Avalglucosidase Alfa Extension Study
- Conditions
- Pompe disease (acid alpha-glucosidase deficiency)MedDRA version: 20.0Level: LLTClassification code 10036143Term: Pompe's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 20.1Level: PTClassification code 10053185Term: Glycogen storage disease type IISystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- EUCTR2013-003321-28-BE
- Lead Sponsor
- Genzyme Corporation
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 21
Patients with Pompe disease who previously completed an avalglucosidase alfa study.
The patient and/or their parent/legal guardian is willing and able to provide signed informed consent, and the patient, if <18 years of age, is willing to provide assent if deemed able to do so.
The patient (and patient’s legal guardian if patient is <18 years of age) must have the ability to comply with the clinical protocol.
The patient, if female and of childbearing potential, must have a negative pregnancy test [urine beta-human chorionic gonadotropin] at baseline
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 17
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 4
The patient is concurrently participating in another clinical study using investigational treatment.
The patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study.
The patient has clinically significant organic disease (with the exception of symptoms relating to Pompe disease), including clinically significant cardiovascular, hepatic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, precludes participation in the study or potentially decreases survival.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: Long-term safety and pharmacokinetics (PK) of avalglucosidase alfa;Secondary Objective: Long-term effect of avalglucosidase alfa on pharmacodynamic and exploratory efficacy variables.;Primary end point(s): 1) Assessment of adverse events (AEs) and treatment-emergent adverse events (TEAEs), including infusion-associated reactions (IARs) and deaths<br>2) Laboratory assessments including hematology, biochemistry and urinalysis<br>3) Vital signs;Timepoint(s) of evaluation of this end point: 1) and 3) Screening/baseline until year 8<br>2) Monthly, from baseline until Year 3 then quaterly until Year 8<br>
- Secondary Outcome Measures
Name Time Method