A Study to Assess Safety and Effectiveness of Adalimumab for Treating Children and Adolescents With Crohn's Disease in Real Life Conditions

Study Overview
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Brief Summary
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The primary objective of this study is to evaluate long-term effectiveness of adalimumab in pediatric participants starting a treatment for Crohn's disease in real life conditions, namely to describe the time to loss of clinical benefit in a time to event approach. Main secondary objectives are to describe growth and pubertal development and to describe long-term safety. The participants will be followed-up up to 10 years.

Official Title
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Assessing Long-term Safety and Effectiveness of Adalimumab for Treating Children and Adolescents With Crohn's Disease in Real Life Conditions-LEA

Conditions
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Crohn's Disease

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: With confirmed diagnosis of Crohn's disease Adalimumab-naïve patient (a patient having received an anti-TNF other than adalimumab may enter the study) Starting a treatment with adalimumab Guardian capable of and willing to grant authorization for use/disclosure of data collected and patient able to comply with the requirements of the study protocol. Exclusion Criteria: Participants with a history of treatment with adalimumab Participants enrolled in a concomitant interventional clinical trial.

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Pediatric participants receiving adalimumab<br>Pediatric participants receiving adalimumab for CD in real-life conditions. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Time to loss of clinical benefit | Loss of clinical benefit will be defined as one of the following: Loss of efficacy leading to adalimumab discontinuation or Introduction / reinforcement of other immunosuppressants (ratio dose/weight) or Introduction / reinforcement of corticosteroids (ratio dose/weight; reinforcement of corticosteroids are allowed within the 4 first months after start of adalimumab) Introduction of enteral nutrition CD-related surgery, discontinuation of adalimumab due to adverse event, death. | Up to 12 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of participants with dose escalation (dose and/or frequency of injections) | Dosing and/or frequency of injections is monitored to assess dose escalation. | Up to 12 years | 
| Median percent change from baseline in C-reactive protein (CRP) | The median percent change from baseline in CRP is assessed at each time point. | From Month 0 to 12 years | 
| Median percent change from baseline in calprotectin | The median percent change from baseline in calprotectin us assessed at each time point. | From Month 0 to 12 years | 
| Change from baseline in weighted Pediatric Crohn's Disease Activity Index (PCDAI) | The Pediatric Activity Index (PCDAI) has become the standard outcome measure in pediatric Crohn's disease (CD) clinical research. The Weighted Pediatric Crohn's Disease Activity Index (wPCDAI) was developed to add weight to the items in the PCDAI and make it more feasible. In the wPCDAI, growth velocity, abdominal examination, and hematocrit are removed. The wPCDAI score can range from 0-125, with higher signifying severe disease activity. | From Month 0 to 12 years | 
| Change in wPCDAI >= 37.5 | A change in wPCDAI >= 37.5 indicates improvement. | From Month 0 to 12 years | 
| Incidence rate of CD-related hospitalizations | Hospitalization will be determined from the health care utilization information. | Up to 12 years | 
| Rate of clinical remission | Clinical remission is weighted PCDAI < 12.5 or Harvey-Bradshaw index (HBI) <5. Rate of clinical remission will be described at each time point | Up to 12 years | 
| Proportion of participants achieving mucosal healing at each time point | Mucosal healing is assessed using SES-CD score (0 or 1). | Up to 12 years | 
| Proportion of participants with steroid-free clinical remission at each time point | The proportion of participants with steroid-free clinical remission is assessed at each time point. | Up to 12 years | 
| Change in weight z-score | Growth is assessed by monitoring changes in weight z-score. | From Month 0 to 12 years | 
| Median percent change from baseline in high sensitivity C-reactive protein (hs-CRP) | The median percent change from baseline in hs-CRP is assessed at each time point. | From Month 0 to 12 years | 
| Assessing Mucosal healing | Mucosal healing is assessed using Simple Endoscopic Score for Cronh's Disease (SES-CD) score (0 or 1). | Up to 12 years | 
| Rate of steroid-free remission | Steroid-free remission is defined as weighted PCDAI < 12.5 or HBI <5 and no daily intake of prednisone (whatever the route). Rate of steroid-free remission will be described at each time point. | Up to 12 years | 
| Proportion of participants with fistula remission (in participants with fistulizing CD at entry) | Fistula remission is defined as closure for at least 2 consecutive visits of all fistulae that were draining at baseline | Up to 12 years | 
| Change in Tanner's staging | Tanner's staging is used to assess growth and pubertal development. | From Month 0 to 12 years | 
| Proportion of participants with immunomodulator-free clinical remission at each time point | The proportion of participants with immunomodulator-free clinical remission is assessed at each time point. | Up to 12 years | 
| Incidence rate of infectious events | The incidence rate of serious and non-serious opportunistic infections is assessed. | Up to 12 years | 
| Incidence rate of all-cause hospitalizations | Hospitalization will be determined from the health care utilization information. | Up to 12 years | 
| Proportion of participants with steroid tapering at each time point (steroids daily dosing lower than at baseline) | The proportion of participants with steroid tapering i.e., steroids daily dosing lower than at baseline (week 0) is assessed. | Up to 12 years | 
| Change in height z-score | Growth is assessed by monitoring changes in height z-score | From Month 0 to 12 years | 
| Proportion of participants with CD-related surgery | CD-related surgery includes subtotal colectomy with ileorectostomy, colectomy with ileo-anal pouch, Koch pouch, ileostomy, small bowel resection, and etc. | Up to 12 years | 
| Incidence rate of CD- or drug-related hospitalizations | Hospitalization will be determined from the health care utilization information. | Up to 12 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Crohn's Disease, Adalimumab, Humira®