Hematopoietic and Lymphoid Cell NeoplasmPhase 4NCT04745910
Pegloticase for the Reduction of Uric Acid in Patients With Tumor Lysis Syndrome
A Phase 4 study testing Pegloticase and Rasburicase in people with Hematopoietic and Lymphoid Cell Neoplasm and Malignant Solid Neoplasm, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
This trial studies the effect of pegloticase in reducing uric acid levels in patients with hyperuricemia (high blood levels of uric acid) caused by tumor lysis syndrome. Tumor lysis syndrome occurs when the breakdown products of cancer cells, such as uric acid, enter the blood stream. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hematopoietic and Lymphoid Cell Neoplasm, Malignant Solid Neoplasm, Tumor Lysis Syndrome
- What is being tested?
- Pegloticase and Rasburicase
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 5 Apr 2022to 27 Apr 2026
- Phase
- Phase 4
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- For inclusion in the study, patients should fulfill the following criteria:
- Signed informed consent prior to any study specific procedures (patient or caregiver)
- Male or female 18 years of age or older
- In the investigator's opinion, expected survival of at least 1 month
- Deemed stable by the investigator
- Serum Uric Acid ≥ 6 mg/dL
- Risk for tumor lysis syndrome based on the MD Anderson TLS risk score
Who cannot join
- Patients will be ineligible for study enrollment if any of the following exclusion criteria are fulfilled:
- Inability or refusal to give informed consent (patient or caregiver)
- Subject unwilling to take study medication
- Known allergy to uricase
- Pregnant
- Breastfeeding
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- Known glucose-6-phosphate dehydrogenase (G6PD) deficiency
- Transfusion in last 7 days (as this interferes with G6PD test), with the exception of platelet transfusions.
- Has received rasburicase during current admission.(Can be considered i24 hours after last Rasburicase administered, current or previous admission)
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Effect of Pegloticase on Reduction of Uric Acid in Patients With Tumor Lysis Syndrome: A Pilot Pragmatic Clinical Trial
This trial studies the effect of pegloticase in reducing uric acid levels in patients with hyperuricemia (high blood levels of uric acid) caused by tumor lysis syndrome. Tumor lysis syndrome occurs when the breakdown products of cancer cells, such as uric acid, enter the blood stream. High levels of uric acid in blood may cause kidney damage and reduce kidney function. The goal of this trial is to learn if pegloticase may lower uric acid levels in blood when given to cancer patients with hyperuricemia caused by tumor lysis syndrome.
Read the detailed description
PRIMARY OBJECTIVE:
- To collect preliminary data on the effectiveness of a single dose of pegloticase in lowering serum uric acid levels to less than 6.0 mg/dl within 24 hours in patients with hyperuricemia in the setting of tumor lysis syndrome.
SECONDARY OBJECTIVES:
- To assess the effect of pegloticase on renal function in patients with hyperuricemia in the setting of tumor lysis syndrome as measured by changes in serum creatinine/estimated glomerular filtration rate (eGFR) and urine output.
II. To estimate the rate of renal replacement therapy initiation for acute kidney injury secondary to tumor lysis syndrome among patients treated with pegloticase.
OUTLINE:
Patients receive pegloticase intravenously (IV) over 120 minutes. Patients whose serum uric acid does not drop below 6 mg/dL within 24 hours receive a second dose of pegloticase IV over 120 minutes on day 2. Patients whose serum uric acid does not drop below 6 mg/dL after two doses of pegloticase receive standard of care rasburicase IV once daily (QD) for 5 days.
- Being tested
- Pegloticase, Rasburicase
- Medical subject headings
- Hematologic Neoplasms, Tumor Lysis Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PegloticaseDrug
Also called Des-(1-6)-(7-threonine,46-threonine,291-lysine,301-serine)uricase (EC 1.7.3.3, urate oxidase) Sus scrofa (pig) tetramer, Non Acetylated, Carbamates with alpha-carboxy-omega-methoxypoly(oxyethylene), Krystexxa, Methoxypolyethylene Glycol Uricase, Oxidase, Urate (Synthetic Sus scrofa variant pigks-DN subunit), homotetramer, amide with alpha-carboxy-omega-methoxypoly(oxy-1,2-ethanediyl)
Given IV
RasburicaseDrug
Also called Elitek, Recombinant Urate Oxidase, SR 29142, SR-29142
Given IV
| Study group | Role | What they receive |
|---|---|---|
| Treatment (pegloticase) | Experimental | Patients receive pegloticase IV over 120 minutes. Patients whose serum uric acid does not drop below 6 mg/dL within 24 hours receive a second dose of pegloticase IV over 120 minutes on day 2. Patients whose serum uric acid does not drop below 6 mg/dL after two doses of pegloticase receive standard of care rasburicase IV QD for 5 days. Receives: Drug: Pegloticase; Drug: Rasburicase |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response rate
Will estimate the 24-hour response rate along with the corresponding 90% confidence interval. In addition, the report will present plots of uric acid levels across all time points.
Measured: Within 24 hours of single dose of pegloticase
Secondary outcomes (2)
Time from pegloticase infusion until serum uric acid levels drop to 6 mg/dL
Will display results in frequency tables and plots of serum uric acid levels across time.
