Study of Pegloticase (KRYSTEXXA®) Plus Methotrexate in Patients With Uncontrolled Gout

NCT ID: NCT03635957

Last Updated: 2024-06-26

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

14 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-09-26

Study Completion Date

2020-10-26

Brief Summary

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The overall objective of the study is to assess the efficacy, safety, tolerability, and pharmacokinetics (PK) of the concomitant use of pegloticase with methotrexate (MTX) to enhance the response rate seen with pegloticase alone in adults with uncontrolled gout.

Detailed Description

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The study design will include: 1) up to a 2-week Screening Period (screening should be complete within 2 weeks prior to Week -4), 2) a 4-week MTX Run in Period (Week - 4 through Day 1); 3) a 52-week Pegloticase + IMM (immunomodulator), (Pegloticase + MTX) Period 4) a Safety Follow-up (Phone/Email/Site Visit) and 5) a 3 and 6 month Post Treatment Follow-up.

Study acquired from Horizon in 2024.

Conditions

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Gout

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Pegloticase With Methotrexate (MTX)

Run-In Period: oral MTX at a dose of 15 mg weekly for 4 weeks prior to the first dose of pegloticase.

Pegloticase + Immunomodulator (IMM) Period: pegloticase 8 mg administered intravenously (IV) every 2 weeks from Day 1 through the Week 50 Visit for a total of 26 infusions. MTX 15 mg weekly on the same day each week, within 1 to 3 days prior to each pegloticase infusion and one additional weekly dose after the last infusion.

Group Type EXPERIMENTAL

Pegloticase

Intervention Type BIOLOGICAL

pegloticase administered intravenously (IV)

Methotrexate (MTX)

Intervention Type DRUG

oral MTX

Standard Gout Flare Prophylaxis

Intervention Type DRUG

It is required that before a subject begins the Pegloticase + IMM Period, he or she has been taking at least one protocol standard gout flare prophylaxis regimen (i.e. colchicine and/or nonsteroidal anti-inflammatory drugs and/or low-dose prednisone ≤10 mg/day) for ≥1 week before the first dose of pegloticase and continues flare prophylaxis per American College of Rheumatology guidelines \[Khanna D et al.2012\] for the greater of 1) 6 months, 2) 3 months after achieving target serum urate (sUA \< 6 mg/dL) for patients with no tophi detected on physical exam, or 3) 6 months after achieving target serum urate (sUA \< 5 mg/dL) for patients with one or more tophi detected on initial physical exam that have since resolved.

Infusion Reaction (IR) Prophylaxis

Intervention Type DRUG

For IR prophylaxis, fexofenadine (60 mg or 180 mg orally based on the Principal Investigator's discretion) will be taken the day before each infusion; fexofenadine (60 mg or 180 mg orally based on the Principal Investigator's discretion) and acetaminophen (1000 mg orally) will be taken the morning of each infusion; and methylprednisolone (125 mg IV) given over the infusion duration 10-30 minutes (recommended) or hydrocortisone (200 mg IV) will be administered immediately prior to each infusion.

Folic Acid

Intervention Type DIETARY_SUPPLEMENT

Subjects will also take folic acid 1 mg orally every day beginning at Week -4 (the start of MTX) and continuing until prior to the Week 52 Visit.

Interventions

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Pegloticase

pegloticase administered intravenously (IV)

Intervention Type BIOLOGICAL

Methotrexate (MTX)

oral MTX

Intervention Type DRUG

Standard Gout Flare Prophylaxis

It is required that before a subject begins the Pegloticase + IMM Period, he or she has been taking at least one protocol standard gout flare prophylaxis regimen (i.e. colchicine and/or nonsteroidal anti-inflammatory drugs and/or low-dose prednisone ≤10 mg/day) for ≥1 week before the first dose of pegloticase and continues flare prophylaxis per American College of Rheumatology guidelines \[Khanna D et al.2012\] for the greater of 1) 6 months, 2) 3 months after achieving target serum urate (sUA \< 6 mg/dL) for patients with no tophi detected on physical exam, or 3) 6 months after achieving target serum urate (sUA \< 5 mg/dL) for patients with one or more tophi detected on initial physical exam that have since resolved.

