Safety and Efficacy Study of Intramuscular Uricase-PEG 20 - Full Text View

Sponsor:	EnzymeRx
Information provided by:	EnzymeRx
ClinicalTrials.gov Identifier:	NCT01038947

Purpose

The purpose of this study is to assess the safety, pharmacokinetics and pharmacodynamics of single and multiple intramuscular doses of Uricase-PEG 20

Condition	Intervention	Phase
Gout Hyperuricemia	Biological: Uricase-PEG 20	Phase I

Study Type:	Interventional
Study Design:	Endpoint Classification: Pharmacokinetics/Dynamics Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	A Cohort Dose-Escalation Phase 1 Study of Intramuscular Injection of Uricase-PEG 20

Resource links provided by NLM:

MedlinePlus related topics: Gout

U.S. FDA Resources

Further study details as provided by EnzymeRx:

Primary Outcome Measures:

Safety (assessment of signs and symptoms and clinical laboratory measurements following administration of Uricase-PEG 20, graded according to the Common Toxicity Criteria for Rheumatology, version 2.0) [ Time Frame: Up to 35 days after dosing ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Pharmacokinetics (Uricase-PEG 20 serum concentration) [ Time Frame: Up to 35 days after dosing ] [ Designated as safety issue: No ]
Pharmacodynamics (plasma uric acid concentration) [ Time Frame: Up to 35 days after dosing ] [ Designated as safety issue: No ]
Immunogenicity [ Time Frame: Up to 35 days after dosing ] [ Designated as safety issue: No ]

Estimated Enrollment:	28
Study Start Date:	December 2009
Estimated Study Completion Date:	May 2010
Estimated Primary Completion Date:	April 2010 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: Uricase-PEG 20 Cohorts will receive ascending doses of Uricase-PEG 20 in a sequential manner. The study will enroll both single dose cohorts and multi-dose cohorts.	Biological: Uricase-PEG 20 Intramuscular injection of Uricase-PEG 20 without premedication

Detailed Description:

Uricase is an enzyme (found in most mammals but not humans) that converts poorly soluble uric acid into highly soluble allantoin. Because humans lack uricase, they are prone to develop elevated levels of uric acid, which can form crystals in the joints and soft tissues. In those which chronically elevated uric acid, gout may develop. In the setting of acute rises in uric acid, seen for example in tumor lysis syndrome, uric acid crystals can damage the renal tubules. Uricase-PEG 20 is a recombinant uricase conjugated with multiple PEG molecules designed to prolong the half-life and decrease the immunogenicity of uricase. This study will characterize the safety, pharmacokinetics and pharmacodynamics of intramuscular Uricase-PEG 20, the anticipated route of administration to be used in future clinical development in gout.

Eligibility

Ages Eligible for Study:	40 Years to 75 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	Yes

Criteria

Inclusion Criteria:

Serum uric acid > 6 mg/dL (men) or > 5 mg/dL (women)
Clinical laboratory values within normal limits or not clinically significant
Women should be menopausal or peri-menopausal

Exclusion Criteria:

Prior exposure to uricase
History of severe allergic reactions, or any allergy to PEG or pegylated products
G6PD or catalase deficiency
Medical condition that may interfere with ability to complete the study (e.g., uncontrolled diabetes or hypertension, congestive heart failure NYHA Class III or IV, history of myocardial infarction, immunosuppression, pregnancy)

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01038947

Contacts

Contact: Rocelle Flores

201-843-4424 ext 203

rflores@enzymerx.com

Locations

United States, Florida
MRA Clinical Research	Recruiting
Miami, Florida, United States, 33143
Contact: Vivian Rosales 305-772-2857 vrosales@miamiresearch.com
Principal Investigator: Patricia Pardo, MD
United States, Pennsylvania
Altoona Center for Clinical Research	Recruiting
Duncansville, Pennsylvania, United States, 16635
Contact: Ellen Kivitz 814-693-0300 ext 224
Principal Investigator: Alan J Kivitz, MD

Sponsors and Collaborators

EnzymeRx

Investigators

Study Director:	Anthony S Fiorino, MD, PhD	EnzymeRx, LLC
Principal Investigator:	Patricia Pardo, MD	MRA Clinical Research

More Information

Additional Information:

Homepage of sponsoring company This link exits the ClinicalTrials.gov site

Publications:

Bomalaski JS, Holtsberg FW, Ensor CM, Clark MA. Uricase formulated with polyethylene glycol (uricase-PEG 20): biochemical rationale and preclinical studies. J Rheumatol. 2002 Sep;29(9):1942-9.

Responsible Party:	Anthony Fiorino, MD, PhD, EnzymeRx, LLC
ClinicalTrials.gov Identifier:	NCT01038947 History of Changes
Other Study ID Numbers:	ENZ-102
Study First Received:	December 22, 2009
Last Updated:	December 22, 2009
Health Authority:	United States: Food and Drug Administration

Additional relevant MeSH terms:

Hyperuricemia
Pathologic Processes

ClinicalTrials.gov processed this record on February 09, 2012