Who is sponsoring NCT00486889?

NCT00486889 is sponsored by Genzyme, a Sanofi Company.

What condition does NCT00486889 study?

NCT00486889 studies Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease.

What drugs are being tested in NCT00486889?

Interventions: alglucosidase alfa.

What is the status of NCT00486889?

NCT00486889 is currently COMPLETED.

Last reviewed 2022-08-26 · How we verify

Growth and Development Study of Alglucosidase Alfa

NCT00486889 PHASE4 COMPLETED Results posted

Pompe disease (also known as glycogen storage disease Type II) is a rare autosomal recessive metabolic muscle disease caused by the deficiency of acid α glucosidase (GAA), an enzyme that degrades lysosomal glycogen. As opposed to the exclusively cytoplasmic accumulation of glycogen that occurs in other glycogen storage disorders, Pompe disease is characterized by organelle bound (lysosomal) and extra-lysosomal accumulation of glycogen in many body tissues, ultimately leading to multisystemic pathology. The overall objective of this study was to evaluate the long-term growth and development of participants with infantile-onset Pompe disease with alglucosidase alfa before 1 year of age. Participants were to be followed for a 10-year period.

Details

Lead sponsor	Genzyme, a Sanofi Company
Phase	PHASE4
Status	COMPLETED
Enrolment	12
Start date	Tue Aug 26 2008 00:00:00 GMT+0000 (Coordinated Universal Time)
Completion	Tue Nov 23 2021 00:00:00 GMT+0000 (Coordinated Universal Time)

Conditions

Pompe Disease
Glycogen Storage Disease Type II (GSD-II)
Acid Maltase Deficiency Disease

Interventions

alglucosidase alfa

Countries

United States