Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
NCT ID: NCT02329769
Last Updated: 2017-12-08
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
TERMINATED
PHASE2
15 participants
INTERVENTIONAL
2014-12-31
2016-08-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Safety and tolerability, pharmacokinetics (PK), pharmacodynamic (PD) and efficacy assessments will be conducted at regular intervals throughout the study.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Keywords
Explore important study keywords that can help with search, categorization, and topic discovery.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
PRO044 SC 6 mg/kg
Weekly subcutaneous (SC) dosing with 6 mg/kg
PRO044 SC 6 mg/kg
PRO044 IV 6 mg/kg
Weekly intravenous (IV) dosing with 6 mg/kg
PRO044 IV 6 mg/kg
PRO044 SC 9 mg/kg
Weekly intravenous (IV) dosing with 9 mg/kg
PRO044 IV 9 mg/kg
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
PRO044 SC 6 mg/kg
PRO044 IV 6 mg/kg
PRO044 IV 9 mg/kg
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
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
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.
9 Years
20 Years
MALE
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
BioMarin Pharmaceutical
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
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
Gothenburg, , Sweden
Countries
Review the countries where the study has at least one active or historical site.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
PRO044-CLIN-02
Identifier Type: -
Identifier Source: org_study_id