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Replagal Enzyme Replacement Therapy for Children With Fabry Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00084084
Recruitment Status : Completed
First Posted : June 7, 2004
Results First Posted : October 10, 2013
Last Update Posted : April 16, 2014
Sponsor:
Information provided by (Responsible Party):
Shire

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Fabry Disease
Intervention Drug: Agalsidase alfa
Enrollment 17
Recruitment Details  
Pre-assignment Details No patient was enrolled in Cohort 2 (ie, no treatment-naive patients were enrolled).
Arm/Group Title Agalsidase Alfa (Cohort 1)
Hide Arm/Group Description Cohort 1 was composed of patients who had completed TKT023. Patients received 0.2 mg/kg agalsidase alfa, IV, every other week.
Period Title: Phase 1 (Treatment With Replagal RB)
Started 17
Completed 16
Not Completed 1
Reason Not Completed
Lost to Follow-up             1
Period Title: Phase 2 (Transition to Replagal AF)
Started 11 [1]
Completed 10
Not Completed 1
Reason Not Completed
Failure to visit clinic as scheduled             1
[1]
The 11 patients remaining in TKT029 at the time Replagal AF was made available continued to Phase 2.
Arm/Group Title Agalsidase Alfa (Cohort 1)
Hide Arm/Group Description 0.2 mg/kg agalsidase alfa infused by IV over 40 (+/- 10) minutes every other week
Overall Number of Baseline Participants 17
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 17 participants
11.99  (3.2464)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants
Female
1
   5.9%
Male
16
  94.1%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 17 participants
White 15
Hispanic 2
Baseline Heart Rate Variability (SDNN)  
Mean (Standard Deviation)
Unit of measure:  Msec
Number Analyzed 17 participants
98.947  (32.324)
1.Primary Outcome
Title Patients Who Experienced At Least One Adverse Event (AE)
Hide Description [Not Specified]
Time Frame 362 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: Patients in Cohort 1 who received at least one dose of Replagal RB in Phase 1.
Arm/Group Title Agalsidase Alfa (Cohort 1)
Hide Arm/Group Description:
Cohort 1 was composed of patients who had completed TKT023. Patients received 0.2 mg/kg agalsidase alfa, IV, every other week.
Overall Number of Participants Analyzed 17
Measure Type: Number
Unit of Measure: participants
17
2.Secondary Outcome
Title Pharmacokinetics - Area Under the Serum Concentration-Time Curve (AUC0-∞)
Hide Description AUC0-∞ is a measure of the total exposure to a drug.
Time Frame 341 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
PK Population: All patients who received at least 1 dose of Replagal (RB or AF) and had at least 1 PK sample drawn.
Arm/Group Title Agalsidase Alfa (Cohort 1)
Hide Arm/Group Description:
Cohort 1 was composed of patients who had completed TKT023. Patients received 0.2 mg/kg agalsidase alfa, IV, every other week.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: min·U/mL
Week 81 245282  (159071)
Week 133 295779  (161058)
Week 159 218078  (73560)
Week 315/341 213193  (119581)
3.Secondary Outcome
Title Pharmacokinetics - Maximum Observed Serum Concentration (Cmax)
Hide Description Cmax is the peak plasma concentration of a drug after administration.
Time Frame 341 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Agalsidase Alfa (Cohort 1)
Hide Arm/Group Description:
Cohort 1 was composed of patients who had completed TKT023. Patients received 0.2 mg/kg agalsidase alfa, IV, every other week.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: U/mL
Week 81 3173  (969)
Week 133 3842  (1235)
Week 159 3462  (1357)
Week315/341 3568  (1492)
4.Other Pre-specified Outcome
Title Heart Rate Variability - Change From Baseline at Week 185 in SDNN
Hide Description Heart rate variability was assessed by 2-hour Holter monitoring. Standard deviation of all filtered RR intervals over the length of the analysis (SDNN) was measured.
Time Frame Week 185
Hide Outcome Measure Data
Hide Analysis Population Description
Number of participants present at Visit Week 185 included for analysis.
Arm/Group Title Agalsidase Alfa (Cohort 1)
Hide Arm/Group Description:
Cohort 1 was composed of patients who had completed TKT023. Patients received 0.2 mg/kg agalsidase alfa, IV, every other week.
