Información:
Error:
ID
18599
Descripción
A Randomized, Open-Label Study to Compare the Efficacy and Safety of AT1001 and Enzyme Replacement Therapy (ERT) in Patients With Fabry Disease and AT1001-Responsive GLA Mutations, Who Were Previously Treated With ERT Visit 1
Palabras clave
Versiones (2)
- 2/11/16 2/11/16 -
- 11/11/16 11/11/16 -
Subido en
11 de noviembre de 2016
DOI
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Licencia
Creative Commons BY-NC 3.0
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Visit 1 CRFs Galafold (migalastat) AT1001-012 NCT01218659
Visit 1 CRFs Galafold (migalastat) AT1001-012 NCT01218659
- StudyEvent: ODM
Similar models
Visit 1 CRFs Galafold (migalastat) AT1001-012 NCT01218659
- StudyEvent: ODM
Name
Tipo
Description | Question | Decode (Coded Value)
Tipo de datos
Alias
Date of visit
Item
Date of visit
text
C1320303 (UMLS CUI [1])
Informed Consent
Item
Date approved Informed Consent signed
date
C0021430 (UMLS CUI [1])
C0011008 (UMLS CUI [2])
C0011008 (UMLS CUI [2])
Subject number
Item
Subject number
integer
C2348585 (UMLS CUI [1])
Full subject number
Item
Full subject number
integer
C2348585 (UMLS CUI [1])
Subject Initials
Item
Subject Initials
text
C2986440 (UMLS CUI [1])
Date of birth
Item
Date of birth
date
C0421451 (UMLS CUI [1])
CL Item
Male (1)
CL Item
Female (2)
CL Item
Hispanic or Latino (1)
CL Item
Not Hispanic or Latino (2)
CL Item
Japanese (3)
Race
Item
Race
text
C0034510 (UMLS CUI [1])
Date of Diagnosis
Item
Date of Diagnosis of Fabry Disease
date
C2316983 (UMLS CUI [1,1])
C0002986 (UMLS CUI [1,2])
C0002986 (UMLS CUI [1,2])
Date of GLA Genotyping obtained
Item
Date of GLA Genotyping obtained
date
C1285573 (UMLS CUI [1,1])
C1439334 (UMLS CUI [1,2])
C0011008 (UMLS CUI [1,3])
C1439334 (UMLS CUI [1,2])
C0011008 (UMLS CUI [1,3])
Item Group
Inclusion and Exclusion Criteria
C1512693 (UMLS CUI-1)
C0680251 (UMLS CUI-2)
C0680251 (UMLS CUI-2)
CL Item
Yes (1)
CL Item
No (2)
Item
Please specify which of the following inclusion criteria were not met
integer
C1512693 (UMLS CUI [1])
CL Item
Male or female, diagnosed with Fabry disease, between 16 to 74 years of age, inclusive V1. Subjects under 18 years of age will be enrolled only at sites with all required regulatory and ethis approvals to do so (1)
CL Item
Confirmed GLA Mutation shown to be responsive to AT1001 in the in vitro HEK-293 cell-based assay, r a confirmed GLA mutation that is non-testable in this assay, but has evidence to suggest the potential response to AT1001 (2)
CL Item
Initiated Treatment with ERT at least 12 months before visit 2 (3)
CL Item
Dose level and regimen of ERT have been stable for the 3 months before visit 2 and is at least 80% of the currently labeled dose and regimen for this time period (4)
CL Item
GFR > 30mL/min/173m2 (5)
CL Item
If taking angiotensin converting enzyme (ACE) Inhibitors or angiotensin receptor blockers (ARBs) has been on stable dose at least the 4 weeks before visit 1 (6)
CL Item
If of reproductive potential, agree to use medically accepted methods of contraception throughout the duration of the study and up to 30 days after last dose of protocol-defined study medication (7)
CL Item
Able to provide written informed consent, and assent if applicable. Subjects under 18 years of age will provide written informed assent, and written informed consent by their parent or legal guardian (8)
Item
Please specify which of the following exclusion criteria were met
integer
C0680251 (UMLS CUI [1])
CL Item
Undergone, or is scheduled to undergo, kidney transplantation or other solid organ transplantation (1)
CL Item
On regular dialysis that is specifically for the treatment of chornic kidney disease (2)
CL Item
Had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 3 months before visit 1 (3)
CL Item
Has clinically significant unstable cardiac diseases in the opinion of the investigator (4)
CL Item
Is pregnant or breastfeeding (5)
CL Item
Has a history of allergy to AT1001 (including excipients) or other iminosugars (e.g., miglustat, miglitol) (6)
