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Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)

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: NCT01037309
Recruitment Status : Completed
First Posted : December 23, 2009
Results First Posted : April 9, 2015
Last Update Posted : October 16, 2018
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Duchenne Muscular Dystrophy
Interventions Drug: PRO044 SC
Drug: PRO044 IV
Enrollment 18
Recruitment Details 3 Subjects were recruited to each of cohorts 1-6 (total n=18) for SC administration PRO044 9 of these 18 subjects were recruited to cohorts 7-9 (n=3 in each cohort) for IV adminstration PRO044
Pre-assignment Details 3 Subjects were recruited to each of cohorts 1-6 (total n=18) for SC administration PRO044 9 of these 18 subjects were recruited to cohorts 7-9 (n=3 in each cohort) for IV adminstration PRO044
Arm/Group Title Subcutaneous PRO044 0.5 mg/kg Subcutaneous PRO044 1.5 mg/kg Subcutaneous PRO044 5 mg/kg Subcutaneous PRO044 8 mg/kg Subcutaneous PRO044 10 mg/kg Subcutaneous PRO044 12 mg/kg Intravenous PRO044 1.5 mg/kg Intravenous PRO044 5 mg/kg Intravenous PRO044 8 mg/kg
Hide Arm/Group Description

Subcutaneous injection of 0.5 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 1.5 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 5 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 8 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 10 mg/kg on day 1, 8, 15, 22 and 29

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 12 mg/kg on day 1, 8, 15, 22 and 29

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Intravenous injection of maximally 1.5 mg/kg on day 1, 8, 15, 22 and 29

PRO044 IV: Intravenous injection, once a week, for five weeks

Intravenous injection of maximally 5 mg/kg on day 1, 8, 15, 22 and 29

PRO044 IV: Intravenous injection, once a week, for five weeks

Intravenous injection of maximally 8 mg/kg on day 1, 8, 15, 22 and 29

PRO044 IV: Intravenous injection, once a week, for five weeks
Period Title: Subcutaneous Cohort 1 - 6
Started 3 3 3 3 3 3 0 0 0
Completed 3 3 3 3 3 3 0 0 0
Not Completed 0 0 0 0 0 0 0 0 0
Period Title: Intravenous Period 7 - 9
Started 0 0 0 0 0 0 3 3 3
Completed 0 0 0 0 0 0 3 3 3
Not Completed 0 0 0 0 0 0 0 0 0
Arm/Group Title PRO044, Cohort 1 PRO044, Cohort 2 PRO044, Cohort 3 PRO044, Cohort 4 PRO044, Cohort 5 PRO044, Cohort 6 Total
Hide Arm/Group Description

Subcutaneous injection of 0.5 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 1.5 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 5 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 8 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 10 mg/kg on day 1, 8, 15, 22 and 29

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 12 mg/kg on day 1, 8, 15, 22 and 29

PRO044 SC: Subcutaneous injection, once a week, for five weeks

 Total of all reporting groups
Overall Number of Baseline Participants 3 3 3 3 3 3 18
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 3 participants 3 participants 3 participants 3 participants 18 participants
<=18 years
3
 100.0%
3
 100.0%
3
 100.0%
3
 100.0%
3
 100.0%
3
 100.0%
18
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 3 participants 3 participants 3 participants 3 participants 3 participants 18 participants
Female
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Male
3
 100.0%
3
 100.0%
3
 100.0%
3
 100.0%
3
 100.0%
3
 100.0%
18
 100.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
 Number Analyzed 3 participants 3 participants 3 participants 3 participants 3 participants 3 participants 18 participants
Belgium 3 1 0 0 1 1 6
Netherlands 0 0 0 1 0 0 1
Italy 0 0 3 1 1 2 7
Sweden 0 2 0 1 1 0 4
1.Primary Outcome
Title Increase in Dystrophin Expression in the Muscle Biopsies by Immunofluorescence Analyses of Cross-sections and by Western Blot Analyses of Total Protein Extracts
Hide Description [Not Specified]
Time Frame Within 13 weeks after 5 weeks of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
For some participants it was not possible to determine dystrophin expression in muscle biopsy
Arm/Group Title PRO044, Cohort 1 PRO044, Cohort 2 PRO044, Cohort 3 PRO044, Cohort 4 PRO044, Cohort 5 PRO044, Cohort 6 PRO044, Cohort 7 PRO044, Cohort 8 PRO044, Cohort 9
Hide Arm/Group Description:

