Replagal Enzyme Replacement Therapy for Children With Fabry Disease
Public ClinicalTrials.gov record NCT00084084. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
An Open Label Clinical Trial of Replagal Enzyme Replacement Therapy In Children With Fabry Disease Who Have Completed Study TKT023 or Who Are Naive to Enzyme Replacement Therapy
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
Primary Objective(s): * To assess the safety of Replagal at a dose of 0.2 mg/kg administered over 40 (+/-10) minutes in children with Fabry disease * To assess the effect of Replagal on heart rate variability in patients 7 to 17 years of age Secondary Objective(s): * To determine the pharmacokinetics of Replagal at baseline and after the initiation of enzyme replacement therapy (ERT) * To determine exploratory measurements of efficacy including renal function (ie, estimated glomerular filtration rate \[eGFR\] and creatinine clearance), clinical outcomes (in Cohorts 1 and 2), and sweating and left ventricular mass index (LVMI) (Cohort 1, Phase 1 only)
Study identification
- NCT ID
- NCT00084084
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 17 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 7 Years to 17 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jun 9, 2004
- Primary completion
- Jun 14, 2011
- Completion
- Jun 14, 2011
- Last update posted
- Jul 29, 2021
2004 – 2011
United States locations
- U.S. sites
- 13
- U.S. states
- 10
- U.S. cities
- 12
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Tucson Access Center of Arizona Kidney Disease Hypertension Center | Tucson | Arizona | 85719 | — |
| University of Arizona Health Sciences Center | Tucson | Arizona | 85724 | — |
| Children's Physician Group | Palm Beach Gardens | Florida | 33410 | — |
| Christus St. Patrick Hospital | Lake Charles | Louisiana | 70601 | — |
| Clinical Center, National Institutes of Health | Bethesda | Maryland | 20892 | — |
| Memorial Hospital | Easton | Maryland | 21601 | — |
| St. Louis Children's Hospital | St Louis | Missouri | 63110 | — |
| NYU School of Medicine | New York | New York | 10016 | — |
| Sacred Heart Hospital | Allentown | Pennsylvania | 18102 | — |
| East Tennessee Children's Hospital | Knoxville | Tennessee | 37916 | — |
| University of Tennessee, Health Science Center | Memphis | Tennessee | 38163 | — |
| Institute of Metabolic Diseases | Dallas | Texas | 75226 | — |
| Office of Michael Cohen | Stafford | Virginia | 22556 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 1 non-U.S. site.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT00084084, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jul 29, 2021 · Synced Sep 3, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT00084084 live on ClinicalTrials.gov.