Trial Outcomes & Findings for A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease (NCT NCT00403494)
NCT ID: NCT00403494
Last Updated: 2021-01-07
Results Overview
This is to assess the effect of oral sapropterin dihydrochloride versus placebo on peak walking time (PWT) in subjects with intermittent claudication (IC) caused by peripheral arterial disease (PAD).
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
190 participants
Primary outcome timeframe
Baseline and up to Week 24
Results posted on
2021-01-07
Participant Flow
This is a multicenter, multinational study. 31 principal investigators (PIs) enrolled subjects at 10 sites in Argentina sites and 21 sites in the United States (US). A total of 190 patients were recruited with 96 subjects randomized to Sapropterin dihydrichloride and 94 to Placebo. 65 subjects were in Argentina - (32 Sapropterin dihydrochloride, 33 Placebo), and 125 subjects in the US - (64 Sapropterin dihydrochloride, 61 Placebo)
Participant milestones
| Measure |
Sapropterin Dihydrochloride
Subjects receive 400 mg oral sapropterin dihydrochloride twice daily for 24 weeks.
|
Placebo
Subjects receive matching oral Placebo twice daily for 24 week.
|
|---|---|---|
|
Overall Study
STARTED
|
96
|
94
|
|
Overall Study
Intent to Treat
|
87
|
83
|
|
Overall Study
Per-protocol
|
69
|
69
|
|
Overall Study
Safety Population
|
97
|
91
|
|
Overall Study
COMPLETED
|
81
|
80
|
|
Overall Study
NOT COMPLETED
|
15
|
14
|
Reasons for withdrawal
| Measure |
Sapropterin Dihydrochloride
Subjects receive 400 mg oral sapropterin dihydrochloride twice daily for 24 weeks.
|
Placebo
Subjects receive matching oral Placebo twice daily for 24 week.
|
|---|---|---|
|
Overall Study
Adverse Event
|
10
|
8
|
|
Overall Study
Withdrew Consent
|
3
|
2
|
|
Overall Study
Inappropriately enrolled
|
0
|
1
|
|
Overall Study
Lost to Follow-up
|
0
|
2
|
|
Overall Study
Protocol noncompliance
|
0
|
1
|
|
Overall Study
Death
|
1
|
0
|
|
Overall Study
Sponsor Request
|
1
|
0
|
Baseline Characteristics
A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease
Baseline characteristics by cohort
| Measure |
Sapropterin Dihydrochloride
n=87 Participants
Subjects receive 400 mg oral sapropterin dihydrochloride twice daily for 24 weeks.
|
Placebo
n=83 Participants
Subjects receive matching oral Placebo twice daily for 24 weeks.
|
Total
n=170 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
68.0 years
STANDARD_DEVIATION 8.9 • n=5 Participants
|
66.6 years
STANDARD_DEVIATION 7.0 • n=7 Participants
|
67.3 years
STANDARD_DEVIATION 8.1 • n=5 Participants
|
|
Sex: Female, Male
Female
|
22 Participants
n=5 Participants
|
23 Participants
n=7 Participants
|
45 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
65 Participants
n=5 Participants
|
60 Participants
n=7 Participants
|
125 Participants
n=5 Participants
|
|
Age
< 65 years
|
28 Participants
n=5 Participants
|
35 Participants
n=7 Participants
|
63 Participants
n=5 Participants
|
|
Age
>= 65 years
|
59 Participants
n=5 Participants
|
48 Participants
n=7 Participants
|
107 Participants
n=5 Participants
|
|
Age
>= 75 years
|
25 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
35 Participants
n=5 Participants
|
|
Race
White
|
80 Participants
n=5 Participants
|
75 Participants
n=7 Participants
|
155 Participants
n=5 Participants
|
|
Race
Black/African American
|
5 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Race
Other
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race
Asian
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity
Not Hispanic or Latino
|
58 Participants
n=5 Participants
|
52 Participants
n=7 Participants
|
110 Participants
n=5 Participants
|
|
Ethnicity
Hispanic or Latino
|
29 Participants
n=5 Participants
|
31 Participants
n=7 Participants
|
60 Participants
n=5 Participants
|
|
Country of Study Site
United States
|
60 Participants
n=5 Participants
|
53 Participants
n=7 Participants
|
113 Participants
n=5 Participants
|
|
Country of Study Site
Argentina
|
27 Participants
n=5 Participants
|
30 Participants
n=7 Participants
|
57 Participants
n=5 Participants
|
|
With/Without Vitamin C
Without Vitamin C
|
41 Participants
n=5 Participants
|
39 Participants
n=7 Participants
|
80 Participants
n=5 Participants
|
|
With/Without Vitamin C
With Vitamin C
|
46 Participants
n=5 Participants
|
44 Participants
n=7 Participants
|
90 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline and up to Week 24Population: Intent-To-Treat Population consisting of all subjects who were randomized, received 1 dose of study drug and had at least 1 post-baselline treadmill test.
