A Study to Evaluate the Safety, Tolerability, PK, and PD Properties of PRX-115 in Adult Volunteers With Elevated Uric Acid Levels

NCT05745727 | Completed Phase 1

• 1 other identifier: PB115-SAD-101
• Study Type: interventional
• Target: 64
• Locations: 1 country
• Sites: 2

Brief Summary

This is a Phase 1, double-blind, placebo-controlled, single ascending dose study in participants with elevated uric acid levels. This study will be conducted in approximately 64 adult male and female participants in the dose escalation phase.

Conditions

Gout

Keywords

Uricase; Gout; Uric acid; PRX-115; hyperuricemia

Outcome Measures

Primary Outcomes (5)

• Number of participants with adverse events receiving PRX-115 compared to placebo

  To assess the safety and tolerability of a single infusion of PRX-115 as assessed by frequency of drug related adverse events, graded by severity.

  Day 0 - Day 85

• Number of participants with abnormal clinically significant clinical laboratory results

  Clinical laboratory tests include hematology, coagulation and biochemistry

  Day 0 - Day 85

• Number of participants with abnormal clinical vital signs

  Vital signs include pulse rate, blood pressure, respiratory rate and tympanic temperature

  Day 0 - Day 85

• Number of participants with abnormal clinically significant results from physical examination

  Day 0 - Day 85

• Number of participants with abnormal clinically significant 12-lead electrocardiogram (ECG) parameters

  Day 0 - Day 85

Secondary Outcomes (9)

• PK of PRX-115: Maximum observed plasma drug concentration (Cmax)

  Day 1 - Day 85

• PK of PRX-115: Area under the plasma concentration versus time curve (AUC 0-t)

  Day 1 - Day 85

• PK of PRX-115: Time to maximum observed plasma drug concentration (Tmax)

  Day 1 - Day 85

• PK of PRX-115: total body clearance (CL)

  Day 1 - Day 85

• PK of PRX-115: volume of distribution during the terminal phase (Vd)

  Day 1 - Day 85

Study Arms (2)

PRX-115

EXPERIMENTAL

Participants will receive a single dose of PRX-115 by IV infusion

Drug: PRX-115

Placebo

PLACEBO COMPARATOR

Participants will receive a single dose of placebo by IV infusion

Drug: Placebo

Interventions

PRX-115 DRUG

Escalating doses of PRX-115 will be given in different cohorts i.e., Cohorts 1 through 8

Also known as: Escalating doses of PRX-115

PRX-115

Placebo DRUG

Escalating doses of Placebo will be given in different cohorts i.e., Cohorts 1 through 8

Also known as: Placebo to match

Placebo

Eligibility Criteria

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

You may qualify if:

• Males or females 18 to 65 years of age, inclusive.
• Serum uric acid greater than 6.0 mg/dL (0.35 mmol/L) at the Screening visit.
• Body mass index within the range 18.5 to 40 kg/m\^2, inclusive, at the Screening visit.
• Women of childbearing potential may be included only if they have a negative beta human chorionic gonadotropin (β-hCG) test result at Screening.
• Men and women of childbearing potential and their partners should use double barrier contraception.

You may not qualify if:

• Has any condition known to have arthritis as a clinical manifestation
• Had greater than or equal to 1 gout flare in the last year prior to either Screening or Day -1.
• Has clinical evidence of subcutaneous tophi at either Screening or Day -1.
• Estimated glomerular filtration rate (eGFR) value less than or equal to 60 mL/min/1.73m\^2
• History of significant renal disease, and/or presence of renal stones at either Screening or Day -1.
• Has a history of anaphylaxis, severe allergic reactions, or severe atopy.
• History of autoimmune disorders, and/or participant is immunocompromised or treated with immunosuppressive medications.
• Has evidence of cardiovascular or cerebrovascular disease.
• History of congestive heart failure, New York Heart Association Class III or IV.
• BP outside the range of 90 to 150 mm Hg for systolic or 50 to 95 mm Hg for diastolic.
• Participants with hypertension who are not on stable medication for at least 6 months.
• Has uncontrolled type 2 diabetes
• Concurrent treatment with urate lowering drugs (ULDs).
• Prior exposure to any experimental or marketed uricase (eg, rasburicase \[Elitek, Fasturtec\], pegloticase \[Krystexxa®\], pegadricase \[SEL-212\]).
• Glucose-6-phosphate dehydrogenase (G6PD) deficiency or known catalase deficiency.

Sponsors & Collaborators

• Protalix: lead

Study Sites (2)

New Zealand Clinical Research

Grafton, Auckland, 1010, New Zealand

Location

New Zealand Clinical Research

Christchurch, Christchurch, 8011, New Zealand

Location

MeSH Terms

Conditions

Gout; Hyperuricemia

Condition Hierarchy (Ancestors)

Arthritis; Joint Diseases; Musculoskeletal Diseases; Crystal Arthropathies; Rheumatic Diseases; Purine-Pyrimidine Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Mark Marshall, Dr.

  New Zealand Clinical Research

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 3, 2023
• First Posted: February 27, 2023
• Study Start: March 23, 2023
• Primary Completion: August 26, 2024
• Study Completion: February 6, 2025
• Last Updated: February 11, 2025

Record last verified: 2025-02

Locations

NZ (2)