Ignite Creation Date:
2024-05-05 @ 11:24 AM
Last Modification Date:
2024-10-26 @ 9:06 AM
Study NCT ID:
NCT00025896
Status:
COMPLETED
Last Update Posted:
2014-11-13
First Post:
2001-10-31
Brief Title:
Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease
Sponsor:
Genzyme a Sanofi Company
Organization:
Sanofi
Study Overview
Official Title:
A Prospective Multinational Multicenter Clinical Trial of the Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase rhGAA in Cross-Reacting Immunologic Material-Positive Patients With Classical Infantile Pompe Disease
Status:
COMPLETED
Status Verified Date:
2014-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Pompe disease is caused by a deficiency of a critical enzyme in the body called acid alpha glucosidase GAA Normally GAA is used by the bodys cells to break down glycogen a stored form of sugar within specialized structures called lysosomes In infants with severe cases of Pompe disease called Classical Infantile Pompe disease an excessive amount of glycogen accumulates and is stored in various tissues especially heart skeletal muscle and liver which prevents their normal function This study being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase rhGAA as a potential enzyme replacement therapy for Pompe disease Patients diagnosed with Classical Infantile Pompe disease who have a small but inactive amount of natural GAA enzyme present in their bodies called Cross-Reacting Immunologic Material-Positive or CRIM patients will be studied
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None