Apremilast Pediatric Study in Children With Active Juvenile Psoriatic Arthritis

Phase 3RecruitingNCT04804553

Amgen60 enrolled

Overview

The study will aim to estimate the efficacy of apremilast compared with placebo in the treatment of juvenile psoriatic arthritis (JPsA) in pediatric participants 5 to less than 18 years of age.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Active Juvenile Psoriatic Arthritis

Interventions

ApremilastDRUG

Participants will receive apremilast orally.

PlaceboDRUG

Participants will receive the matching placebo orally.

Eligibility

Sex: ALLMin age: 5 YearsMax age: 17 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Male or Female participants 5 to \< 18 years of age at the time of randomization. * Participant must have a confirmed diagnosis of juvenile psoriatic arthritis (JPsA) according to the International League of Associations for Rheumatology (ILAR) Edmonton Revision (Petty, 2001) classification criteria of at least 6 months duration: * Arthritis and psoriasis, OR * Arthritis with at least 2 of the following: * Dactylitis * Nail pitting or onycholysis * Psoriasis in a first-degree relative * Active disease: at least 3 active joints. * Inadequate response (at least 2 months) or intolerance to ≥ 1 disease-modifying anti-rheumatic drugs (DMARD), (which may include methotrexate \[MTX\] or biologic agents). Exclusion Criteria: * Exclusions per ILAR Edmonton Revision (Edmonton, 2001) criteria for JPsA include: * Arthritis in an HLA-B27-positive male with arthritis onset after 6 years of age * Ankylosing spondylitis, sacroiliitis with inflammatory bowel disease, Reiter's syndrome, acute anterior uveitis, or a history of one of these disorders in a first-degree relative * History of IgM rheumatoid factor on at least 2 occasions at least 3 months apart * Presence of systemic juvenile idiopathic arthritis (JIA). * Rheumatic autoimmune disease other than psoriatic arthritis (PsA), including, but not limited to: systemic lupus erythematosus, mixed connective tissue disease, scleroderma, polymyositis, or fibromyalgia. * Prior history of or current inflammatory joint disease other than PsA (eg, gout, reactive arthritis, rheumatoid arthritis, ankylosing spondylitis, Lyme disease).

Locations (45)

Outcomes

Primary Outcomes

Number of Participants who Achieve American College of Rheumatology Pediatric (ACR) Pedi 30 Response at Week 16

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 \[CRP\]).

Time frame: Baseline to Week 16

Secondary Outcomes

Change from Baseline in Participants Assessment of Pain at Week 16

The participants assessment of pain will be assessed using a visual analogue scale (VAS). Participants will be asked to place a vertical line on a 100-mm VAS where the left-hand boundary represents "no pain" and the right-hand boundary represents "worst possible pain".

Time frame: Baseline to Week 16

Number of Participants who Achieve ACR Pedi 20, ACR Pedi 50, ACR Pedi 70 and ACR Pedi 90 Response at Week 16

The ACR Pedi 20, 50, 70 and 90 is defined as a minimum of either 20 percent, 50 percent. 70 percent or 90 percent improvement respectively from baseline in a minimum of 3 out of 6 components, with no more than 1 component worsening by \>20 percent, \>50 percent, \>70 percent or \>90 percent respectively. The ACR Pedi consists of 6 core criteria: 1. PGA of disease activity (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 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 (CRP).

Central Contacts

Amgen Call Center

CONTACT

866-572-6436 medinfo@amgen.com

Data from ClinicalTrials.gov

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Landeskrankenhaus Bregenz

Bregenz, Austria

RECRUITING

Universitair Ziekenhuis Gent

Ghent, Belgium

TERMINATED

Centre Hospitalier Regional de la Citadelle

Liège, Belgium

TERMINATED

Ziekenhuis Netwerk Antwerpen Jan Palfijn

Merksem, Belgium

TERMINATED

Hospices Civils de Lyon Hopital Femme Mere Enfant

Bron, France

RECRUITING

Hopital Jeanne de Flandre

Lille, France

RECRUITING

Charite - Universitaetsmedizin Berlin, Campus Virchow

Berlin, Germany

RECRUITING

Universitaetsklinikum Dresden

Dresden, Germany

RECRUITING

An der Schoen Klinik Hamburg Eilbek

Hamburg, Germany

RECRUITING

Asklepios Kinderklinik Sankt Augustin GmbH

Sankt Augustin, Germany

COMPLETED

...and 35 more locations

Time frame: Baseline to Week 16

Change from Baseline in the Physician Global Assessment (PGA) of Disease Activity at Week 16

PGA of disease activity is assessed using a visual analog scale (VAS), where 0 represents no disease activity and 100 represents the most disease activity.

Time frame: Baseline to Week 16

Change from Baseline in the Assessment of Overall Well-being at Week 16

Assessment of overall well-being is assessed using a visual analog scale (VAS), where 0 represents very well and 100 represents very poor for overall well-being. This assessment can be completed by either the participant or the parent/caregiver.

Time frame: Baseline to Week 16

Change from Baseline in the Number of Joints with Active Arthritis at Week 16

Active arthritis is defined as joints with swelling not caused by deformity of joints, in the absence of swelling, with limitation of passive motion accompanied by pain, tenderness, or both.

