Clinical trial • Phase III • Immunology

APREMILAST for Juvenile psoriatic arthritis

Phase III trial of APREMILAST for Juvenile psoriatic arthritis.

Overview

Trial Therapeutic Area
Immunology
Trial Disease
Juvenile psoriatic arthritis
Trial Stage
Phase III
Drug Modality
Small molecule
Paediatric Trial
Yes

Key dates

Initial CTIS Submission Date
19-01-2024
First CTIS Authorization Date
07-03-2024

Trial design

Randomised, placebo (tablet or liquid suspension) matching apremilast; subjects randomized 2:1 to oral apremilast (tablet or liquid suspension) or placebo (tablet or liquid suspension).-controlled Phase III trial across 36 sites in Spain, Belgium, Poland and others.

Randomised
Yes
Comparator
Placebo (tablet or liquid suspension) matching apremilast; subjects randomized 2:1 to oral apremilast (tablet or liquid suspension) or placebo (tablet or liquid suspension).
Target Sample Size
16
Trial Duration For Participant
434

Eligibility

Recruits 16 paediatric patients.

Vulnerable Population
Pediatric subjects aged 5 to <18 years are included and 'isVulnerablePopulationSelected' is true. Age-specific assent and consent documents are provided (adolescent assent and child assent forms, parental/legal guardian informed consent forms, pregnancy ICFs). Multiple language versions and age-stratified ICF/assent documents are available in the document repository.

Inclusion criteria

  • {"criterion_text":"- Male or Female subjects 5 to less than 18 years of age at the time of randomization.\n- Confirmed diagnosis of 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\n- Active disease: at least 3 active joints\n- Inadequate response (at least 2 months) or intolerance to at least 1 DMARD, (which may include MTX or biologic agents).\n- Other protocol-defined inclusion criteria apply."}

Exclusion criteria

  • {"criterion_text":"- Rheumatic autoimmune disease other than PsA, including, but not limited to: systemic lupus erythematosus, mixed connective tissue disease, scleroderma, polymyositis, or fibromyalgia.\n- Prior history of or current inflammatory joint disease other than PsA (eg, gout, reactive arthritis, rheumatoid arthritis, ankylosing spondylitis, Lyme disease).\n- Exclusions per ILAR Edmonton Revision (Edmonton, 2001) criteria for JPsA include: rthritis 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 JIA\n- Other protocol-defined exclusion criteria apply."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Number of participants achieving ACR Pedi 30 from baseline (week 0) to week 16","definition_or_measurement_approach":"ACR Pedi 30 response from baseline to week 16 (American College of Rheumatology Pediatric criteria)."}

Secondary endpoints

  • {"endpoint_text":"- Change in subject's assessment of pain (a)","definition_or_measurement_approach":"Change from baseline to week 16 in subject/parent assessment of pain measured by visual analogue scale (VAS)."}
  • {"endpoint_text":"- Number of participants achieving ACR Pedi 20/50/70/90 (a)","definition_or_measurement_approach":"ACR Pedi 20/50/70/90 responses at week 16 (American College of Rheumatology Pediatric response criteria)."}
  • {"endpoint_text":"- Change in Childhood Heath Assessment Questionnaire (CHAQ) (a)","definition_or_measurement_approach":"Change from baseline to week 16 in CHAQ score (Childhood Health Assessment Questionnaire)."}
  • {"endpoint_text":"- Change in Juvenile Arthritis Disease Activity Score (JADAS) (a)","definition_or_measurement_approach":"Change from baseline to week 16 in JADAS (Juvenile Arthritis Disease Activity Score)."}
  • {"endpoint_text":"- Refer to protocol for fully list of secondary endpoints.","definition_or_measurement_approach":"See protocol for full list and definitions."}
  • {"endpoint_text":"- PASI-75 response at week 16 for subjects with a baseline psoriasis BSA equal or more than 3% (a)","definition_or_measurement_approach":"PASI-75 response (Psoriasis Area Severity Index ≥75% improvement) at week 16 for subjects with baseline psoriasis BSA ≥ 3%."}
  • {"endpoint_text":"- Number of participants who experience PsA flares (a)","definition_or_measurement_approach":"PsA flares defined as ≥ 30% worsening in at least 3 of 6 ACR-Pedi core variables with ≥ 30% improvement in not more than 1 of 6 ACR-Pedi core variables (assessed at week 16)."}

