|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
19 February 2015 |
|
Main ID: |
NCT00765414 |
|
Date of registration:
|
02/10/2008 |
|
Prospective Registration:
|
No |
|
Primary sponsor: |
|
|
Public title:
|
Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.
|
|
Scientific title:
|
An Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid a-Glucosidase (rhGAA) Given as Enyzme Replacement Therapy to a Single Patient With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies |
|
Date of first enrolment:
|
April 2003 |
|
Target sample size:
|
1 |
|
Recruitment status: |
Completed |
|
URL:
|
http://clinicaltrials.gov/show/NCT00765414 |
|
Study type:
|
Interventional |
|
Study design:
|
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
|
|
Phase:
|
Phase 2
|
|
|
Countries of recruitment
|
|
United States
| | | | | | | |
|
Contacts
|
|
Name:
|
Medical Monitor |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
Genzyme, a Sanofi Company |
| | |
|
Key inclusion & exclusion criteria
|
Inclusion Criteria:
- Provide written informed consent prior to participating in any study related
procedures;
- Currently enrolled in Protocol AGLU01402
- Have the ability to comply with the clinical protocol, which required extensive
clinical evaluations for an extended period of time.
Exclusion Criteria:
- Was pregnant or unwilling to use approved birth control during the course of the
study;
- Had experienced any unmanageable AEs under Protocol AGLU01402 (as determined and
agreed upon by the Principal Investigator and sponsor) due to rhGAA that would
preclude continuing ERT;
- Was participating in any other investigational study.
Age minimum:
16 Years
Age maximum:
N/A
Gender:
Female
|
|
Health Condition(s) or Problem(s) studied
|
|
Glycogen Storage Disease Type II GSD II
|
|
Pompe Disease Late-Onset
|
|
Intervention(s)
|
|
Biological: Myozyme
|
|
Primary Outcome(s)
|
|
The objective of this extension study was to monitor the long-term safety and efficacy of a single patient
[Time Frame: 3 years]
|
|
Secondary ID(s)
|
|
AGLU02103
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|
Results
|
|
Results available:
|
|
|
Date Posted:
|
|
|
Date Completed:
|
|
|
URL:
|
|
|
|