Early discontinuation of steroid treatment in negative FDG-PET/CT patients with idiopathic retroperitoneal fibrosis

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] - Immune System Diseases [C20]

Trial duration

25 Nov 2022 → ongoing

Decision date (initial)

2024-07-30

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

French Ministry

External identifiers

EU CT number

2024-514353-30-00

EudraCT number

2022-001692-13

ClinicalTrials.gov

NCT05428826

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Therapy

To compare the cumulative IRF relapse rate 12 months after discontinuation of steroids.

Secondary objectives 3

1. 1. To analyze the characteristics of hypermetabolism of IRF in FDG-PET/CT and their evolution at diagnosis, remission (M9), M21 and relapse,
2. 2. To assess the performance of hypermetabolism of IRF in FDG-PET/CT for diagnosis of disease activity,
3. 3. To compare at M21 the corticosteroids therapy – related adverses events between patients who continue or discontinue the treatment at M9.

Conditions and MedDRA coding

Idiopathic retroperitoneal fibrosis

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

1. • Patient over 18 years old
2. New onset or untreated relapsing of active idiopathic retroperitoneal fibrosis (IRF) defined by the association of: o Related-disease symptoms (Appendix 17.2) or elevated CRP level (>20 mg/l) AND o Retroperitoneal peri-aortic mass that surrounds the abdominal vessels on CT-scan

Exclusion criteria 15

1. • Secondary retroperitoneal fibrosis including drug-related retroperitoneal fibrosis, active infections (such as tuberculosis) or malignancies, systemic vasculitis (such as ANCA-associated vasculitis), Erdheim-Chester disease (Appendix 17.3), patients with IgG4 disease may be enrolled
2. • Contraindication to perform FDG-PET/CT,
3. • Contraindication to perform CT scan with injection of contrast agent,
4. • Contraindication to treatment by prednisone
5. • Active infection,
6. • Acute or chronic liver disease that is deemed sufficiently severe to impair their ability to participate in the trial,
7. • Active or history of malignancy in last 5 years. Individuals with squamous cell or basal cell skin carcinomas and individuals with cervical carcinoma in situ may be enrolled if they have received curative surgical treatment,
8. • Serum creatinine level greater than 400 µmol/L that cannot be attributed to underlying IRF,
9. • Live vaccination received from 4 weeks before inclusion,
10. • Inhaled glucocorticoids (except for patients with documented asthma),
11. • Any previous treatment with rituximab, methotrexate, alemtuzumab, cyclophosphamide, azathiorpine, mycophenolate mofetil, infliximab, adalimumab, etanercept within the past 3 months,
12. • Pregnancy or breastfeeding,
13. • Non-affiliation to a social security regime,
14. • Subject deprived of freedom, subject under a legal protective measure
15. • Refusal to participate

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary endpoint is the cumulate IRF relapse rate measured at the end of the study (M21). The diagnosis of IRF relapse is based on the association of a clinical or biological criterion with a radiological criterion (i.e. composite criteria):

Secondary endpoints 3

1. 1. a. Visual grades of retroperitoneal fibrosis FDG uptake as compared to liver FDG uptake (which consist of one item that yields a score of 0 to III), maximal standardized uptake value (SUVmax) within the retroperitoneal fibrosis (regions of interest- ROI) at diagnosis (M0), remission (M9), M21 and relapse, b. Metabolic volume (i.e. ratio of metabolically active volume (MAV) to global lesion volume) of retroperitoneal fibrosis FDG uptake at diagnosis (M0), remission (M9), M21 and relapse,
2. 2. Diagnostic performance of SUVmax and MAV (area under the curve (AUC) and performance values for the Youden index) for the disease activity,
3. 3. Frequency of diabetes, severe infection, osteoporotic fracture and major cardiovascular events 12 months after remission (M21). Serious cardiovascular adverse events are defined as a composite of nonfatal stroke, nonfatal myocardial infarction, and cardiovascular death and will be assessed at M12,M15 and M21

Investigational products

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

Test 1

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

SACRE Karim

Public contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

SACRE Karim

Locations

1 EU/EEA country · 15 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

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

Documents 5 files

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

Application history

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