Subcutaneous injection of 0.5 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 1.5 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 5 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 8 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 10 mg/kg on day 1, 8, 15, 22 and 29

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 12 mg/kg on day 1, 8, 15, 22 and 29

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Intravenous injection of maximally 1.5 mg/kg on day 1, 8, 15, 22 and 29

PRO044 IV: Intravenous injection, once a week, for five weeks

Intravenous injection of maximally 5 mg/kg on day 1, 8, 15, 22 and 29

PRO044 IV: Intravenous injection, once a week, for five weeks

Intravenous injection of maximally 8 mg/kg on day 1, 8, 15, 22 and 29

PRO044 IV: Intravenous injection, once a week, for five weeks
Overall Number of Participants Analyzed 1 1 3 3 2 3 2 3 3
Measure Type: Count of Participants
Unit of Measure: Participants
1
 100.0%
1
 100.0%
0
   0.0%
1
  33.3%
1
  50.0%
2
  66.7%
2
 100.0%
3
 100.0%
1
  33.3%
2.Primary Outcome
Title Safety and Tolerability of PRO044
Hide Description number of subjects with 1 or more treatment emergent adverse events following SC or IV PRO044
Time Frame During the 5 weeks of treatment and during the 13 weeks after treatment
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title PRO044, Cohort 1 PRO044, Cohort 2 PRO044, Cohort 3 PRO044, Cohort 4 PRO044, Cohort 5 PRO044, Cohort 6 PRO044, Cohort 7 PRO044, Cohort 8 PRO044, Cohort 9
Hide Arm/Group Description:

Subcutaneous injection of 0.5 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 1.5 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 5 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 8 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 10 mg/kg on day 1, 8, 15, 22 and 29

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 12 mg/kg on day 1, 8, 15, 22 and 29

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Intravenous injection of maximally 1.5 mg/kg on day 1, 8, 15, 22 and 29

PRO044 IV: Intravenous injection, once a week, for five weeks

Intravenous injection of maximally 5 mg/kg on day 1, 8, 15, 22 and 29

PRO044 IV: Intravenous injection, once a week, for five weeks

Intravenous injection of maximally 8 mg/kg on day 1, 8, 15, 22 and 29

PRO044 IV: Intravenous injection, once a week, for five weeks
Overall Number of Participants Analyzed 3 3 3 3 3 3 3 3 3
Measure Type: Count of Participants
Unit of Measure: Participants
3
 100.0%
3
 100.0%
3
 100.0%
3
 100.0%
3
 100.0%
3
 100.0%
2
  66.7%
3
 100.0%
3
 100.0%
3.Secondary Outcome
Title PRO044 Pharmacokinetic Cmax (μg/mL) Following Subcutaneous Administration
Hide Description Pharmacokinetic population evaluated for maximum plasma concentration (Cmax)
Time Frame Week 1, Week 5
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic population evaluated
Arm/Group Title PRO044, Cohort 1 PRO044, Cohort 2 PRO044, Cohort 3 PRO044, Cohort 4 PRO044, Cohort 5 PRO044, Cohort 6 PRO044, Cohort 7 PRO044, Cohort 8 PRO044, Cohort 9
Hide Arm/Group Description:

Subcutaneous injection of 0.5 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 1.5 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 5 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 8 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 10 mg/kg on day 1, 8, 15, 22 and 29

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 12 mg/kg on day 1, 8, 15, 22 and 29

PRO044 SC: Subcutaneous injection, once a week, for five weeks

IV injection of maximally 1.5 mg/kg on day 1, 8, 15, 22 and 29

PRO044 SC: IV, once a week, for five weeks

IV injection of maximally 5 mg/kg on day 1, 8, 15, 22 and 29

PRO044 SC: IV, once a week, for five weeks

IV injection of maximally 12 mg/kg on day 1, 8, 15, 22 and 29

PRO044 SC: IV, once a week, for five weeks
Overall Number of Participants Analyzed 3 3 3 3 3 3 3 3 3
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: ug/mL
Week 1
0.4
(42.5%)
1.6
(21.7%)
4.2
(61.5%)
6.2
(53.8%)
4.9
(21.7%)
5.2
(16.0%)
3.1
(28.9%)
4.7
(18.9%)
8.7
(31.3%)
Week 5
0.4
(28.8%)
1.5
(22.9%)
3.4
(31.4%)
5.1
(24.2%)
4.4
(7.0%)
4.8
(35.7%)
2.2
(20.6%)
3.5
(69.5%)
8.7
(33.6%)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title PRO044, Cohort 1 PRO044, Cohort 2 PRO044, Cohort 3 PRO044, Cohort 4 PRO044, Cohort 5 PRO044, Cohort 6 PRO044, Cohort 7 PRO044, Cohort 8 PRO044, Cohort 9
Hide Arm/Group Description

Subcutaneous injection of 0.5 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 1.5 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 5 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 8 mg/kg on day 1, 8, 15, 22 and 29.

