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SNCTP000004392 | NCT04528082 | BASEC2020-02571

Wir untersuchen die Wirksamkeit und Sicherheit von Apremilast bei pädiatrischen Patienten mit aktiven Mundgeschwüren in Verbindung mit der Behçet-Krankheit.

Base di dati: BASEC (Importata da 30.11.2021), WHO (Importata da 18.04.2021)
Cambiato: 18.10.2021
Categoria di malattie: Altro

Descrizione riassuntiva della sperimentazione (Fonte di dati: BASEC)

Ziel der Studie ist es, die Wirksamkeit von Apremilast im Vergleich zu Placebo bei der Behandlung von Mundgeschwüren bei pädiatrischen Probanden im Alter von 2 bis < 18 Jahren im Zusammenhang mit der Behçet-Krankheit bis Woche 12 abzuschätzen. Die Probanden werden untersucht, um zu prüfen, ob sie die Voraussetzungen zur Teilnahme an der Studie erfüllen. Falls sie die Anforderungen erfüllen, werden sie für einen Behandlungszeitraum von 12 Wochen aufgenommen. Während dieser ersten 12 Wochen haben die Versuchspersonen eine Chance von 2 zu 3, Apremilast (entweder in Tabletten- oder Flüssigformulierung) und eine Chance von 1 zu 3, ein Placebo (wie Zuckerpillen/Flüssigkeit) zu erhalten. Dies geschieht nach dem Zufallsprinzip (wie das Werfen einer Münze). Weder die Versuchspersonen noch Ihr Prüfarzt oder die Studienschwestern wissen, welche Behandlung die Versuchspersonen erhalten werden. Nach den 12 Wochen werden alle Versuchspersonen in die aktive Behandlungsgruppe aufgenommen und erhalten Apremilast.

Malattie studiate (Fonte di dati: BASEC)

Diese Studie wird durchgeführt, um mehr über Apremilast bei Kindern und Jugendlichen mit aktiven Mundgeschwüren im Zusammenhang mit der Behçet-Krankheit zu erfahren.

Health conditions (Fonte di dati: WHO)

Behçet Disease

Malattia rara (Fonte di dati: BASEC)

Interventi esaminati (p. es. medicamento, terapia, campagna) (Fonte di dati: BASEC)

Es wird untersucht wie gut Apremilast bei der Behandlung von aktiven Mundgeschwüren im Zusammenhang mit der Behçet-Krankhei wirkt, wie sicher und gut verträglich Apremilast bei Kindern und Jugendlichen ist und ob es irgendwelche Nebenwirkungen verursacht. In dieser Studie werden verschiedene Dosierungen von Apremilast sowohl in Tablettenform als auch in einer flüssigen Suspensionsformulierung von Apremilast untersucht.

Interventions (Fonte di dati: WHO)

Drug: Apremilast
Drug: Placebo

Criteri per la partecipazione alla sperimentazione (Fonte di dati: BASEC)

Um an dieser Studie teilnehmen zu können, muss man zwischen 2 und weniger als 18 Jahre alt sein, während des Screenings und am ersten Tag mindestens zwei Mundgeschwüre haben und zuvor mit mindestens einer nicht-biologischen Therapie gegen die Behçet-Krankheit behandelt worden sein

Criteri di esclusione (Fonte di dati: BASEC)

Von der Studienteilnahme ausgeschlossen sind Personen, bei denen eine aktive Organbeteiligung vorliegt oder die zuvor mit biologischen Therapien gegen die Behçet-Krankheit behandelt wurden. Jegliche zusätzliche Medikation zur Behandlung von Mund- und Genitalgeschwüren im Zusammenhang mit der Behçet-Krankheit sollte vor Beginn der Einnahme des Studienmedikaments abgesetzt werden

Inclusion/Exclusion Criteria (Fonte di dati: WHO)


Key Inclusion Criteria

- Male or Female participants 2 to < 18 years of age at randomization.

- Diagnosed with behçet's disease (BD) meeting the International Study Group for Behçet
Disease (ISGBD) criteria at any time prior to the screening visit.

- Oral ulcers that occurred = 3 times within the 12-month period prior to the screening
visit.

- Participant must have = 2 oral ulcers at both the screening visit and on day 1.

- Participant has had prior treatment with = 1 non-biologic BD therapy, such as, but not
limited to, topical corticosteroids or systemic treatment.

Key Exclusion Criteria

- Behcet's disease-related active major organ involvement - pulmonary (eg, pulmonary artery
aneurysm), vascular (eg, thrombophlebitis), gastrointestinal (eg, ulcers along the
gastrointestinal tract), and central nervous system (CNS) (eg, meningoencephalitis)
manifestations, and ocular lesions (eg, uveitis) requiring immunosuppressive therapy;
however:

- Previous major organ involvement is allowed if it occurred =1 year prior to the
screening visit and is not active at time of enrollment

- Participants with mild BD-related ocular lesions not requiring systemic
immunosuppressive therapy are allowed

- Participants with BD-related arthritis and BD-skin manifestations are also allowed.

