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.
ID
Source
Brief title
Condition
- Inborn errors of metabolism
- Muscle disorders
- Neuromuscular disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
1. Change from Treatment Baseline to End of Study time point in 6MWT - meters
walked
2. Change from Treatment Baseline to End of Study time point in % predicted FVC
in the upright position
Secondary outcome
1. Change from Treatment Baseline to End of Study time point in QMT Leg Score
for % predicted bilateral knee flexors and knee extensors
2. Change from Treatment Baseline to End of Study time point in PCS score of
the MOS SF-36 Health Survey
Background summary
The patients are currently particpating in a placebo controlled study
(AGLU02704) which has recently been extended to 78 weeks of treatment. After
completion of this study, patients will be asked to take part in an open-label
extension study (AGLU03206).
Study 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.
Study design
Open-label treatment with Myozyme.
Intervention
Intravenous infusion with Myozyme (20 mg/kg) qow.
Study burden and risks
Depending on of the type of visit: Day 0 one day; Week 12, Week 26 two days;
all other visits last approximately one day.
During the study the following non-invasive assessments will be conducted:
questionnaires (SF-36 Health survey, Fatigue Severity Scale, Rotterdam 9-Item
handicap Scale); ventilator use diary (only for patients who use a ventilator);
pulmonary function testing; quantitative and manual muscle testing; functional
activities testing; 6 minutes walk test; physical examination; height and
weight; blood pressure, pulse, respiratory rate and temperature; hearing test;
12-lead ECG; urinalysis; echo; telephone contact.
During the study the following invasive assessments will be conducted:
intravenous canula (IV injection) will be used for infusion of Myozyme every
two weeks for a total of 14 times. The infusion volume is dependent on the
weight of the patient. The intravenous canula can be used for blood sample
collection to assess chemistry and hematology (7 ml each time) and anti-Myozyme
antibodies (IgG: 10 ml each time). During the whole study approximately 68 ml
of blood will be drawn. This amount does not include any blood samples drawn
because of medical necessity.
Recently Myozyme received regulatory approval in Europe (29th of March 2006)
and in the United States (28th of April 2006) for the long-term treatment of
patients with Pompe disease. However, the authorities in Europe (EMEA) and the
United States of America (FDA) are requiring Genzyme to complete this extended
study to expand the current knowledge on Myozyme and its effectiveness and
safety for treating Late Onset Pompe patients. With the successful completion
of this study we hope to collect more data on the long-term effects of Myozyme
which support the efficacy and safety in patients with Late Onset Pompe
disease. This information will be submitted to the regulatory authorities in
Europe and the United States of America. Based upon the results in patients
with the infantile-onset of Pompe disease, an improvement in pulmonary function
and muscle strenght can be expected in patients with Late Onset Pompe disease.
We anticipate that the results of the AGLU02704 study will confirm this
hypothesis.
Treatment with Myozyme can cause side-effects. Up to now, infusions with
Myozyme have been well tolerated and the most commonly reported side-effects on
the day of the infusion were hypersensitivityreactions with the following
symptoms: headache, redness in the face, fever, rash, urticaria, change in
blood pressure and increased heart rate.
Other side-effects which are currently unknown can occur. The treatment could
include risks which can currently not be predicted.
Gooimeer 10
1411 DD Naarden
NL
Gooimeer 10
1411 DD Naarden
NL
Listed location countries
Age
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 [β-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.
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.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2006-003644-31-NL |
| CCMO | NL15351.078.07 |