Intervention Type DRUG

Infusion Reaction (IR) Prophylaxis

For IR prophylaxis, fexofenadine (60 mg or 180 mg orally based on the Principal Investigator's discretion) will be taken the day before each infusion; fexofenadine (60 mg or 180 mg orally based on the Principal Investigator's discretion) and acetaminophen (1000 mg orally) will be taken the morning of each infusion; and methylprednisolone (125 mg IV) given over the infusion duration 10-30 minutes (recommended) or hydrocortisone (200 mg IV) will be administered immediately prior to each infusion.

Intervention Type DRUG

Folic Acid

Subjects will also take folic acid 1 mg orally every day beginning at Week -4 (the start of MTX) and continuing until prior to the Week 52 Visit.

Intervention Type DIETARY_SUPPLEMENT

Eligibility Criteria

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Inclusion Criteria

1. Willing and able to give informed consent.
2. Willing and able to comply with the prescribed treatment protocol and evaluations for the duration of the study.
3. Adult men or women ≥18 to ≤65 years of age.
4. Women of childbearing potential (including those with an onset of menopause \<2 years prior to screening, non-therapy-induced amenorrhea for \<12 months prior to screening, or not surgically sterile \[absence of ovaries and/or uterus\]) must have negative serum/urine pregnancy tests during the Screening/(methotrexate) MTX Run in Period; participants must agree to use 2 reliable forms of contraception during the study, one of which is recommended to be hormonal, such as an oral contraceptive. Hormonal contraception must be started ≥1 full cycle prior to Week -4 (start of MTX dosing) and continue for 30 days after the last dose of pegloticase or at least one ovulatory cycle after the last dose of MTX (whichever is the longest duration after the last dose of pegloticase or MTX). Highly effective contraceptive methods (with a failure rate \<1% per year), when used consistently and correctly, include implants, injectables, combined oral contraceptives, some intrauterine devices, sexual abstinence, or vasectomized partner.
5. Men who are not vasectomized must not impregnant their female partner during the study and for at least 3 months after the last dose of MTX.
6. Hyperuricemia at the Screening, Week -4, or Week -2 Visit of the Screening/MTX Run in Period, as documented by sUA ≥6 mg/dL.
7. Uncontrolled gout, defined as meeting the following criteria:

serum uric acid (sUA) ≥6 mg/dL prior to entry into the pegloticase +IMM Period (any laboratory tests during screening up to and including during the MTX Run in Period) and at least 1 of the following: inability to maintain sUA \<6 mg/dL on other urate-lowering therapy; intolerable side effects associated with current urate-lowering therapy; functionally limiting tophaceous deposits (including those detected clinically or by dual-energy computed tomography \[DECT\] imaging)
8. Able to tolerate MTX 15 mg for 4 weeks during the MTX Run-in Period prior to the first dose of pegloticase.

Exclusion Criteria

1. Weight \>160 kg (352 pounds).
2. Any serious acute bacterial infection, unless treated and completely resolved with antibiotics at least 2 weeks prior to the Week -4 Visit of the MTX Run-in Period.
3. Severe chronic or recurrent bacterial infections, such as recurrent pneumonia or chronic bronchiectasis.
4. Current immunocompromised condition, including current or chronic treatment with systemic immunosuppressive agents, including prednisone \>10 mg/day or equivalent dose of other corticosteroid.
5. History of any transplant surgery requiring maintenance immunosuppressive therapy.
6. Known history of hepatitis B virus surface antigen positivity or hepatitis B DNA positivity.
7. Known history of hepatitis C virus RNA positivity.
8. Human immunodeficiency virus (HIV) positivity (tested at the Screening Visit).
9. Glucose-6-phosphate dehydrogenase (G6PD) deficiency (tested at the Screening Visit).
10. Severe chronic renal impairment (glomerular filtration rate \<25 mL/min/1.73 m\^2) or currently on dialysis.
11. Non-compensated congestive heart failure or hospitalization for congestive heart failure within 3 months of the Screening Visit, uncontrolled arrhythmia, treatment for acute coronary syndrome (myocardial infarction or unstable angina), or uncontrolled blood pressure (\>160/100 mmHg) at the end of the Screening/MTX Run-in Period.
12. Pregnant, planning to become pregnant, breastfeeding, planning to impregnant female partner, or not on an effective form of birth control, as determined by the Investigator.
13. Prior treatment with pegloticase (KRYSTEXXA®), another recombinant uricase (rasburicase), or concomitant therapy with a polyethylene glycol-conjugated drug.
14. Known allergy to pegylated products or history of anaphylactic reaction to a recombinant protein or porcine product.
15. Contraindication to MTX treatment or MTX treatment considered inappropriate.
16. Known intolerance to MTX.
17. Receipt of an investigational drug within 4 weeks or 5 half-lives, whichever is longer, prior to MTX administration at Week -4 or plans to take an investigational drug during the study.
18. Current liver disease, as determined by alanine transaminase or aspartate transaminase levels \>3 times upper limit of normal at the Screening Visit.
19. Currently receiving systemic or radiologic treatment for ongoing cancer, excluding non melanoma skin cancer.
20. History of malignancy within 5 years other than non-melanoma skin cancer or in situ carcinoma of cervix.
21. Uncontrolled hyperglycemia with a plasma glucose value \>240 mg/dL at screening that is not subsequently controlled by the end of the Screening/MTX Run-in Period.
22. Diagnosis of osteomyelitis.
23. Known history of hypoxanthine-guanine phosphoribosyl-transferase deficiency, such as Lesch-Nyhan and Kelley-Seegmiller syndrome.
24. Unsuitable candidate for the study, based on the opinion of the Investigator (e.g., cognitive impairment), such that participation might create undue risk to the participant or interfere with the participant's ability to comply with the protocol requirements or complete the study.
25. Alcohol use in excess of 3 alcoholic beverages per week.
26. Currently receiving allopurinol and unable to discontinue medication 7 days prior to MTX dosing at Week -4 and unable to discontinue treatment during the duration of the study.
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Amgen

