A Phase 4 interventional study of Pegloticase and Methotrexate (MTX) in Gout, sponsored by Amgen. Completed at 6 sites in United States. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2024-06-26.
Sponsored by Amgen · Phase 4, Interventional, and Treatment
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.
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.
232 studies on the registry are indexed under Gout; 45 are open to participants now.
This study's enrollment of 14 is below the median of 121 across 202 interventional studies indexed under Gout.
Browse Gout studies →Amgen is the lead sponsor of 1,015 studies on the registry; 49 are open to participants now.
Of its 245 completed or terminated interventional studies of FDA-regulated products, 159 (65%) have results posted.
Counted across the registry records on this site, refreshed daily.
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)
Exclusion Criteria:
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.
Biological: Pegloticase · Drug: Methotrexate (MTX) · Drug: Standard Gout Flare Prophylaxis · Drug: Infusion Reaction (IR) Prophylaxis · Dietary Supplement: Folic Acid
pegloticase administered intravenously (IV)
oral MTX
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.
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.
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.
Percentage of Serum Uric Acid (sUA < 6 mg/dL) Responders During Month 6
Serum uric acid (sUA \< 6 mg/dL) responders are defined as participants achieving and maintaining sUA \< 6 mg/dL for at least 80% of the time during Month 6 (Weeks 20, 22, and 24). Month 6 includes pre-infusion and post-infusion sUA assessments at Week 20, pre-infusion and post-infusion sUA assessments at Week 22, pre-infusion assessments at Week 24, and unscheduled sUA assessments between Week 20 and Week 24.
Time frame: Month 6 (Weeks 20, 22, and 24)
Percentage of Serum Uric Acid (sUA < 6 mg/dL) Responders During Month 3
Serum uric acid (sUA \< 6 mg/dL) responders are defined as participants achieving and maintaining sUA \< 6 mg/dL for at least 80% of the time during Month 3 (Weeks 10, 12, and 14). Month 3 includes pre-infusion and post-infusion sUA assessments at Week 10, pre-infusion and post-infusion sUA assessments at Week 12, pre-infusion assessments at Week 14, and unscheduled assessments between Week 10 and Week 14 pre-infusion.
Time frame: Month 3 (Weeks 10, 12, and 14)
Percentage of Serum Uric Acid (sUA < 6 mg/dL) Overall Responders
Serum uric acid (sUA \< 6 mg/dL) overall responders are defined as participants achieving and maintaining sUA \< 6 mg/dL for at least 80% of the time during Month 3 and Month 6 (Weeks 10, 12, 14, 20, 22, and 24) combined. Participants with more than one sUA result in Month 3 and Month 6 are considered responders if a participant's weighted proportion of hours that sUA is \< 6 mg/dL is greater than or equal to 80%. Participants with the proportion of hours less than 80% are counted as non-responders. Participants with only one value in Month 3 and Month 6 are considered overall responders if they are considered responders in both Month 3 and Month 6.
Time frame: Month 3 and Month 6 combined (Weeks 10, 12, 14, 20, 22, and 24)
Percentage of Serum Uric Acid (sUA < 5 mg/dL) Responders During Month 3
Serum uric acid (sUA \< 5 mg/dL) responders are defined as participants achieving and maintaining sUA \< 5 mg/dL for at least 80% of the time during Month 3. Month 3 includes pre-infusion and post-infusion sUA assessments at Week 10, pre-infusion and post-infusion sUA assessments at Week 12, pre-infusion assessments at Week 14, and unscheduled assessments between Week 10 and Week 14 pre-infusion.
Time frame: Month 3 (Weeks 10, 12, and 14)
Percentage of Serum Uric Acid (sUA < 5 mg/dL) Responders During Month 6
Serum uric acid (sUA \< 5 mg/dL) responders are defined as participants achieving and maintaining sUA \< 5 mg/dL for at least 80% of the time during Month 6. Month 6 includes pre-infusion and post-infusion sUA assessments at Week 20, pre-infusion and post-infusion sUA assessments at Week 22, pre-infusion assessments at Week 24, and unscheduled sUA assessments between Week 20 and Week 24.
Time frame: Month 6 (Weeks 20, 22, and 24)
Percentage of Serum Uric Acid (sUA < 5 mg/dL) Overall Responders
Serum uric acid (sUA \< 5 mg/dL) overall responders are defined as participants achieving and maintaining sUA \< 5 mg/dL for at least 80% of the time during Month 3 and Month 6 (Weeks 10, 12, 14, 20, 22, and 24) combined. Participants with more than one sUA result in Month 3 and Month 6 are considered responders if a participant's weighted proportion of hours that sUA is \< 6 mg/dL is greater than or equal to 80%. Participants with the proportion of hours less than 80% are counted as non-responders. Participants with only one value in Month 3 and Month 6 are considered overall responders if they are considered responders in both Month 3 and Month 6.