Other endpoints

  • {"endpoint_text":"- Estimate impact on HRQoL in pediatric subjects with JPsA","definition_or_measurement_approach":"Health-related quality of life measures (HRQoL) as specified in protocol (e.g., disease-specific or generic HRQoL instruments)."}
  • {"endpoint_text":"- Estimate safety and tolerability of apremilast compared with placebo in pediatric subjects with JPsA","definition_or_measurement_approach":"Safety and tolerability assessments including adverse events, clinical labs, vital signs, and other safety measures per protocol."}
  • {"endpoint_text":"- Characterize pharmacokinetics of apremilast in pediatric subjects with JPsA","definition_or_measurement_approach":"Pharmacokinetic sampling and analysis to characterize apremilast PK in pediatric subjects as defined in protocol."}
  • {"endpoint_text":"- Evaluate taste and acceptability of apremilast tablet and suspension in pediatric subjects with JPsA","definition_or_measurement_approach":"Assessments of taste and acceptability of tablet and suspension formulations (patient/parent-reported measures) as specified in protocol."}

Recruitment

Digital Remote Recruitment
Yes
Planned Sample Size
16
Recruitment Window Months
82
Consent Approach
Informed consent obtained from parent/legal guardian; assent obtained from children/adolescents as appropriate. Age-specific ICF/assent documents exist (child assent forms for younger children, adolescent assent forms, parental/legal guardian ICFs, pregnancy ICFs). Documents available in multiple language versions (examples in repository include EN, FR, NL, DE, ES, PT, IT, LT, RO, GR).

Methods

  • Study-specific flyers and leaflets distributed at sites and clinics
  • Peer-to-peer letters to clinicians (physician-to-physician recruitment letters)
  • GP/Dr-to-Dr referral letters
  • Patient information leaflets and site-specific recruitment brochures
  • Study animation and study video brochure (multimedia digital materials)
  • Site outreach and local pediatric rheumatology clinic recruitment (country/site-specific materials present)

Geography

Total Number Of Sites
36
Total Number Of Participants
51

Spain

Earliest CTIS Part Ii Submission Date
30-01-2024
Latest Decision Or Authorization Date
18-03-2026
Processing Time Days
778
Number Of Sites
6
Number Of Participants
6

Sites

Site Name
Sant Joan De Deu Barcelona Hospital
Department Name
Servicio de Reumatología
Principal Investigator Name
Jordi Anton Lopez
Principal Investigator Email
jordi.anton@sjd.es
Contact Person Name
Jordi Anton Lopez
Contact Person Email
jordi.anton@sjd.es
Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Servicio de Reumatología
Principal Investigator Name
Lucia Lacruz Perez
Principal Investigator Email
lacruz_lucper@gva.es
Contact Person Name
Lucia Lacruz Perez
Contact Person Email
lacruz_lucper@gva.es
Site Name
Hospital Universitario De Canarias
Department Name
Servicio de Reumatología
Principal Investigator Name
Juan Jose Bethencourt Baute
Principal Investigator Email
jbetbau@gobiernodecanarias.org
Contact Person Name
Juan Jose Bethencourt Baute
Contact Person Email
jbetbau@gobiernodecanarias.org
Site Name
Hospital General Universitario Gregorio Maranon
Department Name
Servicio de Reumatología
Principal Investigator Name
Juan Carlos Nieto-González
Principal Investigator Email
juancarlos.nietog@gmail.com
Contact Person Name
Juan Carlos Nieto-González
Contact Person Email
juancarlos.nietog@gmail.com
Site Name
Hospital Universitari Vall D Hebron
Department Name
Servicio de Reumatología
Principal Investigator Name
Estefania Moreno Ruzafa
Principal Investigator Email
reumaped@vallhebron.cat
Contact Person Name
Estefania Moreno Ruzafa
Contact Person Email
reumaped@vallhebron.cat
Site Name
Hospital Universitario Ramon Y Cajal
Department Name
Servicio de Reumatología
Principal Investigator Name
Alina-Lucica Boteanu
Principal Investigator Email
alinalucica.boteanu@salud.madrid.org
Contact Person Name
Alina-Lucica Boteanu

Belgium

Earliest CTIS Part Ii Submission Date
30-01-2024
Latest Decision Or Authorization Date
04-07-2025
Processing Time Days
521
Number Of Sites
3
Number Of Participants
4

Sites

Site Name
Centre Hospitalier Regional De La Citadelle
Department Name
Pediatric Rheumatology
Principal Investigator Name
Benoit Florkin
Principal Investigator Email
Benoit.florkin@chrcitadelle.be
Contact Person Name
Benoit Florkin
Contact Person Email
Benoit.florkin@chrcitadelle.be
Site Name
Het Ziekenhuisnetwerk Antwerpen
Department Name
Rheumatology
Principal Investigator Name
Rik Joos
Principal Investigator Email
Rik.Joos@zna.be
Contact Person Name
Rik Joos
Contact Person Email
Rik.Joos@zna.be
Site Name
Universitair Ziekenhuis Gent
Department Name
Pediatric Rheumatology
Principal Investigator Name
Joke Dehoorne
Principal Investigator Email
joke.dehoorne@uzgent.be
Contact Person Name
Joke Dehoorne
Contact Person Email
joke.dehoorne@uzgent.be

Poland

Earliest CTIS Part Ii Submission Date
30-01-2024
Latest Decision Or Authorization Date
23-03-2026
Processing Time Days
783
Number Of Sites
3
Number Of Participants
4

Sites

Site Name
Narodowy Instytut Geriatrii Reumatologii I Rehabilitacji Im Prof. Dr Hab. Med. Eleonory Reicher
Department Name
Rheumatology
Principal Investigator Name
Joanna Wójtowicz
Principal Investigator Email
agnieszka.paradowska-gorycka@spartanska.pl
Contact Person Name
Joanna Wójtowicz
Site Name
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Centralny Szpital Kliniczny Uniwersytetu Medycznego W Lodzi
Department Name
Rheumatology
Principal Investigator Name
Elżbieta Smolewska
Principal Investigator Email
j.kazmierska@csk.umed.pl
Contact Person Name
Elżbieta Smolewska
Contact Person Email
j.kazmierska@csk.umed.pl
Site Name
Wojewodzki Specjalistyczny Szpital Dzieciecy Im Sw Ludwika W Krakowie
Department Name
Rheumatology
Principal Investigator Name
Zbigniew Żuber
Principal Investigator Email
reumatologia@dzieciecyszpital.pl
Contact Person Name
Zbigniew Żuber

Germany

Earliest CTIS Part Ii Submission Date
30-01-2024
Latest Decision Or Authorization Date
18-03-2026
Processing Time Days
778
Number Of Sites
4
Number Of Participants
2

