Apremilast Pediatric Study in Children With Active Juvenile Psoriatic Arthritis

Browse trials for

• Arthritis, Juvenile 365

Keywords

• Apremilast
• Otezla®
• Juvenile psoriatic arthritis
• Inflammatory disorders

Arthritis, Juvenile

365 studies on the registry are indexed under Arthritis, Juvenile; 101 are open to participants now.

This study's planned enrollment of 60 is above the median of 52 across 230 interventional studies indexed under Arthritis, Juvenile.

Lead sponsor

Amgen is the lead sponsor of 1,047 studies on the registry; 52 are open to participants now.

Of its 246 completed or terminated interventional studies of FDA-regulated products, 166 (67%) have results posted.

Study arms

• Experimental
  Apremilast

  Participants will receive apremilast in the double-blind 16 week treatment phase. Then the participants will continue to receive apremilast in the active 36 weeks treatment phase.

  Drug: Apremilast

• Placebo comparator
  Placebo to Apremilast

  Participants will receive the matching placebo in the double-blind 16 week treatment phase. Then the participants will receive apremilast in the active 36 weeks treatment phase.

  Drug: Apremilast · Drug: Placebo

Interventions

• DrugApremilast

  Participants will receive apremilast orally.

  Also known as: Otezla®

• DrugPlacebo

  Participants will receive the matching placebo orally.

Primary outcomes

1. 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

1. 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

2. 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).

   Time frame: Baseline to Week 16

3. 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

4. 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

5. 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

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

   Time frame: Baseline to Week 16

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

   Time frame: Baseline to Week 16

8. 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

9. 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

10. 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

11. 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

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

    Time frame: Up to Week 56

13. 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

14. 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

15. Change from Baseline in Body Weight at Week 56

    Time frame: Baseline to Week 56

16. Change from Baseline in Height at Week 56

    Time frame: Baseline to Week 56

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

    Time frame: Baseline to Week 56

18. 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

19. 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

Related links

• AmgenTrials clinical trials website ↗

Individual participant data

Plan to share: Yes — De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.

Supporting information: Study protocol, Statistical analysis plan, Informed consent form, Clinical study report

Time frame: Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.

Access criteria: Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors. If not approved, a Data Sharing Independent Review Panel will arbitrate and make the final decision. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the URL below.

Data sharing details ↗ · www.amgen.com