- ICH GCP
- US Clinical Trials Registry
- Klinisk forsøg NCT02461134
Clinical Study to Investigate the Biological Activity, Safety, Tolerability, and Pharmacokinetics of Ponesimod in Subjects With Symptomatic Chronic GVHD
A Phase 2, Open-label, Single-arm, Intra-subject Dose-escalation Study to Investigate the Biological Activity, Safety, Tolerability, and Pharmacokinetics of Ponesimod in Subjects With Symptomatic Moderate or Severe Chronic GVHD Inadequately Responding to First or Second Line Therapy
Chronic graft versus host diseasre (GVHD) is a serious reaction that might occur in a person (the host) who has received cells or organs (graft) from another person because the graft attacks the host's cells. Currently there are no approved therapies for chronic GVHD in the USA, and patients with chroninc GVHD are treated with immunosuppressant drugs. T-lymphocytes (a type of white blood cells) are likely to play a role in the development of chronic GVHD. Due to the capacity of ponesimod to block the traffic of T-lymphocytes, ponesimod may be a new therapeutic approach to treat chroninc GVHD.
The main objective of this study is to assess the effectiveness and safety of several doses of ponesimod in subjects with chronic GVHD who did not respond to standard available treatments.
Studieoversigt
Status
Betingelser
Intervention / Behandling
Undersøgelsestype
Tilmelding (Faktiske)
Fase
- Fase 2
Kontakter og lokationer
Studiesteder
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Arizona
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Scottsdale, Arizona, Forenede Stater, 85258
- Virginia Piper Cancer Institute
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California
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La Jolla, California, Forenede Stater, 92093
- Moore Cancer Center - UCSD
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Los Angeles, California, Forenede Stater, 90095
- David Geffen School of Med at UCLA
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Illinois
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Chicago, Illinois, Forenede Stater, 60611
- Northwestern University
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Indiana
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Indianapolis, Indiana, Forenede Stater, 46202
- Indiana University Simon Cancer Center
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Maryland
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Bethesda, Maryland, Forenede Stater, 20892-1203
- National Cancer Institute
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Minnesota
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Minneapolis, Minnesota, Forenede Stater, 55455
- University of Minnesota - Masonic Cancer CTR CLIN TRIALS CTR
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Missouri
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Saint Louis, Missouri, Forenede Stater, 63110
- Washington Univ School of Med, Oncology (St.Louis)
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New York
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Stony Brook, New York, Forenede Stater, 11794
- Stony Brook Univ. Medical Center
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Washington
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Seattle, Washington, Forenede Stater, 98109-1023
- Fred Hutchinson Cancer Res CTR
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Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
Tager imod sunde frivillige
Køn, der er berettiget til at studere
Beskrivelse
Inclusion Criteria:
- Signed informed consent
- Symptomatic moderate or severe chronic GVHD patients in need of a change of systemic immunosuppressant (IS) therapy
- Women of child bearing potential must have a negative pregnancy test and use reliable methods of contraception
Exclusion Criteria:
- Clinically significant medical conditions including active or uncontrolled infections, new or recurrent malignancy, serious cardiac, pulmonary, or renal disease, and uncontrolled diabetes.
- Karnofsky Performance Score < 60.
- Immunosuppressant therapies other than allowed background therapy
- Anti-arrhythmic and heart rate lowering drugs.
- Any other circumstances or conditions, which, in the opinion of the investigator, may affect full participation in the study or compliance with the protocol
Studieplan
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: N/A
- Interventionel model: Enkelt gruppeopgave
- Maskning: Ingen (Åben etiket)
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
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Eksperimentel: Ponesimod
Study treatment consists of 3 consecutive periods: 5 mg ponesimod treatment period (including up-titration), 10 mg treatment period (including up-titration) and a 20 mg treatment period.
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Oral film-coated tablets at the doses of 2, 3, 4, 5, 6, 7, 8, 9, 10, and 20 mg.
One tablet of ponesimod at any dose will be taken orally once daily.
Andre navne:
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Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
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Change in Peripheral Absolute Lymphocyte Count From Baseline to Week 4, 8 and 12
Tidsramme: From baseline to Week 12
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The primary pharmacodynamic endpoint assesses intra-subject dose response during the first 12 weeks of treatment.
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From baseline to Week 12
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Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
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Incident Rate of Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Tidsramme: From the first study drug intake up to 30 days after last study drug intake (Week 24)
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This outcome measure reports the occurrence of adverse events (AEs), and serious adverse events (SAEs) during the treatment period and the follow-up period, and AEs leading to premature discontinuation of study drug.
A treatment-emergent AE is any AE temporally associated with the use of study treatment whether or not considered by the investigator as related to study treatment.
