An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704

• 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:

Countries of recruitment

France, Germany, Netherlands

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 ID(s)

2006-003644-31-FR

AGLU03206