Pro-RAPID

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

Trial duration

24 May 2024 → ongoing

Decision date (initial)

2024-05-23

Transition trial

Yes

Low-intervention

Yes

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2023-507352-72-00

EudraCT number

2022-002648-35

ClinicalTrials.gov

NCT05552287

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Therapy, Pharmacokinetic

To assess the proportion of patients with IFX trough level ≥ 5 µg/mL at week 12 without treatment escalation

Secondary objectives 1

1. • Proportion of patients with IFX TL ≥ 5 µg/mL at week 24 without the need for treatment escalation • Clinical and biochemical remission at weeks 4, 12, and 24 without the need for treatment escalation in patients with TL ≥ 5 µg/mL and in patients with TL < 5 µg/mL • Predictors of IFX TLs at weeks 4, 12, and 24. Factors included in this analysis will be sex, age, body mass index (BMI), wPCDAI, IBD laboratory values, ATI, dose, and interval of IFX infusions • Development of ATI until week 24 • Prediction of patients who will respond vs. those who will not despite adequate TLs at weeks 12 and 24 based on proteomics analysis by OLINK • Evaluation of quality of life at baseline, week 12, and 24 in all patients • Adverse event rate over time

Conditions and MedDRA coding

Crohn's Disease

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. Anti-TNF-α naïve children (age 1-15 years) with CD or IBD-U and an indication to start IFX treatment will be eligible for inclusion after a diagnosis of CD is made based on the Porto criteria [10]. Indications of starting IFX treatment as per ECCO-ESPGHAN guidelines [9] include non-response after induction with exclusive enteral nutrition or steroids, non-response to immunomodulators, severe growth delay, extensive disease and/or structuring or penetrating disease, with or without perianal disease. Evaluation of the indication to start IFX is performed at the discretion of the attending physician.

Exclusion criteria 1

1. Patients with the following characteristics will be excluded: - Established monogenetic IBD - Diagnosis with UC or IBD-U, ulcerative colitis like - Severe comorbidity (not related to IBD) - Immediate need for surgery (i.e., symptomatic stenosis or stricture in the bowel) - Severe infection such as sepsis or opportunistic infections, positive tuberculin test or a chest radiograph consistent with tuberculosis or malignancy - Pregnancy, suspected or definitive - Treatment with anti-TNF or other biological drugs in the past - Start of corticosteroids or mesalazine less than 2 weeks prior to first IFX infusion - Start of Exclusive Enteral Nutrition less than 2 week prior to first IFX infusion

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary endpoint is the proportion of patients with IFX TL ≥ 5 µg/mL at week 12 without treatment escalation.

Secondary endpoints 1

1. Proportion of patients with IFX TL ≥ 5 µg/mL at week 24 without the need for treatment escalation,proportion of patients in clinical and/or biochemical remission at weeks 4, 12, and week 24 without the need for treatment escalation in patients with TL > 5 µg/mL and in patients with TL < 5 µg/mL, predictors of IFX TLs at weeks 4, 12, and 24

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)

Sponsor organisation

Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)

Address

Dr. Molewaterplein 60

City

Rotterdam

Postcode

3015 GJ

Country

Netherlands

Scientific contact point

Organisation

Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)

Contact name

Johanna Escher

Public contact point

Organisation

Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)

Contact name

Johanna Escher

Locations

1 EU/EEA country · 12 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 7 files

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

Application history

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