Phase 4
N=90
Late-Onset Treatment Study Extension Protocol
Pompe Disease (Late-Onset) · Glycogen Storage Disease Type II (GSD-II) · Glycogenesis Type II · Acid Maltase Deficiency (AMD)
Bottom Line
View on ClinicalTrials.gov: NCT00455195 ↗Enrolled (actual)
90
Serious AEs
19.8%
Results posted
Jan 2012
Primary outcome: Primary: Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) — 60; 37; 21; 15 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- alglucosidase alfa (Biological)
- Age
- Pediatric, Adult, Older Adult · 8+ yrs
- Sex
- All
- Sponsor
- Genzyme, a Sanofi Company
- Primary completion
- Oct 2008
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) |
60; 37; 21; 15; 16; 3 | — |
| PRIMARY 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) |
332.2 | — |
| PRIMARY 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) |
21.3 | — |
| PRIMARY Baseline Values (Week 0) for Percent Predicted Forced Vital Capacity (FVC) For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) |
55.4 | — |
| PRIMARY 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) |
0.8 | — |
| SECONDARY 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) |
37.7 | — |
| SECONDARY 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) |
2.1 | — |
| SECONDARY 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) |
34.33 | — |
| SECONDARY 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) |
0.64 | — |
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.
Data sourced from ClinicalTrials.gov (NCT00455195). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.