Gout ChronicPhase 2RECIPENCT03303989

REduCing Immunogenicity to PegloticasE (RECIPE) Study

A Phase 2 study testing Pegloticase 8 MG/ML [Krystexxa] and MMF in people with Gout Chronic, run by University of Alabama at Birmingham at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Pegloticase treatment for chronic refractory gout is limited by immunogenicity. The investigators propose the REduCing Immunogenicity to PegloticasE (RECIPE) trial to begin to investigate the question of whether a short course of immune modulating therapy with mycophenolate mofetil can significantly and safely… Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Gout Chronic
What is being tested?: Pegloticase 8 MG/ML [Krystexxa] and MMF
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 35 participants
Where: 1 siteUnited States
Study dates: 14 Jun 2018to 31 Mar 2021
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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

Men and women > 18 years of age
Diagnosed with chronic refractory gout*
Defined as: Persons whose signs and symptoms are inadequately controlled with urate lowering therapy (e.g. xanthine oxidase inhibitors or uricosuric agents) at a medically appropriate dose or for whom these drugs are contraindicated.

Who cannot join

Any serious acute bacterial infection (2 weeks prior to Visit 1), unless treated and completely resolved with antibiotics
Severe chronic or recurrent bacterial infections (such as recurrent pneumonia, chronic bronchiectasis)
Current immunocompromised condition, including current or chronic treatment with immunosuppressive agents
Subjects at risk for tuberculosis. Specifically, subjects with: i) current clinical, radiographic or laboratory evidence of active or latent TB; ii) a history of active TB within the last 3 years even if it was treated; iii) a history of active TB greater than 3 years ago unless there is documentation that the prior anti-TB treatment was appropriate in duration and type
Known Hepatitis B surface antigen-positive or Hepatitis B DNA positive subjects
Known Hepatitis C RNA-positive subjects

Show all 23Show fewer

Human Immunodeficiency Virus (HIV) infection
G6PD deficiency (tested at Screening Visit 1)
Severe chronic renal impairment (glomerular filtration rate [GFR] <25 mL/min/1.73 m2) or currently on dialysis
Subjects having any transplant surgery requiring maintenance immunosuppressive therapy
Non-compensated congestive heart failure, uncontrolled arrhythmia, treatment for acute coronary syndrome (myocardial infarction or unstable angina), or hospitalization for congestive heart failure within 3 months of screening or uncontrolled blood pressure (>160/100 mm Hg) at baseline (Screening Visit 1 and Week 0/Baseline visits)
Participants who are pregnant, planning to become pregnant, breastfeeding, or not on an effective form of birth control (defined in Study Protocol section 7.1)
Prior treatment with pegloticase, another recombinant uricase, or concomitant therapy with a polyethylene glycol (PEG)-conjugated drug
Known allergy to pegylated products or history of anaphylactic reaction to a recombinant protein or porcine product
Subjects in whom MMF treatment is contraindicated or considered inappropriate
Recipient of an investigational drug within 4 weeks prior to study drug administration or plans to take an investigational agent during the study
Current liver disease as determined by alanine transaminase ALT or aspartate transaminase (AST) levels >3 times upper limit of normal
Currently receiving treatment for ongoing cancer, excluding non-melanoma skin cancer
History of malignancy within 5 years other than skin cancer or in situ carcinoma of cervix
Uncontrolled hyperglycemia with a plasma glucose value >240 mg/dL at screening
Diagnosed osteomyelitis
Individuals with hypoxanthine-guanine phosphoribosyl-transferase (HGPRT) deficiency such as Lesch-Nyhan and Kelley-Seegmiller syndrome
Not good candidate for the study based on opinion of the Investigator (e.g., cognitive impairment) that might create undue risk to the participant or interfere with the participant's ability to comply with the protocol requirements, or to complete the study

Trial locations and contacts

1 site in 1 country.

United States1 site

University of Alabama at Birmingham
Birmingham, Alabama, 35294

Other trials in United States

About this study

Read the detailed description

Pegloticase is highly efficacious therapy for chronic refractory gout patients (n = > 400K in the US alone). It decreases serum urate (sUA) levels to often undetectable levels and reduces tophi burden. However, its long-term real world effectiveness is severely limited due to its immunogenicity caused by anti-pegloticase antibody formation. REducing Immunogenicity to PEgloticase (RECIPE) is a Phase II, double-blind, placebo controlled, proof-of-concept trial in 32 subjects initiating pegloticase for treatment of chronic refractory gout. RECIPE will investigate the preliminary efficacy and safety of using immune modulating therapy with mycophenolate mofetil (MMF) to prevent immunogenicity conferred by pegloticase. Subjects will be randomized 3:1 to receive MMF vs. placebo in addition to everyone receiving pegloticase. The co-primary aims of the RECIPE trial are to 1) determine if a 12 week course of immune modulating therapy with daily MMF can safely and significantly attenuate immunogenicity to pegloticase as determined by the proportion of participants achieving and maintaining an sUA less than or equal to 6 mg/dL through 12 weeks, compared to concurrent controls, and 2) to assess the incidence and types of adverse events and infusion reaction. After 12 weeks of co-administration, all participants will continue on pegloticase for an additional 12 weeks without combination MMF immune modulating therapy to evaluate the longer term benefits (durability) and safety of this approach. The secondary aims are to: 1) Determine the 6 month durability of immune modulation after discontinuation of the short course of MMF by: a) assessing the absolute change in sUA from baseline to Week 24, and Week 12 to Week 24 and b) determining the proportion of participants with sUA ≤ 6 mg/dL through 24 weeks, and Week 12 to Week 24; 2) Identify and characterize the pegloticase immune response by immunoglobulin isotypes (IgG and IgM), specificities, and antibody titer; and 3) Examine patient reported outcomes (PROs) using the NIH supported Patient Reported Outcomes Measurement Information System (PROMIS) and Gout Impact Scale (GIS) instruments. The University of Alabama at Birmingham (UAB) and the University of Michigan (UM), two large academic gout and immunology research centers, which in aggregate see nearly 10,000 gout patients annually, will serve as the two lead study sites and are very well-positioned to address the clinical and immunologic questions posed.

