Growth and Development Study of Alglucosidase Alfa.
| Status: | Active, not recruiting |
|---|---|
| Conditions: | Diabetes |
| Therapuetic Areas: | Endocrinology |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 4/21/2016 |
| Start Date: | August 2008 |
| End Date: | January 2026 |
A Long-term Study to Evaluate Growth and Development Outcomes in Patients With Infantile-Onset Pompe Disease Who Are Receiving Alglucosidase Alfa.
Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of
a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by
the body's cells to break down glycogen (a stored form of sugar) within specialized
structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen
accumulates and is stored in various tissues, especially heart and skeletal muscle, which
prevents their normal function. The overall objective of this study is to evaluate the
long-term growth and development of patients with infantile-onset Pompe disease with
alglucosidase alfa before 1 year of age. Patients will be followed for 10-year period.
a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by
the body's cells to break down glycogen (a stored form of sugar) within specialized
structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen
accumulates and is stored in various tissues, especially heart and skeletal muscle, which
prevents their normal function. The overall objective of this study is to evaluate the
long-term growth and development of patients with infantile-onset Pompe disease with
alglucosidase alfa before 1 year of age. Patients will be followed for 10-year period.
Inclusion Criteria:
- The patient or 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.
Exclusion Criteria:
- The patient is participating in another clinical study using alglucosidase alfa or
any investigational therapy.
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