Pompe Disease Infantile-OnsetPhase 2NCT00763932
Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies
A Phase 2 study testing Myozyme in people with Pompe Disease Infantile-Onset and Glycogen Storage Disease Type II, run by Genzyme, a Sanofi Company at 8 sites in 3 countries.
Trial summary
This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in patients with infantile-onset Pompe disease who were previously treated with rhGAA derived from the Synpac cell line Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pompe Disease Infantile-Onset, Glycogen Storage Disease Type II
- What is being tested?
- Myozyme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 8 sitesin 3 countries
- Study dates
- Apr 2003to Jul 2006
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- The patient was enrolled in Protocol AGLU-008-01, AGLU-009-02, or AGLU01502
- The patient's legal guardian(s) provided written informed consent prior to any study related procedures being performed
- The patient and his/her guardian(s) were able to comply with the clinical protocol, which required extensive clinical evaluations for an extended period of time.
Who cannot join
- Patients were excluded from this study if they did not meet the specific inclusion criteria, or if the patient experienced any unmanageable AE in Protocol AGLU-008-01, AGLU-009-02, or AGLU01502 (as determined and agreed upon by the Principal Investigator and Genzyme Corporation), due to Synpac rhGAA therapy, that would preclude continuing therapy
Trial locations and contacts
8 sites in 3 countries.
8 sites in 3 countries
United States5 sites
Institute for Genetic Medicine, Saint Peter's University Hospital
New Brunswick, New Jersey
New York University [NYU] School of Medicine
New York, New York
Children's Hospital of Oakland
Oakland, California
University of Nebraska Medical Center
Omaha, Nebraska
Children's Hospital of Orange Country
Orange, California
France2 sites
Pediatrique Hospital Debrousse
Lyon
Hôpital Porte Madeleine
Orléans
About this study
Official title: A Multicenter, Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid α-Glucosidase (rhGAA) in Patients With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies
This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in patients with infantile-onset Pompe disease who were previously treated with rhGAA derived from the Synpac cell line
- Being tested
- Myozyme
- Medical subject headings
- Glycogen Storage Disease Type II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MyozymeBiological
Also called Alglucosidase alfa
10 mg/kg or 20 mg/kg qw OR 20 mg/kg or 40 mg/kg qow
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Not described Receives: Biological: Myozyme |
What is being measured
The primary outcome is the main question the study is designed to answer.
The objective of this extension study was to monitor the long-term safety and efficacy
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Oct 2008 (estimated)
- Study startApr 2003
- Primary completionJun 2006
- Study completionJul 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pompe Disease Infantile-OnsetStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Medical Monitor, Study director, Genzyme, a Sanofi Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00763932
- Sponsor's protocol ID
- AGLU02003
- Status last verified
- Feb 2014
- First submitted
- 30 Sep 2008
- Last updated
- 5 Feb 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid α-Glucosidase (rhGAA) in Patients With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies
- ClinicalTrials.gov ID
- NCT00763932
- Acronym / sponsor ID
- AGLU02003
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- Apr 2003
- Primary completion
- Jun 2006 (actual)
- Study completion
- Jul 2006 (actual)
- First posted
- 1 Oct 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in patients with infantile-onset Pompe disease who were previously treated with rhGAA derived from the Synpac cell line
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Biological: Myozyme |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- The patient was enrolled in Protocol AGLU-008-01, AGLU-009-02, or AGLU01502
- The patient's legal guardian(s) provided written informed consent prior to any study related procedures being performed
- The patient and his/her guardian(s) were able to comply with the clinical protocol, which required extensive clinical evaluations for an extended period of time.
Key exclusion criteria (1 listed)
- Patients were excluded from this study if they did not meet the specific inclusion criteria, or if the patient experienced any unmanageable AE in Protocol AGLU-008-01, AGLU-009-02, or AGLU01502 (as determined and agreed upon by the Principal Investigator and Genzyme Corporation), due to Synpac rhGAA therapy, that would preclude continuing therapy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MyozymeAlso: Alglucosidase alfa10 mg/kg or 20 mg/kg qw OR 20 mg/kg or 40 mg/kg qow | Biological | 1 |
Primary outcome
The objective of this extension study was to monitor the long-term safety and efficacy
Time frame: 3 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Feb 2014.
Related trials for Pompe Disease Infantile-Onset
Recruiting
Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
Rare Genetic Muscle Diseases, Muscular Dystrophy, Duchenne
Active, not recruitingObservational study
A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)
Glycogen Storage Disease Type II
RecruitingObservational study
Registry of Patients Diagnosed With Lysosomal Storage Diseases
Mucopolysaccharidosis I, Mucopolysaccharidosis II
Active, not recruitingPhase 3
Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa
Glycogen Storage Disease Type II
RecruitingPhase 4
China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
Glycogen Storage Disease Type II, Pompe's Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Monitor study director
Genzyme, a Sanofi Company
8 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Myozyme in people with Pompe Disease Infantile-Onset and Glycogen Storage Disease Type II, run by Genzyme, a Sanofi Company at 8 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Feb 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States, France, South Africa. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The objective of this extension study was to monitor the long-term safety and efficacy (time frame: 3 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Feb 2014.