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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MD

Role: STUDY_DIRECTOR

Amgen

Locations

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Orthopedic Physicians Alaska

Anchorage, Alaska, United States

Site Status

Arizona Arthritis & Rheumatology -West Valley

Glendale, Arizona, United States

Site Status

Arizona Arthritis & Rheumatology -East Valley

Mesa, Arizona, United States

Site Status

Avail Clinical Research

DeLand, Florida, United States

Site Status

Western Washington Arthritis Clinic

Bothell, Washington, United States

Site Status

Arthritis Northwest PLLC

Spokane, Washington, United States

Site Status

Countries

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United States

References

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Botson JK, Obermeyer K, LaMoreaux B, Zhao L, Weinblatt ME, Peterson J. Improved joint and patient-reported health assessments with pegloticase plus methotrexate co-therapy in patients with uncontrolled gout: 12-month exploratory outcomes of the MIRROR open-label trial. Arthritis Res Ther. 2022 Dec 27;24(1):281. doi: 10.1186/s13075-022-02979-4.

Reference Type DERIVED
PMID: 36575505 (View on PubMed)

Botson JK, Tesser JRP, Bennett R, Kenney HM, Peloso PM, Obermeyer K, Song Y, LaMoreaux B, Zhao L, Xin Y, Chamberlain J, Ramanathan S, Weinblatt ME, Peterson J. A multicentre, efficacy and safety study of methotrexate to increase response rates in patients with uncontrolled gout receiving pegloticase (MIRROR): 12-month efficacy, safety, immunogenicity, and pharmacokinetic findings during long-term extension of an open-label study. Arthritis Res Ther. 2022 Aug 25;24(1):208. doi: 10.1186/s13075-022-02865-z.

Reference Type DERIVED
PMID: 36008814 (View on PubMed)

Dalbeth N, Becce F, Botson JK, Zhao L, Kumar A. Dual-energy CT assessment of rapid monosodium urate depletion and bone erosion remodelling during pegloticase plus methotrexate co-therapy. Rheumatology (Oxford). 2022 Nov 28;61(12):4898-4904. doi: 10.1093/rheumatology/keac173.

Reference Type DERIVED
PMID: 35293984 (View on PubMed)

Botson JK, Tesser JRP, Bennett R, Kenney HM, Peloso PM, Obermeyer K, LaMoreaux B, Weinblatt ME, Peterson J. Pegloticase in Combination With Methotrexate in Patients With Uncontrolled Gout: A Multicenter, Open-label Study (MIRROR). J Rheumatol. 2021 May;48(5):767-774. doi: 10.3899/jrheum.200460. Epub 2020 Sep 15.

Reference Type DERIVED
PMID: 32934137 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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HZNP-KRY-201

Identifier Type: -

Identifier Source: org_study_id

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