Sites

Site Name
Asklepios Klinik Sankt Augustin GmbH
Department Name
Center of General Paediatrics and Neonatology
Principal Investigator Name
Gerd Horneff
Principal Investigator Email
g.horneff@asklepios.com
Contact Person Name
Gerd Horneff
Contact Person Email
g.horneff@asklepios.com
Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Children's university hospital Charite, Campus Virchow, SPZ
Principal Investigator Name
Kirsten Minden
Principal Investigator Email
kirsten.minden@charite.de
Contact Person Name
Kirsten Minden
Contact Person Email
kirsten.minden@charite.de
Site Name
Technische Universitat Dresden
Department Name
Children's Hospital at Technical Unversity Dresden
Principal Investigator Name
Normi Brueck
Principal Investigator Email
Normi.Brueck@uniklinikum-dresden.de
Contact Person Name
Normi Brueck
Site Name
Hamburger Zentrum für Kinder- und Jugendrheumatologie
Department Name
Kinder- und Jugendrheumatologie
Principal Investigator Name
Ivan Foeldvari
Principal Investigator Email
foeldvari@t-online.de
Contact Person Name
Ivan Foeldvari
Contact Person Email
foeldvari@t-online.de

Italy

Earliest CTIS Part Ii Submission Date
30-01-2024
Latest Decision Or Authorization Date
20-03-2026
Processing Time Days
780
Number Of Sites
4
Number Of Participants
7

Sites

Site Name
Asst Centro Specialistico Ortopedico Traumatologico Gaetano Pini Cto
Department Name
Pediatric rheumatology
Principal Investigator Name
Achille Marino
Principal Investigator Email
Achille.Marino@asst-pini-cto.it
Contact Person Name
Achille Marino
Site Name
Giannina Gaslini Institute For Scientific Hospitalization And Care
Department Name
Pediatric rheumatology
Principal Investigator Name
Roberta Caorsi
Principal Investigator Email
robertacaorsi@gaslini.org
Contact Person Name
Roberta Caorsi
Contact Person Email
robertacaorsi@gaslini.org
Site Name
Azienda Sanitaria Locale 2 Lanciano Vasto Chieti
Department Name
pediatric clinic
Principal Investigator Name
Luciana Breda
Principal Investigator Email
luciana.bredach@gmail.com
Contact Person Name
Luciana Breda
Contact Person Email
luciana.bredach@gmail.com
Site Name
Bambino Gesu Childrens Hospital
Department Name
Rheumatology
Principal Investigator Name
Silvia Magni Manzoni
Principal Investigator Email
silvia.magnimanzoni@opbg.net
Contact Person Name
Silvia Magni Manzoni
Contact Person Email
silvia.magnimanzoni@opbg.net

France

Earliest CTIS Part Ii Submission Date
30-01-2024
Latest Decision Or Authorization Date
17-03-2026
Processing Time Days
777
Number Of Sites
2
Number Of Participants
3

Sites

Site Name
Hospices Civils De Lyon
Department Name
Pediatric Rheumatology
Principal Investigator Name
Heloise REUMAUX
Principal Investigator Email
heloise.reumaux@chu-lille.fr
Contact Person Name
Heloise REUMAUX
Contact Person Email
heloise.reumaux@chu-lille.fr
Site Name
Centre Hospitalier Universitaire De Lille
Department Name
Pediatric Rheumatology
Principal Investigator Name
Alexandre BELOT
Principal Investigator Email
alexandre.belot@chu-lyon.fr
Contact Person Name
Alexandre BELOT
Contact Person Email
alexandre.belot@chu-lyon.fr

Lithuania

Earliest CTIS Part Ii Submission Date
30-01-2024
Latest Decision Or Authorization Date
18-03-2026
Processing Time Days
778
Number Of Sites
1
Number Of Participants
3

Sites

Site Name
Vilniaus Universiteto Ligonine Santaros Klinikos Vsi
Department Name
Pediatrijos centras
Principal Investigator Name
Skirmante Rusoniene
Principal Investigator Email
skirmante.rusoniene@santa.lt
Contact Person Name
Skirmante Rusoniene
Contact Person Email
skirmante.rusoniene@santa.lt

Austria

Earliest CTIS Part Ii Submission Date
30-01-2024
Latest Decision Or Authorization Date
23-03-2026
Processing Time Days
783
Number Of Sites
1
Number Of Participants
3

