A Long-term Continuation Study of Patients with Infantile-Onset Pompe Disease who were previously enrolled in Protocol AGLU01602

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2005-001629-27-DE

Enrollment

Registered

2005-06-09

Start date

2005-08-03

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

Pompe disease is a rare metabolic muscle disease inherited in an autosomal recessive fashion. Pompe disease is caused by a deficiency of GAA, which is needed for the degradation of lysosomal glycogen. Pompe disease is characterized by organelle bound (lysosomal) accumulation of glycogen in many body tissues. In general, there is an inverse correlation between the amount of residual GAA activity in patients with Pompe disease and the severity of the disease.

Interventions

Product Name: Myozyme Pharmaceutical Form: Powder for solution for infusion INN or Proposed INN: Alglucosidase alfa CAS Number: n.a. Current Sponsor code: rhGAA Other descriptive name: n.a. Concentr

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: (1) the patient’s legal guardian(s) must provide written informed consent prior to any study-related procedures being performed; (2) the patient and his/her legal guardian(s) must have the ability to comply with the clinical protocol; and (3) the patient must have completed Protocol AGLU01602. 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: A patient will be excluded from this study if he/she has experienced any unmanageable AE in Protocol AGLU01602 (as determined and agreed upon by the Principal Investigator and Genzyme Corporation) due to Myozyme that would preclude continuing recombinant human acid a-glucosidase (rhGAA) therapy.

Design outcomes

Primary

Measure	Time frame
Main Objective: The overall objective is to evaluate the long-term safety and efficacy of rhGAA treatment in patients with infantile-onset Pompe disease;Secondary Objective: ;Primary end point(s): The proportion of patients treated with Myozyme (20 mg/kg dose group, 40 mg/kg dose group, and both dose groups combined) who are alive and free of invasive ventilatory support after each 52-week Maintenance Phase Module and at the end of the study will be estimated using the Kaplan-Meier methodology.	—

Measure

Time frame

Main Objective: The overall objective is to evaluate the long-term safety and efficacy of rhGAA treatment in patients with infantile-onset Pompe disease;Secondary Objective: ;Primary end point(s): The proportion of patients treated with Myozyme (20 mg/kg dose group, 40 mg/kg dose group, and both dose groups combined) who are alive and free of invasive ventilatory support after each 52-week Maintenance Phase Module and at the end of the study will be estimated using the Kaplan-Meier methodology.

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Countries

Germany, Italy

Outcome results

None listed

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