Trial record 1 of 1 for:
AGLU06909
Alglucosidase Alfa Pompe Safety Sub-Registry
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT01710813 |
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Recruitment Status :
Completed
First Posted : October 19, 2012
Last Update Posted : July 13, 2021
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Sponsor:
Genzyme, a Sanofi Company
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
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Brief 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.
| Condition or disease | Intervention/treatment |
|---|---|
| Pompe Disease | Biological: alglucosidase alfa |
| Study Type : | Observational [Patient Registry] |
| Actual Enrollment : | 110 participants |
| Observational Model: | Cohort |
| Time Perspective: | Prospective |
| Target Follow-Up Duration: | 4 Years |
| 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 |
| Actual Study Start Date : | March 20, 2015 |
| Actual Primary Completion Date : | April 20, 2021 |
| Actual Study Completion Date : | April 20, 2021 |
Resource links provided by the National Library of Medicine
Drug Information available for:
Alglucosidase Alfa
| Group/Cohort | Intervention/treatment |
|---|---|
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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
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Biological: alglucosidase alfa
Alglucosidase alfa IV infusion of 20 mg/kg; qow
Other Name: Myozyme; Lumizyme |
Primary Outcome Measures :
- 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
Information from the National Library of Medicine
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| Ages Eligible for Study: | Child, Adult, Older Adult |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Probability Sample |
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.
Criteria
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01710813
Locations
Show 18 study locations
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
| Study Director: | Medical Monitor | Genzyme, a Sanofi Company |
Additional Information:
| Responsible Party: | Genzyme, a Sanofi Company |
| ClinicalTrials.gov Identifier: | NCT01710813 |
| Other Study ID Numbers: |
AGLU06909 LTS13930 ( Other Identifier: Other company study code ) |
| First Posted: | October 19, 2012 Key Record Dates |
| Last Update Posted: | July 13, 2021 |
| Last Verified: | July 2021 |
Keywords provided by Sanofi ( Genzyme, a Sanofi Company ):
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pompe disease alglucosidase alfa anaphylaxis severe cutaneous systemic immune complex-mediated reactions |
Additional relevant MeSH terms:
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Glycogen Storage Disease Type II Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases |
Metabolism, Inborn Errors Genetic Diseases, Inborn Glycogen Storage Disease Carbohydrate Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases |