Dose Escalation Study of Intravenous PRX004 in Subjects with Amyloid Transthyretin (ATTR) Amyloidosis

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Updated on 19 February 2024

Summary

You are being asked to take part in this research study because you have ATTR amyloidosis with peripheral neuropathy (nerve damage that occurs in the extremities of the body and may cause pain, such as foot pain or hand pain). Transthyretin (TTR) is a naturally occurring protein. 


In ATTR amyloidosis, TTR misfolds (folds incorrectly) and forms toxic deposits called fibrils (amyloid) which build up in tissues (e.g. heart, nerves, gastrointestinal tract), and disrupt organ shape and function. ATTR amyloidosis can run in families and this is known as hereditary ATTR amyloidosis. Hereditary ATTR amyloidosis usually affects the nerves or the heart, or both. PRX004 is a type of drug called an antibody. Antibodies are proteins that make up part of the immune system, your body’s natural defense system. Antibodies recognize foreign or unwanted material, such as infections or even some cancers and help destroy the foreign or unwanted material while causing little or less harm to normal cells.

PRX004 was developed to target the abnormal protein (amyloid) that is involved in ATTR amyloidosis. PRX004 has not been previously tested in humans.
 The purpose of this study is to: 
• Determine the highest dose of PRX004 up to a set maximum that can safely be given without unacceptable (intolerable) side effects to subjects with hereditary ATTR amyloidosis.
 • Determine what dose or doses of PRX004 should be used in future research studies on subjects with hereditary ATTR amyloidosis by looking at how safe it is, how well tolerated it is, how it affects the body and how it moves through the body.
 • To gain more information on how safe and well-tolerated PRX004 is in subjects with hereditary ATTR amyloidosis.

Description

This is Phase 1, open label, dose escalation study of intravenous PRX004. The purpose of the study is to know how safe and well tolerated PRX004 is in subjects with hereditary ATTR amyloidosis. This study consist of 2 parts: Dose escalation phase- in which subjects will be enrolled at each dose level to receive IV PRX004 once every 28 days, up to 3 doses. Long term extension phase this is an option to continue with monthly infusions for up to 15 additional months.

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Details
Condition Amyloid Transthyretin (ATTR) Amyloidosis
Age 18years - 99years
Clinical Study Identifier03336580
Last Modified on19 February 2024

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