age up to 4 Years
Item
Alter bis 4 Jahre
boolean
C0001779 (UMLS CUI 2011AA)
397669002 (SNOMED CT 2011_0131)
Male or female PKU infants and young children <4 years of age at the scheduled Day 1 visit of the 26-week Study Period (taking into consideration the maximum of 21 days in the Screening Period).
Item
Male or female PKU infants and young children <4 years of age at the scheduled Day 1 visit of the 26-week Study Period (taking into consideration the maximum of 21 days in the Screening Period).
boolean
C0024554 (UMLS CUI 2011AA)
248153007 (SNOMED CT 2011_0131)
M (HL7 V3 2006_05)
C0015780 (UMLS CUI 2011AA)
248152002 (SNOMED CT 2011_0131)
F (HL7 V3 2006_05)
C0031485 (UMLS CUI 2011AA)
C0011900 (UMLS CUI 2011AA)
439401001 (SNOMED CT 2011_0131)
MTHU008876 (LOINC Version 232)
C0021270 (UMLS CUI 2011AA)
133931009 (SNOMED CT 2011_0131)
10021731 (MedDRA 14.1)
C0008059 (UMLS CUI 2011AA)
67822003 (SNOMED CT 2011_0131)
CHILD (HL7 V3 2006_05)
Confirmed clinical and biochemical PKU, including at least two previous blood Phe levels ≥ 400 micromol/L obtained on 2 separate occasions.
Item
Confirmed clinical and biochemical PKU, including at least two previous blood Phe levels ≥ 400 micromol/L obtained on 2 separate occasions.
boolean
C0750484 (UMLS CUI 2011AA)
C0205210 (UMLS CUI 2011AA)
58147004 (SNOMED CT 2011_0131)
C0205474 (UMLS CUI 2011AA)
80534008 (SNOMED CT 2011_0131)
C0031485 (UMLS CUI 2011AA)
C0205156 (UMLS CUI 2011AA)
9130008 (SNOMED CT 2011_0131)
C0005768 (UMLS CUI 2011AA)
C0428204 (UMLS CUI 2011AA)
365727002 (SNOMED CT 2011_0131)
Previously responded, as assessed by the Investigator, to a BH4 test, if all 3 of the following criteria are satisfied:
Item
Previously responded, as assessed by the Investigator, to a BH4 test, if all 3 of the following criteria are satisfied:
boolean
C0205156 (UMLS CUI 2011AA)
9130008 (SNOMED CT 2011_0131)
C0871261 (UMLS CUI 2011AA)
C0048897 (UMLS CUI 2011AA)
432859002 (SNOMED CT 2011_0131)
MTHU035851 (LOINC Version 232)
C0022885 (UMLS CUI 2011AA)
15220000 (SNOMED CT 2011_0131)
10059938 (MedDRA 14.1)
C0750557 (UMLS CUI 2011AA)
C1550543 (UMLS CUI 2011AA)
FLFS (HL7 V3 2006_05)
C0332282 (UMLS CUI 2011AA)
255260001 (SNOMED CT 2011_0131)
C0205449 (UMLS CUI 2011AA)
421291004 (SNOMED CT 2011_0131)
CL414621 (UMLS CUI 2011AA)
The BH4 dose was 20 mg/kg/day.
Item
The BH4 dose was 20 mg/kg/day.
boolean
C0048897 (UMLS CUI 2011AA)
432859002 (SNOMED CT 2011_0131)
MTHU035851 (LOINC Version 232)
C0178602 (UMLS CUI 2011AA)
260911001 (SNOMED CT 2011_0131)
The duration of the test was at least for 24 hours.
Item
The duration of the test was at least for 24 hours.
boolean
C0429930 (UMLS CUI 2011AA)
252129004 (SNOMED CT 2011_0131)
C0439091 (UMLS CUI 2011AA)
276138003 (SNOMED CT 2011_0131)
GE (HL7 V3 2006_05)
C0205447 (UMLS CUI 2011AA)
38112003 (SNOMED CT 2011_0131)
C0439228 (UMLS CUI 2011AA)
258703001 (SNOMED CT 2011_0131)
D (HL7 V3 2006_05)
A 30% decrease in blood Phe levels.
