Pompe Disease (Infantile-Onset)Phase 4NCT01597596
A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
A Phase 4 study testing alglucosidase alfa in people with Pompe Disease (Infantile-Onset) and Glycogen Storage Disease Type II (GSD II), run by Genzyme, a Sanofi Company at 17 sites in 3 countries.
Trial summary
A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa manufactured at the 160 litre (L) and 4000 L scales in participants who had been diagnosed with infantile-onset Pompe disease. Read more about this study
- Who is it for?
- Ages Up to 12 months
- Conditions the study is for
- Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenosis 2
- What is being tested?
- alglucosidase alfa
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 17 sitesin 3 countries
- Study dates
- Aug 2012to Dec 2014
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 Up to 12 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- The participant's parent/legal guardian was willing and able to provide signed informed consent.
- The participant might be less than or equal to 12 months of age.
- The participant might have documented GAA enzyme deficiency from blood, skin, or muscle tissue.
- The participant might be naïve to treatment with alglucosidase alfa.
Who cannot join
- The participant was cross-reactive immunologic material negative.
- The participant required invasive ventilator support at the time of enrollment.
- The participant had decompensated clinical heart failure.
- The participant had a major congenital abnormality, excluding cardiac hypertrophy.
- The participant had a clinically significant organ disease (excluding the signs and symptoms of Pompe disease).
- The participant was currently receiving any investigational product.
- The participant was participating in another clinical study.
- The participant and/or the patient's parent/legal guardian was unable to adhere to the requirements of the study.
Trial locations and contacts
17 sites in 3 countries.
17 sites in 3 countries
United States14 sites
Study site
Cambridge, Massachusetts
Study site
Chicago, Illinois
Study site
Cincinnati, Ohio
Study site
Decatur, Georgia
Study site
Detroit, Michigan
Study site
Durham, North Carolina
Study site
Fort Worth, Texas
Study site
Gainsville, Florida
Study site
Las Vegas, Nevada
Study site
Little Rock, Arkansas
Study site
New Brunswick, New Jersey
Study site
New York, New York
Study site
Oakland, California
Study site
Seattle, Washington
About this study
Official title: A Phase 3/4, Prospective, Multinational, Open-label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa manufactured at the 160 litre (L) and 4000 L scales in participants who had been diagnosed with infantile-onset Pompe disease. Participants were treated with alglucosidase alfa 160 L scale product in the United States (US) and 4000 L scale product in the regions outside the US.
- Conditions
- Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenosis 2, Acid Maltase Deficiency
- Being tested
- alglucosidase alfa
- 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.
alglucosidase alfaBiological
Also called Lumizyme
Intravenous (IV) infusion of alglucosidase alfa (4000 L material) 20 mg/kg every other week (QOW)
alglucosidase alfaBiological
Also called Myozyme
IV infusion of alglucosidase alfa (160 L material) 20 mg/kg QOW.
| Study group | Role | What they receive |
|---|---|---|
| Alglucosidase Alfa 4000 L material (Non-US participants) | Experimental | Alglucosidase alfa 4000 L material for 52 weeks. Receives: Biological: alglucosidase alfa |
| Alglucosidase Alfa 160 L material (US participants) | Active comparator | Alglucosidase alfa 160 L material for 52 weeks. Receives: Biological: alglucosidase alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Cardiac Function at Week 52
Cardiac function was measured by the left ventricular mass Z-score (LVM-Z). Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative change from baseline indicates a decrease and positive change from baseline indicates an increase in LVM Z-score. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy.
Measured: Baseline, Week 52
Secondary outcomes (3)
Percentage of Participants With Estimated Probability of Survival
Measured: Up to Week 52
Number of Participants With Invasive Ventilator-Free Survival
Invasive ventilator-free survival was defined as the time during which the participant is alive and not invasively ventilated. Number of Participants with invasive ventilator-free survival were reported.
Measured: Up to Week 52
Change From Baseline in Motor Development Status at Week 52
Motor development status was assessed by the Gross Motor Function Measure - 88 Scale (GMFM-88) total percent scores. GMFM-88 is an 88-item measure to detect gross motor function. It consists of 5 categories: lying and rolling; sitting; crawling and kneeling; standing; walking, running and jumping. Each item was scored on a 4-point Likert scale (0 = cannot do; 1 = initiates [<10% of the task]; 2 = partially completes [10% to <100% of the task]; 3 = task completion). The score for each dimension was expressed as a percentage of the maximum score for that dimension. Total score ranges from 0% to 100%, where higher scores indicate better motor functions.
