Stop Treatment in gout

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Assistance Publique Hopitaux De Paris

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nutritional and Metabolic Diseases [C18]

Decision date (initial)

2025-03-07

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

Ministery of Health PHRC 2022

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Efficacy, Safety

to demonstrate that oral ULT withdrawal in patients with gout in remission, monitored with US scans, is not inferior to maintaining oral ULT for the risk of flares at 2 years

Secondary objectives 12

1. • The proportion of patients experiencing one or more flares and mean flare rates
2. • The incidence of US features of gout
3. • The SUA levels
4. • The Outcome Measures in Rheumatology (OMERACT) core outcome domains for long-term gout studies
5. • The incidence of major cardiovascular events
6. • The incidence of comorbidities and mortality
7. • The kidney function (eGFR)
8. • The consumption of colchicine, NSAIDs, steroids over the study period
9. • The tolerance and adverse events of medications
10. • The adherence to ULT
11. • The cost-effectiveness of withdrawal ULT with US monitoring.
12. • To assess the agreement between the local and central reading of ultrasound features

Conditions and MedDRA coding

Gout adult patients

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. • Age ≥ 18 years
2. • Gout, defined according to the 2015 ACR/EULAR classification criteria
3. • No flares for at least 2 years
4. • No tophi
5. • Currently receiving allopurinol or febuxostat taken for at least 2 years and SUA levels ≤ 60 mg/l
6. • No urate deposit on ultrasound (score 0/24) at inclusion visit at both MTPs 1 and knees
7. • Ability to provide informed consent
8. • Health Insurance.

Exclusion criteria 9

1. • Unstable systemic medical condition (e.g., New York Heart Association stage IV heart failure, recent myocardial infarction, advanced cancer)
2. • History of allergy to allopurinol or febuxostat or one of the excipients • Association with
3. • Association with azathioprine, mercaptopurine (cytostatics-antimetabolites)
4. • Contraindications to experimental medicinal products or auxiliary medicinal products
5. • CKD stage 4 (eGFR less than 30 ml/mn/1.73 m2)
6. • Ongoing treatment with uricosurics (benzbromarone and probenecid) or uricase
7. • Patient on SMA (state medical aid-AME)
8. • Participation in other clinical trial on medicinal product for human use
9. • Women of childbearing age.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. the proportion of patients experiencing one or more flares at two years (M24) according to the Gaffo’s criteria.

Secondary endpoints 16

1. • The proportion of patients experiencing one or more flares at M6, M12, M18, M30 and M36 according to both the Gaffo's criteria and the patient-reported Gout Attack Intensity Score (GAIS)
2. • Mean flare rates at M6, M12, M18, M24, M30, M36 according to both the Gaffo's criteria and the patient-reported Gout Attack Intensity Score (GAIS)
3. • US features of gout at both MPT1s (double contour sign, tophi, aggregates) and both knees (double contour sign) at M6, M12, M18, M24, M30, M36 in the withdrawal arm and at M24 in the maintenance arm.
4. • Urate levels at M6, M12, M18, M24, M30, M36
5. • Change from baseline to M6, M12, M18, M24, M30 and M36 in Outcome Measures in Rheumatology (OMERACT) core outcome domains for long-term gout studies: Pain (VAS 0–100 mm), patient’s global assessment of disease activity (VAS 0–100mm); Health-related quality of life using the EuroQol 5-domain-3L (EQ-5D-3L) questionnaire; Activity limitation using the Health Assessment Questionnaire II
6. • Incidence of Major CardioVascular events (nonfatal stroke, nonfatal myocardial infarction, cardiovascular death) at M6, M12, M18, M24, M30, M36
7. • Renal function (eGFR) at M6, M12, M18, M24, M30, M36
8. • Incidence of comorbidities at M6, M12,M18, M24,M30 and M36
9. • Overall Survival at M12, M24 and M36
10. • Consumption of colchicine, NSAIDs, steroids using a patient self-reported notebook at M6, M12, M18, M24, M30, M36
11. • Adverse events and serious adverse events at M6, M12, M18, M24, M30, M36 according to the CTCAE V5.0 classification
12. • Adherence to ULT by the questionnaire MARS at M6, M12, M18, M24, M30 and M36
13. • Incremental Cost effectiveness ratios estimating cost per QALY gained
14. • Incremental Cost effectiveness ratios estimating cost per flare avoided.
15. Objective of any potential ancillary study: Blood samples for translational researches will be taken only from patients recruited at the Lariboisière centre at D0, M6, M12 and M24. The aim is to study the effects of the increase in urate levels on the innate immune system, the inflammatory pathways, particularly those involving monocytes, the proteome, the metabolome, the lipidome, the transcriptome and the epigenetic.
16. • Agreement between the local and central reading of ultrasound features through the study period (M0 to M36): kappa value for the DC sign, tophi and aggregates at MTP1 and knees.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Assistance Publique Hopitaux De Paris

Sponsor organisation

Assistance Publique Hopitaux De Paris

Address

Porte 23, 1 Avenue Claude Vellefaux 1 Avenue Claude Vellefaux

City

Paris Cedex 10

Postcode

75475

Country

France

Scientific contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

Pr Pascal RICHETTE

Public contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

Pr Pascal RICHETTE

Locations

1 EU/EEA country · 23 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 12 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications