Alpha-Galactosidase A Replacement Therapy for Fabry Disease


Phase 2 Results N/A

Eligibility Criteria

INCLUSION CRITERIA - Patient has Fabry Disease:
Patient is a male hemizygote, age 18 or above, with evidence of Fabry Disease, defined as Alpha-galactosidase A enzyme deficiency. Alpha-galactosidase A deficiency is defined as a plasma or serum enzyme level less than 1.2 nmoles/hr/mL.
Patient is a heterozygous carrier female, aged 18 or above, with evidence of Fabry Disease defined as a mutation in the Alpha-galactosidase A gene.
Patient has clinical evidence of Fabry Disease. For patients with compromised renal function or a history of renal transplant as a result of Fabry Disease, the renal disease must be consistent with Fabry Disease. For other patients, clinical evidence is defined as at least one (1) of the following:
neurologic disease (neuropathic pain)
cardiac disease (cadiomyopathy)
cerebrovascular disease (history of stroke)
dermatologic disease (angiokeratomas)
gastrointestinal disease (malabsorption and weight loss).
Patient is not eligible for an ongoing TKT sponsored placebo-controlled clinical trial that is accruing patients. The selection criteria for ongoing Study TKT010 appear in Appendix D. If another TKT sponsored placebo-controlled clinical trial is initiated during this study, the selection criteria for that study will be appended to this study.
Patient has a disease other than Fabry that is the cause of the patient's renal dysfunction (for example, diabetes or hypertension).
Patient has received another investigational therapeutic agent for Fabry Disease.
Patient has received a renal transplant as a result of renal dysfunction caused by a disease other than Fabry Disease.
Patient is unable to comply with the protocol, eg, is uncooperative with protocol schedule, refuses to agree to all of the study procedures, is unable to return for safety evaluations, or is otherwise unlikely to complete the study as determined by the investigator or the medical monitor.