Pompe Disease Registry

This study is currently recruiting participants.

See

Contacts and Locations

Verified June 2017 by Sanofi ( Genzyme, a Sanofi Company )

Sponsor:

Information provided by (Responsible Party):

Sanofi ( Genzyme, a Sanofi Company )

ClinicalTrials.gov Identifier:

NCT00231400

First received: September 30, 2005

Last updated: June 13, 2017

Last verified: June 2017

History of Changes

Tracking Information

First Received Date ^ICMJE

September 30, 2005

Last Updated Date

June 13, 2017

Actual Start Date ^ICMJE

August 21, 2004

Estimated Primary Completion Date

September 30, 2026 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

Understanding of the variability, progression , identification and natural history of the manifestations of Pompe disease [ Time Frame: 15 Years ]

Original Primary Outcome Measures ^ICMJE

Not Provided

Change History

Complete list of historical versions of study NCT00231400 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Not Provided

Original Secondary Outcome Measures ^ICMJE

Not Provided

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

Pompe Disease Registry

Official Title ^ICMJE

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 ^ICMJE

Observational

Study Design ^ICMJE

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 ^ICMJE

Glycogen Storage Disease Type II
Pompe Disease

Intervention ^ICMJE

Not Provided

Study Groups/Cohorts

Not Provided

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 ^ICMJE

Recruiting

Estimated Enrollment ^ICMJE

2000

Estimated Completion Date

September 30, 2026

Estimated Primary Completion Date

September 30, 2026 (Final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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, Senior

Accepts Healthy Volunteers

Contacts ^ICMJE

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 ^ICMJE

Brazil, Canada, China, Hong Kong, Italy, Korea, Republic of, Malaysia, Philippines, Singapore, Slovakia, Taiwan, Thailand, United States

Removed Location Countries

Administrative Information

NCT Number ^ICMJE

NCT00231400

Other Study ID Numbers ^ICMJE

Pompe Registry
DIREGC07005 ( Other Identifier: Sanofi )

Has Data Monitoring Committee

U.S. FDA-regulated Product

Not Provided

Plan to Share Data

Not Provided

IPD Description

Not Provided

Responsible Party

Sanofi ( Genzyme, a Sanofi Company )

Study Sponsor ^ICMJE

Genzyme, a Sanofi Company

Collaborators ^ICMJE

Not Provided

Investigators ^ICMJE

Study Director:

Medical Monitor

Genzyme, a Sanofi Company

PRS Account

Sanofi

Verification Date

June 2017

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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