'country': 'France', 'facility': 'Research Site', 'geoPoint': {'lat': 49.18585, 'lon': -0.35912}}, {'city': 'Dijon', 'country': 'France', 'facility': 'Research Site', 'geoPoint': {'lat': 47.31344, 'lon': 5.01391}}, {'city': 'Lille', 'country': 'France', 'facility': 'Research Site', 'geoPoint': {'lat': 50.63391, 'lon': 3.05512}}, {'city': 'Limoges', 'country': 'France', 'facility': 'Research Site', 'geoPoint': {'lat': 45.83362, 'lon': 1.24759}}, {'city': 'Marseille', 'country': 'France', 'facility': 'Research Site', 'geoPoint': {'lat': 43.29695, 'lon': 5.38107}}, {'city': 'Nantes', 'country': 'France', 'facility': 'Research Site', 'geoPoint': {'lat': 47.21725, 'lon': -1.55336}}, {'city': 'Nice', 'country': 'France', 'facility': 'Research Site', 'geoPoint': {'lat': 43.70313, 'lon': 7.26608}}, {'city': 'Paris', 'country': 'France', 'facility': 'Research Site - 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