Late-Onset Treatment Study Extension Protocol
Information source: Sanofi
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Pompe Disease (Late-Onset); Glycogen Storage Disease Type II (GSD-II); Glycogenesis Type II; Acid Maltase Deficiency (AMD)
Intervention: alglucosidase alfa (Biological)
Phase: Phase 4
Status: Completed
Sponsored by: Genzyme, a Sanofi Company Official(s) and/or principal investigator(s): Medical Monitor, Study Director, Affiliation: Genzyme, a Sanofi Company
Summary
Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of
a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by
the body's cells to break down glycogen (a stored form of sugar) within specialized
structures called lysosomes. In patients with Pompe disease, an excessive amount of
glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle,
which prevents their normal function. The objective of this extension study is to assess
the long-term safety and efficacy of alglucosidase alfa treatment in patients with
Late-Onset Pompe Disease who were previously treated under the placebo-controlled,
double-blind study AGLU02704 (NCT00158600).
Clinical Details
Official title: An Open-Label Extension Study of Patients With Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704
Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600)Baseline Values (Week 0) of Functional Endurance as Measured by Six-Minute Walk Test (6MWT) For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) Change From Baseline (Week 0) in the Six-Minute Walk Test (6MWT) at Week 104 For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) Baseline Values (Week 0) for Percent Predicted Forced Vital Capacity (FVC) For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) Change From Baseline (Week 0) in the Percent Predicted Forced Vital Capacity (FVC) at Week 104 For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600)
Secondary outcome: Baseline Values (Week 0) for Percent Predicted Proximal Muscle Strength of the Lower Limbs as Measured by Quantitative Muscle Testing (QMT) For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600)Change From Baseline (Week 0) for Percent Predicted Proximal Muscle Strength of the Lower Limbs as Measured by Quantitative Muscle Testing (QMT) at Week 104 For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) Baseline Values (Week 0) for Quality of Life Related to Physical Component Score (PCS) as Measured by the Medical Outcomes Study (MOS) Short Form-36 Health Survey For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) Change From Baseline in Quality of Life Related to Physical Component Score (PCS) as Measured by the Medical Outcomes Study (MOS) Short Form-36 Health Survey For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600)
Eligibility
Minimum age: 8 Years.
Maximum age: N/A.
Gender(s): Both.
Criteria:
Inclusion Criteria:
- Patient must have completed protocol AGLU02704 (NCT00158600)
- Patient must provide signed, informed consent prior to performing any study-related
procedures
- Patient (and patient's legal guardian if patient is under 18 years of age) must have
the ability to comply with the clinical protocol
- A female patient of childbearing potential must have a negative pregnancy test at
Baseline. (note: all female patients of childbearing potential and sexually mature
males must use a medically accepted method of contraception throughout the study.)
Exclusion Criteria:
- The patient has a medical condition, serious intercurrent illness, or other
extenuating circumstance that, in the opinion of the Investigator, would preclude
treatment with alglucosidase alfa.
Locations and Contacts
Westmead, Australia
Paris, France
Rotterdam, Netherlands
Anchorage, Alaska, United States
Calgary, Alberta, Canada
Phoenix, Arizona, United States
Beverly Hills, California, United States
San Diego, California, United States
Washington, District of Columbia, United States
Coral Springs, Florida, United States
Gainesville, Florida, United States
Sarasota, Florida, United States
Chicago, Illinois, United States
Kansas City, Kansas, United States
New Orleans, Louisiana, United States
Boston, Massachusetts, United States
Omaha, Nebraska, United States
New York, New York, United States
Durham, North Carolina, United States
Cincinnati, Ohio, United States
Toledo, Ohio, United States
Tulsa, Oklahoma, United States
Portland, Oregon, United States
Philadelphia, Pennsylvania, United States
Pittsburgh, Pennsylvania, United States
Sherbrooke, Quebec, Canada
Greenville, South Carolina, United States
Dallas, Texas, United States
San Antonio, Texas, United States
Seattle, Washington, United States
Green Bay, Wisconsin, United States
Additional Information
Starting date: March 2007
Last updated: February 4, 2014
|