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Trial record 35 of 103 for:    Pompe Disease

Pompe Disease Registry

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ClinicalTrials.gov Identifier: NCT00231400
Recruitment Status : Recruiting
First Posted : October 4, 2005
Last Update Posted : June 18, 2019
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Tracking Information
First Submitted Date September 30, 2005
First Posted Date October 4, 2005
Last Update Posted Date June 18, 2019
Actual Study Start Date August 21, 2004
Estimated Primary Completion Date January 31, 2034   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: January 30, 2013)
Understanding of the variability, progression , identification and natural history of the manifestations of Pompe disease [ Time Frame: 15 Years ]
Original Primary Outcome Measures Not Provided
Change History Complete list of historical versions of study NCT00231400 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 Pompe Disease Registry
Official Title Pompe Disease Registry
Brief Summary

The Pompe Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Pompe disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician.

The objectives of the Registry are:

  • To enhance the understanding of the variability, progression, and natural history of the key manifestations of Pompe disease;
  • To assist the Pompe medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care;
  • To characterize and describe the Pompe disease population as a whole; and
  • To evaluate the long-term effectiveness and safety of available treatment options including ERT(Enzyme Replacement Therapy) with Myozyme®.
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Other
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Patients diagnosed with Pompe disease
Condition
  • Glycogen Storage Disease Type II
  • Pompe Disease
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: January 30, 2013)
2000
Original Enrollment Not Provided
Estimated Study Completion Date January 31, 2034
Estimated Primary Completion Date January 31, 2034   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Patient must have a confirmed diagnosis of Pompe disease, documented by GAA(Glucosidase Alpha Acid) enzyme deficiency or GAA gene mutation

Exclusion Criteria:

  • There are no exclusion criteria
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Contact: Trial Transparency email recommended (Toll free number for US & Canada) 800-633-1610 ext 1 then # Contact-Us@sanofi.com
Contact: Medical Information 617-252-7832 medinfo@genzyme.com
Listed Location Countries Argentina,   Australia,   Belgium,   Brazil,   Canada,   Chile,   China,   Colombia,   Denmark,   France,   Germany,   Greece,   Hong Kong,   Hungary,   India,   Italy,   Japan,   Korea, Republic of,   Kuwait,   Malaysia,   Netherlands,   Philippines,   Poland,   Portugal,   Russian Federation,   Serbia,   Singapore,   Slovakia,   Taiwan,   Thailand,   United Arab Emirates,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT00231400
Other Study ID Numbers Pompe Registry
DIREGC07005 ( Other Identifier: Sanofi )
Has Data Monitoring Committee No
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 June 2019