Denne side blev automatisk oversat, og nøjagtigheden af ​​oversættelsen er ikke garanteret. Der henvises til engelsk version for en kildetekst.

Safety and Efficacy of BAF312 in Dermatomyositis

9. december 2020 opdateret af: Novartis Pharmaceuticals

A Double Blind, Randomized, Placebo-controlled Study to Evaluate, Safety, Tolerability, Efficacy and Preliminary Dose-response of BAF312 in Patients With Active Dermatomyositis (DM)

This study investigated the dose response relationship for the efficacy and safety of BAF312 compared to placebo in active DM patients over a treatment period of 6+6 months and to determine the minimum dose required for a maximal clinical effect. The study was composed of 2 periods: a double-blind period 1 with BAF312 administered at different daily doses (0.5, 2, 10 mg and placebo) and a fixed-dose Period 2 in which BAF312 was administered at the dose of 2 mg daily .

Studieoversigt

Status

Afsluttet

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

The study was prematurely terminated based on the results of an interim analysis where BAF312 did not demonstrate superior efficacy over placebo and a dose-response relationship was not observed. There were no safety concerns. Approximately 56 participants were planned to be randomized. A total of 17 participants were enrolled and randomized by the time the study was terminated.

Undersøgelsestype

Interventionel

Tilmelding (Faktiske)

17

Fase

  • Fase 2

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiesteder

  • Forenede Stater
    • Arizona
      • Phoenix, Arizona, Forenede Stater, 85028
        • Novartis Investigative Site
    • California
      • Los Angeles, California, Forenede Stater, 90095
        • Novartis Investigative Site
      • Orange, California, Forenede Stater, 92868
        • Novartis Investigative Site
    • Florida
      • Miami, Florida, Forenede Stater, 33136
        • Novartis Investigative Site
    • Kansas
      • Kansas City, Kansas, Forenede Stater, 66160
        • Novartis Investigative Site
    • Massachusetts
      • Boston, Massachusetts, Forenede Stater, 02115
        • Novartis Investigative Site
  • Japan
    • Chiba
      • Chiba-city, Chiba, Japan, 260-8712
        • Novartis Investigative Site
    • Miyagi
      • Sendai city, Miyagi, Japan, 980-8574
        • Novartis Investigative Site
  • Tjekkiet
    • Czech Republic
      • Prague 2, Czech Republic, Tjekkiet, 128 50
        • Novartis Investigative Site

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

18 år til 75 år (Voksen, Ældre voksen)

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

Alle

Beskrivelse

Key Inclusion Criteria:

Written informed consent must be obtained before any assessment is performed.

  • Patients who have been defined as "definite" or "probable" based on the criteria of Bohan and Peter (Bohan and Peter 1975) for dermatomyositis at least 3 months before screening
  • Patients must have active disease as defined by muscle weakness
  • Patients may be on a stable dose of corticosteroid (up/equal to 20 mg once daily prednisone equivalent)
  • Patients currently treated with oral or subcutaneous MTX must have been a stable dose of no more/equal to than 25 mg per week
  • Patients currently treated with Azathioprine must have been a stable maintenance dose of no more/equal to 3 mg/kg/day
  • Negative cancer screening conducted in the 12 months prior to screening visit

Key Exclusion Criteria

  • Dermatomyositis patients having overlap myositis or any other type of myositis including paraneoplastic myositis, drug-induced myopathy, necrotizing myositis
  • Preexisting severe cardiac or pulmonary conditions, malignancy of any organ system or significant eye diseases.
  • Uncontrolled diabetes mellitus or diabetes complicated with organ involvement.
  • Pregnant or nursing (lactating) women

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Tredobbelt

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: BAF312 0.5mg
During period 1, participants were uptitrated daily from BAF312 0.25 mg to 0.5 mg over a 10 day period. After, participants continued on 0.5 mg daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.
Medicin: BAF312
BAF312 was provided as film-coated tablets in strengths of 0.25, 0,5, 1 and 2 mg for oral administration.
Andre navne:
  • Siponimod
Eksperimentel: BAF312 2mg
During period 1, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.
Medicin: BAF312
BAF312 was provided as film-coated tablets in strengths of 0.25, 0,5, 1 and 2 mg for oral administration.
Andre navne:
  • Siponimod
Eksperimentel: BAF312 10 mg
During period 1, participants were uptitrated daily from BAF312 0.25 mg to 10.0 mg over a 10 day period. After, participants continued on 10.0 mg daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.
Medicin: BAF312
BAF312 was provided as film-coated tablets in strengths of 0.25, 0,5, 1 and 2 mg for oral administration.
Andre navne:
  • Siponimod
Placebo komparator: Placebo
During period 1, participants received matching placebo daily for up to 24 weeks. During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period. After, participants continued on 2.0 mg daily for up to 24 weeks.
Medicin: Placebo
Matching placebo to BAF312 as tablets for oral administration.

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score
Tidsramme: Baseline, 6 months
Each muscles tested was evaluated on a 0 - 10 scale where 0 indicated the weakest muscle score and 10 indicated the strongest muscle score. The total MMT24 score ranged from 0 - 240, where an increasing trend in the values indicates improvement. A positive change from baseline indicates improvement.
Baseline, 6 months

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
BAF312 Plasma Concentration
Tidsramme: 6 months
6 months
Peripheral Blood Lymphocyte Counts
Tidsramme: baseline, 6 months
Absolute lymphocyte counts
baseline, 6 months
Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score
Tidsramme: baseline, 3 months
Each muscles tested was evaluated on a 0-10 scale where 0 indicated the weakest muscle score and 10 indicated the strongest muscle score. the total MMT24 score ranged from 0-240, where an increasing trend in the values indicates improvement. A positive change from baseline indicates improvement.
baseline, 3 months
Change From Baseline in 6 Minutes Walking Distance (6-MWD) Test
Tidsramme: baseline, 6 months
baseline, 6 months

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Novartis Pharmaceuticals

Publikationer og nyttige links

Den person, der er ansvarlig for at indtaste oplysninger om undersøgelsen, leverer frivilligt disse publikationer. Disse kan handle om alt relateret til undersøgelsen.

Hjælpsomme links

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

25. august 2013

Primær færdiggørelse (Faktiske)

17. februar 2016

Studieafslutning (Faktiske)

17. februar 2016

Datoer for studieregistrering

Først indsendt

2. december 2013

Først indsendt, der opfyldte QC-kriterier

6. januar 2014

Først opslået (Skøn)

7. januar 2014

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

5. januar 2021

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

9. december 2020

Sidst verificeret

1. december 2018

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • CBAF312X2206
  • 2013-001799-39 (EudraCT nummer)

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

Kliniske forsøg med Active Dermatomyositis

Kliniske forsøg med BAF312

Søg i lignende forsøg

Sponsorer og samarbejdspartnere

Medicinske tilstande

Narkotikainterventioner

CROs by country

CROs in Russia

Betingelser

Sjældne sygdomme

Narkotikainterventioner

Kosttilskud

Sponsor / samarbejdspartnere

Placeringer

Abonner