CL Item
Has absolute contraindication to iohexol and/or inability to undergo iohexol GFR testing (7)
CL Item
Requires treatment with Glyset (miglitol), or Zavesca (miglustat) (8)
CL Item
Received any investigational/experimental drug, biologic or device within 30 days of Visit 1 (9)
CL Item
Has any intercurrent illness or condition that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study (10)
CL Item
Is otherwise unsuitable for the study, in the opinion of the investigator (11)
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
Item
Immunological/Allergies
integer
C0021053 (UMLS CUI [1])
C0020517 (UMLS CUI [2])
C0020517 (UMLS CUI [2])
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
Item
Endocrine/Metabolic
integer
C0014130 (UMLS CUI [1])
C0025517 (UMLS CUI [2])
C0025517 (UMLS CUI [2])
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
Date of Assessment
Item
Date of Assessment
date
C2985720 (UMLS CUI [1])
Weight
Item
Body weight
float
C0005910 (UMLS CUI [1])
Height
Item
Body height
float
C0005890 (UMLS CUI [1])
Blood pressure
Item
Blood pressure measurement systolic
integer
C0005823 (UMLS CUI [1])
Blood pressure
Item
Blood pressure measurement diastolic
integer
C0005823 (UMLS CUI [1])
Heart rate
Item
Heart rate
integer
C0018810 (UMLS CUI [1])
Respiration Rate
Item
Respiration rate, Breathing rate (BR)
integer
C0231832 (UMLS CUI [1])
CL Item
Yes (1)
CL Item
No (2)
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
Item
Lymphatics/Lymph Nodes
integer
C0024235 (UMLS CUI [1])
C0024204 (UMLS CUI [2])
C0024204 (UMLS CUI [2])
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not Done (3)
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not done (3)
CL Item
Normal (1)
CL Item
Abnormal (2)
CL Item
Not Done (3)
ECG Date
Item
Date ECG was performed
date
C1623258 (UMLS CUI [1,1])
C0011008 (UMLS CUI [1,2])
C0011008 (UMLS CUI [1,2])
CL Item
normal (1)
CL Item
abnormal, not clinically significant (2)
CL Item
abnormal, clinically significant (3)
PR interval
Item
PR interval
float
C0429087 (UMLS CUI [1])
Hematology Sample Collection Date
Item
Hematology Sample Collection Date
date
C0474523 (UMLS CUI [1,1])
C0011008 (UMLS CUI [1,2])
C0011008 (UMLS CUI [1,2])
Item
Are there any laboratory abnormalities, deemed clinically significant by the PI, noted for Hematology?
integer
C0474523 (UMLS CUI [1,1])
C0587081 (UMLS CUI [1,2])
C0587081 (UMLS CUI [1,2])
Code List
Are there any laboratory abnormalities, deemed clinically significant by the PI, noted for Hematology?
CL Item
Yes (1)
CL Item
No (2)
Item Group
Hematology: clinically significant abnormal laboratory values, please sepcify
C0474523 (UMLS CUI-1)
C1853129 (UMLS CUI-2)
C2826293 (UMLS CUI-3)
C1853129 (UMLS CUI-2)
C2826293 (UMLS CUI-3)
Item
Hematology: clinically significant abnormal laboratory values, please sepcify
integer
C0474523 (UMLS CUI [1,1])
C1853129 (UMLS CUI [1,2])
C2826293 (UMLS CUI [1,3])
C1853129 (UMLS CUI [1,2])
C2826293 (UMLS CUI [1,3])
Code List
Hematology: clinically significant abnormal laboratory values, please sepcify
CL Item
Total Hemoglobin (1)
CL Item
Hematocrit (2)
CL Item
Erythrocyte (3)
CL Item
Platelet Count (4)
CL Item
Leukocyte (5)
CL Item
MCV (6)
CL Item
MCH (7)
CL Item
MCHC (8)
CL Item
RDW (9)
CL Item
Neutrophils (10)
CL Item
Lymphocytes (11)
CL Item
Monocytes (12)
CL Item
Eosinophils (13)
CL Item
Basophils (14)
CL Item
Neutrophils, ABS (15)
CL Item
Lymphocytes, ABS (16)
CL Item
Monocytes, ABS (17)
CL Item
Eosinophils, ABS (18)
CL Item
Basophils, ABS (19)
CL Item
Morphology (20)
CL Item
Co-Ag, INR (Screening only) (21)
CL Item
Co-Ag, aPTT (Screening only) (22)
Item Group
Chemistry, Laboratory findings
C0007996 (UMLS CUI-1)
C0022885 (UMLS CUI-2)
C0022885 (UMLS CUI-2)
Chemistry Sample Collection Date
Item
Chemistry Sample Collection Date
date
C0007996 (UMLS CUI [1,1])
C0022877 (UMLS CUI [1,2])
C0011008 (UMLS CUI [1,3])
C0022877 (UMLS CUI [1,2])
C0011008 (UMLS CUI [1,3])
Item
Are there any laboratory abnormalities, deemed clinically significant by the PI, noted for Chemistry?
integer
C0007996 (UMLS CUI [1,1])
C0587081 (UMLS CUI [1,2])
C0587081 (UMLS CUI [1,2])
Code List
Are there any laboratory abnormalities, deemed clinically significant by the PI, noted for Chemistry?
CL Item
Yes (1)
CL Item
No (2)
Item Group
Chemistry: clinically significant abnormal laboratory values, please specify
C0005774 (UMLS CUI-1)
C2826293 (UMLS CUI-2)
C2826293 (UMLS CUI-2)
Item
Chemistry: clinically significant abnormal laboratory values, please specify
integer
C0005774 (UMLS CUI [1,1])
C2826293 (UMLS CUI [1,2])
C2826293 (UMLS CUI [1,2])
Code List
Chemistry: clinically significant abnormal laboratory values, please specify
CL Item
AST (1)
CL Item
ALT (2)
CL Item
Alkaline Phosphate (3)
CL Item
Total Bilirubin (4)
CL Item
Creatinine (5)
CL Item
Urea (6)
CL Item
Non-Fasting Glucose (7)
CL Item
Calcium (8)
CL Item
Calcium (Albumin Corrected) (9)
CL Item
Sodium (10)
CL Item
Potassium (11)
CL Item
Magnesium (12)
CL Item
Total Protein (13)
CL Item
Albumin (14)
CL Item
Bicarbonate (15)
CL Item
LDH (16)
CL Item
Blood Urea Nitrogen (BUN) (17)
CL Item
Chloride (18)
CL Item
Phosphorus (19)
CL Item
eGFR MDRD, ID MS Traceable (20)
CL Item
Cholesterol (21)
CL Item
Creatinine Phosphokinase (CPK) (22)
CL Item
Glutamyltransferase (23)
CL Item
Triglyceride (24)
CL Item
Uric Acid (25)
Urinalysis Sample Collection Date
Item
Urinalysis Sample Collection Date
date
C0042014 (UMLS CUI [1,1])
C0022877 (UMLS CUI [1,2])
C0011008 (UMLS CUI [1,3])
C0022877 (UMLS CUI [1,2])
C0011008 (UMLS CUI [1,3])
Item
Are there any laboratory abnormalities, deemed clinically significant by the PI, noted for Urinalysis?
integer
C0042014 (UMLS CUI [1,1])
C0587081 (UMLS CUI [1,2])
C0587081 (UMLS CUI [1,2])
Code List
Are there any laboratory abnormalities, deemed clinically significant by the PI, noted for Urinalysis?