Measured: Baseline, Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2012 (estimated)
- Study startAug 2012
- Primary completionDec 2014
- Study completionDec 2014
Why it stopped: Study was stopped due to approved label expansion of alglucosidase alfa.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Pompe Disease (Infantile-Onset)Studies 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 4
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Genzyme, a Sanofi Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01597596
- Sponsor's protocol ID
- AGLU07510
- Other IDs
- 2011-005595-42
- Status last verified
- Dec 2015
- First submitted
- 10 May 2012
- Last updated
- 18 Jan 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 18 Jan 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3/4, Prospective, Multinational, Open-label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
- ClinicalTrials.gov ID
- NCT01597596
- Acronym / sponsor ID
- AGLU07510
- Conditions
- Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenosis 2, Acid Maltase Deficiency
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study was stopped due to approved label expansion of alglucosidase alfa.
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- Aug 2012
- Primary completion
- Dec 2014 (actual)
- Study completion
- Dec 2014 (actual)
- First posted
- 14 May 2012 (estimated)
- Results
- Posted 18 Jan 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa manufactured at the 160 litre (L) and 4000 L scales in participants who had been diagnosed with infantile-onset Pompe disease. Participants were treated with alglucosidase alfa 160 L scale product in the United States (US) and 4000 L scale product in the regions outside the US.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Alglucosidase Alfa 4000 L material (Non-US participants) | Experimental | Biological: alglucosidase alfaAlglucosidase alfa 4000 L material for 52 weeks. |
| Alglucosidase Alfa 160 L material (US participants) | Active comparator | Biological: alglucosidase alfaAlglucosidase alfa 160 L material for 52 weeks. |
Who may qualify
- Ages
- Up to 12 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- The participant's parent/legal guardian was willing and able to provide signed informed consent.
- The participant might be less than or equal to 12 months of age.
- The participant might have documented GAA enzyme deficiency from blood, skin, or muscle tissue.
- The participant might be naïve to treatment with alglucosidase alfa.
Key exclusion criteria (8 listed)
- The participant was cross-reactive immunologic material negative.
- The participant required invasive ventilator support at the time of enrollment.
- The participant had decompensated clinical heart failure.
- The participant had a major congenital abnormality, excluding cardiac hypertrophy.
- The participant had a clinically significant organ disease (excluding the signs and symptoms of Pompe disease).
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 |
|---|---|---|
| alglucosidase alfaAlso: LumizymeIntravenous (IV) infusion of alglucosidase alfa (4000 L material) 20 mg/kg every other week (QOW) | Biological | Alglucosidase Alfa 4000 L material (Non-US participants) |
| alglucosidase alfaAlso: MyozymeIV infusion of alglucosidase alfa (160 L material) 20 mg/kg QOW. | Biological | Alglucosidase Alfa 160 L material (US participants) |
Primary outcome
Change From Baseline in Cardiac Function at Week 52
Time frame: Baseline, Week 52
Cardiac function was measured by the left ventricular mass Z-score (LVM-Z). Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative change from baseline indicates a decrease and positive change from baseline indicates an increase in LVM Z-score. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy.
3 secondary outcomes
Percentage of Participants With Estimated Probability of Survival
Time frame: Up to Week 52
Number of Participants With Invasive Ventilator-Free Survival
Time frame: Up to Week 52
Change From Baseline in Motor Development Status at Week 52
Time frame: Baseline, Week 52
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jan 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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
17 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing alglucosidase alfa in people with Pompe Disease (Infantile-Onset) and Glycogen Storage Disease Type II (GSD II), run by Genzyme, a Sanofi Company at 17 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 4 inclusion and 8 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: stopped early, as of the sponsor's last update on 18 Jan 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in United States, Germany, Taiwan. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 18 Jan 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Change From Baseline in Cardiac Function at Week 52 (time frame: Baseline, Week 52). 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 18 Jan 2016.