Item
A 30% decrease in blood Phe levels.
boolean
C0547047 (UMLS CUI 2011AA)
260370003 (SNOMED CT 2011_0131)
C0005768 (UMLS CUI 2011AA)
C0428204 (UMLS CUI 2011AA)
365727002 (SNOMED CT 2011_0131)
Defined level of dietary Phe tolerance consistent with the diagnosis of PKU;
Item
Defined level of dietary Phe tolerance consistent with the diagnosis of PKU;
boolean
C1704788 (UMLS CUI 2011AA)
C0456079 (UMLS CUI 2011AA)
276625007 (SNOMED CT 2011_0131)
C0012155 (UMLS CUI 2011AA)
41829006 (SNOMED CT 2011_0131)
C0031453 (UMLS CUI 2011AA)
63004003 (SNOMED CT 2011_0131)
MTHU003552 (LOINC Version 232)
C0013220 (UMLS CUI 2011AA)
228432001 (SNOMED CT 2011_0131)
10052804 (MedDRA 14.1)
C0332290 (UMLS CUI 2011AA)
385433004 (SNOMED CT 2011_0131)
C0011900 (UMLS CUI 2011AA)
439401001 (SNOMED CT 2011_0131)
MTHU008876 (LOINC Version 232)
C0031485 (UMLS CUI 2011AA)
Good adherence to dietary treatment, including prescribed dietary Phe restriction and prescribed amounts of Phe-free protein supplements and low-Phe foods.
Item
Good adherence to dietary treatment, including prescribed dietary Phe restriction and prescribed amounts of Phe-free protein supplements and low-Phe foods.
boolean
C0205170 (UMLS CUI 2011AA)
20572008 (SNOMED CT 2011_0131)
C0012155 (UMLS CUI 2011AA)
41829006 (SNOMED CT 2011_0131)
C1171369 (UMLS CUI 2011AA)
C0457976 (UMLS CUI 2011AA)
182927005 (SNOMED CT 2011_0131)
C0770246 (UMLS CUI 2011AA)
226282004 (SNOMED CT 2011_0131)
Maintenance of blood Phe levels within the therapeutic target range of 120-360 micromol/L (defined as ≥120 to < 360 micromol/L) over a 1-month period prior to Screening, as assessed by the Investigator.
Item
Maintenance of blood Phe levels within the therapeutic target range of 120-360 micromol/L (defined as ≥120 to < 360 micromol/L) over a 1-month period prior to Screening, as assessed by the Investigator.
boolean
C0024501 (UMLS CUI 2011AA)
C0005768 (UMLS CUI 2011AA)
C0428204 (UMLS CUI 2011AA)
365727002 (SNOMED CT 2011_0131)
C0332285 (UMLS CUI 2011AA)
18720000 (SNOMED CT 2011_0131)
C0039798 (UMLS CUI 2011AA)
C1521840 (UMLS CUI 2011AA)
C1514721 (UMLS CUI 2011AA)
Parent(s) and/or guardian(s) willing to comply with all study procedures, maintain strict adherence to the diet, and willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to any study procedures.
Item
Parent(s) and/or guardian(s) willing to comply with all study procedures, maintain strict adherence to the diet, and willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to any study procedures.
boolean
C0030551 (UMLS CUI 2011AA)
40683002 (SNOMED CT 2011_0131)
PARNT (HL7 V3 2006_05)
C1274041 (UMLS CUI 2011AA)
394619001 (SNOMED CT 2011_0131)
MTHU014954 (LOINC Version 232)
C0600109 (UMLS CUI 2011AA)
225466006 (SNOMED CT 2011_0131)
C0525058 (UMLS CUI 2011AA)
C0012155 (UMLS CUI 2011AA)
41829006 (SNOMED CT 2011_0131)
C1321605 (UMLS CUI 2011AA)
405078008 (SNOMED CT 2011_0131)
C0085732 (UMLS CUI 2011AA)
371150009 (SNOMED CT 2011_0131)
C0184704 (UMLS CUI 2011AA)
55149002 (SNOMED CT 2011_0131)
Use of Kuvan, Biopten, or any unregistered preparation of tetrahydrobiopterin within the previous 30 days, unless for the purposes of a BH4 responsiveness test.
Item
Use of Kuvan, Biopten, or any unregistered preparation of tetrahydrobiopterin within the previous 30 days, unless for the purposes of a BH4 responsiveness test.
boolean
C1524063 (UMLS CUI 2011AA)
260676000 (SNOMED CT 2011_0131)
C1995652 (UMLS CUI 2011AA)
434239009 (SNOMED CT 2011_0131)
C1518422 (UMLS CUI 2011AA)
C1514821 (UMLS CUI 2011AA)
C0013227 (UMLS CUI 2011AA)
C0048897 (UMLS CUI 2011AA)
432859002 (SNOMED CT 2011_0131)
MTHU035851 (LOINC Version 232)
C1554961 (UMLS CUI 2011AA)
E (HL7 V3 2006_05)
C0048897 (UMLS CUI 2011AA)
432859002 (SNOMED CT 2011_0131)
MTHU035851 (LOINC Version 232)
C0552602 (UMLS CUI 2011AA)
MTHU002705 (LOINC Version 232)
C0022885 (UMLS CUI 2011AA)
15220000 (SNOMED CT 2011_0131)
10059938 (MedDRA 14.1)
Previous exposure to Kuvan, Biopten, or any unregistered preparation of tetrahydrobiopterin for >30 days.