Time frame: Baseline to Week 16

Change from Baseline in the Number of Joints with Limited Range of Motion at Week 16

Time frame: Baseline to Week 16

Change from Baseline in the Laboratory Marker of Inflammation (C-reactive Protein) at Week 16

Time frame: Baseline to Week 16

Change from Baseline in Childhood Health Assessment Questionnaire (CHAQ) at Week 16

The CHAQ will be used to assess physical ability and functional status of participants as well as their quality of life. The CHAQ consists of 20 items concerning difficulty in performing the following 8 activities of daily living: dressing and grooming, arising, eating, walking, reaching, personal hygiene, gripping, and activities. Subjects will choose from 4 responses ranging from 0 (without any difficulty) to 3 (unable to do). A lower score indicates a better outcome. The subject's/parent's/caregiver's assessment of arthritis-related pain will also be assessed on a VAS that is part of the CHAQ.

Time frame: Baseline to Week 16

Change from Baseline in Juvenile Arthritis Disease Activity Score (JADAS) at Week 16

JADAS is a composite score of participant well-being visual analog scale (VAS) score, physician global assessment (PGA) VAS score, active joint count, and laboratory marker of inflammation (C-reactive protein \[CRP\]). There are 4 components of the JADAS: 1. Active joint count (71 joints) 2. PGA (0 to 100) measured on a VAS 3. Patient/parent global assessment of well-being (0 to 100) measured on a VAS 4. CRP (normalized to a 0 to 10 scale) The active joint count is taken from 71 joints: Temporomandibular joints, cervical spine, glenohumeral joints, acromioclavicular joints, sternoclavicular joints, elbows, wrists, metacarpophalangeal joints, interphalangeal joints, proximal interphalangeal joints, distal interphalangeal joints, hips, subtalar joints, midfoot joints, knees and ankles . The total score is calculated by adding all 4 components of the JADAS. The score for the JADAS ranges from 0 to 101 where a lower score indicates a better outcome.

Time frame: Baseline to Week 16

Number of Participants who Experience Psoriatic Arthritis (PsA) Flares at Week 16

PsA flares are defined as more than or equal to 30 percent worsening in at least 3 of 6 American College of Rheumatology Pediatric (ACR Pedi) core set variables with a more than or equal to 30 percent improvement in not more than 1 of 6 ACR Pedi core set variables.

Time frame: Baseline to Week 16

Psoriasis Area Severity Index (PASI)-75 Response at Week 16 for Participants With a Baseline Psoriasis Body Surface Area (BSA) ≥ 3 percent

PASI scores range from 0 to 72, with higher scores reflecting greater disease severity. The PASI score is determined only for subjects whose BSA involved by psoriasis is ≥3 percent. PASI scores range from 0 to 72, with higher scores reflecting greater disease severity. Erythema, thickness, and scaling are scored on a scale of 0 (none) to 4 (very severe) on the 4 anatomic regions of the body: head, trunk, upper limbs, and lower limbs. Degree of involvement on each of the 4 anatomic regions is scored on a scale of 0 (no involvement) to 6 (90 percent \< 100 percent involvement). The sum of scores for erythema, thickness, and scaling is multiplied by the degree of involvement for each anatomic region, and then multiplied by a constant corresponding to the region's percentage of BSA. The resultant values for each anatomic region are then summed to yield the PASI score.

Time frame: Baseline to Week 16

Number of Participants who Experience One or More Treatment-Emergent Adverse Events (TEAEs)

Time frame: Up to Week 56

Number of Participants With Suicidal Ideation or Behaviour Assessed Via the Columbia Suicide Severity Rating Scale (C-SSRS)

The C-SSRS is an assessment tool that evaluates suicidal ideation and behavior. Number of participants with suicidal ideation or behavior is defined as the number of participants who answer "yes" at any time during the study (up to end of safety follow-up, Week 56) to one of the 10 categories: Category 1: Wish to be dead Category 2: Non-specific active suicidal thoughts Category 3: Active suicidal ideation with any methods (not plan) without intent to act Category 4: Active suicidal ideation with some intent to act, without specific plan Category 5: Active suicidal ideation with specific plan and intent Category 6: Preparatory acts or behavior Category 7: Aborted attempt Category 8: Interrupted attempt Category 9: Actual attempt (non-fatal) Category 10: Completed suicide

Time frame: Up to Week 56

Change from Baseline in Tanner Staging at Week 52

Tanner Staging of sexual development assessment will be used to assess sexual maturity. Tanner Staging assessment consists of 3 domains (pubic hair, breast development, and other changes) for girls and 4 domains (pubic hair, penis development, testes development, and other changes) for boys. Stages range from 1-5, with 1 indicating preadolescent and 5 adult.

Time frame: Baseline to Week 52

Change from Baseline in Body Weight at Week 56

Time frame: Baseline to Week 56

Change from Baseline in Height at Week 56

Time frame: Baseline to Week 56

Change from Baseline in Body Mass Index (BMI) at Week 56

Time frame: Baseline to Week 56

Plasma Concentrations of Apremilast

Time frame: Week 2: 0-5 hours post dose; Week 8: 2 hours post dose; Week 16: 4 hours post-dose; Week 28: pre dose; Week 40: pre dose; Week 52: pre dose

Taste and Acceptability of Apremilast

Taste and acceptability will be assessed using a questionnaire with a 7-point faces Likert Scale, with 1 ranging from "super bad" to 7 "super good" and questions to determine whether the participants are able to take the treatment medication.

Time frame: Baseline and Week 2