This is to assess the effect of oral sapropterin dihydrochloride versus placebo on peak walking time (PWT) in subjects with intermittent claudication (IC) caused by peripheral arterial disease (PAD).
Outcome measures
| Measure |
Sapropterin Dihydrochloride
n=87 Participants
Intent to Treat Population. Subjects receive 400 mg oral sapropterin dihydrochloride twice daily for 24 weeks
|
Placebo
n=83 Participants
Intent to Treat Population. Subjects receive matching oral Placebo twice daily for 24 weeks.
|
|---|---|---|
|
Change in Peak Walking Time (PWT) From Baseline
Baseline
|
5.394 minutes
Standard Deviation 2.803
|
5.712 minutes
Standard Deviation 2.876
|
|
Change in Peak Walking Time (PWT) From Baseline
Change from Baseline to Week 24
|
1.121 minutes
Standard Deviation 2.775
|
1.493 minutes
Standard Deviation 2.597
|
PRIMARY outcome
Timeframe: Up to 24-weeksPopulation: Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
Adverse events were described and summarized with focus on treatment- emergent events (TEAEs). A TEAE was defined as any AE that presented , increased in frequency or worsened in severity following initiation of study drug administration. If the onset of an AE was missing then the AE was considered treatment emergent. Drug-related AEs are AEs classified by the investigator as possibly or probably related to study drug.
Outcome measures
| Measure |
Sapropterin Dihydrochloride
n=97 Participants
Intent to Treat Population. Subjects receive 400 mg oral sapropterin dihydrochloride twice daily for 24 weeks
|
Placebo
n=91 Participants
Intent to Treat Population. Subjects receive matching oral Placebo twice daily for 24 weeks.
|
|---|---|---|
|
Number of Subjects With Adverse Events (AEs)
Any AEs
|
84 Participants
|
66 Participants
|
|
Number of Subjects With Adverse Events (AEs)
Study Drug-related AEs
|
29 Participants
|
30 Participants
|
|
Number of Subjects With Adverse Events (AEs)
Study-Drug-related SAEs
|
0 Participants
|
1 Participants
|
|
Number of Subjects With Adverse Events (AEs)
Any AEs leading to Study Withdrawal
|
11 Participants
|
7 Participants
|
|
Number of Subjects With Adverse Events (AEs)
Study Drug-related AEs leading to Study WIthdrawal
|
4 Participants
|
5 Participants
|
|
Number of Subjects With Adverse Events (AEs)
Any SAEs Leading to Study Withdrawal
|
4 Participants
|
1 Participants
|
|
Number of Subjects With Adverse Events (AEs)
Study Drug-related SAEs Leading to Study Withdrawal
|
0 Participants
|
1 Participants
|
|
Number of Subjects With Adverse Events (AEs)
Deaths
|
1 Participants
|
0 Participants
|
|
Number of Subjects With Adverse Events (AEs)
Any SAEs
|
14 Participants
|
11 Participants
|
SECONDARY outcome
Timeframe: Baseline and up to Week 24Population: Intent-To-Treat Population
Time, in minutes, when the subject first begins to experience claudication symptoms, regardless of whether this is manifested as muscle pain, ache, cramp, numbness or fatigue.
