Fabry Disease MedDRA version: 6.1 Level: PT Classification code 10016016
Conditions
Interventions
Trade Name: Replagal
Product Name: Replagal
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: agalsidase alfa
Concentration unit: mg/ml milligram(s)/millilitre
Concentrat
Sponsors
TKT Inc
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Subject is a male hemizygote, age 18 years or older with confirmed diagnosis of Fabry Disease. Diagnosis of Fabry disease may be confirmed by proof of a mutation of the a-Galactosidase A gene compatible with Fabry Disease and/or a deficiency of a-Galactosidase A ( 1.0 mg/dl or proteinuria > 300 mg/24 hours. 3. Subject must have voluntarily signed an Institutional Review Board (IRB) approved informed consent form after all relevant aspects of the study have been explained and discussed with the subject. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Subject has been previously treated with Replagal or any other enzyme replacement therapy for Fabry Disease. If the patient has previously been treated with Replagal or another enzyme replacement therapy then they must have been off the therapy for at least 30 days and must have a base-line Day -14 antibody blood sample drawn and that test must be negative for antiagalsidase alfa IgG and IgE antibodies and not experienced a prior severe infusion reactions with prior enzyme replacement therapy. 2. Subject has been enrolled in another clinical investigative study in the past 30 days 3. Subject is unable to give informed consent or is deemed unable to comply with all aspects of the clinical trial. 4. Subject has plasma Gb3 drawn on Day –14 (base-line) less than 4.0 nmol/mL. 5. Subject is undergoing dialysis or who has received a renal transplant. 6. Subjects who cannot tolerate the study procedures or who are unable or unwilling to travel to the study center as required by this protocol. 7. Subjects with an inter-current medical condition that would render them unsuitable for the study (e.g. HIV, diabetes) by confounding an assessment of the effects of the experimental therapy and its adverse events. 8. Subjects who in the opinion of the investigator (for whatever reason) are thought to be unsuitable for the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to assess the pharmacodynamic effects of alternative weekly and every 2 week dosing regimens (i.e. drug given every other week) of Replagal in comparison to the current standard Replagal treatment regimen of 0.2 mg/kg every 2 weeks.;Secondary Objective: Secondary objectives are to evaluate the safety and pharmacokinetics at each of the dose levels and regimens.;Primary end point(s): The pharmacodynamic parameter to be assessed is plasma globotriaosylceramide (Gb3). Clinical parameters including sweating, heart rate variability, proteinuria, severity of neuropathic pain, pain and anti-diarrheal medication usage, frequency and severity of abdominal pain, and frequency of diarrhea also will be assessed. | — |
Countries
Czech Republic
Outcome results
None listed