World Health Organization site
Skip Navigation Links

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: 19 March 2012
Main ID:  EUCTR2006-003644-31-DE
Date of registration: 11/07/2007
Prospective Registration: Yes
Primary sponsor: Genzyme Europe B.V.
Public title: An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704
Scientific title: An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704
Date of first enrolment: 03/08/2007
Target sample size: 82
Recruitment status: Not Recruiting
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2006-003644-31
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: no Randomised: Open: Single blind: Double blind: Parallel group: Cross over: Other: If controlled, specify comparator, Other Medicinial Product: Placebo: Other:  
Phase: 
Countries of recruitment
France Germany Netherlands
Contacts
Name:    
Address: 
Telephone:
Email:
Affiliation: 
Name:    
Address: 
Telephone:
Email:
Affiliation: 
Key inclusion & exclusion criteria
Inclusion criteria:
1. The patient must have completed Protocol AGLU02704, “A Randomized, Double-Blind, Multicenter, Multinational, Placebo-Controlled Study of the Safety, Efficacy, and Pharmacokinetics of Myozyme, Recombinant Human Acid alpha-Glucosidase (rhGAA), Treatment in Patients with Late-Onset Pompe Disease”, OR for patients who reside in a region where Myozyme is not available through government reimbursement or charitable access mechanisms, the patient must have completed a minimum of 52 weeks in Protocol AGLU02704;
2. The patient must provide signed, informed consent prior to performing any study-related procedures. Consent of a legally authorized guardian(s) is (are) required for patients under 18 years of age. If the patient is under 18 years of age and can understand the written informed consent, signature will be required from both the patient and the authorized guardian(s);
3. The patient (and patient’s legal guardian if patient is under 18 years of age) must have the ability to comply with the clinical protocol;
4. A female patient of childbearing potential must have a negative pregnancy test (urine beta-human chorionic gonadotropin [ß-hCG]) at Baseline. Note: All female patients of childbearing potential and sexually mature males must use a medically accepted method of contraception throughout the study.
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:
1. The patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that, in the opinion of the Investigator, would preclude treatment with Myozyme.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Glycogen Storage Disease type II (Pompe's disease)
MedDRA version: 9.1 Level: LLT Classification code 10036143 Term: Pompe's disease
Intervention(s)

Trade Name: Myozyme
Product Name: Myozyme
Product Code: rhGAA, acid alpha glucosidase
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: Alglucosidase alfa
CAS Number: n.a.
Current Sponsor code: rhGAA
Other descriptive name: alglucosidase alpha
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 5-

Primary Outcome(s)
Main Objective: The objective of this extension study is to assess the long-term safety and efficacy of Myozyme treatment in patients with Late-Onset Pompe Disease who were previously treated under the placebo-controlled, double-blind study AGLU02704.

The primary objectives of the study are: 1) to evaluate the safety profile of Myozyme; 2) to determine the effect of Myozyme treatment on functional endurance as measured by the Six Minute Walk Test (6MWT); and 3) to determine the effect of Myozyme treatment on respiratory muscle weakness as measured by Forced Vital Capacity (FVC) in the upright position.
Primary end point(s): See objective section (E2).
Secondary Objective: Secondary objectives are: 1) to determine the effect of Myozyme treatment on proximal muscle weakness in the lower limbs as measured by Quantitative Muscle Testing (QMT) in bilateral knee flexors (hamstrings) and knee extensors (quadriceps) and 2) to determine the effect of Myozyme treatment on health-related quality of life as measured by the Physical Component Summary (PCS) score of the Medical Outcomes Study (MOS) SF-36 Health Survey.
Secondary Outcome(s)
Secondary ID(s)
AGLU03206
2006-003644-31-FR
Source(s) of Monetary Support
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
Disclaimer: Trials posted on this search portal are not endorsed by WHO, but are provided as a service to our users. In no event shall the World Health Organization be liable for any damages arising from the use of the information linked to in this section. None of the information obtained through use of the search portal should in any way be used in clinical care without consulting a physician or licensed health professional. WHO is not responsible for the accuracy, completeness and/or use made of the content displayed for any trial record.
Copyright - World Health Organization - Version 3.6 - Version history