- Participants with BD-related arthritis and BD-skin manifestations are also allowed.
Previous exposure to biologic therapies for the treatment of BD oral ulcers, previous
biologic exposure is allowed for other indications (including other manifestations of
BD).

Minimum age: 2 Years
Maximum age: 17 Years
Sex: All

Altri dati sulla sperimentazione nel registro primario dell’OMS

http://www.who.int/trialsearch/Trial2.aspx?TrialID=NCT04528082

Altri dati sulla sperimentazione dalla banca dati dell’OMS (ICTRP)

https://trialsearch.who.int/Trial2.aspx?TrialID=NCT04528082

Altre informazioni sulla sperimentazione

Data di registrazione della sperimentazione

24.08.2020

Inserimento del primo partecipante

15.07.2021

Stato di reclutamento

Not recruiting

Data globale di conclusione della sperimentazione

30.11.-0001

Titolo scientifico (Fonte di dati: WHO)

A Phase 3, Multicenter, Double-blind, Randomized, Placebo-controlled, Parallel Group Study, Followed by an Active Treatment Phase to Evaluate the Efficacy and Safety of Apremilast in Children From 2 to Less Than 18 Years of Age With Active Oral Ulcers Associated With Behçet's Disease (BEAN)

Tipo di sperimentazione (Fonte di dati: WHO)

Interventional

Disegno della sperimentazione (Fonte di dati: WHO)

Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).

Fase (Fonte di dati: WHO)

Phase 3

Punti finali primari (Fonte di dati: WHO)

Area Under the Curve for the Number of Oral Ulcers from Week 0 Through Week 12 (AUCw0-12)

Punti finali secondari (Fonte di dati: WHO)

Change from Week 0 to Week 12 in Disease Activity
Change from Week 0 to Week 12 in the Pain of Oral Ulcers
Change from Week 0 to Week 12 of the 10-item Short Form Survey (SF-10)
Change from Week 0 to Week 52 in Body Mass Index (BMI)
Change from Week 0 to Week 52 in Body Weight
Change from Week 0 to Week 52 in Height
Change from Week 0 to Week 52 in Tanner Staging
Complete Response Rate for Genital Ulcers
Complete Response Rate for Oral Ulcers
Number of Oral Ulcers from Week 0 to Week 12
Number of Participants Reporting One or More Common Terminology Criteria for Adverse Events (CTCAE) = Grade 3 Adverse Events
Number of Participants Reporting One or More Treatment-related Adverse Events
Number of Participants who Experience a Clinically Significant Change from Week 0 in Clinical Laboratory Tests
Number of Participants who Experience a Clinically Significant Change from Week 0 in Physical Examinations
Number of Participants who Experience a Clinically Significant Change from Week 0 in Vital Signs
Number of Participants who Experience One or More Serious Adverse Events (SAEs)
Number of Participants who Experience One or More Treatment-emergent Adverse Events (TEAEs)
Number of Participants Who Have New-onset or Recurrence of Behçet's-related Manifestations (Other than Oral and Genital Ulcers)
Number of Participants who Require Protocol-prohibited Medications due to Worsening of Behçet's Disease
Number of Participants whose Number of Oral Ulcers is Reduced by = 50% from Week 0
Number of Participants With Suicidal Ideation or Behaviour Assessed Via the Columbia Suicide Severity Rating Scale (C-SSRS)
Plasma Concentrations of Apremilast
Taste and Acceptability

Contatto per informazioni (Fonte di dati: WHO)

Please refer to primary and secondary sponsors

Risultati della sperimentazione (Fonte di dati: WHO)

Sintesi dei risultati

ancora nessuna informazione disponibile

Collegamento ai risultati nel registro primario

ancora nessuna informazione disponibile

Informazioni sulla disponibilità dei dati dei singoli partecipanti

ancora nessuna informazione disponibile

Siti di esecuzione della sperimentazione

Siti di esecuzione in Svizzera (Fonte di dati: BASEC)

Losanna

Contatto per maggiori informazioni sulla sperimentazione

Dati della persona di contatto in Svizzera (Fonte di dati: BASEC)

Prof. Dr. med. Michael Hofer
+41 79 556 46 82
Michael.Hofer@chuv.ch

Contatto per informazioni generali (Fonte di dati: WHO)

Amgen Call Center
866-572-6436
medinfo@amgen.com

Contatto per informazioni scientifiche (Fonte di dati: WHO)

MD
Amgen

Responsabile della sperimentazione

Sponsor principale (Fonte di dati: WHO)

Amgen

Autorizzazione da parte della commissione d’etica (Fonte di dati: BASEC)

Nome della commissione d’etica che rilascia l’autorizzazione (nel caso di studi multicentrici solo la commissione direttiva)

Commission cantonale d’Éthique de la Recherche sur l’être humain Vaud (CER-VD)

Data di autorizzazione da parte della commissione d’etica

04.05.2021

Altri numeri di identificazione delle sperimentazioni

Numero di identificazione della sperimentazione della commissione d’etica (BASEC-ID) (Fonte di dati: BASEC)

2020-02571

Secondary ID (Fonte di dati: WHO)

2019-002787-27
20190530