|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
EUCTR |
|
Last refreshed on:
|
24 February 2025 |
|
Main ID: |
EUCTR2007-001375-11-NL |
|
Date of registration:
|
24/05/2007 |
|
Prospective Registration:
|
No |
|
Primary sponsor: |
|
|
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 |
|
Target sample size:
|
100 |
|
Recruitment status: |
Not Recruiting |
|
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
|
|
Phase:
|
Human pharmacology (Phase I): no
Therapeutic exploratory (Phase II): no
Therapeutic confirmatory - (Phase III): no
Therapeutic use (Phase IV): yes
|
|
|
Countries of recruitment
|
|
Netherlands
| | | | | | | |
|
Contacts
|
|
Name:
|
|
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
|
|
|
Name:
|
|
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
|
| |
|
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.
|
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 Objective: not applicable
|
|
Secondary ID(s)
|
|
not applicable
|
|
not applicable
|
|
Source(s) of Monetary Support
|
|
Ethics review
|
Status: Approved
Approval date: 16/05/2007
Contact:
|
|
Results
|
|
Results available:
|
|
|
Date Posted:
|
|
|
Date Completed:
|
|
|
URL:
|
|
|
|