Clinical trial NCT00455195: Late-Onset Treatment Study Extension Protocol
Official title: An Open-Label Extension Study of Patients With Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Pompe Disease (Late-Onset)
Glycogen Storage Disease Type II (GSD-II)
Glycogenesis Type II
Acid Maltase Deficiency (AMD)
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2007-04-03
Enrollment: 81
Sex: ALL
Minimum age (years): 8
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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).


Eligibility 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.

Primary outcomes:
measure: Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600)
timeFrame: Week 0 to 2.5 years
measure: 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)
timeFrame: Week 0
measure: 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)
timeFrame: Week 0, Week 104
measure: Baseline Values (Week 0) for Percent Predicted Forced Vital Capacity (FVC) For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600)
timeFrame: Week 0
measure: 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)
timeFrame: Week 0, Week 104
Secondary outcomes:
measure: 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)
timeFrame: Week 0
measure: 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)
timeFrame: Week 0, Week 104
measure: 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)
timeFrame: Week 0
measure: 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)
timeFrame: Week 0 , Week 104
Other outcomes:
Locations:
city: Anchorage
country: United States
state: Alaska
city: Phoenix
country: United States
state: Arizona
city: Beverly Hills
country: United States
state: California
city: San Diego
country: United States
state: California
city: Washington
country: United States
state: District of Columbia
city: Coral Springs
country: United States
state: Florida
city: Gainesville
country: United States
state: Florida
city: Sarasota
country: United States
state: Florida
city: Chicago
country: United States
state: Illinois
city: Kansas City
country: United States
state: Kansas
city: New Orleans
country: United States
state: Louisiana
city: Boston
country: United States
state: Massachusetts
city: Omaha
country: United States
state: Nebraska
city: New York
country: United States
state: New York
city: Durham
country: United States
state: North Carolina
city: Cincinnati
country: United States
state: Ohio
city: Toledo
country: United States
state: Ohio
city: Tulsa
country: United States
state: Oklahoma
city: Portland
country: United States
state: Oregon
city: Philadelphia
country: United States
state: Pennsylvania
city: Pittsburgh
country: United States
state: Pennsylvania
city: Greenville
country: United States
state: South Carolina
city: Dallas
country: United States
state: Texas
city: San Antonio
country: United States
state: Texas
city: Seattle
country: United States
state: Washington
city: Green Bay
country: United States
state: Wisconsin
city: Westmead
country: Australia
city: Calgary
country: Canada
state: Alberta
city: Sherbrooke
country: Canada
state: Quebec
city: Paris
country: France
city: Rotterdam
country: Netherlands
Study officials:
affiliation: Genzyme, a Sanofi Company
name: Medical Monitor
role: STUDY_DIRECTOR
References: