|
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 October 2017 |
|
Main ID: |
NCT00074958 |
|
Date of registration:
|
24/12/2003 |
|
Prospective Registration:
|
No |
|
Primary sponsor: |
|
|
Public title:
|
A Study of Fabrazyme in Pediatric Patients With Fabry Disease
|
|
Scientific title:
|
A Multi-center, Phase 2, Open-Label Study of Fabrazyme (Recombinant Human a-Galactosidase A) Replacement Therapy in Pediatric Patients With Fabry Disease |
|
Date of first enrolment:
|
October 2002 |
|
Target sample size:
|
16 |
|
Recruitment status: |
Completed |
|
URL:
|
https://clinicaltrials.gov/show/NCT00074958 |
|
Study type:
|
Interventional |
|
Study design:
|
|
|
Phase:
|
Phase 2
|
|
|
Countries of recruitment
|
|
France
|
Italy
|
Poland
|
United Kingdom
|
United States
| | | |
|
Contacts
|
|
Name:
|
Medical Monitor |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
Genzyme, a Sanofi Company |
| | |
|
Key inclusion & exclusion criteria
|
Inclusion criteria:
- Patient or legal guardian must provide written informed consent
- Patients must have a clinical diagnosis of Fabry disease and active Fabry disease
(clinical signs and symptoms)
- Patients must be at least 7 years of age but no older than 15 years of age at time of
enrollment
- Patients must be Tanner Stage = III
- Female patients must have a negative pregnancy test prior to each infusion and use a
medically accepted form of contraception throughout the study
Exclusion Criteria:
- Patient has a clinically significant organic disease (with the exception of symptoms
relating to Fabry disease) that in the opinion of the investigator would preclude
participation in the trial
- Patient has participated in a study employing investigational drug within 30 days of
the start of this study
- Patient has received prior treatment with enzyme replacement therapy
- Patient is unable to comply with the clinical protocol
Age minimum:
7 Years
Age maximum:
15 Years
Gender:
All
|
|
Health Condition(s) or Problem(s) studied
|
|
Fabry Disease
|
|
Intervention(s)
|
|
Biological: Fabrazyme (agalsidase beta)
|
|
Primary Outcome(s)
|
|
Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin
[Time Frame: Baseline, Week 24 and Week 48]
|
|
Secondary Outcome(s)
|
|
Plasma GL-3
[Time Frame: Baseline, Week 24 and Week 48]
|
|
Secondary ID(s)
|
|
AGAL-016-01
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|