Sites

Site Name
Vorarlberger Krankenhaus-Betriebsgesellschaft mbH
Department Name
Division of Paediatric and Adolescent Medicine
Principal Investigator Name
Christian Huemer
Principal Investigator Email
christian.huemer@lkhb.at
Contact Person Name
Christian Huemer
Contact Person Email
christian.huemer@lkhb.at

Greece

Earliest CTIS Part Ii Submission Date
30-01-2024
Latest Decision Or Authorization Date
19-03-2026
Processing Time Days
779
Number Of Sites
3
Number Of Participants
10

Sites

Site Name
Nosokomeio Paidon I Agia Sofia
Department Name
A University Pediatric Clinic, Pediatric Rheumatology Unit
Principal Investigator Name
Christina Kanaka-Gantenbein
Principal Investigator Email
chriskan@med.uoa.gr
Contact Person Name
Christina Kanaka-Gantenbein
Contact Person Email
chriskan@med.uoa.gr
Site Name
University General Hospital Attikon General Hospital Of West Attica H Agia Varvara
Department Name
3rd Department of Pediatrics
Principal Investigator Name
Lampros Fotis
Principal Investigator Email
lampros.fotis@outlook.com
Contact Person Name
Lampros Fotis
Contact Person Email
lampros.fotis@outlook.com
Site Name
Ippokratio General Hospital Of Thessaloniki
Department Name
A Pediatric Clinic
Principal Investigator Name
Vasiliki Sgouropoulou
Principal Investigator Email
vsgouro@hotmail.com
Contact Person Name
Vasiliki Sgouropoulou
Contact Person Email
vsgouro@hotmail.com

Portugal

Earliest CTIS Part Ii Submission Date
30-01-2024
Latest Decision Or Authorization Date
20-03-2026
Processing Time Days
780
Number Of Sites
4
Number Of Participants
3

Sites

Site Name
Unidade Local De Saude De Gaia/Espinho E.P.E.
Department Name
Servico de Reumatologia
Principal Investigator Name
Patrícia Pinto
Principal Investigator Email
patricia.pinto@ulsge.min-saude.pt
Contact Person Name
Patrícia Pinto
Site Name
Unidade Local De Saude Do Alto Minho E.P.E.
Department Name
Servico de Reumatologia
Principal Investigator Name
Daniela Peixoto
Principal Investigator Email
helena.pires@blueclinical.pt
Contact Person Name
Daniela Peixoto
Contact Person Email
helena.pires@blueclinical.pt
Site Name
Centro Hospitalar De Lisboa Ocidental E.P.E.
Department Name
Serviço de Reumatologia
Principal Investigator Name
Filipa Mourão
Principal Investigator Email
afmourao@chlo.min-saude.pt
Contact Person Name
Filipa Mourão
Contact Person Email
afmourao@chlo.min-saude.pt
Site Name
Unidade Local De Saude De Santa Maria E.P.E.
Department Name
Servico de Reumatologia
Principal Investigator Name
Filipa Oliveira Ramos
Principal Investigator Email
filipa.ramos@chln.min-saude.pt
Contact Person Name
Filipa Oliveira Ramos
Contact Person Email
filipa.ramos@chln.min-saude.pt

Romania

Earliest CTIS Part Ii Submission Date
30-01-2024
Latest Decision Or Authorization Date
15-05-2026
Processing Time Days
836
Number Of Sites
2
Number Of Participants
3

Sites

Site Name
Spitalul Clinic De Urgenta Pentru Copii Cluj-Napoca
Department Name
Pediatrics
Principal Investigator Name
Mihaela Nicoleta Sparchez
Principal Investigator Email
pediatrie2@spitcocluj.ro
Contact Person Name
Mihaela Nicoleta Sparchez
Contact Person Email
pediatrie2@spitcocluj.ro
Site Name
Spitalul Clinic De Urgenta Pentru Copii Louis Turcanu Timisoara
Department Name
Pediatrics
Principal Investigator Name
Otilia Marginean
Principal Investigator Email
secretariat@spitalcopiitm.ro
Contact Person Name
Otilia Marginean
Contact Person Email
secretariat@spitalcopiitm.ro

