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An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165 (Prism301)

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ClinicalTrials.gov Identifier: NCT01819727
Recruitment Status : Completed
First Posted : March 28, 2013
Results First Posted : September 10, 2018
Last Update Posted : September 10, 2018
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Phenylketonuria
Intervention Drug: BMN 165
Enrollment 261

Recruitment Details  
Pre-assignment Details  
Arm/Group Title BMN 165, 20mg/Day BMN 165, 40mg/Day
Hide Arm/Group Description Subjects who meet the eligibility criteria will be randomized 1:1 to titrate to one of two dose regimens: 20 or 40 mg/day. Randomization will be stratified by the last available blood Phe concentration prior to Day 1 (600-900 μmol/L and > 900 μmol/L). All subjects will initiate IP at a fixed-dose of 2.5 mg/week for 4 weeks (Induction). After the IP, subjects will enter the Titration Period (Weeks 5 up to 34) where they will increase their weekly dose to a daily dose regimen of 20 or 40 mg/day. The Titration Period will be individualized to each subject based on a minimum of 6 weeks (the amount of time it takes to reach a dose regimen of 20 mg/day with no dose interruptions) and up to 30 weeks (accounts for dose reduction or interruption due to AEs). Subjects will stop titration once they have achieved either the 20 or 40 mg/day dose regimen. The majority of subjects will maintain the 20 or 40 mg/day dose regimen for at least an additional 2 weeks until 26 weeks-36 weeks in the study. Subjects who meet the eligibility criteria will be randomized 1:1 to titrate to one of two dose regimens: 20 or 40 mg/day. Randomization will be stratified by the last available blood Phe concentration prior to Day 1 (600-900 μmol/L and > 900 μmol/L). All subjects will initiate IP at a fixed-dose of 2.5 mg/week for 4 weeks (Induction). After the IP, subjects will enter the Titration Period (Weeks 5 up to 34) where they will increase their weekly dose to a daily dose regimen of 20 or 40 mg/day. The Titration Period will be individualized to each subject based on a minimum of 6 weeks (the amount of time it takes to reach a dose regimen of 20 mg/day with no dose interruptions) and up to 30 weeks (accounts for dose reduction or interruption due to AEs). Subjects will stop titration once they have achieved either the 20 or 40 mg/day dose regimen. The majority of subjects will maintain the 20 or 40 mg/day dose regimen for at least an additional 2 weeks until 26 weeks-36 weeks in the study.
Period Title: Overall Study
Started 131 130
Completed 111 102
Not Completed 20 28
Reason Not Completed
Adverse Event             8             9
Death             1             0
Lost to Follow-up             1             1
Physician Decision             3             2
Pregnancy             1             0
Protocol Violation             0             2
Study Terminated by Sponsor             0             1
Withdrawal by Subject             6             11
Unable to demostrate ability self-inject             0             1
Quit due to lack of health benifits             0             1
Arm/Group Title BMN 165, 20mg/Day BMN 165, 40mg/Day Total
Hide Arm/Group Description Subjects who meet the eligibility criteria will be randomized 1:1 to titrate to one of two dose regimens: 20 or 40 mg/day. Randomization will be stratified by the last available blood Phe concentration prior to Day 1 (600-900 μmol/L and > 900 μmol/L). All subjects will initiate IP at a fixed-dose of 2.5 mg/week for 4 weeks (Induction). After the IP, subjects will enter the Titration Period (Weeks 5 up to 34) where they will increase their weekly dose to a daily dose regimen of 20 or 40 mg/day. The Titration Period will be individualized to each subject based on a minimum of 6 weeks (the amount of time it takes to reach a dose regimen of 20 mg/day with no dose interruptions) and up to 30 weeks (accounts for dose reduction or interruption due to AEs). Subjects will stop titration once they have achieved either the 20 or 40 mg/day dose regimen. The majority of subjects