Trials / Completed
CompletedNCT00486889
Growth and Development Study of Alglucosidase Alfa
A Long-term Study to Evaluate Growth and Development Outcomes in Patients With Infantile-Onset Pompe Disease Who Are Receiving Alglucosidase Alfa
- Status
- Completed
- Phase
- Phase 4
- Study type
- Interventional
- Enrollment
- 12 (actual)
- Sponsor
- Genzyme, a Sanofi Company · Industry
- Sex
- All
- Age
- 24 Months
- Healthy volunteers
- Not accepted
Summary
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | alglucosidase alfa | Dose: 20 mg/kg every 2 weeks; Route of administration: Intravenous infusion |
Timeline
- Start date
- 2008-08-26
- Primary completion
- 2021-11-23
- Completion
- 2021-11-23
- First posted
- 2007-06-15
- Last updated
- 2022-08-26
- Results posted
- 2022-08-26
Locations
3 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT00486889. Inclusion in this directory is not an endorsement.