Pompe DiseaseNCT01710813
Alglucosidase Alfa Pompe Safety Sub-Registry
An observational study of Pompe Disease, run by Genzyme, a Sanofi Company at 18 sites in 6 countries.
Trial summary
To collect uniform and meaningful data on patients with Pompe disease who experience anaphylaxis, severe allergic reactions, and/or signals of severe cutaneous and/or systemic immune complex-mediated reactions following treatment with alglucosidase alfa. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Pompe Disease
- What is being tested?
- alglucosidase alfa
- Type of treatment
- Injection or infusion
- How many people
- 110 participants
- Where
- 18 sitesin 6 countries
- Study dates
- 20 Mar 2015to 20 Apr 2021
- How long will I take part?
- 4 Years
- Phase
- Observational
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients who are enrolled in the Pompe Registry will be enrolled in this sub-registry include patients with infantile-onset Pompe disease, as well as those with late-onset Pompe disease. An approximately equal proportion of currently treated and treatment-naïve patients is targeted for enrollment at each site.
Who may be able to join
- The patient must be enrolled in the Pompe Registry;
- Provide a signed patient information and authorization form;
- Have a confirmed diagnosis of Pompe disease (confirmation of diagnosis is defined as documented GAA enzyme deficiency from any tissue source and/or documentation of 2 GAA gene mutations);
- Be naïve to and plan to be treated with alglucosidase alfa at or prior to enrollment, or are being treated with alglucosidase alfa.
Who cannot join
- Patients will be excluded if they have received an investigational drug (excluding alglucosidase alfa) within 30 days prior to signing a Safety Sub-Registry Patient Information and Authorization form, or if they are taking or plan to take any investigational product while enrolled in the Safety Sub-Registry.
Trial locations and contacts
18 sites in 6 countries.
18 sites in 6 countries
United States6 sites
Investigational Site Number 840002
Durham, North Carolina, 27710
Investigational Site Number 840001
Fairfax, Virginia, 22030
Investigational Site Number 840004
Hershey, Pennsylvania, 17033
Investigational Site Number 840016
Phoenix, Arizona, 85013
Investigational Site Number 840014
Pittsburgh, Pennsylvania, 15213
Investigational Site Number 840008
Salt Lake City, Utah, 84113
Italy6 sites
Investigational Site Number 380001
Brescia, 25123
Investigational Site Number 380002
Cagliari, 09126
Investigational Site Number 380006
Messina, 98125
Investigational Site Number 380005
Milan, 20133
Investigational Site Number 380004
Padova, 35128
Investigational Site Number 380003
Roma, 00168
Belgium3 sites
Investigational Site Number 056001
Ghent, 9000
Investigational Site Number 056002
Leuven, 3000
Investigational Site Number 056003
Leuven, 3000
About this study
Official title: A Prospective Safety Sub-Registry to Assess Anaphylaxis and Severe Allergic Reactions, and Severe Cutaneous and Systemic Immune Complex Mediated Reactions With Alglucosidase Alfa Treatment
To collect uniform and meaningful data on patients with Pompe disease who experience anaphylaxis, severe allergic reactions, and/or signals of severe cutaneous and/or systemic immune complex-mediated reactions following treatment with alglucosidase alfa.
- Conditions
- Pompe Disease
- Being tested
- alglucosidase alfa
- Keywords
- pompe disease, alglucosidase alfa, anaphylaxis, severe cutaneous, systemic immune complex-mediated reactions
- Medical subject headings
- Glycogen Storage Disease Type II, Anaphylaxis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
alglucosidase alfaBiological
Also called Myozyme; Lumizyme
Alglucosidase alfa IV infusion of 20 mg/kg; qow
| Study group | Role | What they receive |
|---|---|---|
| pompe safety sub-registry | patients are selected from those who are enrolled in the Pompe Registry, and will be followed for safety evaluation in this sub-registry Receives: Biological: alglucosidase alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
number of patients experience anaphylaxis, severe allergic reactions and/or signals of severe cutaneous and/or systematic immune complex-mediated reactions
collect meaningful data on patients with these outcomes following treatment with alglucosidase alfa
Measured: 4 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 2012 (estimated)
- Study start20 Mar 2015
- Primary completion20 Apr 2021
- Study completion20 Apr 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pompe DiseaseStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 110 participants (actual)
- Target duration
- 4 Years
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
- NCT01710813
- Sponsor's protocol ID
- AGLU06909
- Other IDs
- LTS13930
- Status last verified
- Jul 2021
- First submitted
- 17 Oct 2012
- Last updated
- 13 Jul 2021
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 13 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Safety Sub-Registry to Assess Anaphylaxis and Severe Allergic Reactions, and Severe Cutaneous and Systemic Immune Complex Mediated Reactions With Alglucosidase Alfa Treatment
- ClinicalTrials.gov ID
- NCT01710813
- Acronym / sponsor ID
- AGLU06909
- Conditions
- Pompe Disease
- Keywords
- pompe disease, alglucosidase alfa, anaphylaxis, severe cutaneous, systemic immune complex-mediated reactions
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 110 participants (actual)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- 20 Mar 2015 (actual)
- Primary completion
- 20 Apr 2021 (actual)
- Study completion
- 20 Apr 2021 (actual)
- First posted
- 19 Oct 2012 (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.
To collect uniform and meaningful data on patients with Pompe disease who experience anaphylaxis, severe allergic reactions, and/or signals of severe cutaneous and/or systemic immune complex-mediated reactions following treatment with alglucosidase alfa.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 4 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| pompe safety sub-registry | – | Biological: alglucosidase alfapatients are selected from those who are enrolled in the Pompe Registry, and will be followed for safety evaluation in this sub-registry |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients who are enrolled in the Pompe Registry will be enrolled in this sub-registry include patients with infantile-onset Pompe disease, as well as those with late-onset Pompe disease. An approximately equal proportion of currently treated and treatment-naïve patients is targeted for enrollment at each site.
Key inclusion criteria (4 listed)
- The patient must be enrolled in the Pompe Registry;
- Provide a signed patient information and authorization form;
- Have a confirmed diagnosis of Pompe disease (confirmation of diagnosis is defined as documented GAA enzyme deficiency from any tissue source and/or documentation of 2 GAA gene mutations);
- Be naïve to and plan to be treated with alglucosidase alfa at or prior to enrollment, or are being treated with alglucosidase alfa.
Key exclusion criteria (1 listed)
- Patients will be excluded if they have received an investigational drug (excluding alglucosidase alfa) within 30 days prior to signing a Safety Sub-Registry Patient Information and Authorization form, or if they are taking or plan to take any investigational product while enrolled in the Safety Sub-Registry.
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: Myozyme; LumizymeAlglucosidase alfa IV infusion of 20 mg/kg; qow | Biological | pompe safety sub-registry |
Primary outcome
number of patients experience anaphylaxis, severe allergic reactions and/or signals of severe cutaneous and/or systematic immune complex-mediated reactions
Time frame: 4 Years
collect meaningful data on patients with these outcomes following treatment with alglucosidase alfa
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 13 Jul 2021.
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Pompe 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
18 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Pompe Disease, run by Genzyme, a Sanofi Company at 18 sites in 6 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 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 13 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in United States, Italy, Belgium, Czechia, Germany and 1 more country. 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.
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?
number of patients experience anaphylaxis, severe allergic reactions and/or signals of severe cutaneous and/or systematic immune complex-mediated reactions (time frame: 4 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 13 Jul 2021.