## Protocol Section
### Identification Module
**NCT ID:** NCT06431750

**Acronym:** Toddler

**Brief Title:** Prospective Observational Study to Evaluate Secukinumab Treatment Effectiveness in Pediatric Patients With Active Juvenile Enthesitis-related or Psoriatic Arthritis

**Official Title:** Prospective Observational Study to Evaluate Secukinumab Treatment Effectiveness in Pediatric Patients With Active Juvenile Enthesitis-related or Psoriatic Arthritis (Toddler)

#### Organization Study ID Info
**ID:** CAIN457FRU02
#### Organization
**Class:** INDUSTRY
**Full Name:** Novartis
### Status Module
#### Completion Date
**Date:** 2028-08-30

**Type:** ESTIMATED

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-05-29

**Type:** ACTUAL

**Last Update Submit Date:** 2024-05-22

**Overall Status:** NOT_YET_RECRUITING

#### Primary Completion Date
**Date:** 2028-08-30

**Type:** ESTIMATED

#### Start Date
**Date:** 2025-04-30

**Type:** ESTIMATED

**Status Verified Date:** 2024-05

#### Study First Post Date
**Date:** 2024-05-29
**Type:** ACTUAL
**Study First Submit Date:** 2024-04-30
**Study First Submit QC Date:** 2024-05-22
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** INDUSTRY

**Name:** Novartis Pharmaceuticals

#### Responsible Party

**Type:** SPONSOR

### Description Module

**Brief Summary:** This is a multicenter, non-interventional, cohort study in pediatric patients with active juvenile enthesitis-related or psoriatic arthritis

**Detailed Description:** Rheumatologists and pediatricians actively managing pediatric Enthesitis-related Arthritis (ERA) and Juvenile Psoriatic Arthritis (jPsA) in different regions of Russia are planned to participate in the study. To be include in this study, physicians must consult more than 10 pediatric patients with ERA/jPsA in a typical month. Any pediatric patients with active ERA/jPsA who started secukinumab treatment (index date) within 4-8 weeks prior to inclusion can be enrolled. At the inclusion retrospective data collection is planned, then data will be collected 3 months of treatment), 6, 12, 18, and 24 months after index date. Therefore, maximum duration of prospective observation is expected to be 23 months. Data collection schedule fits routine (Juvenile Idiopathic Arthritis) JIA management guidelines for patients receiving biologics stating that treatment effectiveness should be evaluated at 3 and 6 months after biologic initiation and every 6 months thereafter. At each visit effectiveness and tolerability parameters, as well as the patients' Quality of life (QoL) will be documented. Retrospective data will be collected for medical history and JIA treatment, including secukinumab before inclusion, as well to describe all necessary parameters since the Index date. Expected look-back period is 12 months.

Initiation of secukinumab treatment will be considered the index date.

### Conditions Module

**Conditions:**

- Juvenile Idiopathic Arthritis

**Keywords:**

- Juvenile Idiopathic Arthritis

- JIA

- Secukinumab

- Cosentyx

### Design Module

#### Design Info

**Observational Model:** COHORT

**Time Perspective:** PROSPECTIVE

#### Enrollment Info

**Count:** 150

**Type:** ESTIMATED

**Study Type:** OBSERVATIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** pediatric patients with active ERA/jPsA for whom routine treatment with secukinumab according to the approved national label is initiated during 4 to 8 weeks prior to enrollment

**Intervention Names:**

- Other: Secukinumab

**Label:** Secukinumab

### Interventions

#### Intervention 1

**Arm Group Labels:**

- Secukinumab

**Description:** This is an observational study, there is no treatment allocation.

**Name:** Secukinumab

**Type:** OTHER

### Outcomes Module

#### Primary Outcomes

**Description:** Drug survival is defined as time from the index date (defined as secukinumab treatment initiation) until discontinuation of secukinumab

**Measure:** Drug survival rate

**Time Frame:** 24 months

**Description:** The ACR Pedi 30/50/70/90 is defined as a minimum of 30/50/70/90 percent improvement from baseline in a minimum of 3 out of 6 components, with no more than 1 component worsening by \>30/50/70/90 percent.

