Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
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|ClinicalTrials.gov Identifier: NCT02329769|
Recruitment Status : Terminated
First Posted : January 1, 2015
Last Update Posted : December 8, 2017
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|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|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||15 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|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|
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
- Efficacy of PRO044 (composite of several measures) [ Time Frame: After 48 weeks of treatment ]
- 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.
- Pulmonary Function (Spirometry)
- Handheld myometry.
- Performance Upper Limb (PUL).
- Patient Reported Outcome measure (PROM).
- 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
- Assess the pharmacokinetics of PRO044 (composite of several measures) [ Time Frame: After 48 weeks of treatment ]
- t ½
- AUC: 0-24h, 0-∞ (where applicable)
- 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|
- Subjects previously treated with PRO044.
- 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
- Current, or history of, liver or renal disease.
- Acute illness within 4 weeks prior to the first dose of PRO044 (Week 1) which may interfere with the measurements.
- Severe cardiac myopathy which in the opinion of the Investigator prohibits participation in this study
- Need for daytime mechanical ventilation.
- Screening aPTT above the upper limit of normal (ULN).
- Screening platelet count below the lower limit of normal (LLN).
- Use of anticoagulants, antithrombotics or antiplatelet agents.
- Use of any investigational product within 6 months prior to the start of Screening for the study.
- Current or history of drug and/or alcohol abuse.
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
|Policlinico Universitario Agostino Gemelli|
|Leids Universitair Medisch Centrum|
|Drottning Silvias Barn- ochungdomssjukhus|
|Responsible Party:||BioMarin Pharmaceutical|
|Other Study ID Numbers:||
|First Posted:||January 1, 2015 Key Record Dates|
|Last Update Posted:||December 8, 2017|
|Last Verified:||December 2017|
Duchenne muscular dystrophy
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked