Clinical trial NCT02698475: An Efficacy, Safety, and Pharmacokinetics Study of Subcutaneously Administered Ustekinumab in the Treatment of Moderate to Severe Chronic Plaque Psoriasis in Pediatric Participants Greater Than or Equal to 6 to Less Than 12 Years of Age
Official title: A Phase 3 Open-label Study to Assess the Efficacy, Safety, and Pharmacokinetics of Subcutaneously Administered Ustekinumab in the Treatment of Moderate to Severe Chronic Plaque Psoriasis in Pediatric Subjects Greater Than or Equal to 6 to Less Than 12 Years of Age
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Psoriasis
Keywords: Psoriasis
Ustekinumab
STELARA
Lead sponsor: Janssen Research & Development, LLC
Sponsor class: INDUSTRY
First posted: 2016-03-03
Enrollment: 44
Sex: ALL
Minimum age (years): 6
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the efficacy and safety of ustekinumab in pediatric participants aged greater than or equal to (\>=) 6 through less than (\<) 12 years with moderate to severe chronic plaque psoriasis

Detailed description: This is an open label (identity of study drug will be known to participant and study staff) and multicenter (when more than one hospital or medical school team work on a medical research study) study. The participant population will be comprised of boys and girls who have had a diagnosis of plaque psoriasis for at least 6 months prior to first study drug administration and who have moderate to severe disease defined by Psoriasis Area and Severity Index score (PASI) \>=12, Physician's Global Assessment (PGA) \>=3, and Body Surface Area (BSA) \>=10 percent (%). The study consists of Screening Phase (up to 10 weeks before administration of the study drug), Treatment Period (Week 0 up to Week 52) and Safety follow up (Week 56). Participants will be primarily evaluated for efficacy, pharmacokinetics (PK) and safety. Following completion of the Week 52 visit, participants who have had a beneficial response from ustekinumab treatment as determined by the investigator, and who have not yet reached the age of 12 years or older in countries where marketing authorization for ustekinumab has been granted for the treatment of psoriasis in adolescent participants (12-17 years), and are willing to continue ustekinumab treatment, may enter the long-term extension period (from Week 56 through Week 264) of the study.

Eligibility criteria: Inclusion Criteria:

* Participants who have a diagnosis of plaque-type psoriasis with or without psoriatic arthritis (PsA) for at least 6 months prior to first administration of study drug, with widespread lesions defined by Psoriasis Area and Severity Index score (PASI) greater than or equal to (\>=) 12, Physician's Global Assessment (PGA) \>=3, and involved body surface area (BSA) \>=10 percent (%)
* Participants who are candidates for phototherapy or systemic treatment of psoriasis (either naive or history of previous treatment) or have psoriasis considered by the investigator as poorly controlled with topical therapy after an adequate dose and duration of therapy
* Participants who are considered eligible according to the protocol defined tuberculosis (TB) screening criteria
* Participants must have positive protective antibody titers to varicella and measles prior to the first administration of study drug. In the absence of positive protective antibody titers, the participant must have documentation of age-appropriate vaccination for varicella and/or measles (that includes both doses of each vaccine) or verification of past varicella and/or measles infection documented by a health care provider
* Participants must agree not to receive a live virus or live bacterial vaccination at least 2 weeks (or longer as indicated in the package insert of the relevant vaccine) prior to the first administration of study drug, during the study, or within 15 weeks after the last administration of study drug
* Participants must agree not to receive a Bacille Calmette-Guerin (BCG) vaccination within 12 months of screening, during the study, or within 12 months after the last administration of study drug

Exclusion Criteria:

* Participants who currently have nonplaque forms of psoriasis (example, erythrodermic, guttate, or pustular)
* Have received any systemic immunosuppressants (example methotrexate \[MTX\], azathioprine, cyclosporine, 6-thioguanine, mercaptopurine, mycophenolate mofetil, hydroxyurea, and tacrolimus) within 4 weeks of the first administration of study drug
* Have received any biologic agent (example ENBREL, HUMIRA) within the previous 3 months or 5 times the t1/2 of the agent, whichever is longer
* Have a history of chronic or recurrent infectious disease
* Have a history of latent or active granulomatous infection
* Have any known malignancy or have a history of malignancy
* Have a known history of lymphoproliferative disease, including lymphoma, or signs and symptoms suggestive of possible lymphoproliferative disease

