At a glance
ClinicalIndex Comparison Record- ✓Prior enrollment in protocol 00-N-0185/TKT011 or 02-N-0220/TKT015
- ✓GFR decline >5 mL/min/year despite ≥2.5 years of agalsidase alfa in TKT003/006/011 OR ≥1.0 years ERT in TKT010/015
- ✓At least twice demonstrated significant improvement or normalization of sweat function (QSART or thermoregulatory sweat test) or ≥10% reduction in serum creatinine, followed by return to pre-infusion state before next biweekly infusion
- ✓Written informed consent
- ✕Not enrolled in prior protocols 00-N-0185/TKT011 or 02-N-0220/TKT015
- ✕Stable serum creatinine or lesser rise in serum creatinine than specified, without objective evidence of incomplete response (e.g., loss of sweat function improvement between infusions)
- ✕On dialysis or status post renal transplantation
- ✕Cannot tolerate study procedures or unable/unwilling to travel to study center
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
Study of Weekly Dosing Regimens of Replagal in Patients With Fabry Disease With Incomplete Clinical Response to Long-Term Therapy
In Brief
A Phase 2 clinical trial evaluating Replagal for Fabry Disease. Completed, enrolled 13 participants across 1 site.
Detailed Summary
This study will determine the safety and effectiveness of increasing Replagal infusions in certain patients with Fabry disease. Replagal is a genetically engineered form of Alpha-galactosidase A, an enzyme that normally breaks down a fatty substance called globotriaosylceramide (Gb3). In patients with Fabry disease, Alpha-galactosidase A does not function properly and, therefore, Gb3 builds up, causing problems with the kidneys, heart, nerves, and blood vessels. Patients with Fabry disease who are participating in NIH protocol 00-N-0185 or 02-N-0220 may be eligible for this study. This includes patients who are currently taking Replagal but whose kidney function continues to worsen, or patients who have certain test results that are much improved after Replagal infusion. Participants will receive Replagal infusions (0.2 mg/kg body weight) through a vein once a week (as opposed to the previous dosage of once every 2 weeks) for up to 2 years. The first infusion, and some others, are given at the NIH Clinical Center, but most are administered by the patient's local doctor. Vital signs are measured before, immediately after, and 1 hour after each infusion. Baseline evaluations are done on an inpatient basis at the NIH Clinical Center over a 1-week period before and after the first Replagal infusion and at 6-month intervals during the study. Tests include a check of vital signs (temperature, respiratory rate, pulse rate, and blood pressure); weight measurement; physical and neurological examinations; routine blood and urine tests; 24-hour urine collection; electrocardiogram; and review of treatment side effects. In addition, the following tests are done: * Quantitative sensory testing: This is a non-invasive test to measure the ability to sense warm, cold and vibration in the hand and foot. * QSART: This test measures the amount of sweat in a particular area of skin that did not sweat enough. A small amount of a medicine called acetylcholine is put on the skin and made to enter the skin using a very small electric current. * Doppler skin blood flow: This test measures blood flow to the blood vessels of the skin. A machine takes pictures of blood flow in the skin of the forearm using a laser beam. Pictures are taken before and during application of medicines that cause blood vessels to dilate. Acetylcholine is used on one forearm and nitroprusside is used on the other. The medication is made to enter the skin using a small el...
Study Details
Timeline
Interventions
enzyme replacement therapy