A Safety Study of Ustekinumab in the Treatment of Pediatric Participants Aged 6 Years and Older With Moderate to Severe Plaque Psoriasis

Study Overview
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Brief Summary
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The purpose of this study is to monitor the long-term safety of ustekinumab in pediatric participants (6 years to 17 years of age at the time of inclusion) with moderate to severe plaque psoriasis, through monitoring for the following adverse events potentially related to immune modulation: serious infections, malignancies and autoimmunity; and to monitor the long-term effects of ustekinumab on growth (weight, height, body mass index) and development (sexual maturity based on the Tanner Scale).

Official Title
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An Observational Post-authorization Safety Study of Ustekinumab in the Treatment of Pediatric Patients Aged 6 Years and Older With Moderate to Severe Plaque Psoriasis

Conditions
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Psoriasis

Intervention / Treatment
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* Drug: Ustekinumab


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Have a confirmed diagnosis of moderate to severe chronic plaque psoriasis Either start therapy with ustekinumab for the treatment of psoriasis within 2 months after the first assessment in the study or have started therapy with ustekinumab in the 12-week period before the first assessment in the study; a. the treatment decision must have been taken independently of and prior to a participant's inclusion in the study; b. where participants have started therapy with ustekinumab before the first assessment in the study, appropriate baseline data at the start of ustekinumab treatment must be documented, including psoriasis area and severity index (PASI), physician global assessment of disease (PGA), body surface area (BSA) and children's dermatology life quality index (CDLQI) scores where available Participants (and/or a legally-acceptable representative/guardian where applicable) must sign a participation agreement/informed consent form (ICF) allowing source data collection and verification in accordance with local requirements and the participants (and/or a legally-acceptable representative/guardian where applicable) must be able to understand and complete the requested patient-reported outcomes (PROs) Be willing to participate in the study Exclusion Criteria: Is enrolled in an 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 |
| --- | --- |
| Participants 6-18 years of Age With Moderate to Severe Plaque Psoriasis<br>All Participants diagnosed with moderate to severe plaque psoriasis who will either start therapy with ustekinumab within 2 months after the first assessment in the study or have started therapy with ustekinumab in the 12-week period before the first assessment in the study as per routine clinical practice, will be monitored for the long-term safety of ustekinumab and long-term effects of ustekinumab on growth and development. The primary data source for the study will be the medical records of participants and standardized questionnaires (completed by the physician and by the participant/parent). | Drug: Ustekinumab<br>* Participants will not receive any intervention as part of this study. Participants with moderate to severe plaque psoriasis who are initiating treatment with ustekinumab in clinical practice (patients should either start therapy with ustekinumab within 2 months after the first assessment in the study or have started therapy with ustekinumab in the 12-week period before the first assessment in the study for the treatment of psoriasis) will be observed for the long-term safety of ustekinumab and the long-term effects of ustekinumab on growth and development.<br>* Other names: STELARA;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Adverse Events | An adverse event is any untoward medical occurrence in a patient administered a medicinal product. An adverse event does not necessarily have a causal relationship with the treatment. An adverse event can be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal product, whether or not related to that medicinal product. All participants will be monitored for the long-term safety of ustekinumab for the frequency and severity of adverse events potentially related to immune modulation and of clinical interest such as: serious infections, malignancies, and autoimmunity. | Baseline up to end of data collection (approximately 8 years) | 
| Evaluation of Growth: Height | Growth will be based on height recorded at baseline and throughout the observational period. | Baseline up to end of data collection (approximately 8 years) | 
| Evaluation of Growth: Weight | Growth will be based on body weight recorded at baseline and throughout the observational period. | Baseline up to end of data collection (approximately 8 years) | 