Item
Previous exposure to Kuvan, Biopten, or any unregistered preparation of tetrahydrobiopterin for >30 days.
boolean
C0205156 (UMLS CUI 2011AA)
9130008 (SNOMED CT 2011_0131)
C0332157 (UMLS CUI 2011AA)
24932003 (SNOMED CT 2011_0131)
C1995652 (UMLS CUI 2011AA)
434239009 (SNOMED CT 2011_0131)
C1518422 (UMLS CUI 2011AA)
C1514821 (UMLS CUI 2011AA)
C0013227 (UMLS CUI 2011AA)
C0048897 (UMLS CUI 2011AA)
432859002 (SNOMED CT 2011_0131)
MTHU035851 (LOINC Version 232)
C0439093 (UMLS CUI 2011AA)
276140008 (SNOMED CT 2011_0131)
GT (HL7 V3 2006_05)
C0205447 (UMLS CUI 2011AA)
38112003 (SNOMED CT 2011_0131)
C0439231 (UMLS CUI 2011AA)
258706009 (SNOMED CT 2011_0131)
MO (HL7 V3 2006_05)
Known hypersensitivity to Kuvan or its excipients.
Item
Known hypersensitivity to Kuvan or its excipients.
boolean
C0020517 (UMLS CUI 2011AA)
418634005 (SNOMED CT 2011_0131)
T78.40 (ICD-10-CM Version 2010)
C1995652 (UMLS CUI 2011AA)
434239009 (SNOMED CT 2011_0131)
C0015237 (UMLS CUI 2011AA)
360215002 (SNOMED CT 2011_0131)
Known hypersensitivity to other approved or non-approved formulations of tetrahydrobiopterin.
Item
Known hypersensitivity to other approved or non-approved formulations of tetrahydrobiopterin.
boolean
C0020517 (UMLS CUI 2011AA)
418634005 (SNOMED CT 2011_0131)
T78.40 (ICD-10-CM Version 2010)
C0205394 (UMLS CUI 2011AA)
74964007 (SNOMED CT 2011_0131)
C1705957 (UMLS CUI 2011AA)
C0048897 (UMLS CUI 2011AA)
432859002 (SNOMED CT 2011_0131)
MTHU035851 (LOINC Version 232)
Previous diagnosis of BH4 deficiency.
Item
Previous diagnosis of BH4 deficiency.
boolean
C0332132 (UMLS CUI 2011AA)
48318009 (SNOMED CT 2011_0131)
C2057687 (UMLS CUI 2011AA)
10069116 (MedDRA 14.1)
Current use of methotrexate, trimethoprim, or other dihydrofolate reductase inhibitors.
Item
Current use of methotrexate, trimethoprim, or other dihydrofolate reductase inhibitors.
boolean
C1524063 (UMLS CUI 2011AA)
260676000 (SNOMED CT 2011_0131)
C0025677 (UMLS CUI 2011AA)
387381009 (SNOMED CT 2011_0131)
MTHU005020 (LOINC Version 232)
C0041041 (UMLS CUI 2011AA)
32792001 (SNOMED CT 2011_0131)
MTHU002305 (LOINC Version 232)
C2911817 (UMLS CUI 2011AA)
Current use of medications that are known to affect nitric oxide synthesis, metabolism or action.
Item
Current use of medications that are known to affect nitric oxide synthesis, metabolism or action.
boolean
C1524063 (UMLS CUI 2011AA)
260676000 (SNOMED CT 2011_0131)
C0013227 (UMLS CUI 2011AA)
C0392760 (UMLS CUI 2011AA)
247591002 (SNOMED CT 2011_0131)
C1157570 (UMLS CUI 2011AA)
C1158468 (UMLS CUI 2011AA)
Current use of levodopa.
Item
Current use of levodopa.
boolean
C1524063 (UMLS CUI 2011AA)
260676000 (SNOMED CT 2011_0131)
C0699991 (UMLS CUI 2011AA)
15383004 (SNOMED CT 2011_0131)
MTHU015998 (LOINC Version 232)
Current use of experimental/other investigational or unregistered drugs that may affect the study outcomes.
Item
Current use of experimental/other investigational or unregistered drugs that may affect the study outcomes.
boolean
C1524063 (UMLS CUI 2011AA)
260676000 (SNOMED CT 2011_0131)
C0013230 (UMLS CUI 2011AA)
CL415205 (UMLS CUI 2011AA)
C0392760 (UMLS CUI 2011AA)
247591002 (SNOMED CT 2011_0131)
C0683954 (UMLS CUI 2011AA)
Inability to comply with study procedures.