Outcome measures
| Measure |
Sapropterin Dihydrochloride
n=87 Participants
Intent to Treat Population. Subjects receive 400 mg oral sapropterin dihydrochloride twice daily for 24 weeks
|
Placebo
n=83 Participants
Intent to Treat Population. Subjects receive matching oral Placebo twice daily for 24 weeks.
|
|---|---|---|
|
Change in Claudication Onset Time (COT) From Baseline
Baseline
|
2.159 minutes
Standard Deviation 1.611
|
2.313 minutes
Standard Deviation 1.689
|
|
Change in Claudication Onset Time (COT) From Baseline
Change from Baseline to Week 24
|
0.903 minutes
Standard Deviation 1.900
|
1.015 minutes
Standard Deviation 1.902
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline up to 24 weeksPopulation: Intent-to-Treat Population
The first 50% of subjects enrolled in the study were randomized to receive sapropterin dihydrochloride at 400 mg BID or oral placebo BID alone (without vitamin C). When approximately 50% enrollment was met, 1000 mg/day vitamin C was added to the dose regimen of newly enrolled subjects in both sapropterin dihydrochloride and placebo treatment groups given orally in two divided doses of 500 mg with study drug.
Outcome measures
| Measure |
Sapropterin Dihydrochloride
n=87 Participants
Intent to Treat Population. Subjects receive 400 mg oral sapropterin dihydrochloride twice daily for 24 weeks
|
Placebo
n=83 Participants
Intent to Treat Population. Subjects receive matching oral Placebo twice daily for 24 weeks.
|
|---|---|---|
|
Change in Peak Walking Time From Baseline With and Without Vitamin C
Baseline without vitamin C
|
4.776 minutes
Standard Deviation 2.650
|
5.201 minutes
Standard Deviation 2.781
|
|
Change in Peak Walking Time From Baseline With and Without Vitamin C
Change from Baseline week 24; without vitamin C
|
0.264 minutes
Standard Deviation 2.064
|
0.607 minutes
Standard Deviation 2.166
|
|
Change in Peak Walking Time From Baseline With and Without Vitamin C
Baseline with vitamin C
|
5.944 minutes
Standard Deviation 2.849
|
6.164 minutes
Standard Deviation 2.914
|
|
Change in Peak Walking Time From Baseline With and Without Vitamin C
Change from Baseline week 24; with vitamin C
|
1.826 minutes
Standard Deviation 3.093
|
2.255 minutes
Standard Deviation 2.716
|
Adverse Events
Sapropterin Dihydrochloride
Serious events: 14 serious events
Other events: 84 other events
Deaths: 1 deaths
Placebo
Serious events: 11 serious events
Other events: 66 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Sapropterin Dihydrochloride
n=97 participants at risk
Subjects receive 400 mg oral sapropterin dihydrochloride twice daily for 24 weeks.
|
Placebo
n=91 participants at risk
Subjects receive matching oral Placebo twice daily for 24 weeks.