The ACR Pedi consists of 6 core criteria:

1. physician global assessment (PGA) of disease activity (visual analog scale \[VAS\]) where 0 represents no disease activity and 100 represents the most disease activity
2. assessment of overall well-being (VAS) where 0 represents very well and 100 represents very poor for overall well-being
3. functional ability (assessed using the Childhood Health Assessment Questionnaire \[CHAQ\]);
4. number of joints with active arthritis (defined as joints with swelling not caused by deformity or joints, in the absence of swelling, with limitation of passive motion accompanied by pain, tenderness, or both)
5. number of joints with limited range of motion
6. laboratory marker of inflammation (C-reactive protein \[CRP\]).

**Measure:** American College of Rheumatology pediatric (ACR Pedi) 30/50/70/90 achievement rates

**Time Frame:** Month 3, Month 6, Month 12, Month 18, and Month 24

**Description:** To state the absence of disease activity (inactive phase of the disease), the patient must meet all of the listed criteria.

* absence of joints with active arthritis,
* absence of fever, rash, serositis, splenomegaly or generalized lymphadenopathy typical of juvenile arthritis;
* absence of active uveitis,
* normal ESR and (or) CRP,
* absence of disease activity according to the doctor's general assessment (VAS),
* morning stiffness less than 15 minutes.

**Measure:** Clinical remission/inactive disease rates by Wallace criteria

**Time Frame:** Month 3, Month 6, Month 12, Month 18, and Month 24

#### Secondary Outcomes

**Description:** Describes the child's usual activities in eight domains over the past week. It include dressing, getting up, eating, walking, hygiene, reaching overhead objects, grip and activities. Each question is scored from 0 to 3 (0 = no difficulty, 1 = some difficulty, 2 = much difficulty and 3 = unable to do). The score for each of the eight functional areas will be averaged to calculate the disability index.

**Measure:** Childhood Health Assessment Questionnaire (CHAQ) score

**Time Frame:** Up to 24 months

**Description:** Physician global assessment of disease activity assessed on a VAS scale of 0 \[no activity\] to 10 \[extreme activity\]

**Measure:** Physician global assessment of disease activity

**Time Frame:** Up to 24 months

**Description:** Patient/legal representative global assessment of disease activity assessed on a VAS scale of 0 \[no activity\] to 10 \[extreme activity\]

**Measure:** Patient/legal representative global assessment of disease activity

**Time Frame:** Up to 24 months

**Description:** Time to treatment failure defined as time from index date to loss of ACR Pedi 30, 50, and 70 response.

**Measure:** Time to treatment failure

**Time Frame:** Up to 24 months

**Description:** The ACR Pedi 30 is defined as a minimum of 30 percent improvement from baseline in a minimum of 3 out of 6 components, with no more than 1 component worsening by \>30 percent.

The ACR Pedi consists of 6 core criteria:

1. physician global assessment (PGA) of disease activity (visual analog scale \[VAS\]) where 0 represents no disease activity and 100 represents the most disease activity
2. assessment of overall well-being (VAS) where 0 represents very well and 100 represents very poor for overall well-being
3. functional ability (assessed using the Childhood Health Assessment Questionnaire \[CHAQ\]);
4. number of joints with active arthritis (defined as joints with swelling not caused by deformity or joints, in the absence of swelling, with limitation of passive motion accompanied by pain, tenderness, or both)
5. number of joints with limited range of motion
6. laboratory marker of inflammation (C-reactive protein \[CRP\]).

**Measure:** Proportion of patients not achieving ACR Pedi 30 response

**Time Frame:** Month 3

**Description:** Score to evaluate the number of joints inflamed. A joint is considered to be inflamed when at leats two of the following criteria are present:

* pain
* Swelling
* limited motion. The score range (total) is from 0-101, where higher scores indicate worse outcome.