will maintain the 20 or 40 mg/day dose regimen for at least an additional 2 weeks until 26 weeks-36 weeks in the study. Subjects who meet the eligibility criteria will be randomized 1:1 to titrate to one of two dose regimens: 20 or 40 mg/day. Randomization will be stratified by the last available blood Phe concentration prior to Day 1 (600-900 μmol/L and > 900 μmol/L). All subjects will initiate IP at a fixed-dose of 2.5 mg/week for 4 weeks (Induction). After the IP, subjects will enter the Titration Period (Weeks 5 up to 34) where they will increase their weekly dose to a daily dose regimen of 20 or 40 mg/day. The Titration Period will be individualized to each subject based on a minimum of 6 weeks (the amount of time it takes to reach a dose regimen of 20 mg/day with no dose interruptions) and up to 30 weeks (accounts for dose reduction or interruption due to AEs). Subjects will stop titration once they have achieved either the 20 or 40 mg/day dose regimen. The majority of subjects will maintain the 20 or 40 mg/day dose regimen for at least an additional 2 weeks until 26 weeks-36 weeks in the study. Total of all reporting groups
Overall Number of Baseline Participants 131 130 261
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 131 participants 130 participants 261 participants
30.2  (8.63) 28.1  (8.77) 29.2  (8.75)
Age, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 131 participants 130 participants 261 participants
16 - <18 years
5
   3.8%
6
   4.6%
11
   4.2%
18 - <66 years
126
  96.2%
124
  95.4%
250
  95.8%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 131 participants 130 participants 261 participants
Female
62
  47.3%
68
  52.3%
130
  49.8%
Male
69
  52.7%
62
  47.7%
131
  50.2%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 131 participants 130 participants 261 participants
Hispanic or Latino
6
   4.6%
1
   0.8%
7
   2.7%
Not Hispanic or Latino
125
  95.4%
128
  98.5%
253
  96.9%
Unknown or Not Reported
0
   0.0%
1
   0.8%
1
   0.4%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 131 participants 130 participants 261 participants
American Indian or Alaska Native
0
   0.0%
1
   0.8%
1
   0.4%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
1
   0.8%
2
   1.5%
3
   1.1%
White
130
  99.2%
124
  95.4%
254
  97.3%
Other
0
   0.0%
2
   1.5%
2
   0.8%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 131 participants 130 participants 261 participants
131
 100.0%
130
 100.0%
261
 100.0%
1.Primary Outcome
Title Number of Participants With Hypersensitivity Adverse Reaction
Hide Description

Hypersensitivity AEs will be identified in two ways:

  • Broad Algorithmic anaphylactic reaction Standardized MedDRA Queries (SMQ)
  • Modified Hypersensitivity SMQ to include above additional preferred terms
Time Frame baseline and 36 weeks
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The safety population will consist of all subjects who receive any pegvaliase throughout the study duration. The safety population will be analyzed according to the treatment assignment actually received.
Arm/Group Title BMN 165, 20mg/Day BMN 165, 40mg/Day
Hide Arm/Group Description:
Subjects who meet the eligibility criteria will be randomized 1:1 to titrate to one of two dose regimens: 20 or 40 mg/day. Randomization will be stratified by the last available blood Phe concentration prior to Day 1 (600-900 μmol/L and > 900 μmol/L). All subjects will initiate IP at a fixed-dose of 2.5 mg/week for 4 weeks (Induction). After the IP, subjects will enter the Titration Period (Weeks 5 up to 34) where they will increase their weekly dose to a daily dose regimen of 20 or 40 mg/day. The Titration Period will be individualized to each subject based on a minimum of 6 weeks (the amount of time it takes to reach a dose regimen of 20 mg/day with no dose interruptions) and up to 30 weeks (accounts for dose reduction or interruption due to AEs). Subjects will stop titration once they have achieved either the 20 or 40 mg/day dose regimen. The majority of subjects will maintain the 20 or 40 mg/day dose regimen for at least an additional 2 weeks until 26 weeks-36 weeks in the study.
