Pegloticase for the Reduction of Uric Acid in Patients With Tumor Lysis Syndrome

NCT04745910 | Completed Phase 4 DMC FDA Drug

• 2 other identifiers: 2020-0330NCI-2020-13890
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Hematopoietic and Lymphoid Cell Neoplasm; Malignant Solid Neoplasm; Tumor Lysis Syndrome

Outcome Measures

Primary Outcomes (1)

• 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.

  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

  Up to 14 days after pegloticase administration

• Renal function

  Up to 14 days after pegloticase administration

Other Outcomes (1)

• Serum uric acid level

  Up to 14 days after pegloticase administration

Study Arms (1)

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.

Drug: Pegloticase; Drug: Rasburicase

Interventions

Pegloticase DRUG

Given IV

Also known as: 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), Polyethylene Glycol-uricase, Puricase

Treatment (pegloticase)

Rasburicase DRUG

Given IV

Also known as: Elitek, Recombinant Urate Oxidase, SR 29142, SR-29142, Urate Oxidase

Treatment (pegloticase)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• Inability or refusal to give informed consent (patient or caregiver)
• Subject unwilling to take study medication
• Known allergy to uricase
• Pregnant
• Breastfeeding
• 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)

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead

Study Sites (1)

M D Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Links

• M D Anderson Cancer Center

MeSH Terms

Conditions

Hematologic Neoplasms; Tumor Lysis Syndrome

Interventions

Pegloticase; Urate Oxidase; Carbamates; Oxidoreductases; Uric Acid; Amides; rasburicase

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Intervention Hierarchy (Ancestors)

Enzymes; Enzymes and Coenzymes; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Xanthines; Alkaloids; Heterocyclic Compounds; Purinones; Purines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Sreedhar Mandayam, MD

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 3, 2021
• First Posted: February 9, 2021
• Study Start: April 5, 2022
• Primary Completion: April 27, 2026
• Study Completion: April 27, 2026
• Last Updated: May 6, 2026

Record last verified: 2026-04

Locations

US (1)