Effects and health economic aspects of enzyme therapy in children and adults with Pompe disease Long-term follow-up of patients receiving commercially available Myozyme - not applicable

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2007-001375-11-NL
• Date of registration: 24/05/2007
• Prospective Registration: No
• Public title: Effects and health economic aspects of enzyme therapy in children and adults with Pompe disease Long-term follow-up of patients receiving commercially available Myozyme - not applicable
• Scientific title: Effects and health economic aspects of enzyme therapy in children and adults with Pompe disease Long-term follow-up of patients receiving commercially available Myozyme - not applicable
• Date of first enrolment: 16/05/2007
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2007-001375-11
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

Netherlands

Key inclusion & exclusion criteria

Inclusion criteria:
1. Patients should have a proven diagnosis of Pompe disease, which means that the diagnosis is confirmed by deficiency of a-glucosidase activity in leukocytes or skeletal muscle, or by a documented mutation in the a-glucosidase gene.
2. The patient must have symptoms of Pompe disease being: Signs of skeletal muscle weakness, or decreased pulmonary function (FVC < 80% in sitting position), or Cardiac hypertrophy

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
Patients who are not likely to benefit from treatment


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Pompe disease (glycogen storage disease type II) is a genetic, lysosomal storage disorder with a frequency of 1 in 40.000 newborns. The disease is caused by deficiency of alpha-glucosidase, a lysosomal hydrolase involved in the degradation of glycogen.

Intervention(s)

Trade Name: Myozyme
Product Name: Myozyme
Product Code: MYOZYME®
Pharmaceutical Form: Intravenous infusion

Primary Outcome(s)

Main Objective: The objective of the study is to gather more information about the long-term effect of enzyme therapy in patients with Pompe disease with a different severity of disease. The goal is also to set guideline for start and stop of treatment and to evaluate health economic aspects. Furthermore, it is important to find an optimal dosing regimen.

Secondary Objective: not applicable

Primary end point(s): - Survival
- Muscle strength and function
- Motor and mental outcome
- Pulmonary function
- Cardiac hypertrophy and function
- Hearing loss
- Disease specific symptoms, handicap, quality of life and fatigue
- Muscle mass and regeneration
- Costs

Secondary ID(s)

not applicable