Primary outcomes:
measure: Percentage of Participants With Physician's Global Assessment (PGA) Score of Cleared (0) or Minimal (1) at Week 12
timeFrame: Week 12
Secondary outcomes:
measure: Percentage of Participants Who Achieved Psoriasis Area and Severity Index (PASI) 75 Response at Week 12
timeFrame: Week 12
measure: Change From Baseline in Children Dermatology Life Quality Index (CDLQI) Score at Week 12
timeFrame: Baseline and Week 12
measure: Percentage of Participants Who Achieved PASI 90 Response at Week 12
timeFrame: Week 12
measure: Percentage of Participants With a PGA Score of Cleared (0), Cleared (0) or Minimal (1), Mild (2) at Weeks 4, 8, 12, 16, 28, 40, and 52
timeFrame: Weeks 4, 8, 12, 16, 28, 40, and 52
measure: Percentage of Participants Who Achieved a PASI 50, PASI 75, PASI 90 and PASI 100 Response at Weeks 4, 8, 12, 16, 28, 40, and 52
timeFrame: Weeks 4, 8, 12, 16, 28, 40, and 52
measure: Percent Change From Baseline in PASI Score at Weeks 4, 8, 12, 16, 28, 40, and 52
timeFrame: Baseline and Weeks 4, 8, 12, 16, 28, 40, 52
measure: Percentage of Participants Who Achieved PASI 100, PASI 90, PASI 75 or PASI 50 Response in PASI Components (Induration, Erythema, and Scaling) and Region Components (Head, Trunk, Upper Extremities, and Lower Extremities) at Week 12
timeFrame: Week 12
measure: Change From Baseline in Children Dermatology Life Quality Index (CDLQI) Score at Weeks 4, 12, 28, and 52
timeFrame: Baseline and Weeks 4, 12, 28, 52
measure: Percentage of Participants With a CDLQI Score of 0 or 1 at Week 12 in Participants With a Baseline CDLQI Score Greater Than (>) 1
timeFrame: Week 12
measure: Percentage of Participants With a CDLQI Score of 0 or 1 at Weeks 4, 12, 28 and 52 in Participants With a Baseline CDLQI Score > 1
timeFrame: Weeks 4, 12, 28, and 52
measure: Change From Baseline in CDLQI Component Scores at Week 12
timeFrame: Baseline and Week 12
measure: Percentage of Participants With PGA Score of Cleared (0), Cleared (0) or Minimal (1), Cleared (0) or Minimal (1) or Mild (2) at Weeks 80, 104, 128, 152, and 176
timeFrame: Weeks 80, 104, 128, 152, 176
Other outcomes:
Locations:
city: San Diego
country: United States
state: California
city: Chicago
country: United States
state: Illinois
city: Indianapolis
country: United States
state: Indiana
city: Saint Louis
country: United States
state: Missouri
city: Arlington
country: United States
state: Texas
city: Dallas
country: United States
state: Texas
city: Norfolk
country: United States
state: Virginia
city: Brussels
country: Belgium
city: Gent
country: Belgium
city: Liege
country: Belgium
city: Calgary
country: Canada
state: Alberta
city: St. John's
country: Canada
state: Newfoundland and Labrador
city: Berlin
country: Germany
city: Bonn
country: Germany
city: Dresden
country: Germany
city: Frankfurt
country: Germany
city: Budapest
country: Hungary
city: Debrecen
country: Hungary
city: Kaposvar
country: Hungary
city: Kecskemet
country: Hungary
city: Szeged
country: Hungary
city: Bundang
country: Korea, Republic of
city: Incheon
country: Korea, Republic of
city: Seoul
country: Korea, Republic of
city: Nijmegen
country: Netherlands
city: Lodz
country: Poland
city: Warszawa
country: Poland
city: Wroclaw
country: Poland
city: Kaohsiung
country: Taiwan
city: Taipei
country: Taiwan
city: Taoyuan
country: Taiwan
Study officials:
affiliation: Janssen Research & Development, LLC
name: Janssen Research & Development, LLC Clinical Trial
role: STUDY_DIRECTOR
References:
citation: Leu JH, Shiff NJ, Clark M, Bensley K, Lomax KG, Berezny K, Nelson RM, Zhou H, Xu Z. Intravenous Golimumab in Patients with Polyarticular Juvenile Idiopathic Arthritis and Juvenile Psoriatic Arthritis and Subcutaneous Ustekinumab in Patients with Juvenile Psoriatic Arthritis: Extrapolation of Data from Studies in Adults and Adjacent Pediatric Populations. Paediatr Drugs. 2022 Nov;24(6):699-714. doi: 10.1007/s40272-022-00533-y. Epub 2022 Sep 28.