GoutPhase 1NCT01021241
Safety and Efficacy Study of Intravenous Uricase-PEG 20
A Phase 1 study testing Uricase-PEG 20 in people with Gout and Hyperuricemia, run by EnzymeRx at 2 sites in United States.
Trial summary
The purpose of this study is to assess the safety, pharmacokinetics and pharmacodynamics of single intravenous doses of Uricase-PEG 20. Read more about this study
- Who is it for?
- Ages 40 to 75 yearsHealthy volunteers can take part
- Conditions the study is for
- Gout, Hyperuricemia
- What is being tested?
- Uricase-PEG 20
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 2 sitesUnited States
- Study dates
- Oct 2009to Mar 2010
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 40 to 75 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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)
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Altoona Center for Clinical Research
Duncansville, Pennsylvania, 16635
MRA Clinical Research
Miami, Florida, 33143
About this study
Official title: A Cohort Dose-Escalation Phase 1 Study of Intravenous Infusion of Uricase-PEG 20
The purpose of this study is to assess the safety, pharmacokinetics and pharmacodynamics of single intravenous doses of Uricase-PEG 20.
Read the 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 intravenous Uricase-PEG 20, the anticipated route of administration to be used in future clinical development in tumor lysis syndrome.
- Conditions
- Gout, Hyperuricemia
- Being tested
- Uricase-PEG 20
- Medical subject headings
- Gout, Hyperuricemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Uricase-PEG 20Biological
Intravenous infusion of Uricase-PEG 20 over one hour; no premedication
| Study group | Role | What they receive |
|---|---|---|
| Uricase-PEG 20 | Experimental | Cohorts will receive ascending doses of Uricase-PEG 20 in a sequential manner Receives: Biological: Uricase-PEG 20 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: Through Day 35 after dosing
Pharmacokinetics (Uricase-PEG 20 serum concentration)
Measured: Through Day 35 after dosing
Pharmacodynamics (plasma uric acid concentration)
Measured: Through Day 35 after dosing
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Nov 2009 (estimated)
- Study startOct 2009
- Primary completionMar 2010 (estimated)
- Study completionMar 2010 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for GoutStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- EnzymeRx
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Anthony S Fiorino, MD, PhD, Study director, EnzymeRx, LLCAlan Kivitz, MD, Principal investigator, Altoona Center for Clinical Research
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01021241
- Sponsor's protocol ID
- ENZ-101
- Status last verified
- Feb 2010
- First submitted
- 24 Nov 2009
- Last updated
- 23 Feb 2010
- Results posted
- No
- Data monitoring committee
- No
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.
PubMed 12233890
Study at a glance
From the registry record, last updated by the sponsor on 23 Feb 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Cohort Dose-Escalation Phase 1 Study of Intravenous Infusion of Uricase-PEG 20
- ClinicalTrials.gov ID
- NCT01021241
- Acronym / sponsor ID
- ENZ-101
- Conditions
- Gout, Hyperuricemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- EnzymeRxIndustry (pharma, biotech, devices)
- Study start
- Oct 2009
- Primary completion
- Mar 2010 (estimated)
- Study completion
- Mar 2010 (estimated)
- First posted
- 26 Nov 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to assess the safety, pharmacokinetics and pharmacodynamics of single intravenous doses of Uricase-PEG 20.
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 intravenous Uricase-PEG 20, the anticipated route of administration to be used in future clinical development in tumor lysis syndrome.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Uricase-PEG 20 | Experimental | Biological: Uricase-PEG 20Cohorts will receive ascending doses of Uricase-PEG 20 in a sequential manner |
Who may qualify
- Ages
- 40 to 75 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (4 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Uricase-PEG 20Intravenous infusion of Uricase-PEG 20 over one hour; no premedication | Biological | Uricase-PEG 20 |
Primary outcome
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: Through Day 35 after dosing
Pharmacokinetics (Uricase-PEG 20 serum concentration)
Time frame: Through Day 35 after dosing
Pharmacodynamics (plasma uric acid concentration)
Time frame: Through Day 35 after dosing
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Feb 2010.
- Background: PubMed 12233890Bomalaski 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.
- Abstract describing animal pharmacokinetic studiesLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Anthony S Fiorino, MD, PhD study director
EnzymeRx, LLC
Alan Kivitz, MD principal investigator
Altoona Center for Clinical Research
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Uricase-PEG 20 in people with Gout and Hyperuricemia, run by EnzymeRx at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 23 Feb 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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: Through Day 35 after dosing). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01021241, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Feb 2010.