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From the first study drug intake up to 30 days after last study drug intake (Week 24)
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Andre resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
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Assessment of a Partial or Complete Overall Response at Week 24
Tidsramme: At Week 24
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The exploratory efficacy endpoint is based on the 2014 NIH Consensus Development Project response criteria.
A complete overall response is defined as a resolution of all reversible manifestations due to chronic GVHD in each organ as defined per NIH Consensus Development Project response criteria.
A partial overall response is defined as improvement in a measure for at least one organ without progression in measures for any other organ.
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At Week 24
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Samarbejdspartnere og efterforskere
Sponsor
Efterforskere
- Studiestol: Daniele D'Ambrosio, MD, PhD, Actelion
Datoer for undersøgelser
Studer store datoer
Studiestart (Faktiske)
Primær færdiggørelse (Faktiske)
Studieafslutning (Faktiske)
Datoer for studieregistrering
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først opslået (Skøn)
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- AC-058C202
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
Studerer et amerikansk FDA-reguleret lægemiddelprodukt
Studerer et amerikansk FDA-reguleret enhedsprodukt
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Kliniske forsøg med Kronisk graft versus værtssygdom
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Jules Bordet InstituteMacopharma; Belgian Hematological SocietyRekrutteringRefractory Chronic Graft Versus Host Disease (cGVHD)Belgien
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Jazz PharmaceuticalsAfsluttetAkut-graft-versus-host sygdom | Graft-versus-host-sygdomForenede Stater, Belgien, Det Forenede Kongerige, Grækenland, Tyskland, Spanien, Frankrig, Italien, Østrig, Canada, Bulgarien, Kroatien, Polen, Portugal
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John LevineAfsluttetGVHD | Lavrisiko akut graft-versus-host-sygdom | Graft-versus-host-sygdomForenede Stater
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Regimmune CorporationAfsluttetGraft vs værtssygdom | Graft-versus-host-sygdom | Akut-graft-versus-host sygdom | Forebyggelse af aGVHDForenede Stater
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Peking Union Medical College HospitalIkke rekrutterer endnuVaginalt mikrobiom | Graft Versus Host Reaktion
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Victor AquinoLedigTransplantationsrelateret lidelse | GVH - Graft Versus Host ReactionForenede Stater
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Fundación Pública Andaluza para la gestión de la...Instituto de Salud Carlos IIIAktiv, ikke rekrutterendeGraft-versus-host-sygdomSpanien
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)Trukket tilbageGraft-versus-host sygdomsforebyggelseForenede Stater
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Shenzhen Xbiome Biotech Co., Ltd.Beijing Improve-Quality Tech.Co., Ltd.Rekruttering
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Cytopeutics Sdn. Bhd.Universiti Tunku Abdul RahmanAktiv, ikke rekrutterendeAkut-graft-versus-host sygdomMalaysia
Kliniske forsøg med Ponesimod
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ActelionAfsluttetMultipel sclerosePolen, Ukraine, Det Forenede Kongerige, Forenede Stater, Tyskland, Spanien, Bulgarien, Rumænien, Serbien, Canada, Finland, Sverige, Den Russiske Føderation, Ungarn, Tjekkiet, Holland, Israel, Schweiz, Østrig, Frankrig
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ActelionAfsluttetSund og raskDet Forenede Kongerige
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ActelionAfsluttetSund og raskForenede Stater
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Janssen Pharmaceutica N.V., BelgiumAfsluttet
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ActelionAfsluttetMultipel scleroseForenede Stater, Spanien, Ungarn, Israel, Portugal, Tyskland, Den Russiske Føderation, Hviderusland, Polen, Serbien, Letland, Kalkun, Kroatien, Canada, Tjekkiet, Bulgarien, Bosnien-Hercegovina, Finland, Frankrig, Georgien, Græken... og mere
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ActelionAfsluttetMultipel scleroseForenede Stater, Hviderusland, Bosnien-Hercegovina, Bulgarien, Canada, Kroatien, Tjekkiet, Finland, Frankrig, Georgien, Tyskland, Grækenland, Ungarn, Israel, Italien, Letland, Litauen, Mexico, Polen, Portugal, Rumænien, Den Russiske... og mere
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ActelionAfsluttet
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ActelionAfsluttetMultipel scleroseForenede Stater, Tyskland, Ungarn, Polen, Det Forenede Kongerige, Australien, Østrig, Belgien, Bulgarien, Canada, Tjekkiet, Danmark, Frankrig, Grækenland, Italien, Mexico, Portugal, Den Russiske Føderation, Spanien, Schweiz
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ActelionAfsluttetPsoriasisØstrig, Belgien, Bulgarien, Tjekkiet, Danmark, Frankrig, Ungarn, Italien, Litauen, Rumænien, Den Russiske Føderation, Slovakiet, Spanien, Sverige, Schweiz, Ukraine, Det Forenede Kongerige
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ActelionAfsluttet