Active Studies

This Phase 2a clinical study is designed to provide data on OCR-002 in patients with acute
liver failure/acute liver injury (ALF/ALI) in regard to:

- safety and tolerability;

- metabolism of the compound to glutamine and phenylacetylglutamine (PAGN);

- its effect on circulating ammonia levels and neurological function in patients with and
without impaired renal function after continuous infusion at different infusion rates.

Subjects will receive up to 120 hours (5 days) of drug infusion, followed by a 30 day
follow-up visit post infusion. It is anticipated that this early safety and tolerability
study, with appropriate PK/PD data, will lead to a development program for the use of
OCR-002 in the treatment of hyperammonemia either due to ALF or possibly other liver
conditions. The hypotheses are:

- Treatment with OCR-002 is safe and tolerable in patients with acute liver failure/acute
liver injury due to acetaminophen overdose or drug-induced liver injury, autoimmune
hepatitis, viral hepatitis or indeterminate etiologies.

- A dose of 10g/24h (0.42g/h) will achieve steady state plasma concentrations within
6-12h with little additional accumulation in the ALI/ALF setting.

- Treatment with OCR-002 will improve neurological function in patients with acute liver
failure/severe acute liver injury due to acetaminophen overdose.

RATIONALE: Lenalidomide and bortezomib may stop the growth of multiple myeloma by blocking
blood flow to the tumor. Also, bortezomib may stop the growth of tumor cells by blocking
some of the enzymes needed for cell growth. Monoclonal antibodies, such as elotuzumab, can
block cancer growth in different ways. Some block the ability of cancer cells to grow and
spread. Others find cancer cells and help kill them or carry cancer-killing substances to
them. Drugs used in chemotherapy, such as lenalidomide and dexamethasone also work in
different ways to kill tumor cells or stop them from growing. Giving elotuzumab together
with chemotherapy may be a better way to block cancer growth.

PURPOSE: This phase I/II trial is studying the side effects and best dose of elotuzumab and
to see how well it works when given with lenalidomide, bortezomib, and dexamethasone in
treating patients with multiple myeloma.

Purpose:
The purpose of this study is to understand cognitive impairment in end stage renal disease before and after a kidney transplant.

Prospective observational drug registry developed to characterize the safety profile (including primarily potential serious hepatic risks) and to describe clinical characteristics and outcomes of patients newly treated with Opsumit in the post-marketing setting.

 

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