**Measure:** Disease activity index in 71 joints Juvenile Arthritis Disease Activity Score (JADAS71)

**Time Frame:** Up to 24 months

**Description:** The JSpADA score is a comprehensive

clinical assessment tool designed to measure the impact of juvenile spondyloarthritis on the patient. The JSpADA scale includes 8 indicators that assess the signs and symptoms of spondyloarthritis. Patients receive an overall score based on scores on each of 8 indicators. There are two options for assessing morning stiffness, clinical sacroiliitis, uveitis and back mobility: presence or absence. Number of joints with active arthritis: 0 joints = 0; 1-2 joints = 0.5; \> 2 joints = 1; Number of active enthesites: 0 = 0; 1-2 = 0.5; \> 2 = 1; Patient-Reported Pain Score (VAS 0-10): 0 = 0; 1-4 = 0.5; 5-10 = 1; Assessment of ESR or CRP level: normal = 0; 1-2x = 0.5; \> 2x = 1.

As a result, we add up the scores for the obtained indicators and get an overall score, and the higher it is, the higher the disease activity.

**Measure:** Juvenile Spondyloarthritis (JSpA) Disease Activity - JSpADA

**Time Frame:** Up to 24 months

**Description:** Psoriasis Area and Severity Index (PASI) for patients with Juvenile Psoriatic Arthritis (jPsA) to be provided. Score can range from 0 (no disease) to 72 (maximal disease).

**Measure:** Psoriasis Area and Severity Index (PASI) for patients with Juvenile Psoriatic Arthritis (jPsA)

**Time Frame:** Up to 24 months

**Description:** Reasons for discontinuation:

* lack of efficacy,
* adverse events
* administrative reasons (no medication available in the hospital, etc.)
* lack of patient's adherence
* patient's or legal representative's wish
* other.

**Measure:** Number of participants by reasons of secukinumab discontinuation

**Time Frame:** Up to 24 months

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

1. Written informed consent and legal representative's permission for study participation obtained prior to beginning of participation in the study.
2. Age ≥6 to \<18 years old.
3. Recognized physician diagnosis of active ERA or jPsA:

   * ERA per ILAR criteria:

     * Peripheral arthritis and enthesitis, or
     * Arthritis or enthesitis, plus ≥ 3 months of inflammatory back pain and sacroiliitis on imaging, or
     * Arthritis or enthesitis plus 2 of the following: sacroiliac joint tenderness; inflammatory back pain; presence of HLA-B27 antigen; acute (symptomatic) anterior uveitis; and history of a spondyloarthritis in a first-degree relative.
   * jPsA per ILAR criteria

     * Arthritis and psoriasis, or
     * arthritis and at least 2 of the following:

       * Dactylitis
       * Nail pitting or onycholysis
       * Psoriasis in a first-degree relative.
4. Patient was prescribed with secukinumab within 4-8 weeks before inclusion.
5. Decision for secukinumab prescription was made by the attending physician according to the approved national label during routine clinical practice, regardless of this non-interventional study conduct.
6. The Purified Protein Derivative (PPD) Skin Test for Tuberculosis and/or Negative T-SPOT test and/or TB-feron test before secukinumab treatment and every 6 months.

Exclusion Criteria:

1. Known or suspected severe hypersensitivity for secukinumab, formulation excipients, or injection device components (i.e., latex).
2. Chronic recurrent infections.
3. Clinically significant infection exacerbation, including active tuberculosis.
4. Age \<6 years or ≥18 years.
5. Pregnancy and breastfeeding.
6. Patients participating in parallel in an interventional clinical trial.
7. Patients participating in parallel in other Novartis-sponsored non-interventional study generating primary data for secukinumab.
8. Patients within the safety follow-up phase of interventional study.
9. Active inflammatory bowel disease at inclusion.
10. Patients who received any vaccine within 4 weeks prior to secukinumab initiation.
11. Any medical or psychological condition in the investigator's opinion which may prevent the study participation.
12. Concomitant conditions (Candida infections, other infections, inflammatory bowel disease \[IBD\], uveitis, skin and nail psoriasis for ERA patients, hepatitis B, hepatitis C, tuberculosis).