Conditions: Gout Chronic
Being tested: Pegloticase 8 MG/ML [Krystexxa], MMF, Placebo

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Pegloticase 8 MG/ML [Krystexxa]Drug
Also called Krystexxa
Participants randomized to the pegloticase + MMF arm will start two week run-in on 1) mycophenolate mofetil at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period, Mycophenolate mofetil therapy will continue for 12 weeks at the highest tolerated dose. After the 12-week combination mycophenolate mofetil and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months.
MMFDrug
Also called Cell Cept
Participants randomized to the pegloticase + MMF arm will start two week run-in on 1) mycophenolate mofetil at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period, Mycophenolate mofetil therapy will continue for 12 weeks at the highest tolerated dose. After the 12-week combination mycophenolate mofetil and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months.
PlaceboDrug
Participants randomized to the pegloticase + placebo arm will start two week run-in on 1) placebo at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period. After the 12-week combination placebo and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months.
Pegloticase 8 MG/ML [Krystexxa]Drug
Also called Krystexxa
Participants randomized to the pegloticase + placebo arm will start two week run-in on 1) placebo at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period. After the 12-week combination placebo and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months.

Study groups in NCT03303989
Study group	Role	What they receive
pegloticase + MMF	Experimental	Participants randomized to this arm will receive pegloticase + mycophenolate mofetil. Receives: Drug: Pegloticase 8 MG/ML [Krystexxa]; Drug: MMF
pegloticase + placebo	Placebo comparator	Participants randomized to this arm will receive pegloticase + placebo Receives: Drug: Placebo; Drug: Pegloticase 8 MG/ML [Krystexxa]

What is being measured

The primary outcome is the main question the study is designed to answer.

Proportion of Participants Achieving and Maintaining an sUA ≤ to 6 Milligram Per Deciliter (mg/dL) Through 12 Weeks
Proportion of participants achieving and maintaining an sUA ≤ to 6 mg/dL through 12 weeks, compared to concurrent controls.
Measured: 12 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

14 Jun 201831 Mar 2021

First posted6 Oct 2017
Study start14 Jun 2018
Primary completion27 Apr 2020
Study completion31 Mar 2021

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Gout ChronicStudies of the same condition, open and under way.
More trials run by University of Alabama at BirminghamThe 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 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Model description: Phase II, double blind, placebo controlled multisite proof-of-concept trial in subjects initiating pegloticase for treatment of chronic gout
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 35 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03303989
Sponsor's protocol ID: 300000591
Acronym: RECIPE
Status last verified: Mar 2022
First submitted: 29 Sep 2017
Last updated: 16 Mar 2022
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Undecided

Publications

Khanna PP, Khanna D, Cutter G, Foster J, Melnick J, Jaafar S, Biggers S, Rahman AKMF, Kuo HC, Feese M, Kivitz A, King C, Shergy W, Kent J, Peloso PM, Danila MI, Saag KG. Reducing Immunogenicity of Pegloticase With Concomitant Use of Mycophenolate Mofetil in Patients With Refractory Gout: A Phase II, Randomized, Double-Blind, Placebo-Controlled Trial. Arthritis Rheumatol. 2021 Aug;73(8):1523-1532. doi: 10.1002/art.41731. Epub 2021 May 19.
PubMed 33750034

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Study at a glance

From the registry record, last updated by the sponsor on 16 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: REduCing Immunogenicity to PegloticasE (RECIPE) Study
ClinicalTrials.gov ID: NCT03303989
Acronym / sponsor ID: RECIPE / 300000591
Conditions: Gout Chronic
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 35 participants (actual)
Lead sponsor: University of Alabama at BirminghamUniversity, hospital or other
Collaborators: University of Michigan
Study start: 14 Jun 2018 (actual)
Primary completion: 27 Apr 2020 (actual)
Study completion: 31 Mar 2021 (actual)
First posted: 6 Oct 2017 (actual)
Results: Posted 10 Nov 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 2
Model description: Phase II, double blind, placebo controlled multisite proof-of-concept trial in subjects initiating pegloticase for treatment of chronic gout