Time frame: Month 3 and Month 6 combined (Weeks 10, 12, 14, 20, 22, and 24)
Mean Change in sUA From Pegloticase Baseline to Weeks 14, 24, 36, 52
The mean change from baseline is based on observed values in participants remaining on treatment at given time point. For sUA values less than the lower limit of detection (up to 1.5 mg/dL), 0 is used in the analysis.
Time frame: Baseline (defined as the last measurement taken prior to the first infusion of pegloticase in the pegloticase + IMM period), Pre- and Post-Infusion at Weeks 14, 24, 36 and Week 52
| Milestone | All Participants |
|---|---|
| Started | 15 |
| Completed | 14 |
| Not completed | 1 |
| Withdrew: Lost to follow-up | 1 |
| Milestone | All Participants |
|---|---|
| Started | 14 |
| Completed study at 24 weeks-amendment 1 | 1 |
| Completed study at 52 weeks-amendment 2 | 10 |
| Discontinued study treatment early | 5 |
| Had 3-month follow-up | 7 |
| Had 6-month follow-up | 6 |
| Completed | 11 |
| Not completed | 3 |
| Withdrew: Lack of efficacy | 3 |
Serum uric acid (sUA \< 6 mg/dL) responders are defined as participants achieving and maintaining sUA \< 6 mg/dL for at least 80% of the time during Month 6 (Weeks 20, 22, and 24). Month 6 includes pre-infusion and post-infusion sUA assessments at Week 20, pre-infusion and post-infusion sUA assessments at Week 22, pre-infusion assessments at Week 24, and unscheduled sUA assessments between Week 20 and Week 24.
| percentage of participants | Pegloticase With Methotrexate (MTX) |
|---|---|
| Percentage of Serum Uric Acid (sUA < 6 mg/dL) Responders During Month 6 | 78.6 (49.2 to 95.3) |
Serum uric acid (sUA \< 6 mg/dL) responders are defined as participants achieving and maintaining sUA \< 6 mg/dL for at least 80% of the time during Month 3 (Weeks 10, 12, and 14). Month 3 includes pre-infusion and post-infusion sUA assessments at Week 10, pre-infusion and post-infusion sUA assessments at Week 12, pre-infusion assessments at Week 14, and unscheduled assessments between Week 10 and Week 14 pre-infusion.
| percentage of participants | Pegloticase With Methotrexate (MTX) |
|---|---|
| Percentage of Serum Uric Acid (sUA < 6 mg/dL) Responders During Month 3 | 78.6 (49.2 to 95.3) |
Serum uric acid (sUA \< 6 mg/dL) overall responders are defined as participants achieving and maintaining sUA \< 6 mg/dL for at least 80% of the time during Month 3 and Month 6 (Weeks 10, 12, 14, 20, 22, and 24) combined. Participants with more than one sUA result in Month 3 and Month 6 are considered responders if a participant's weighted proportion of hours that sUA is \< 6 mg/dL is greater than or equal to 80%. Participants with the proportion of hours less than 80% are counted as non-responders. Participants with only one value in Month 3 and Month 6 are considered overall responders if they are considered responders in both Month 3 and Month 6.
| percentage of participants | Pegloticase With Methotrexate (MTX) |
|---|---|
| Percentage of Serum Uric Acid (sUA < 6 mg/dL) Overall Responders | 78.6 (49.2 to 95.3) |
Serum uric acid (sUA \< 5 mg/dL) responders are defined as participants achieving and maintaining sUA \< 5 mg/dL for at least 80% of the time during Month 3. Month 3 includes pre-infusion and post-infusion sUA assessments at Week 10, pre-infusion and post-infusion sUA assessments at Week 12, pre-infusion assessments at Week 14, and unscheduled assessments between Week 10 and Week 14 pre-infusion.
| percentage of participants | Pegloticase With Methotrexate (MTX) |
|---|---|
| Percentage of Serum Uric Acid (sUA < 5 mg/dL) Responders During Month 3 | 78.6 (49.2 to 95.3) |
Serum uric acid (sUA \< 5 mg/dL) responders are defined as participants achieving and maintaining sUA \< 5 mg/dL for at least 80% of the time during Month 6. Month 6 includes pre-infusion and post-infusion sUA assessments at Week 20, pre-infusion and post-infusion sUA assessments at Week 22, pre-infusion assessments at Week 24, and unscheduled sUA assessments between Week 20 and Week 24.
