A Phase I-II Pharmacokinetic/Pharmacodynamic Study of Replagal to Assess the Effects of Alternative Dose and Regimen in Patients with Fabry Disease (TKT027) - TKT027

TKT Inc0 sites20 target enrollmentJune 28, 2004

Overview

No summary available.

Registry

who.int

Start Date

June 28, 2004

End Date

TBD

Last Updated

11 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

Male

Sponsor

TKT Inc

•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 (\< 4\.0 nmol/mL/hour in plasma or serum or \< 8% of average mean
•normal in leukocytes).
•2\. Subject must have one or more clinical manifestations of Fabry disease including neuropathic pain, angiokeratoma, corneal verticillata, cardiomyopathy, hypo\- or anhydrosis, abdominal pain and/or diarrhea, serum creatinine \> 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

•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.