Arm	Type	Receives
pegloticase + MMF	Experimental	Drug: Pegloticase 8 MG/ML [Krystexxa], Drug: MMFParticipants randomized to this arm will receive pegloticase + mycophenolate mofetil.
pegloticase + placebo	Placebo comparator	Drug: Placebo, Drug: Pegloticase 8 MG/ML [Krystexxa]Participants randomized to this arm will receive pegloticase + placebo

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (3 listed)

Men and women > 18 years of age
Diagnosed with chronic refractory gout*

Key exclusion criteria (23 listed)

Any serious acute bacterial infection (2 weeks prior to Visit 1), unless treated and completely resolved with antibiotics
Severe chronic or recurrent bacterial infections (such as recurrent pneumonia, chronic bronchiectasis)
Current immunocompromised condition, including current or chronic treatment with immunosuppressive agents
Subjects at risk for tuberculosis. Specifically, subjects with: i) current clinical, radiographic or laboratory evidence of active or latent TB; ii) a history of active TB within the last 3 years even if it was treated; iii) a history of active TB greater than 3 years ago unless there is documentation that the prior anti-TB treatment was appropriate in duration and type
Known Hepatitis B surface antigen-positive or Hepatitis B DNA positive subjects

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
Pegloticase 8 MG/ML [Krystexxa]Also: KrystexxaParticipants randomized to the pegloticase + MMF arm will start two week run-in on 1) mycophenolate mofetil at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period, Mycophenolate mofetil therapy will continue for 12 weeks at the highest tolerated dose. After the 12-week combination mycophenolate mofetil and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months.	Drug	pegloticase + MMF
MMFAlso: Cell CeptParticipants randomized to the pegloticase + MMF arm will start two week run-in on 1) mycophenolate mofetil at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period, Mycophenolate mofetil therapy will continue for 12 weeks at the highest tolerated dose. After the 12-week combination mycophenolate mofetil and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months.	Drug	pegloticase + MMF
PlaceboParticipants randomized to the pegloticase + placebo arm will start two week run-in on 1) placebo at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period. After the 12-week combination placebo and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months.	Drug	pegloticase + placebo
Pegloticase 8 MG/ML [Krystexxa]Also: KrystexxaParticipants randomized to the pegloticase + placebo arm will start two week run-in on 1) placebo at 500 mg/BID or the first week, titrating dose up to 1000mg/BID for the second week of run-in prior to the first infusion; and 2) Pegloticase 8 mg IV every two weeks following 2 week run-in period. After the 12-week combination placebo and pegloticase study period, participants will continue open label pegloticase therapy for an additional three months.	Drug	pegloticase + placebo

Primary outcome

Proportion of Participants Achieving and Maintaining an sUA ≤ to 6 Milligram Per Deciliter (mg/dL) Through 12 Weeks
Time frame: 12 weeks
Proportion of participants achieving and maintaining an sUA ≤ to 6 mg/dL through 12 weeks, compared to concurrent controls.

Results and safety

Results posted to the registry on 10 Nov 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Pegloticase + MMF	24	22
Pegloticase + Placebo	11	10

Primary outcome: Proportion of Participants Achieving and Maintaining an sUA ≤ to 6 Milligram Per Deciliter (mg/dL) Through 12 Weeks

Time frame: 12 weeks. Count of participants. Unit: Participants

Measure	Pegloticase + MMF n = 22	Pegloticase + Placebo n = 10
Yes	19	4
No	3	6

Pegloticase + MMF vs Pegloticase + Placebo: p <0.01, Fisher Exact

Population analysed: 32 participants received at least one dose of pegloticase and were included in modified intention-to-treat analyses.

Adverse events

Time frame: 36 weeks. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Pegloticase + MMF	2 of 22 (9.1%)	15 of 22 (68.2%)	0 of 22
Pegloticase + Placebo	1 of 10 (10%)	7 of 10 (70%)	0 of 10

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 16 Mar 2022.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 33750034Khanna PP, Khanna D, Cutter G, Foster J, Melnick J, Jaafar S, Biggers S, Rahman AKMF, Kuo HC, Feese M, Kivitz A, King C, Shergy W, Kent J, Peloso PM, Danila MI, Saag KG. Reducing Immunogenicity of Pegloticase With Concomitant Use of Mycophenolate Mofetil in Patients With Refractory Gout: A Phase II, Randomized, Double-Blind, Placebo-Controlled Trial. Arthritis Rheumatol. 2021 Aug;73(8):1523-1532. doi: 10.1002/art.41731. Epub 2021 May 19.
Individual participant dataSharing planned: Undecided

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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.

Kenneth G Saag, MD principal investigator

Professor

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Pegloticase 8 MG/ML [Krystexxa] and MMF in people with Gout Chronic, run by University of Alabama at Birmingham 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 3 inclusion and 23 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 16 Mar 2022. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 10 Nov 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Proportion of Participants Achieving and Maintaining an sUA ≤ to 6 Milligram Per Deciliter (mg/dL) Through 12 Weeks (time frame: 12 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03303989, which also lists 1 linked publication.

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 16 Mar 2022.