Subjects who meet the eligibility criteria will be randomized 1:1 to titrate to one of two dose regimens: 20 or 40 mg/day. Randomization will be stratified by the last available blood Phe concentration prior to Day 1 (600-900 μmol/L and > 900 μmol/L). All subjects will initiate IP at a fixed-dose of 2.5 mg/week for 4 weeks (Induction). After the IP, subjects will enter the Titration Period (Weeks 5 up to 34) where they will increase their weekly dose to a daily dose regimen of 20 or 40 mg/day. The Titration Period will be individualized to each subject based on a minimum of 6 weeks (the amount of time it takes to reach a dose regimen of 20 mg/day with no dose interruptions) and up to 30 weeks (accounts for dose reduction or interruption due to AEs). Subjects will stop titration once they have achieved either the 20 or 40 mg/day dose regimen. The majority of subjects will maintain the 20 or 40 mg/day dose regimen for at least an additional 2 weeks until 26 weeks-36 weeks in the study.
Overall Number of Participants Analyzed 131 130
Measure Type: Count of Participants
Unit of Measure: Participants
111
  84.7%
119
  91.5%
2.Secondary Outcome
Title Blood Phenylalanine Concentration
Hide Description Plasma phenylalanine (Phe) concentration
Time Frame baseline and 36 weeks
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The intent-to-treat (ITT) population will consist of all subjects who are randomized to study treatment whether or not treatment was received.
Arm/Group Title BMN 165, 20mg/Day BMN 165, 40mg/Day
Hide Arm/Group Description:
Subjects who meet the eligibility criteria will be randomized 1:1 to titrate to one of two dose regimens: 20 or 40 mg/day. Randomization will be stratified by the last available blood Phe concentration prior to Day 1 (600-900 μmol/L and > 900 μmol/L). All subjects will initiate IP at a fixed-dose of 2.5 mg/week for 4 weeks (Induction). After the IP, subjects will enter the Titration Period (Weeks 5 up to 34) where they will increase their weekly dose to a daily dose regimen of 20 or 40 mg/day. The Titration Period will be individualized to each subject based on a minimum of 6 weeks (the amount of time it takes to reach a dose regimen of 20 mg/day with no dose interruptions) and up to 30 weeks (accounts for dose reduction or interruption due to AEs). Subjects will stop titration once they have achieved either the 20 or 40 mg/day dose regimen. The majority of subjects will maintain the 20 or 40 mg/day dose regimen for at least an additional 2 weeks until 26 weeks-36 weeks in the study.
Subjects who meet the eligibility criteria will be randomized 1:1 to titrate to one of two dose regimens: 20 or 40 mg/day. Randomization will be stratified by the last available blood Phe concentration prior to Day 1 (600-900 μmol/L and > 900 μmol/L). All subjects will initiate IP at a fixed-dose of 2.5 mg/week for 4 weeks (Induction). After the IP, subjects will enter the Titration Period (Weeks 5 up to 34) where they will increase their weekly dose to a daily dose regimen of 20 or 40 mg/day. The Titration Period will be individualized to each subject based on a minimum of 6 weeks (the amount of time it takes to reach a dose regimen of 20 mg/day with no dose interruptions) and up to 30 weeks (accounts for dose reduction or interruption due to AEs). Subjects will stop titration once they have achieved either the 20 or 40 mg/day dose regimen. The majority of subjects will maintain the 20 or 40 mg/day dose regimen for at least an additional 2 weeks until 26 weeks-36 weeks in the study.
Overall Number of Participants Analyzed 131 130
Mean (Standard Deviation)
Unit of Measure: umol/L
Baseline 1241.0  (389.70) 1224.4  (384.28)
Week 36 868.4  (501.78) 624.4  (530.58)
3.Other Pre-specified Outcome
Title Dietary Phenylalanine
Hide Description All patients will complete a 3-day diet diary to be submitted baseline (predose on Day 1), Week 4, and every 4 weeks thereafter.
Time Frame baseline and 36 weeks
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The intent-to-treat (ITT) population will consist of all subjects who are randomized to study treatment whether or not treatment was received.