Overall Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: msec
20.256  (29.060)
Time Frame 362 weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Safety Population RB Transition Safety Population
Hide Arm/Group Description Patients in Cohort 1 who received at least 1 dose of Replagal RB in Phase 1 - no data from Phase 2 (Replagal AF) included. A subset of patients from the Safety Population RB who additionally received at least 1 dose of Replagal AF in Phase 2.
All-Cause Mortality
Safety Population RB Transition Safety Population
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Safety Population RB Transition Safety Population
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/17 (11.76%)      2/11 (18.18%)    
Congenital, familial and genetic disorders     
Pectus Excavatum  1  0/17 (0.00%)  0 1/11 (9.09%)  1
Ear and labyrinth disorders     
Vertigo Positional  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Injury, poisoning and procedural complications     
Facial Bones Fracture  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Renal Injury  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Road Traffic Accident  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Traumatic Liver Injury  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Metabolism and nutrition disorders     
Dehydration  1  1/17 (5.88%)  1 0/11 (0.00%)  0
Nervous system disorders     
Neuralgia  1  1/17 (5.88%)  1 0/11 (0.00%)  0
Cerebrovascular Accident  1  1/17 (5.88%)  2 1/11 (9.09%)  2
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (12.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Safety Population RB Transition Safety Population
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   17/17 (100.00%)      11/11 (100.00%)    
Blood and lymphatic system disorders     
Anaemia  1  2/17 (11.76%)  2 1/11 (9.09%)  1
Lymphadenopathy  1  2/17 (11.76%)  2 2/11 (18.18%)  2
Cardiac disorders     
Arrhythmia Supraventricular  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Atrioventricular Block First Degree  1  1/17 (5.88%)  1 0/11 (0.00%)  0
Palpitations  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Sinus Tachycardia  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Tachycardia  1  1/17 (5.88%)  1 2/11 (18.18%)  3
Pericardial Effusion  1  0/17 (0.00%)  0 1/11 (9.09%)  1
Congenital, familial and genetic disorders     
Pectus Excavatum  1  0/17 (0.00%)  0 1/11 (9.09%)  2
Ear and labyrinth disorders     
Ear Pain  1  4/17 (23.53%)  6 1/11 (9.09%)  1
Cerumen Impaction  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Deafness Unilateral  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Middle Ear Effusion  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Otorrhoea  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Tinnitus  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Tympanic Membrane Perforation  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Vertigo Positional  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Eye disorders     
Diplopia  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Eye Pain  1  1/17 (5.88%)  2 1/11 (9.09%)  2
Eye Swelling  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Gaze Palsy  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Myopia  1  1/17 (5.88%)  2 1/11 (9.09%)  2
Strabismus  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Refraction Disorder  1  0/17 (0.00%)  0 1/11 (9.09%)  1
Visual Acuity Reduced  1  0/17 (0.00%)  0 1/11 (9.09%)  1
Gastrointestinal disorders     
Abdominal Pain  1  8/17 (47.06%)  13 8/11 (72.73%)  14
Nausea  1  7/17 (41.18%)  11 5/11 (45.45%)  15
Abdominal Pain Upper  1  6/17 (35.29%)  10 6/11 (54.55%)  12
Vomiting  1  6/17 (35.29%)  13 6/11 (54.55%)  12
Diarrhoea  1  4/17 (23.53%)  11 6/11 (54.55%)  17
Abdominal Discomfort  1  2/17 (11.76%)  2 3/11 (27.27%)  3
Toothache  1  2/17 (11.76%)  2 2/11 (18.18%)  2
Aphthous Stomatitis  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Dental Caries  1  1/17 (5.88%)  1 0/11 (0.00%)  0
