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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
BioMarin Nederland BV
ClinicalTrials.gov Identifier:
NCT01037309
First received: December 21, 2009
Last updated: March 26, 2015
Last verified: March 2015
Results First Received: September 1, 2014  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: PRO044 SC
Drug: PRO044 IV

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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

Reporting Groups
  Description
Subcutaneous PRO044 0.5 mg/kg

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 PRO044 1.5 mg/kg

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 PRO044 5 mg/kg

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 PRO044 8 mg/kg

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 PRO044 10 mg/kg

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 PRO044 12 mg/kg

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 PRO044 1.5 mg/kg

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 PRO044 5 mg/kg

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 PRO044 8 mg/kg

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


Participant Flow for 2 periods

Period 1:   Subcutaneous Cohort 1 - 6
    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  
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 2:   Intravenous Period 7 - 9
    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  
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  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
PRO044, Cohort 1

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

PRO044, Cohort 2

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

PRO044, Cohort 3

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

PRO044, Cohort 4

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

PRO044, Cohort 5

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

PRO044, Cohort 6

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 Total of all reporting groups

Baseline Measures
    PRO044, Cohort 1     PRO044, Cohort 2     PRO044, Cohort 3     PRO044, Cohort 4     PRO044, Cohort 5     PRO044, Cohort 6     Total  
Number of Participants  
[units: participants]
  3     3     3     3     3     3     18  
Age  
[units: participants]
             
<=18 years     3     3     3     3     3     3     18  
Between 18 and 65 years     0     0     0     0     0     0     0  
>=65 years     0     0     0     0     0     0     0  
Gender  
[units: participants]
             
Female     0     0     0     0     0     0     0  
Male     3     3     3     3     3     3     18  
Region of Enrollment  
[units: 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  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Increase in Dystrophin Expression in the Muscle Biopsies by Immunofluorescence Analyses of Cross-sections and by Western Blot Analyses of Total Protein Extracts   [ Time Frame: Within 13 weeks after 5 weeks of treatment ]

2.  Primary:   Safety and Tolerability of PRO044   [ Time Frame: During the 5 weeks of treatment and during the 13 weeks after treatment ]

3.  Primary:   Determine the Pharmacokinetics of PRO044   [ Time Frame: During the 5 weeks of treatment and during the 13 weeks after treatment ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information
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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.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Fiona Lawrence
Organization: Prosensa
phone: +31(0)713322100
e-mail: f.lawrence@prosensa.nl


No publications provided


Responsible Party: BioMarin Nederland BV
ClinicalTrials.gov Identifier: NCT01037309     History of Changes
Other Study ID Numbers: PRO044-CLIN-01
Study First Received: December 21, 2009
Results First Received: September 1, 2014
Last Updated: March 26, 2015
Health Authority: United States: Food and Drug Administration
Belgium: Federal Agency for Medicinal Products and Health Products
Italy: National Institute of Health
Netherlands: Ministry of Health, Welfare and Sport
Sweden: Medical Products Agency