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An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid alpha- Glucosidase (rhGAA) Treatment in Patients > 6 and <= 36 Months Old with Infantile-Onset Pompe Disease (Glycogen Storage Disease Type II)

An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid alpha- Glucosidase (rhGAA) Treatment in Patients > 6 and <= 36 Months Old with Infantile-Onset Pompe Disease (Glycogen Storage Disease Type II)

Status
Active, not recruiting
Phases
Unknown
Study type
Interventional
Source
EU CTR
Registry ID
EUCTR2004-002168-59-IT
Enrollment
20
Registered
2005-01-03
Start date
2004-10-14
Completion date
Unknown
Last updated
2012-03-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Treatment for Type II Glycogenosis MedDRA version: 6.1 Level: PT Classification code 10053185

Interventions

Product Name: Recombinant human acid alfa-glucosidase (rhGAA) Product Code: NA Pharmaceutical Form: Powder for infusion* INN or Proposed INN: alglucosidase alfa CAS Number: 420784-05-0 Current Sponsor

Sponsors

GENZYME
Lead Sponsor

Eligibility

Sex/Gender
All

Inclusion criteria

Inclusion criteria: Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria:

Design outcomes

Primary

MeasureTime frame
Main Objective: ;Secondary Objective: ;Primary end point(s):—

Countries

Italy

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026