**Maximum Age:** 18 Years

**Minimum Age:** 6 Years

**Sampling Method:** NON_PROBABILITY_SAMPLE

**Sex:** ALL

**Standard Ages:**

- CHILD

- ADULT

**Study Population:** Pediatric patients with active ERA/jPsA for whom routine treatment with secukinumab according to the approved national label is initiated during 4 to 8 weeks prior to enrollment

### Contacts Locations Module

#### Central Contacts

**Contact 1:**

**Email:** novartis.email@novartis.com

**Name:** Novartis Pharmaceuticals

**Phone:** +41613241111

**Role:** CONTACT

**Contact 2:**

**Name:** Novartis Pharmaceuticals

**Role:** CONTACT

#### Overall Officials

**Official 1:**

**Affiliation:** Novartis Pharmaceuticals

**Name:** Novartis Pharmaceuticals

**Role:** STUDY_DIRECTOR

### IPD Sharing Statement Module

**IPD Sharing:** NO

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000007592

- Term: Joint Diseases

- ID: D000009140

- Term: Musculoskeletal Diseases

- ID: D000025242

- Term: Spondylarthropathies

- ID: D000025241

- Term: Spondylarthritis

- ID: D000013166

- Term: Spondylitis

- ID: D000013122

- Term: Spinal Diseases

- ID: D000001847

- Term: Bone Diseases

- ID: D000011565

- Term: Psoriasis

- ID: D000017444

- Term: Skin Diseases, Papulosquamous

- ID: D000012871

- Term: Skin Diseases

- ID: D000012216

- Term: Rheumatic Diseases

- ID: D000003240

- Term: Connective Tissue Diseases

- ID: D000001327

- Term: Autoimmune Diseases

- ID: D000007154

- Term: Immune System Diseases

### Condition Browse Module - Browse Branches

- Abbrev: BC05

- Name: Musculoskeletal Diseases

- Abbrev: All

- Name: All Conditions

- Abbrev: BC17

- Name: Skin and Connective Tissue Diseases

- Abbrev: BC20

- Name: Immune System Diseases

- Abbrev: BC01

- Name: Infections

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M4476

- Name: Arthritis

- Relevance: HIGH

- As Found: Arthritis

- ID: M18178

- Name: Arthritis, Psoriatic

- Relevance: HIGH

- As Found: Psoriatic Arthritis

- ID: M4479

- Name: Arthritis, Juvenile

- Relevance: HIGH

- As Found: Juvenile Idiopathic Arthritis

- ID: M10621

- Name: Joint Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M12097

- Name: Musculoskeletal Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M23036

- Name: Spondylarthropathies

- Relevance: LOW

- As Found: Unknown

- ID: M15961

- Name: Spondylitis

- Relevance: LOW

- As Found: Unknown

- ID: M23035

- Name: Spondylarthritis

- Relevance: LOW

- As Found: Unknown

- ID: M15919

- Name: Spinal Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M5126

- Name: Bone Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M14422

- Name: Psoriasis

- Relevance: LOW

- As Found: Unknown

- ID: M15674

- Name: Skin Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M19713

- Name: Skin Diseases, Papulosquamous

- Relevance: LOW

- As Found: Unknown

- ID: M15045

- Name: Rheumatic Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M6323

- Name: Collagen Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M6464

- Name: Connective Tissue Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M4629

- Name: Autoimmune Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M10200

- Name: Immune System Diseases

- Relevance: LOW

- As Found: Unknown

- ID: T5412

- Name: Spondylarthropathy

- Relevance: LOW

- As Found: Unknown

### Condition Browse Module - Meshes

- ID: D000001168

- Term: Arthritis

- ID: D000015535

- Term: Arthritis, Psoriatic

- ID: D000001171

- Term: Arthritis, Juvenile

### Misc Info Module

- Version Holder: 2024-05-31