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 10 mg/kg on day 1, 8, 15, 22 and 29

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Subcutaneous injection of maximally 12 mg/kg on day 1, 8, 15, 22 and 29

PRO044 SC: Subcutaneous injection, once a week, for five weeks

Intravenous injection of maximally 1.5 mg/kg on day 1, 8, 15, 22 and 29

PRO044 IV: Intravenous injection, once a week, for five weeks

Intravenous injection of maximally 5 mg/kg on day 1, 8, 15, 22 and 29

PRO044 IV: Intravenous injection, once a week, for five weeks

Intravenous injection of maximally 8 mg/kg on day 1, 8, 15, 22 and 29

PRO044 IV: Intravenous injection, once a week, for five weeks
All-Cause Mortality
PRO044, Cohort 1 PRO044, Cohort 2 PRO044, Cohort 3 PRO044, Cohort 4 PRO044, Cohort 5 PRO044, Cohort 6 PRO044, Cohort 7 PRO044, Cohort 8 PRO044, Cohort 9
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--      --/--      --/--      --/--      --/--      --/--      --/--    
Hide Serious Adverse Events
PRO044, Cohort 1 PRO044, Cohort 2 PRO044, Cohort 3 PRO044, Cohort 4 PRO044, Cohort 5 PRO044, Cohort 6 PRO044, Cohort 7 PRO044, Cohort 8 PRO044, Cohort 9
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/3 (33.33%)      0/3 (0.00%)      1/3 (33.33%)      0/3 (0.00%)      0/3 (0.00%)      0/3 (0.00%)      0/3 (0.00%)      0/3 (0.00%)      0/3 (0.00%)    
Blood and lymphatic system disorders                   
International normalised ratio increased  0/3 (0.00%)  0 0/3 (0.00%)  0 1/3 (33.33%)  1 0/3 (0.00%)  0 0/3 (0.00%)  0 0/3 (0.00%)  0 0/3 (0.00%)  0 0/3 (0.00%)  0 0/3 (0.00%)  0
activated partial thromboplastin time prolonged  0/3 (0.00%)  0 0/3 (0.00%)  0 1/3 (33.33%)  1 0/3 (0.00%)  0 0/3 (0.00%)  0 0/3 (0.00%)  0 0/3 (0.00%)  0 0/3 (0.00%)  0 0/3 (0.00%)  0
Injury, poisoning and procedural complications                   
Concussion [1]  1/3 (33.33%)  1 0/3 (0.00%)  0 0/3 (0.00%)  0 0/3 (0.00%)  0 0/3 (0.00%)  0 0/3 (0.00%)  0 0/3 (0.00%)  0 0/3 (0.00%)  0 0/3 (0.00%)  0
Renal and urinary disorders                   
orthostatic proteinuria  0/3 (0.00%)  0 0/3 (0.00%)  0 1/3 (33.33%)  1 0/3 (0.00%)  0 0/3 (0.00%)  0 0/3 (0.00%)  0 0/3 (0.00%)  0 0/3 (0.00%)  0 0/3 (0.00%)  0
[1]
fell over in playground
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
PRO044, Cohort 1 PRO044, Cohort 2 PRO044, Cohort 3 PRO044, Cohort 4 PRO044, Cohort 5 PRO044, Cohort 6 PRO044, Cohort 7 PRO044, Cohort 8 PRO044, Cohort 9
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/3 (100.00%)      3/3 (100.00%)      3/3 (100.00%)      3/3 (100.00%)      3/3 (100.00%)      3/3 (100.00%)      2/3 (66.67%)      3/3 (100.00%)      3/3 (100.00%)    
Gastrointestinal disorders                   
Abdominal pain  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  2/3 (66.67%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Abdominal pain upper  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%) 
Diarrhea  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Dyspepsia  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Vomiting  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
General disorders                   
Injection site hematoma  1/3 (33.33%)  3/3 (100.00%)  3/3 (100.00%)  3/3 (100.00%)  3/3 (100.00%)  3/3 (100.00%)  1/3 (33.33%)  1/3 (33.33%)  0/3 (0.00%) 
Injection site erythema  0/3 (0.00%)  3/3 (100.00%)  3/3 (100.00%)  3/3 (100.00%)  2/3 (66.67%)  3/3 (100.00%)  0/3 (0.00%)  2/3 (66.67%)  0/3 (0.00%) 
Injection site edema  0/3 (0.00%)  0/3 (0.00%)  3/3 (100.00%)  2/3 (66.67%)  1/3 (33.33%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Injection site pain  0/3 (0.00%)  2/3 (66.67%)  1/3 (33.33%)  2/3 (66.67%)  1/3 (33.33%)  2/3 (66.67%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Performance status decreased  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Application site erythema  1/3 (33.33%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Influenza like illness  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Pyrexia  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  1/3 (33.33%)  1/3 (33.33%)  1/3 (33.33%)  0/3 (0.00%) 
Injection site discoloration  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Injection site pruritus  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Injection site urticaria  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Asthenia  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Fatigue  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Injection site induration  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Localized edema  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Edema peripheral  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Infections and infestations                   
Nasopharyngitis  1/3 (33.33%)  3/3 (100.00%)  0/3 (0.00%)  1/3 (33.33%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  3/3 (100.00%) 
Influenza  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  1/3 (33.33%)  1/3 (33.33%)  2/3 (66.67%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Pharyngitis  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Pneumonia  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%) 
Hookworm infection  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%) 
Upper respiratory tract infection  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Gastroenteritis  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Rhinitis  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Varicella  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Injury, poisoning and procedural complications                   
Procedural pain  0/3 (0.00%)  2/3 (66.67%)  2/3 (66.67%)  1/3 (33.33%)  1/3 (33.33%)  2/3 (66.67%)  1/3 (33.33%)  0/3 (0.00%)  3/3 (100.00%) 
Contusion  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  2/3 (66.67%)  2/3 (66.67%) 
Fall  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Post procedural edema  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Post-traumatic pain  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Investigations                   
Complement factor decreased  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
aPTT prolonged  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
INR increased  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Monocyte count decreased  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Neutrophil count increased  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
RBC urine positive  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Laboratory test abnormal (elevated MCP-1 levels)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  2/3 (66.67%)  1/3 (33.33%)  0/3 (0.00%) 
Complement factor C3 decreased  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Cystatin C increased  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Electrocardiogram T wave amplitude decreased  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%) 
GLDH increased  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
Urine protein/creatinine ratio increased  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%) 
WBC count increased  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Musculoskeletal and connective tissue disorders                   
Muscle spasms  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%) 
Back pain  2/3 (66.67%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Pain in extremity  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Arthralgia  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Nervous system disorders                   
Headache  1/3 (33.33%)  1/3 (33.33%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  1/3 (33.33%)  1/3 (33.33%)  0/3 (0.00%)  1/3 (33.33%) 
Dizziness  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Psychiatric disorders                   
Insomnia  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Renal and urinary disorders                   
Proteinuria  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  2/3 (66.67%)  2/3 (66.67%)  2/3 (66.67%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Dysuria  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Glycosuria  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Microalbuminuria  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Myoglobinuria  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Orthostatic proteinuria  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Reproductive system and breast disorders                   
Acquired hydrocele  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Respiratory, thoracic and mediastinal disorders                   
Epistaxis  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%) 
Oropharyngeal pain  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Cough  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  2/3 (66.67%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Dysphonia  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Skin and subcutaneous tissue disorders                   
Erythema  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  1/3 (33.33%)  1/3 (33.33%) 
Rash  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%) 
Skin swelling  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%) 
Panniculitis  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
Vascular disorders                   
Pallor  0/3 (0.00%)  0/3 (0.00%)  1/3 (33.33%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%)  0/3 (0.00%) 
[Not Specified]
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
Layout table for Results Point of Contact information
Name/Title: Fiona Lawrence
Organization: Prosensa
Phone: +31(0)713322100
EMail: f.lawrence@prosensa.nl
Layout table for additonal information
Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT01037309    
Other Study ID Numbers: PRO044-CLIN-01
First Submitted: December 21, 2009
First Posted: December 23, 2009
Results First Submitted: September 1, 2014
Results First Posted: April 9, 2015
Last Update Posted: October 16, 2018