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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

Page last updated: August 23, 2015

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