Item
Inability to comply with study procedures.
boolean
C1321605 (UMLS CUI 2011AA)
371151008 (SNOMED CT 2011_0131)
Inability to tolerate oral intake.
Item
Inability to tolerate oral intake.
boolean
C1299582 (UMLS CUI 2011AA)
371151008 (SNOMED CT 2011_0131)
C1514756 (UMLS CUI 2011AA)
C1527415 (UMLS CUI 2011AA)
26643006 (SNOMED CT 2011_0131)
_OralRoute (HL7 V3 2006_05)
C1512806 (UMLS CUI 2011AA)
History of organ transplantation.
Item
History of organ transplantation.
boolean
CL421546 (UMLS CUI 2011AA)
392521001 (SNOMED CT 2011_0131)
C0029216 (UMLS CUI 2011AA)
10061890 (MedDRA 14.1)
MTHU000173 (LOINC Version 232)
Concurrent disease or condition that would interfere with study participation or increase the risk for adverse events, including seizure disorders, corticosteroid administration, active malignancy, diabetes mellitus, severe congenital heart disease, renal or hepatic failure.
Item
Concurrent disease or condition that would interfere with study participation or increase the risk for adverse events, including seizure disorders, corticosteroid administration, active malignancy, diabetes mellitus, severe congenital heart disease, renal or hepatic failure.
boolean
C0012634 (UMLS CUI 2011AA)
64572001 (SNOMED CT 2011_0131)
C0348080 (UMLS CUI 2011AA)
260905004 (SNOMED CT 2011_0131)
COND (HL7 V3 2006_05)
C0521102 (UMLS CUI 2011AA)
78235001 (SNOMED CT 2011_0131)
C2348568 (UMLS CUI 2011AA)
C0205217 (UMLS CUI 2011AA)
U (HL7 V3 2006_05)
C0683481 (UMLS CUI 2011AA)
C0014544 (UMLS CUI 2011AA)
84757009, 313307000 (SNOMED CT 2011_0131)
10015037 (MedDRA 14.1)
MTHU020586 (LOINC Version 232)
G40.9 (ICD-10-CM Version 2010)
345.9 (ICD-9-CM Version 2011)
C0001617 (UMLS CUI 2011AA)
79440004 (SNOMED CT 2011_0131)
C1533734 (UMLS CUI 2011AA)
416118004 (SNOMED CT 2011_0131)
C0006826 (UMLS CUI 2011AA)
363346000 (SNOMED CT 2011_0131)
10028997 (MedDRA 14.1)
MTHU010328 (LOINC Version 232)
C00-C96 (ICD-10-CM Version 2010)
C0011849 (UMLS CUI 2011AA)
191044006 (SNOMED CT 2011_0131)
C0152021 (UMLS CUI 2011AA)
13213009 (SNOMED CT 2011_0131)
10019273 (MedDRA 14.1)
Q24.9 (ICD-10-CM Version 2010)
C1565489 (UMLS CUI 2011AA)
10038474 (MedDRA 14.1)
C0085605 (UMLS CUI 2011AA)
59927004 (SNOMED CT 2011_0131)
10019663 (MedDRA 14.1)
K72.9 (ICD-10-CM Version 2010)
E11211 (CTCAE 1105E)
Other significant disease that in the Investigator's opinion would exclude the subject from the trial.
Item
Other significant disease that in the Investigator's opinion would exclude the subject from the trial.
boolean
C0750502 (UMLS CUI 2011AA)
386134007 (SNOMED CT 2011_0131)
C0012634 (UMLS CUI 2011AA)
64572001 (SNOMED CT 2011_0131)
C0332196 (UMLS CUI 2011AA)
77765009 (SNOMED CT 2011_0131)
C0681850 (UMLS CUI 2011AA)
C0008976 (UMLS CUI 2011AA)
110465008 (SNOMED CT 2011_0131)
Any condition that, in the view of the Principal Investigator renders the subject at high risk for failure to comply with treatment or to complete the study.
Item
Any condition that, in the view of the Principal Investigator renders the subject at high risk for failure to comply with treatment or to complete the study.
boolean
C0348080 (UMLS CUI 2011AA)
260905004 (SNOMED CT 2011_0131)
COND (HL7 V3 2006_05)
C1882509 (UMLS CUI 2011AA)
C0681850 (UMLS CUI 2011AA)
C0332167 (UMLS CUI 2011AA)
15508007 (SNOMED CT 2011_0131)
C0457432 (UMLS CUI 2011AA)
258143003 (SNOMED CT 2011_0131)
C0680095 (UMLS CUI 2011AA)
C1554962 (UMLS CUI 2011AA)
C (HL7 V3 2006_05)
C0008976 (UMLS CUI 2011AA)
110465008 (SNOMED CT 2011_0131)