| Evaluation of Growth: Body Mass Index (BMI) | Growth will be based on body weight recorded at baseline and throughout the observational period. Sex and age adjusted BMI will be calculated by dividing the body weight (in kilograms) by the square of height (in meters). | Baseline up to end of data collection (approximately 8 years) | 
| Sexual Maturity Based on the Tanner scale | The Tanner scale is used to measure visible changes during puberty commonly referred to as Tanner stages. Female participants are evaluated for breast development and pubic hair distribution and male participants are evaluated for development of external genitalia and pubic hair distribution, based on a 5-stage ordinal scale ranging from TS 1 (prepubertal/preadolescent characteristics) to TS 5 (mature or adult characteristics). | Baseline up to end of data collection (approximately 8 years) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants Achieving Psoriasis Area and Severity Index (PASI) 50 Response | The PASI is a measure for assessing and grading the severity and extent of psoriatic lesions and their response to therapy. The PASI measure also accounts for body surface area of psoriasis involvement. In the PASI measure, the body is divided into 4 regions: the head, trunk, upper extremities, and lower extremities. Each of these areas is assessed separately for erythema, induration and scaling, which are each rated on a scale of 0 to 4. Total PASI score ranges from 0 to 72. A PASI 50 response represents at least 50 percent improvement from baseline in the PASI score. | Baseline up to end of data collection (approximately 8 years) | 
| Percentage of Participants Achieving PASI 75 Response | The PASI is a measure for assessing and grading the severity and extent of psoriatic lesions and their response to therapy. The PASI measure also accounts for body surface area of psoriasis involvement. In the PASI measure, the body is divided into 4 regions: the head, trunk, upper extremities, and lower extremities. Each of these areas is assessed separately for erythema, induration and scaling, which are each rated on a scale of 0 to 4. Total PASI score ranges from 0 to 72. A PASI 75 response represents at least 75 percent improvement from baseline in the PASI score. | Baseline up to end of data collection (approximately 8 years) | 
| Percentage of Participants Achieving PASI 90 Response | The PASI is a measure for assessing and grading the severity and extent of psoriatic lesions and their response to therapy. The PASI measure also accounts for body surface area of psoriasis involvement. In the PASI measure, the body is divided into 4 regions: the head, trunk, upper extremities, and lower extremities. Each of these areas is assessed separately for erythema, induration and scaling, which are each rated on a scale of 0 to 4. Total PASI score ranges from 0 to 72. A PASI 90 response represents at least 90 percent improvement from baseline in the PASI score. | Baseline up to end of data collection (approximately 8 years) | 
| Percentage of Participants Achieving a Physician's Global Assessment (PGA) Score of 0 or 1 | The PGA documents the physician's assessment of the severity of the participant's psoriasis lesions at a given time on a 5-point scale, where (0) = cleared, (1) = minimal, (2) = mild, (3) = moderate, (4) = marked, and (5) = severe. Overall lesions are graded for induration, erythema, and scaling. The sum of the 3 scores will be divided by 3 to obtain a final PGA score. Higher scores indicate greater severity of disease. | Baseline up to end of data collection (approximately 8 years) | 
| Percentage of Participant's Body Surface Area (BSA) Covered by Plaque-type Psoriasis | Percentage of participant's body surface area covered by plaque-type psoriasis was estimated using the palm method: the area equivalent to the participant's palm extending to the proximal interphalangeal joints and thumb = 1 percent (%) of BSA. The total BSA affected was the summation of the BSA of the individual regions affected. | Baseline up to end of data collection (approximately 8 years) | 
| Change From Baseline in Children's Dermatology Life Quality Index (CDLQI) | The Children's Dermatology Quality Life Index (CDLQI) questionnaire is used to assess the participant's perspective on the impact of skin disorders on daily living. It is a 10 item instrument with 4-item response options on a scale from 0 (Not at all) to 3 (Very much) and a recall period of 1 week. The total score ranges from 0 to 30, with lower scores indicating better quality of life. | Baseline up to end of data collection (approximately 8 years) | 
| Number of Participants With Comorbidities | Participants are assessed for pre-existing and new comorbidities associated with pediatric plaque psoriasis. | Baseline up to end of data collection (approximately 8 years) |