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Open Label, Extension 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: NCT02329769
Recruitment Status : Terminated
First Posted : January 1, 2015
Last Update Posted : December 8, 2017
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Brief Summary:
The purpose of this study is to see whether PRO044 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 44 in the DNA for the dystrophin protein.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: PRO044 SC 6 mg/kg Drug: PRO044 IV 6 mg/kg Drug: PRO044 IV 9 mg/kg Phase 2

Detailed Description:

A Phase II, open-label, extesion study. Following a Screening period of up to one month, subjects previously treated with PRO044, and eligible for enrolment in PRO044-CLIN-02, will be allocated to one of three groups to receive either 6 mg/kg or 9 mg/kg PRO044 weekly by IV infusion or 6 mg/kg weekly by SC injection for 48 weeks.

Safety and tolerability, pharmacokinetics (PK), pharmacodynamic (PD) and efficacy assessments will be conducted at regular intervals throughout the study.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 15 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II, Open Label, Extension Study to Assess the Effect of PRO044 in Patients With Duchenne Muscular Dystrophy
Actual Study Start Date : December 2014
Actual Primary Completion Date : July 1, 2016
Actual Study Completion Date : August 31, 2016

Arm Intervention/treatment
Experimental: PRO044 SC 6 mg/kg
Weekly subcutaneous (SC) dosing with 6 mg/kg
Drug: PRO044 SC 6 mg/kg
Experimental: PRO044 IV 6 mg/kg
Weekly intravenous (IV) dosing with 6 mg/kg
Drug: PRO044 IV 6 mg/kg
Experimental: PRO044 SC 9 mg/kg
Weekly intravenous (IV) dosing with 9 mg/kg
Drug: PRO044 IV 9 mg/kg

Primary Outcome Measures :
  1. Efficacy of PRO044 (composite of several measures) [ Time Frame: After 48 weeks of treatment ]

    Efficacy parameters:

    Muscle Function

    • 6 Minute Walk Distance (6MWD)
    • North Star Ambulatory Assessment
    • Timed tests (10-meter walk/run, rising from floor, stair climb)
    • DMD Functional Outcomes Questionnaire (DMD-FOS) -for ambulant subjects only
    • Egen Klassification - for non-ambulant subjects.

    Muscle strength

    • Pulmonary Function (Spirometry)
    • Handheld myometry.


    • Performance Upper Limb (PUL).
    • Patient Reported Outcome measure (PROM).

  2. Safety and tolerability of PRO044 (treatement emergent adverse events) [ Time Frame: After 48 weeks of treatment ]
    Number of subjects with 1 or more treatement emergent adverse events following SC or IV PRO044 dosing

Secondary Outcome Measures :
  1. Assess the pharmacokinetics of PRO044 (composite of several measures) [ Time Frame: After 48 weeks of treatment ]

    Pharmacokinetic parameters:

    • t ½
    • AUC: 0-24h, 0-∞ (where applicable)
    • Cmax
    • tmax
    • CL (for IV subjects) or CL/F (for SC subjects)
    • PRO044 concentrations in muscle tissue.

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Ages Eligible for Study:   9 Years to 20 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Subjects previously treated with PRO044.
  2. Continued use of glucocorticoids for a minimum of 60 days prior to study entry with a reasonable expectation that the subject will remain on steroids for the duration of the study. Changes to the dose regimen or cessation of glucocorticoids will be at the discretion of the Principle Investigator (PI) in consultation with the subject/parent and the Medical Monitor. If the subject is not on steroids, involvement in the study needs to be discussed with the medical monitor

Exclusion Criteria:

  1. Current, or history of, liver or renal disease.
  2. Acute illness within 4 weeks prior to the first dose of PRO044 (Week 1) which may interfere with the measurements.
  3. Severe cardiac myopathy which in the opinion of the Investigator prohibits participation in this study
  4. Need for daytime mechanical ventilation.
  5. Screening aPTT above the upper limit of normal (ULN).
  6. Screening platelet count below the lower limit of normal (LLN).
  7. Use of anticoagulants, antithrombotics or antiplatelet agents.
  8. Use of any investigational product within 6 months prior to the start of Screening for the study.
  9. Current or history of drug and/or alcohol abuse.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02329769

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UZ Leuven
Leuven, Belgium
S.Anna Hospital
Ferrara, Italy
Policlinico Universitario Agostino Gemelli
Roma, Italy
Leids Universitair Medisch Centrum
Leiden, Netherlands
Drottning Silvias Barn- ochungdomssjukhus
Goteborg, Sweden
Sponsors and Collaborators
BioMarin Pharmaceutical
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Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT02329769    
Other Study ID Numbers: PRO044-CLIN-02
First Posted: January 1, 2015    Key Record Dates
Last Update Posted: December 8, 2017
Last Verified: December 2017
Keywords provided by BioMarin Pharmaceutical:
Duchenne muscular dystrophy
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked