Phase 3
N=245
Efficacy and Safety Study of Apremilast (CC-10004) in Pediatric Subjects From 6 Through 17 Years of Age With Moderate to Severe Plaque Psoriasis
Psoriasis
Bottom Line
View on ClinicalTrials.gov: NCT03701763 ↗Enrolled (actual)
245
Serious AEs
1.1%
Results posted
Dec 2023
Primary outcome: Primary: Percentage of Participants With a Static Physician Global Assessment (sPGA) Response at Week 16 — 11.5; 33.1 percentage of participants — p=<0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Apremilast (CC-10004) (Drug); Placebo (Other)
- Age
- Pediatric · 6+ yrs
- Sex
- All
- Sponsor
- Amgen
- Primary completion
- Apr 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With a Static Physician Global Assessment (sPGA) Response at Week 16 |
11.5; 33.1 | <0.0001 sig |
| SECONDARY Percentage of Participants Who Achieved At Least 75% Reduction in Psoriasis Area Severity Index (PASI-75) From Baseline at Week 16 |
16.1; 45.4 | <0.0001 sig |
| SECONDARY Percentage of Participants Who Achieved At Least 50% Reduction in Psoriasis Area Severity Index (PASI-50) From Baseline at Week 16 |
32.1; 70.5 | <0.0001 sig |
| SECONDARY Percentage Change From Baseline in Total PASI Score at Week 16 |
-37.49; -64.52 | <0.0001 sig |
| SECONDARY Percentage Change From Baseline in Body Surface Area (BSA) Affected by Psoriasis at Week 16 |
-20.56; -55.44 | < 0.0001 sig |
| SECONDARY Percentage of Participants Who Achieved a Children's Dermatology Life Quality Index (CDLQI) Score of 0 or 1 at Week 16 |
31.3; 35.4 | 0.5616 |
| SECONDARY Change From Baseline in CDLQI Score at Week 16 |
-2.7; -5.3 | 0.0009 sig |
| SECONDARY Number of Participants Who Experienced a Treatment-emergent Adverse Event (TEAE) During the Placebo-controlled Phase |
33; 58; 48 | — |
| SECONDARY Number of Participants Who Experienced a TEAE During the Apremilast Exposure Period |
88; 80 | — |
| SECONDARY Number of Participants Who Experienced a Treatment-emergent Serious Adverse Event (TESAE) During the Placebo-controlled Phase |
1; 2; 0 | — |
| SECONDARY Number of Participants Who Experienced a TESAE During the Apremilast Exposure Period |
2; 1 | — |
| SECONDARY Number of Participants Who Experienced a Treatment-related Adverse Event (TRAE) During the Placebo-controlled Phase |
12; 36; 32 | — |
| SECONDARY Number of Participants Who Experienced a TRAE During the Apremilast Exposure Period |
45; 47 | — |
| SECONDARY Number of Participants With Diarrhea During the Placebo-controlled Phase |
25; 67; 20; 51; 14; 41 | — |
| SECONDARY Number of Participants With Diarrhea During the Apremilast Exposure Period |
36; 47; 30; 36; 22; 32 | — |
| SECONDARY Number of Participants With Diarrhea Symptoms During the Placebo-controlled Phase |
3; 32; 2; 13; 3; 16 | — |
| SECONDARY Number of Participants With Diarrhea Symptoms During the Apremilast Exposure Period |
17; 21; 10; 4; 7; 9 | — |
| SECONDARY Number of Participants With Suicidal Ideation or Behavior Per the Columbia-Suicide Severity Rating Scale (C-SSRS) During the Placebo-controlled Phase |
0; 0 | — |
| SECONDARY Number of Participants With Suicidal Ideation or Behavior Per the C-SSRS During the Apremilast-extension Phase |
0; 0 | — |
| SECONDARY Number of Female Participants at Stage I-V of Sexual Development Per Tanner Staging of Sexual Development |
8; 17; 3; 9; 3; 9 | — |
| SECONDARY Number of Male Participants at Stage I-V of Sexual Development Per Tanner Staging of Sexual Development |
11; 15; 5; 5; 4; 10 | — |
| SECONDARY Mean Body Weight of Participants During the Placebo-controlled Phase |
52.36; 52.04; 54.18; 51.95 | — |
| SECONDARY Mean Body Weight of Participants During the Apremilast Exposure Period |
36.81; 67.94; 39.04; 67.79 | — |
| SECONDARY Mean Height of Participants During the Placebo-controlled Phase |
153.29; 153.33; 154.54; 154.40 | — |
| SECONDARY Mean Height of Participants During the Apremilast Exposure Period |
140.86; 166.13; 144.90; 167.84 | — |
| SECONDARY Mean Body Mass Index (BMI) of Participants During the Placebo-controlled Phase |
21.41; 21.33; 21.87; 20.98 | — |
| SECONDARY Mean BMI of Participants During the Apremilast Exposure Period |
18.32; 24.52; 18.30; 23.95 | — |
| SECONDARY Number of Participants Who Experienced a Psoriasis Flare During the Placebo-controlled Phase |
3; 2 | — |
| SECONDARY Number of Participants Who Experienced a Psoriasis Flare During the Apremilast Exposure Period |
7; 11 | — |
| SECONDARY Number of Participants Who Experienced a Psoriasis Rebound |
0; 0; 4 | — |
Summary
This is a Phase 3, multicenter, randomized, placebo-controlled, double-blind study of the efficacy and safety of apremilast (CC-10004) in pediatric subjects with moderate to severe plaque psoriasis.
At least 230 pediatric subjects (ages 6 through 17 years) will be randomized 2:1 to receive either apremilast or placebo for the first 16 weeks and then all subjects will receive apremilast during the 36 week Extension Phase for a total of 52 weeks. Randomization to apremilast arm or placebo arm will be stratified by age group (6 to 11 years or 12 to 17 years). Subjects will receive apremilast treatment of either 20 mg twice daily (BID) or 30 mg BID, depending on weight. This Phase 3 study is being conducted to evaluate the safety and efficacy of apremilast in the treatment of pediatric subjects.
Eligibility Criteria
Inclusion Criteria
- Males or female subjects 6 to 17 years of age, inclusive, at the time the informed consent form is signed by the legal guardian
- Subjects must have a weight of ≥ 20 kg
- Diagnosis of chronic plaque psoriasis for at least 6 months prior to Screening.
- Has moderate to severe plaque psoriasis at Screening and Baseline as defined by:
- PASI score ≥ 12; and
- Body surface area (BSA) ≥ 10%; and
- sPGA ≥ 3 (moderate to severe)
- Disease inadequately controlled by or inappropriate for topical therapy for psoriasis
- Candidate for systemic therapy or phototherapy
Exclusion Criteria
- Guttate, erythrodermic, or pustular psoriasis at Screening and Baseline
- Psoriasis flare or rebound within 4 weeks prior to Screening
- Prior history of suicide attempt at any time in the subject's lifetime prior to Screening or randomization in the study, or major psychiatric illness requiring hospitalization within 3 years prior to signing the assent and informed consent
- Answer "Yes" to any question on the Columbia-Suicide Severity Rating Scale during Screening or at Baseline
- Current or planned concurrent use of the following therapies that may have a possible effect on psoriasis
a. Topical therapy within 2 weeks prior to randomization (including but not limited to topical corticosteroids, topical retinoid or vitamin D analog preparations, tacrolimus, pimecrolimus, or anthralin/dithranol)
Exceptions*:
i. Low potency or weak corticosteroids (please refer to the Investigators' Manual) will be allowed as background therapy for treatment of the face, axillae and groin in accordance with manufacturer's suggested usage ii. Unmedicated skin moisturizer (eg, Eucerin®) will also be permitted for body lesions
*Subjects should not use these topical treatments within 24 hours prior to the clinic visit.
b. Conventional systemic therapy for psoriasis within 4 weeks prior to randomization c. Phototherapy treatment (ie, ultraviolet B [UVB], PUVA) within 4 weeks prior to randomization d. Biologic therapy within 4 weeks prior to randomization or 5 PK/PD half-lives (whichever is longer).
Data sourced from ClinicalTrials.gov (NCT03701763). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.