| NCT ID | NCT00231400 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Genzyme, a Sanofi Company |
| Condition | Glycogen Storage Disease Type II |
| Study Type | OBSERVATIONAL |
| Enrollment | 2,000 participants |
| Start Date | 2004-09-15 |
| Primary Completion | 2034-01-31 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 2,000 participants in total. It began in 2004-09-15 with a primary completion date of 2034-01-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both treated and not. Data from the Registry are also used to fulfill various global regulatory commitments, to support product development/reimbursement, and for other research and non-research related purposes. The objectives of the Registry are: * To enhance understanding of the variability, progression, identification, and natural history of Pompe disease, with the ultimate goal of better guiding and assessing therapeutic intervention. * To assist the Pompe medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care. * To characterize the Pompe disease population. * To evaluate the long-term effectiveness of alglucosidase alfa.
Eligibility Criteria
Inclusion Criteria: All patients with a confirmed diagnosis of Pompe disease who have signed the informed consent and authorization form(s) are eligible for inclusion. Confirmed diagnosis is defined as documented GAA enzyme deficiency from blood, skin, or muscle tissue and/or documentation of 2 GAA gene mutations. Exclusion Criteria: There are no exclusion criteria in this Registry
Contact & Investigator
Trial Transparency email recommended (Toll free number for US & Canada)
✉ contact-us@sanofi.com📞 800-633-1610
Study Director
STUDY DIRECTOR
Genzyme, a Sanofi Company
Frequently Asked Questions
Who can join the NCT00231400 clinical trial?
This trial is open to participants of all sexes, studying Glycogen Storage Disease Type II. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT00231400 currently recruiting?
Yes, NCT00231400 is actively recruiting participants. Contact the research team at contact-us@sanofi.com for enrollment information.
Where is the NCT00231400 trial being conducted?
This trial is being conducted at Birmingham, United States, Phoenix, United States, Phoenix, United States, Tucson, United States and 11 additional locations.
Who is sponsoring the NCT00231400 clinical trial?
NCT00231400 is sponsored by Genzyme, a Sanofi Company. The principal investigator is Study Director at Genzyme, a Sanofi Company. The trial plans to enroll 2,000 participants.