CL Item
Yes (1)
CL Item
No (2)
Item Group
Urinalysis: clinically significant abnormal laboratory values, please specify
C0042014 (UMLS CUI-1)
C1853129 (UMLS CUI-2)
C2826293 (UMLS CUI-3)
C1853129 (UMLS CUI-2)
C2826293 (UMLS CUI-3)
Item
Urinalysis: clinically significant abnormal laboratory values, please specify
integer
C0042014 (UMLS CUI [1,1])
C1853129 (UMLS CUI [1,2])
C2826293 (UMLS CUI [1,3])
C1853129 (UMLS CUI [1,2])
C2826293 (UMLS CUI [1,3])
Code List
Urinalysis: clinically significant abnormal laboratory values, please specify
CL Item
Specific Gravity (1)
CL Item
pH (2)
CL Item
Protein (3)
CL Item
Glucose (4)
CL Item
Ketones (5)
CL Item
Blood (6)
CL Item
Leukocyte Esterase (7)
CL Item
Nitrite (8)
CL Item
Bilirubin (9)
CL Item
Urobilinogen (10)
CL Item
WBC (11)
CL Item
RBC (12)
CL Item
Color (13)
CL Item
Apperance (14)
CL Item
Microalbumin, 24-HR Urine (mg/day) (15)
CL Item
Protein, 24-HR Urine (mg/day) (16)
CL Item
Microscopic (17)
CL Item
WBC Casts (18)
CL Item
Epithelial Cells (19)
CL Item
Renal Tubular Cells (20)
CL Item
Transitional Epithelial Cells (21)
CL Item
Bacterial Casts (22)
CL Item
Epithelial Casts (23)
CL Item
Fatty Casts (24)
CL Item
Granular Casts (25)
CL Item
Hyaline Casts (26)
CL Item
Mixed Casts (27)
CL Item
RBC Casts (28)
CL Item
Waxy Casts (29)
CL Item
Amorphous Urate Crystals (30)
CL Item
Acid Urate Crystals (31)
CL Item
Ammonium Biurate Crystals (32)
CL Item
Amorphous Crystals (33)
CL Item
Ammonium Oxalate Crystals (34)
CL Item
Ammonium Phosphate Crystals (35)
CL Item
Bilirubin Crystals (36)
CL Item
Calcium Oxalate Crystals (37)
CL Item
Calcium Phospate Crystals (38)
CL Item
Calcium Carbonate Crystals (39)
CL Item
Cystine Crystals (40)
CL Item
Cholesterol Crystals (41)
CL Item
Hippuric Acid Crystals (42)
CL Item
Leucine Crystals (43)
CL Item
Monosodium Urate Crystals (44)
CL Item
Sulfa Crystals (45)
CL Item
Triple Phosphate Crystals (46)
CL Item
Tyrosine Crystals (47)
CL Item
Uric Acid Crystals (48)
CL Item
Bacteria (49)
CL Item
Fat Bodies (50)
CL Item
Starch Granules (51)
CL Item
Trichomonas (52)
CL Item
Yeast (53)
CL Item
Microalbumin, Urine Conc (54)
CL Item
Creatinine, Urine 24-Hr (55)
CL Item
Creatinine, Urine Conc (56)
CL Item
Protein, Urine 24-Hr (57)
CL Item
Urine total protein, random (58)
24-Hr Urine Collection Start Date
Item
24-Hr Urine Collection Start Date
date
C0455060 (UMLS CUI [1,1])
C0808070 (UMLS CUI [1,2])
C0808070 (UMLS CUI [1,2])
24-Hr Urine Collection Start Time
Item
24-Hr Urine Collection Start Time
time
C0455060 (UMLS CUI [1,1])
C1301880 (UMLS CUI [1,2])
C1301880 (UMLS CUI [1,2])
24-Hr Urine Collection Stop Date
Item
24-Hr Urine Collection Stop Date
date
C0455060 (UMLS CUI [1,1])
C0806020 (UMLS CUI [1,2])
C0806020 (UMLS CUI [1,2])
24-Hr Urine Collection Stop Time
Item
24-Hr Urine Collection Stop Time
time
C0455060 (UMLS CUI [1,1])
C1522314 (UMLS CUI [1,2])
C1522314 (UMLS CUI [1,2])
24-Hr Urine Collection Total Volume
Item
24-Hr Urine Collection Total Volume
float
C0455060 (UMLS CUI [1,1])
C2700258 (UMLS CUI [1,2])
C2700258 (UMLS CUI [1,2])
Date sent to Laboratory
Item
24-Hr Urine Collection Date Sent to Laboratory
date
C0455060 (UMLS CUI [1,1])
C1515023 (UMLS CUI [1,2])
C0011008 (UMLS CUI [1,3])
C1515023 (UMLS CUI [1,2])
C0011008 (UMLS CUI [1,3])
reference date
Item
Reference date
date
C1719096 (UMLS CUI [1])
Visit
Item
Visit Type
text
C0545082 (UMLS CUI [1])
eCRF
Item
electronic case report form
text
C1516308 (UMLS CUI [1])
Comment
Item
Comment
text
C0947611 (UMLS CUI [1])