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Trial record 79 of 103 for:    Pompe Disease

Alglucosidase Alfa Pompe Safety Sub-Registry

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
Recruitment Status : Active, not recruiting
First Posted : October 19, 2012
Last Update Posted : August 13, 2019
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Tracking Information
First Submitted Date October 17, 2012
First Posted Date October 19, 2012
Last Update Posted Date August 13, 2019
Study Start Date March 20, 2015
Estimated Primary Completion Date March 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: February 17, 2015)
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
Original Primary Outcome Measures
 (submitted: October 17, 2012)
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 manufactured at the 4000-L scale.
Change History Complete list of historical versions of study NCT01710813 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Alglucosidase Alfa Pompe Safety Sub-Registry
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
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.
Detailed Description Not Provided
Study Type Observational [Patient Registry]
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration 4 Years
Biospecimen Not Provided
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.

Condition Pompe Disease
Intervention Biological: alglucosidase alfa
Alglucosidase alfa IV infusion of 20 mg/kg; qow
Other Name: Myozyme; Lumizyme
Study Groups/Cohorts 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
Intervention: Biological: alglucosidase alfa
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Active, not recruiting
Actual Enrollment
 (submitted: July 18, 2019)
110
Original Estimated Enrollment
 (submitted: October 17, 2012)
100
Estimated Study Completion Date April 2021
Estimated Primary Completion Date March 2021   (Final data collection date for primary outcome measure)
Eligibility 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.
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Belgium,   Czechia,   Germany,   Italy,   Taiwan,   United States
Removed Location Countries Czech Republic
 
Administrative Information
NCT Number NCT01710813
Other Study ID Numbers AGLU06909
LTS13930 ( Other Identifier: Other company study code )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Sanofi ( Genzyme, a Sanofi Company )
Study Sponsor Genzyme, a Sanofi Company
Collaborators Not Provided
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
PRS Account Sanofi
Verification Date August 2019