Arm/Group Title BMN 165, 20mg/Day BMN 165, 40mg/Day
Hide Arm/Group Description:
Subjects who meet the eligibility criteria will be randomized 1:1 to titrate to one of two dose regimens: 20 or 40 mg/day. Randomization will be stratified by the last available blood Phe concentration prior to Day 1 (600-900 μmol/L and > 900 μmol/L). All subjects will initiate IP at a fixed-dose of 2.5 mg/week for 4 weeks (Induction). After the IP, subjects will enter the Titration Period (Weeks 5 up to 34) where they will increase their weekly dose to a daily dose regimen of 20 or 40 mg/day. The Titration Period will be individualized to each subject based on a minimum of 6 weeks (the amount of time it takes to reach a dose regimen of 20 mg/day with no dose interruptions) and up to 30 weeks (accounts for dose reduction or interruption due to AEs). Subjects will stop titration once they have achieved either the 20 or 40 mg/day dose regimen. The majority of subjects will maintain the 20 or 40 mg/day dose regimen for at least an additional 2 weeks until 26 weeks-36 weeks in the study.
Subjects who meet the eligibility criteria will be randomized 1:1 to titrate to one of two dose regimens: 20 or 40 mg/day. Randomization will be stratified by the last available blood Phe concentration prior to Day 1 (600-900 μmol/L and > 900 μmol/L). All subjects will initiate IP at a fixed-dose of 2.5 mg/week for 4 weeks (Induction). After the IP, subjects will enter the Titration Period (Weeks 5 up to 34) where they will increase their weekly dose to a daily dose regimen of 20 or 40 mg/day. The Titration Period will be individualized to each subject based on a minimum of 6 weeks (the amount of time it takes to reach a dose regimen of 20 mg/day with no dose interruptions) and up to 30 weeks (accounts for dose reduction or interruption due to AEs). Subjects will stop titration once they have achieved either the 20 or 40 mg/day dose regimen. The majority of subjects will maintain the 20 or 40 mg/day dose regimen for at least an additional 2 weeks until 26 weeks-36 weeks in the study.
Overall Number of Participants Analyzed 131 130
Mean (Standard Deviation)
Unit of Measure: mg
Baseline 1750.8  (1168.91) 1647.9  (1222.68)
Week 36 1851.8  (1145.06) 2057.9  (1448.21)
Time Frame Week 0- Week 36
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title BMN 165, 20mg/Day BMN 165, 40mg/Day
Hide Arm/Group Description Subjects who meet the eligibility criteria will be randomized 1:1 to titrate to one of two dose regimens: 20 or 40 mg/day. Randomization will be stratified by the last available blood Phe concentration prior to Day 1 (600-900 μmol/L and > 900 μmol/L). All subjects will initiate IP at a fixed-dose of 2.5 mg/week for 4 weeks (Induction). After the IP, subjects will enter the Titration Period (Weeks 5 up to 34) where they will increase their weekly dose to a daily dose regimen of 20 or 40 mg/day. The Titration Period will be individualized to each subject based on a minimum of 6 weeks (the amount of time it takes to reach a dose regimen of 20 mg/day with no dose interruptions) and up to 30 weeks (accounts for dose reduction or interruption due to AEs). Subjects will stop titration once they have achieved either the 20 or 40 mg/day dose regimen. The majority of subjects will maintain the 20 or 40 mg/day dose regimen for at least an additional 2 weeks until 26 weeks-36 weeks in the study. Subjects who meet the eligibility criteria will be randomized 1:1 to titrate to one of two dose regimens: 20 or 40 mg/day. Randomization will be stratified by the last available blood Phe concentration prior to Day 1 (600-900 μmol/L and > 900 μmol/L). All subjects will initiate IP at a fixed-dose of 2.5 mg/week for 4 weeks (Induction). After the IP, subjects will enter the Titration Period (Weeks 5 up to 34) where they will increase their weekly dose to a daily dose regimen of 20 or 40 mg/day. The Titration Period will be individualized to each subject based on a minimum of 6 weeks (the amount of time it takes to reach a dose regimen of 20 mg/day with no dose interruptions) and up to 30 weeks (accounts for dose reduction or interruption due to AEs). Subjects will stop titration once they have achieved either the 20 or 40 mg/day dose regimen. The majority of subjects will maintain the 20 or 40 mg/day dose regimen for at least an additional 2 weeks until 26 weeks-36 weeks in the study.
All-Cause Mortality
BMN 165, 20mg/Day BMN 165, 40mg/Day
Affected / at Risk (%) Affected / at Risk (%)
Total   1/131 (0.76%)      0/130 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
BMN 165, 20mg/Day BMN 165, 40mg/Day
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   7/131 (5.34%)      19/130 (14.62%)    
Cardiac disorders     
Pericardial effusion  1  0/131 (0.00%)  0 1/130 (0.77%)  1
General disorders     
Electrocution  1  1/131 (0.76%)  1 0/130 (0.00%)  0
Injection site reaction  1  1/131 (0.76%)  1 0/130 (0.00%)  0
Puncture site haemorrhage  1  0/131 (0.00%)  0 1/130 (0.77%)  1
Immune system disorders     
Anaphylactic reaction  1  2/131 (1.53%)  2 6/130 (4.62%)  6
Anaphylactoid reaction  1  0/131 (0.00%)  0 2/130 (1.54%)  2
Hypersensitivity  1  0/131 (0.00%)  0 3/130 (2.31%)  3
Serum sickness  1  1/131 (0.76%)  1 1/130 (0.77%)  1
Infections and infestations     
Gastroenteritis viral  1  0/131 (0.00%)  0 1/130 (0.77%)  1
Pyelonephritis acute  1  0/131 (0.00%)  0 1/130 (0.77%)  1
Investigations     
Blood creatine phosphokinase increased  1  1/131 (0.76%)  1 1/130 (0.77%)  1
Metabolism and nutrition disorders     
Hypokalaemia  1  0/131 (0.00%)  0 1/130 (0.77%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Papilloma  1  0/131 (0.00%)  0 1/130 (0.77%)  1
Pregnancy, puerperium and perinatal conditions     
Abortion missed  1  1/131 (0.76%)  1 0/130 (0.00%)  0
Psychiatric disorders     
Anxiety  1  0/131 (0.00%)  0 2/130 (1.54%)  2
Vascular disorders     
Deep vein thrombosis  1  1/131 (0.76%)  1 0/130 (0.00%)  0
1
Term from vocabulary, MedDRA 18.0
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
BMN 165, 20mg/Day BMN 165, 40mg/Day
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   130/131 (99.24%)      129/130 (99.23%)    
Blood and lymphatic system disorders     
Lymphadenopathy  1  12/131 (9.16%)  21 10/130 (7.69%)  18
Gastrointestinal disorders     
Abdominal discomfort  1  11/131 (8.40%)  13 5/130 (3.85%)  5
Abdominal pain  1  9/131 (6.87%)  10 5/130 (3.85%)  8
Abdominal pain upper  1  10/131 (7.63%)  13 5/130 (3.85%)  6
Diarrhoea  1  13/131 (9.92%)  16 11/130 (8.46%)  13
Dyspepsia  1  8/131 (6.11%)  13 9/130 (6.92%)  11
Nausea  1  29/131 (22.14%)  38 22/130 (16.92%)  28
Vomiting  1  21/131 (16.03%)  33 16/130 (12.31%)  20
General disorders     
Chills  1  15/131 (11.45%)  22 9/130 (6.92%)  13
Fatigue  1  21/131 (16.03%)  58 13/130 (10.00%)  27
Injection site bruising  1  24/131 (18.32%)  65 26/130 (20.00%)  44
Injection site erythema  1  57/131 (43.51%)  323 61/130 (46.92%)  210
Injection site haemorrhage  1  7/131 (5.34%)  9 4/130 (3.08%)  6
Injection site oedema  1  0/131 (0.00%)  0 7/130 (5.38%)  112
Injection site pain  1  27/131 (20.61%)  63 29/130 (22.31%)  55
Injection site pruritus  1  29/131 (22.14%)  87 36/130 (27.69%)  119
Injection site rash  1  22/131 (16.79%)  96 28/130 (21.54%)  66
Injection site reaction  1  80/131 (61.07%)  700 68/130 (52.31%)  495
Injection site swelling  1  23/131 (17.56%)  70 20/130 (15.38%)  45