| percentage of participants | Pegloticase With Methotrexate (MTX) |
|---|---|
| Percentage of Serum Uric Acid (sUA < 5 mg/dL) Responders During Month 6 | 78.6 (49.2 to 95.3) |
Serum uric acid (sUA \< 5 mg/dL) overall responders are defined as participants achieving and maintaining sUA \< 5 mg/dL for at least 80% of the time during Month 3 and Month 6 (Weeks 10, 12, 14, 20, 22, and 24) combined. Participants with more than one sUA result in Month 3 and Month 6 are considered responders if a participant's weighted proportion of hours that sUA is \< 6 mg/dL is greater than or equal to 80%. Participants with the proportion of hours less than 80% are counted as non-responders. Participants with only one value in Month 3 and Month 6 are considered overall responders if they are considered responders in both Month 3 and Month 6.
| percentage of participants | Pegloticase With Methotrexate (MTX) |
|---|---|
| Percentage of Serum Uric Acid (sUA < 5 mg/dL) Overall Responders | 78.6 (49.2 to 95.3) |
The mean change from baseline is based on observed values in participants remaining on treatment at given time point. For sUA values less than the lower limit of detection (up to 1.5 mg/dL), 0 is used in the analysis.
| mg/dL | Pegloticase With Methotrexate (MTX) |
|---|---|
| Change at Week 14 - pre-infusion | -9.27 ± 2.810 |
| Change at Week 14 - post-infusion | -9.31 ± 2.959 |
| Change at Week 24 - pre-infusion | -9.27 ± 2.810 |
| Change at Week 24 - post-infusion | -9.48 ± 4.135 |
| Change at Week 36 - pre-infusion | -8.13 ± 3.975 |
| Change at Week 36 - post-infusion | -9.41 ± 3.293 |
| Change at Week 52 | -8.15 ± 4.086 |
Collected over From first dose of study drug through the end of treatment plus 30 days. Mean duration of MTX Treatment during the MTX Run-in Period was 23.0 days, and during the Pegloticase+IMM Period was 242.4 days. Mean duration of pegloticase treatment during the Pegloticase + IMM Period was 242.4 days.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Run-In Period: MTX | 0/15 (0%) | 0/15 (0%) | 10/15 (66.7%) |
| Pegloticase + IMM Period: Pegloticase + MTX | 0/14 (0%) | 1/14 (7.1%) | 14/14 (100%) |
| Event | Run-In Period: MTX | Pegloticase + IMM Period: Pegloticase + MTX |
|---|---|---|
| Bacterial SepsisInfections and infestations | 0/15 | 1/14 |
| Event | Run-In Period: MTX | Pegloticase + IMM Period: Pegloticase + MTX |
|---|---|---|
| GoutMetabolism and nutrition disorders | 5/15 | 13/14 |
| DiarrhoeaGastrointestinal disorders | 1/15 | 3/14 |
| NasopharyngitisInfections and infestations | 1/15 | 3/14 |
| Upper Respiratory Tract InfectionInfections and infestations | 0/15 | 3/14 |
| Muscle StrainInjury, poisoning and procedural complications | 0/15 | 3/14 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 0/15 | 3/14 |
| SinusitisInfections and infestations | 0/15 | 2/14 |
| HypertensionVascular disorders | 0/15 | 2/14 |
| Abdominal DiscomfortGastrointestinal disorders | 2/15 | 0/14 |
| NauseaGastrointestinal disorders | 2/15 | 1/14 |
Modified intention-to-treat (mITT) population: all enrolled participants who received ≥1 dose of pegloticase.
| Age, Continuous(years) | Pegloticase With Methotrexate (MTX) |
|---|---|
| Mean | 49.3 ± 8.66 |
| Sex: Female, Male(Participants) | Pegloticase With Methotrexate (MTX) |
|---|---|
| Female | 0 |
| Male | 14 |
| Ethnicity (NIH/OMB)(Participants) | Pegloticase With Methotrexate (MTX) |
|---|---|
| Hispanic or Latino | 1 |
| Not Hispanic or Latino | 13 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Pegloticase With Methotrexate (MTX) |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 2 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 12 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Serum Uric Acid (sUA)(mg/dL) | Pegloticase With Methotrexate (MTX) |
|---|---|
| Mean | 9.16 ± 2.486 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: Yes — De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.
Supporting information: Study protocol, Sap, Icf, Csr
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