An Open-Label, Multicenter, Multinational Extension Study Of The Long-Term Safety And Pharmacokinetics Of Repeated Biweekly Infusions Of Avalglucosidase Alfa In Patients With Pompe Disease

Who is this study for? Patients with Pompe disease
What treatments are being studied? GZ402666
Status: Active, not recruiting
Location: See all (17) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2/Phase 3
SUMMARY

Objective: Long-term safety and pharmacokinetics (PK) of avalglucosidase alfa Secondary Objective: Long-term effect of avalglucosidase alfa on pharmacodynamic and exploratory efficacy variables

Eligibility
Participation Requirements
Sex: All
Healthy Volunteers: No
View:

• Patients with Pompe disease who previously completed a an avalglucosidase 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.

Locations
United States
Arizona
Investigational Site Number 840006
Phoenix
Florida
Investigational Site Number 840010
Jacksonville
Kansas
Investigational Site Number 840001
Kansas City
Missouri
Investigational Site Number 840008
Saint Louis
North Carolina
Investigational Site Number 840002
Durham
Ohio
Investigational Site Number 840011
Cincinnati
Texas
Investigational Site Number 840009
Dallas
Virginia
Investigational Site Number 840003
Fairfax
Other Locations
Belgium
Investigational Site Number 056001
Leuven
Denmark
Investigational Site Number 208001
København Ø
France
Investigational Site Number 250003
Nice
Investigational Site Number 250002
Paris
Germany
Investigational Site Number 276003
Mainz
Investigational Site Number 276001
München
Investigational Site Number 276002
Münster
Netherlands
Investigational Site Number 528001
Rotterdam
United Kingdom
Investigational Site Number 826003
Newcastle Upon Tyne
Time Frame
Start Date: February 27, 2014
Estimated Completion Date: November 10, 2022
Participants
Target number of participants: 19
Treatments
Experimental: Avalglucosidase alfa
administered intravenously every 2 weeks
Related Therapeutic Areas
Sponsors
Leads: Genzyme, a Sanofi Company

This content was sourced from clinicaltrials.gov

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