Faecal Incontinence  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Frequent Bowel Movements  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Gastritis  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Lip Swelling  1  1/17 (5.88%)  2 1/11 (9.09%)  3
Retching  1  1/17 (5.88%)  1 0/11 (0.00%)  0
Stomatitis  1  1/17 (5.88%)  1 2/11 (18.18%)  2
General disorders     
Pyrexia  1  12/17 (70.59%)  27 9/11 (81.82%)  19
Chest Pain  1  6/17 (35.29%)  9 7/11 (63.64%)  13
Fatigue  1  4/17 (23.53%)  5 4/11 (36.36%)  6
Pain  1  3/17 (17.65%)  5 4/11 (36.36%)  8
Gait Disturbance  1  2/17 (11.76%)  3 2/11 (18.18%)  4
Non-Cardiac Chest Pain  1  2/17 (11.76%)  2 5/11 (45.45%)  7
Oedema Peripheral  1  2/17 (11.76%)  2 2/11 (18.18%)  3
Asthenia  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Chest Discomfort  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Chills  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Infusion Site Pain  1  1/17 (5.88%)  1 0/11 (0.00%)  0
Irritability  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Temperature Intolerance  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Influenza Like Illness  1  0/17 (0.00%)  0 2/11 (18.18%)  2
Immune system disorders     
Drug Hypersensitivity  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Infections and infestations     
Gastroenteritis Viral  1  8/17 (47.06%)  12 5/11 (45.45%)  6
Nasopharyngitis  1  7/17 (41.18%)  12 6/11 (54.55%)  12
Upper Respiratory Tract Infection  1  4/17 (23.53%)  11 5/11 (45.45%)  16
Influenza  1  3/17 (17.65%)  3 3/11 (27.27%)  3
Bronchitis  1  2/17 (11.76%)  2 0/11 (0.00%)  0
Ear Infection  1  2/17 (11.76%)  3 2/11 (18.18%)  3
Sinusitis  1  2/17 (11.76%)  5 4/11 (36.36%)  8
Acarodermatitis  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Cellulitis  1  1/17 (5.88%)  2 1/11 (9.09%)  2
Hordeolum  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Impetigo  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Infected Bites  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Infectious Mononucleosis  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Lung Infection  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Otitis Media  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Pharyngitis Streptococcal  1  1/17 (5.88%)  2 1/11 (9.09%)  2
Pneumonia  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Rocky Mountain Spotted Fever  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Tinea Pedis  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Viral Infection  1  1/17 (5.88%)  1 3/11 (27.27%)  3
Gastroenteritis  1  0/17 (0.00%)  0 2/11 (18.18%)  2
Abscess  1  0/17 (0.00%)  0 1/11 (9.09%)  1
Blister Infected  1  0/17 (0.00%)  0 1/11 (9.09%)  1
Body Tinea  1  0/17 (0.00%)  0 1/11 (9.09%)  1
Localised Infection  1  0/17 (0.00%)  0 1/11 (9.09%)  1
Molluscum Contagiosum  1  0/17 (0.00%)  0 1/11 (9.09%)  1
Rhinitis  1  0/17 (0.00%)  0 1/11 (9.09%)  1
Tonsillitis  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Injury, poisoning and procedural complications     
Contusion  1  5/17 (29.41%)  15 5/11 (45.45%)  16
Excoriation  1  5/17 (29.41%)  16 5/11 (45.45%)  16
Post-Traumatic Pain  1  4/17 (23.53%)  5 4/11 (36.36%)  5
Fall  1  3/17 (17.65%)  8 3/11 (27.27%)  8
Skin Laceration  1  3/17 (17.65%)  9 3/11 (27.27%)  10
Joint Injury  1  2/17 (11.76%)  3 2/11 (18.18%)  3
Joint Sprain  1  2/17 (11.76%)  2 1/11 (9.09%)  1
Limb Injury  1  2/17 (11.76%)  4 2/11 (18.18%)  4
Procedural Pain  1  2/17 (11.76%)  2 4/11 (36.36%)  4
Arthropod Bite  1  1/17 (5.88%)  1 1/11 (9.09%)  3
Facial Bones Fracture  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Head Injury  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Heat Exhaustion  1  1/17 (5.88%)  1 0/11 (0.00%)  0
