EUCTR2021-005552-11-Outside-EU/EEA
Active, not recruiting
Phase 1
A Long-term Study to Evaluate Growth and Development Outcomes in Patients With Infantile-Onset Pompe Disease Who Are Receiving Alglucosidase Alfa.
ConditionsPompe diseaseMedDRA version: 20.1Level: PTClassification code 10053185Term: Glycogen storage disease type IISystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
DrugsMyozyme
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Pompe disease
- Sponsor
- Genzyme Corporation
- Status
- Active, not recruiting
- Last Updated
- 4 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •The patient's legal guardian must provide signed, informed consent prior to performing any study\-related procedures;
- •The patient must have a confirmed diagnosis of Pompe disease as determined by deficient endogenous acid alpha\-glucosidase (GAA) activity or GAA mutation analysis; and
- •The patient must be \<1 year of age at time of study enrollment (and receive alglucosidase alfa treatment before 1 year of age), or the patient must be between 1 year and 24 months of age and must have initiated alglucosidase alfa treatment prior to turning 1 year of age.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 15
- •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
- •The patient is participating in another clinical study using alglucosidase alfa or any investigational therapy.
Outcomes
Primary Outcomes
Not specified
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