Trials / Terminated
TerminatedNCT01597596
A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
A Phase 3/4, Prospective, Multinational, Open-label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
- Status
- Terminated
- Phase
- Phase 4
- Study type
- Interventional
- Enrollment
- 4 (actual)
- Sponsor
- Genzyme, a Sanofi Company · Industry
- Sex
- All
- Age
- 12 Months
- Healthy volunteers
- Not accepted
Summary
A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa manufactured at the 160 litre (L) and 4000 L scales in participants who had been diagnosed with infantile-onset Pompe disease. Participants were treated with alglucosidase alfa 160 L scale product in the United States (US) and 4000 L scale product in the regions outside the US.
Conditions
- Pompe Disease (Infantile-Onset)
- Glycogen Storage Disease Type II (GSD II)
- Glycogenosis 2
- Acid Maltase Deficiency
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | alglucosidase alfa | Intravenous (IV) infusion of alglucosidase alfa (4000 L material) 20 mg/kg every other week (QOW) |
| BIOLOGICAL | alglucosidase alfa | IV infusion of alglucosidase alfa (160 L material) 20 mg/kg QOW. |
Timeline
- Start date
- 2012-08-01
- Primary completion
- 2014-12-01
- Completion
- 2014-12-01
- First posted
- 2012-05-14
- Last updated
- 2016-01-18
- Results posted
- 2016-01-18
Locations
17 sites across 3 countries: United States, Germany, Taiwan
Source: ClinicalTrials.gov record NCT01597596. Inclusion in this directory is not an endorsement.