Measured: Up to 14 days after pegloticase administration
Renal function
Will summarize serum creatinine and estimated glomerular filtration rate at each time point and plot these lab values across time. Finally, will calculate the rate and 90% confidence interval for the initiation of renal replacement therapy within the 14-day observation window.
Measured: Up to 14 days after pegloticase administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Feb 2021
- Study start5 Apr 2022
- Primary completion27 Apr 2026
- Study completion27 Apr 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematopoietic and Lymphoid Cell NeoplasmStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Sreedhar Mandayam, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04745910
- Sponsor's protocol ID
- 2020-0330
- Other IDs
- NCI-2020-13890, 2020-0330
- Status last verified
- Apr 2026
- First submitted
- 3 Feb 2021
- Last updated
- 6 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Pegloticase on Reduction of Uric Acid in Patients With Tumor Lysis Syndrome: A Pilot Pragmatic Clinical Trial
- ClinicalTrials.gov ID
- NCT04745910
- Acronym / sponsor ID
- 2020-0330
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- 5 Apr 2022 (actual)
- Primary completion
- 27 Apr 2026 (actual)
- Study completion
- 27 Apr 2026 (actual)
- First posted
- 9 Feb 2021 (actual)
- 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.
This trial studies the effect of pegloticase in reducing uric acid levels in patients with hyperuricemia (high blood levels of uric acid) caused by tumor lysis syndrome. Tumor lysis syndrome occurs when the breakdown products of cancer cells, such as uric acid, enter the blood stream. High levels of uric acid in blood may cause kidney damage and reduce kidney function. The goal of this trial is to learn if pegloticase may lower uric acid levels in blood when given to cancer patients with hyperuricemia caused by tumor lysis syndrome.
Detailed description
PRIMARY OBJECTIVE:
- To collect preliminary data on the effectiveness of a single dose of pegloticase in lowering serum uric acid levels to less than 6.0 mg/dl within 24 hours in patients with hyperuricemia in the setting of tumor lysis syndrome.
SECONDARY OBJECTIVES:
- To assess the effect of pegloticase on renal function in patients with hyperuricemia in the setting of tumor lysis syndrome as measured by changes in serum creatinine/estimated glomerular filtration rate (eGFR) and urine output.
II. To estimate the rate of renal replacement therapy initiation for acute kidney injury secondary to tumor lysis syndrome among patients treated with pegloticase.
OUTLINE:
Patients receive pegloticase intravenously (IV) over 120 minutes. Patients whose serum uric acid does not drop below 6 mg/dL within 24 hours receive a second dose of pegloticase IV over 120 minutes on day 2. Patients whose serum uric acid does not drop below 6 mg/dL after two doses of pegloticase receive standard of care rasburicase IV once daily (QD) for 5 days.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (pegloticase) | Experimental | Drug: Pegloticase, Drug: RasburicasePatients receive pegloticase IV over 120 minutes. Patients whose serum uric acid does not drop below 6 mg/dL within 24 hours receive a second dose of pegloticase IV over 120 minutes on day 2. Patients whose serum uric acid does not drop below 6 mg/dL after two doses of pegloticase receive standard of care rasburicase IV QD for 5 days. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Signed informed consent prior to any study specific procedures (patient or caregiver)
- Male or female 18 years of age or older
- In the investigator's opinion, expected survival of at least 1 month
- Deemed stable by the investigator
- Serum Uric Acid ≥ 6 mg/dL
Key exclusion criteria (9 listed)
- Patients will be ineligible for study enrollment if any of the following exclusion criteria are fulfilled:
- Inability or refusal to give informed consent (patient or caregiver)
- Subject unwilling to take study medication
- Known allergy to uricase
- Pregnant
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 |
|---|---|---|
| PegloticaseAlso: Des-(1-6)-(7-threonine,46-threonine,291-lysine,301-serine)uricase (EC 1.7.3.3, urate oxidase) Sus scrofa (pig) tetramer, Non Acetylated, Carbamates with alpha-carboxy-omega-methoxypoly(oxyethylene), Krystexxa, Methoxypolyethylene Glycol Uricase, Oxidase, Urate (Synthetic Sus scrofa variant pigks-DN subunit), homotetramer, amide with alpha-carboxy-omega-methoxypoly(oxy-1,2-ethanediyl)Given IV | Drug | Treatment (pegloticase) |
| RasburicaseAlso: Elitek, Recombinant Urate Oxidase, SR 29142, SR-29142Given IV | Drug | Treatment (pegloticase) |
Primary outcome
Response rate
Time frame: Within 24 hours of single dose of pegloticase
Will estimate the 24-hour response rate along with the corresponding 90% confidence interval. In addition, the report will present plots of uric acid levels across all time points.
2 secondary outcomes
Time from pegloticase infusion until serum uric acid levels drop to 6 mg/dL
Time frame: Up to 14 days after pegloticase administration
Renal function
Time frame: Up to 14 days after pegloticase administration
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 May 2026.
- M D Anderson Cancer CenterLink 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.
Sreedhar Mandayam, MD principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Pegloticase and Rasburicase in people with Hematopoietic and Lymphoid Cell Neoplasm and Malignant Solid Neoplasm, run by M.D. Anderson Cancer Center at 1 site 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 7 inclusion and 9 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: finished, as of the sponsor's last update on 6 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Response rate (time frame: Within 24 hours of single dose of pegloticase). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 6 May 2026.