Injection site urticaria  1  10/131 (7.63%)  27 14/130 (10.77%)  25
Oedema peripheral  1  6/131 (4.58%)  7 9/130 (6.92%)  22
Pain  1  22/131 (16.79%)  31 7/130 (5.38%)  11
Pyrexia  1  26/131 (19.85%)  43 8/130 (6.15%)  13
Immune system disorders     
Hypersensitivity  1  4/131 (3.05%)  4 7/130 (5.38%)  7
Infections and infestations     
Nasopharyngitis  1  24/131 (18.32%)  33 25/130 (19.23%)  29
Sinusitis  1  9/131 (6.87%)  10 13/130 (10.00%)  13
Upper respiratory tract infection  1  22/131 (16.79%)  27 20/130 (15.38%)  24
Injury, poisoning and procedural complications     
Contusion  1  17/131 (12.98%)  26 9/130 (6.92%)  11
Investigations     
Blood cortisol decreased  1  1/131 (0.76%)  1 8/130 (6.15%)  8
Blood creatine phosphokinase increased  1  7/131 (5.34%)  7 4/130 (3.08%)  4
C-reactive protein increased  1  12/131 (9.16%)  16 10/130 (7.69%)  10
Complement factor C3 decreased  1  11/131 (8.40%)  12 7/130 (5.38%)  7
Complement factor C4 decreased  1  12/131 (9.16%)  12 6/130 (4.62%)  6
Red blood cell sedimentation rate increased  1  7/131 (5.34%)  8 1/130 (0.77%)  1
Musculoskeletal and connective tissue disorders     
Arthralgia  1  78/131 (59.54%)  376 92/130 (70.77%)  350
Back pain  1  21/131 (16.03%)  34 20/130 (15.38%)  26
Joint stiffness  1  8/131 (6.11%)  9 6/130 (4.62%)  10
Joint swelling  1  8/131 (6.11%)  15 5/130 (3.85%)  6
Musculoskeletal pain  1  12/131 (9.16%)  18 19/130 (14.62%)  24
Myalgia  1  12/131 (9.16%)  18 6/130 (4.62%)  9
Neck pain  1  5/131 (3.82%)  7 10/130 (7.69%)  12
Pain in extremity  1  20/131 (15.27%)  42 26/130 (20.00%)  52
Pain in jaw  1  8/131 (6.11%)  9 6/130 (4.62%)  8
Nervous system disorders     
Dizziness  1  20/131 (15.27%)  24 21/130 (16.15%)  32
Headache  1  46/131 (35.11%)  112 36/130 (27.69%)  98
Psychiatric disorders     
Anxiety  1  4/131 (3.05%)  11 10/130 (7.69%)  11
Respiratory, thoracic and mediastinal disorders     
Cough  1  11/131 (8.40%)  12 10/130 (7.69%)  13
Nasal congestion  1  9/131 (6.87%)  10 7/130 (5.38%)  7
Oropharyngeal pain  1  22/131 (16.79%)  23 12/130 (9.23%)  14
Skin and subcutaneous tissue disorders     
Alopecia  1  8/131 (6.11%)  8 12/130 (9.23%)  12
Erythema  1  11/131 (8.40%)  17 12/130 (9.23%)  28
Pruritus  1  22/131 (16.79%)  39 25/130 (19.23%)  65
Rash  1  27/131 (20.61%)  68 40/130 (30.77%)  83
Rash erythematous  1  3/131 (2.29%)  3 8/130 (6.15%)  17
Urticaria  1  18/131 (13.74%)  71 30/130 (23.08%)  101
1
Term from vocabulary, MedDRA 18.0
Indicates events were collected by systematic assessment
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.
Results Point of Contact
Name/Title: Principal Scientist I, Clinical Sciences
Organization: BioMarin Pharmaceutical Inc.
Phone: 415-506-6348
Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT01819727     History of Changes
Other Study ID Numbers: 165-301
Prism301 ( Other Identifier: Phase 3 "brand" name )
First Submitted: March 18, 2013
First Posted: March 28, 2013
Results First Submitted: June 22, 2018
Results First Posted: September 10, 2018
Last Update Posted: September 10, 2018