Laceration  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Lumbar Vertebral Fracture  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Muscle Strain  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Renal Injury  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Road Traffic Accident  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Scratch  1  1/17 (5.88%)  1 2/11 (18.18%)  2
Thermal Burn  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Traumatic Haematoma  1  1/17 (5.88%)  2 1/11 (9.09%)  2
Traumatic Liver Injury  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Lower Limb Fracture  1  0/17 (0.00%)  0 1/11 (9.09%)  1
Neck Injury  1  0/17 (0.00%)  0 1/11 (9.09%)  1
Investigations     
Albumin Urine Present  1  1/17 (5.88%)  1 0/11 (0.00%)  0
Blood Albumin Increased  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Blood Pressure Increased  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Blood Triglycerides Increased  1  1/17 (5.88%)  1 1/11 (9.09%)  1
C-Reactive Protein Increased  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Nuclear Magnetic Resonance Imaging Brain Abnormal  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Pulmonary Function Test Decreased  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Sputum Abnormal  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Weight Decreased  1  1/17 (5.88%)  2 1/11 (9.09%)  2
Metabolism and nutrition disorders     
Decreased Appetite  1  3/17 (17.65%)  3 3/11 (27.27%)  3
Anorexia  1  1/17 (5.88%)  1 2/11 (18.18%)  2
Dehydration  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Obesity  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Musculoskeletal and connective tissue disorders     
Arthralgia  1  8/17 (47.06%)  9 5/11 (45.45%)  9
Pain in Extremity  1  8/17 (47.06%)  59 8/11 (72.73%)  67
Back Pain  1  2/17 (11.76%)  6 2/11 (18.18%)  9
Groin Pain  1  2/17 (11.76%)  2 2/11 (18.18%)  2
Muscular Weakness  1  2/17 (11.76%)  2 2/11 (18.18%)  2
Musculoskeletal Chest Pain  1  2/17 (11.76%)  3 3/11 (27.27%)  4
Myalgia  1  2/17 (11.76%)  2 2/11 (18.18%)  2
Neck Pain  1  2/17 (11.76%)  3 2/11 (18.18%)  4
Foot Deformity  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Limb Discomfort  1  1/17 (5.88%)  2 1/11 (9.09%)  2
Musculoskeletal Pain  1  1/17 (5.88%)  1 2/11 (18.18%)  2
Posture Abnormal  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Muscle Spasms  1  0/17 (0.00%)  0 1/11 (9.09%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Melanocytic Naevus  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Skin Papilloma  1  0/17 (0.00%)  0 1/11 (9.09%)  1
Nervous system disorders     
Headache  1  9/17 (52.94%)  33 7/11 (63.64%)  46
Neuralgia  1  9/17 (52.94%)  35 7/11 (63.64%)  32
Burning Sensation  1  6/17 (35.29%)  16 5/11 (45.45%)  15
Paraesthesia  1  3/17 (17.65%)  7 3/11 (27.27%)  7
Balance Disorder  1  2/17 (11.76%)  2 2/11 (18.18%)  3
Dizziness  1  1/17 (5.88%)  1 2/11 (18.18%)  2
Amnesia  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Cerebral Infarction  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Cerebrovascular Accident  1  1/17 (5.88%)  5 1/11 (9.09%)  5
Coordination Abnormal  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Cranial Nerve Disorder  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Dysarthria  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Facial Paresis  1  1/17 (5.88%)  3 1/11 (9.09%)  3
Hypersomnia  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Migraine  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Poor Quality Sleep  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Presyncope  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Reflexes Abnormal  1  1/17 (5.88%)  3 1/11 (9.09%)  3
Sinus Headache  1  2/17 (11.76%)  2 1/11 (9.09%)  1
Thalamic Infarction  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Tremor  1  0/17 (0.00%)  0 1/11 (9.09%)  1
Disturbance in Attention  1  1/17 (5.88%)  1 0/11 (0.00%)  0
Psychiatric disorders     
Attention Deficit / Hyperactivity Disorder  1  3/17 (17.65%)  4 2/11 (18.18%)  3
Anxiety  1  1/17 (5.88%)  2 1/11 (9.09%)  2
Decreased Activity  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Depression  1  1/17 (5.88%)  1 1/11 (9.09%)  3
Panic Disorder  1  1/17 (5.88%)  2 1/11 (9.09%)  2
Agitation  1  0/17 (0.00%)  0 1/11 (9.09%)  1
Renal and urinary disorders     
Dysuria  1  2/17 (11.76%)  2 1/11 (9.09%)  1
Urethral Meatus Stenosis  1  2/17 (11.76%)  2 2/11 (18.18%)  2
Haematuria  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Nephrolithiasis  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Proteinuria  1  0/17 (0.00%)  0 1/11 (9.09%)  1
Reproductive system and breast disorders     
Testicular Pain  1  2/17 (11.76%)  2 2/11 (18.18%)  2
Balanitis  1  1/17 (5.88%)  2 1/11 (9.09%)  2
Scrotal Pain  1  1/17 (5.88%)  2 1/11 (9.09%)  2
Testicular Oedema  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Dysmenorrhoea  1  0/17 (0.00%)  0 1/11 (9.09%)  1
Respiratory, thoracic and mediastinal disorders     
Cough  1  13/17 (76.47%)  27 10/11 (90.91%)  23
Nasal Congestion  1  10/17 (58.82%)  16 6/11 (54.55%)  14
Oropharyngeal Pain  1  5/17 (29.41%)  8 5/11 (45.45%)  14
Rhinitis Allergic  1  4/17 (23.53%)  5 4/11 (36.36%)  5
Dyspnoea  1  3/17 (17.65%)  4 6/11 (54.55%)  10
Upper Respiratory Tract Congestion  1  3/17 (17.65%)  4 3/11 (27.27%)  4
Asthma  1  2/17 (11.76%)  2 2/11 (18.18%)  2
Sinus Congestion  1  2/17 (11.76%)  2 2/11 (18.18%)  2
Productive Cough  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Respiratory Tract Congestion  1  1/17 (5.88%)  1 1/11 (9.09%)  4
Rhinorrhoea  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Pleural Effusion  1  0/17 (0.00%)  0 1/11 (9.09%)  1
Wheezing  1  0/17 (0.00%)  0 1/11 (9.09%)  1
Skin and subcutaneous tissue disorders     
Dermatitis Contact  1  3/17 (17.65%)  5 3/11 (27.27%)  6
Erythema  1  3/17 (17.65%)  5 3/11 (27.27%)  5
Acne  1  1/17 (5.88%)  1 2/11 (18.18%)  2
Angiokeratoma  1  1/17 (5.88%)  2 3/11 (27.27%)  4
Ecchymosis  1  1/17 (5.88%)  3 1/11 (9.09%)  3
Eczema  1  1/17 (5.88%)  2 2/11 (18.18%)  3
Hyperhidrosis  1  1/17 (5.88%)  1 0/11 (0.00%)  0
Petechiae  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Pruritus  1  1/17 (5.88%)  2 1/11 (9.09%)  2
Pruritus Generalised  1  1/17 (5.88%)  2 1/11 (9.09%)  2
Rash  1  1/17 (5.88%)  3 3/11 (27.27%)  5
Rash Generalised  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Rash Papular  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Rash Pruritic  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Skin Lesion  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Urticaria  1  1/17 (5.88%)  5 1/11 (9.09%)  6
Vasculitic Rash  1  1/17 (5.88%)  3 0/11 (0.00%)  0
Angioedema  1  0/17 (0.00%)  0 1/11 (9.09%)  1
Vascular disorders     
Flushing  1  2/17 (11.76%)  2 2/11 (18.18%)  6
Haematoma  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Haemorrhage  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Hypertension  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Pallor  1  1/17 (5.88%)  1 1/11 (9.09%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (12.0)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Shire's agreements with investigators vary. All agreements provide Shire the right to embargo communications regarding trial results prior to public release for a period ≤ 180 days from the time submitted to Shire for review. Shire does not prohibit publication but can require the removal of confidential information (excluding trial results) and can request postponement of a single-center publication until after disclosure of the trial's multi-center publication.
Results Point of Contact
Name/Title: Anna Wijatyk, MD, Medical Director
Organization: Shire Human Genetic Therapies
Phone: 781-482-9622
Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT00084084     History of Changes
Other Study ID Numbers: TKT029
First Submitted: June 5, 2004
First Posted: June 7, 2004
Results First Submitted: August 1, 2013
Results First Posted: October 10, 2013
Last Update Posted: April 16, 2014