|
|---|---|---|
|
Cardiac disorders
Atrial flutter
|
1.0%
1/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
0.00%
0/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Cardiac disorders
Angina pectoris
|
0.00%
0/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
1.1%
1/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Vascular disorders
Acute coronary syndrome
|
0.00%
0/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
1.1%
1/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Vascular disorders
Angina unstable
|
1.0%
1/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
0.00%
0/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Vascular disorders
Arterial occlusive disease
|
1.0%
1/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
0.00%
0/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Vascular disorders
Cerebrovascular accident
|
1.0%
1/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
0.00%
0/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Vascular disorders
Coronary artery disease
|
1.0%
1/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
0.00%
0/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Vascular disorders
Gastric haemorrhage
|
0.00%
0/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
1.1%
1/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Vascular disorders
Ischaemic limb pain
|
1.0%
1/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
0.00%
0/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Vascular disorders
Peripheral vascular disorder
|
0.00%
0/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
1.1%
1/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Vascular disorders
Renal artery stenosis
|
0.00%
0/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
1.1%
1/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Cardiac disorders
Myocardial infarction
|
2.1%
2/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
0.00%
0/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Cardiac disorders
Cardiac failure
|
0.00%
0/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
1.1%
1/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Cardiac disorders
Cardiac failure congestive
|
1.0%
1/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
0.00%
0/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Cardiac disorders
Myocardial ischaemia
|
0.00%
0/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
1.1%
1/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Cardiac disorders
Tachyarrhythmia
|
1.0%
1/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
0.00%
0/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Infections and infestations
Pneumonia
|
2.1%
2/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
0.00%
0/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Infections and infestations
Urinary tract infection
|
1.0%
1/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
0.00%
0/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
1.1%
1/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Gastrointestinal disorders
Retroperitoneal neoplasm
|
1.0%
1/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
0.00%
0/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Investigations
Electrocardiogram abnormal
|
0.00%
0/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
1.1%
1/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Investigations
Prostate examination abnormal
|
1.0%
1/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
0.00%
0/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Respiratory, thoracic and mediastinal disorders
Chronic obstructive pulmonary disease
|
0.00%
0/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
1.1%
1/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
0.00%
0/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
1.1%
1/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
General disorders
Death
|
1.0%
1/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
0.00%
0/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Nervous system disorders
Transient ischaemic attack
|
1.0%
1/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
0.00%
0/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Psychiatric disorders
Alcoholism
|
0.00%
0/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
1.1%
1/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Skin and subcutaneous tissue disorders
Cellulitis
|
0.00%
0/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
1.1%
1/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
Other adverse events
| Measure |
Sapropterin Dihydrochloride
n=97 participants at risk
Subjects receive 400 mg oral sapropterin dihydrochloride twice daily for 24 weeks.
|
Placebo
n=91 participants at risk
Subjects receive matching oral Placebo twice daily for 24 weeks.
|
|---|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
4.1%
4/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
7.7%
7/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Gastrointestinal disorders
Nausea
|
5.2%
5/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
4.4%
4/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
General disorders
Pharmaceutical product complaint
|
4.1%
4/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
7.7%
7/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
9.3%
9/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
4.4%
4/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
5.2%
5/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
6.6%
6/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
6.2%
6/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
1.1%
1/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Infections and infestations
Nasopharyngitis
|
6.2%
6/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
3.3%
3/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Nervous system disorders
Headache
|
4.1%
4/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
5.5%
5/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Vascular disorders
Hypotension
|
8.2%
8/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
0.00%
0/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Vascular disorders
Intermittent claudication
|
6.2%
6/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
1.1%
1/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Gastrointestinal disorders
Abdominal pain
|
4.1%
4/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
3.3%
3/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Gastrointestinal disorders
Abdominal Pain Upper
|
4.1%
4/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
1.1%
1/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
General disorders
Fatigue
|
4.1%
4/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
4.4%
4/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Vascular disorders
Hypertension
|
4.1%
4/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
4.4%
4/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
General disorders
Oedema peripheral
|
4.1%
4/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
4.4%
4/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Infections and infestations
Upper respiratory tract infection
|
4.1%
4/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
3.3%
3/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Infections and infestations
Bronchitis
|
3.1%
3/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
1.1%
1/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
3.1%
3/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
1.1%
1/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Nervous system disorders
Insomnia
|
3.1%
3/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
3.3%
3/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
3.1%
3/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
3.3%
3/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal discomfort
|
3.1%
3/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
0.00%
0/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
3.1%
3/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
0.00%
0/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Infections and infestations
Urinary tract infection
|
3.1%
3/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
3.3%
3/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Gastrointestinal disorders
Flatulence
|
2.1%
2/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
5.5%
5/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Gastrointestinal disorders
Dyspepsia
|
0.00%
0/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
5.5%
5/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
3.1%
3/97 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
1.1%
1/91 • Up to 25 Weeks
Of the 188 patients (96 randomized to study drug and 94 to placebo.) 2 subjects randomized to receive placebo, received at least 1 dose of study drug and were included in the study drug group. 1 participant from each group was excluded from the safety analysis because they did not have at least 1 post-Baseline safety assessment.
|
Additional Information
Joshua Lilienstein/Medical Director, Global Medical Affairs
BioMarin Pharmaceutical Inc.
Phone: 651.523.0310
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60