Netherlands

Earliest CTIS Part Ii Submission Date
30-01-2024
Latest Decision Or Authorization Date
24-04-2026
Processing Time Days
815
Number Of Sites
3
Number Of Participants
3

Sites

Site Name
Amsterdam UMC
Department Name
Pediatrics - Pediatric Rheumatology
Principal Investigator Name
J. Merlijn van den Berg
Principal Investigator Email
ctis@amsterdamumc.nl
Contact Person Name
J. Merlijn van den Berg
Contact Person Email
ctis@amsterdamumc.nl
Site Name
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Department Name
Rheumatology
Principal Investigator Name
Philomine van Pelt
Principal Investigator Email
p.vanpelt@erasmusmc.nl
Contact Person Name
Philomine van Pelt
Contact Person Email
p.vanpelt@erasmusmc.nl
Site Name
Wilhelmina Childrens Hospital
Department Name
Department of Pediatric Immunology and Rheumatology
Principal Investigator Name
Joost Swart
Principal Investigator Email
J.F.Swart@umcutrecht.nl
Contact Person Name
Joost Swart
Contact Person Email
J.F.Swart@umcutrecht.nl

Sponsor

Primary sponsor

Full Name
Amgen Inc.
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Contract research organisations

Name
Almac Clinical Technologies LLC
Responsibilities
sponsorDuties code: 3
Name
Excelya Greece CRO Single Member S.A.
Responsibilities
Administrative activities in Greece
Name
WCG Clinical Inc.
Responsibilities
Site Training Portal to provide training to investigators on the following assessments: Joint Count; PASI/BAS; Tanner Stages; C-SSRS
Name
QPS LLC
Responsibilities
sponsorDuties code: 4
Name
Cytel Inc.
Responsibilities
Independent Biostatics Group (Data Monitoring Committee)

Third parties

  • {"country":"United States","full_name":"Almac Clinical Technologies LLC","duties_or_roles":"sponsorDuties code: 3","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"WCG Clinical Inc.","duties_or_roles":"Site Training Portal to provide training to investigators on the following assessments: Joint Count; PASI/BAS; Tanner Stages; C-SSRS","organisation_type":"Pharmaceutical company"}
  • {"country":"Greece","full_name":"Excelya Greece CRO Single Member S.A.","duties_or_roles":"Administrative activities in Greece","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Subject cost reimbursement for the following EU countries: Belgium, Netherlands, Germany, Spain and Greece","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Medical Equipment Supplies And Management Limited","duties_or_roles":"Provision of equipment and ancillary supplies to clinical trial sites","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"sponsorDuties code: 7","organisation_type":"Pharmaceutical company"}
  • {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services S.a.r.l.","duties_or_roles":"Central laboratory services (contact: ctasubmissions@labcorp.com)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"QPS LLC","duties_or_roles":"sponsorDuties code: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Cytel Inc.","duties_or_roles":"Independent Biostatics Group (Data Monitoring Committee)","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"France","full_name":"Quipment","duties_or_roles":"Provision of equipment and ancillary supplies to clinical trial sites","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Azenta US Inc.","duties_or_roles":"Back-up samples long term storage at end of study.","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
Apremilast (ORAL SOLUTION)
Active Substance
APREMILAST
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Authorisation Status
Marketing authorisation exists (prodAuthStatus 1, MIA number 108520 F)
Maximum Dose
60 mg (max daily dose amount)
Investigational Product Name
Apremilast (FILM COATED TABLET / TABLETS)
Active Substance
APREMILAST
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Authorisation Status
Marketing authorisation exists (prodAuthStatus 1, MIA number 108520 F)
Maximum Dose
60 mg (max daily dose amount)
Investigational Product Name
Apremilast placebo product (10, 20 and 30 mg tablets and 5 mg/mL oral solution)
Modality
Other

Related trials

Other published trials that may interest you.