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En åben-label undersøgelse for deltagere, der er ikke-responderere ved slutningen af ​​behandlingsvurdering på VRDN-001-101 og VRDN-001-301 pivotale undersøgelser (OLE)

22. juli 2026 opdateret af: Viridian Therapeutics, Inc.
Et åbent studie for deltagere, der ikke reagerer ved slutningen af ​​behandlingsperiodens vurdering (dvs. 15 uger) i VRND-001-101 (THRIVE) og VRDN-001-301 (THRIVE-2) pivotale undersøgelser

Studieoversigt

Status

Afsluttet

Betingelser

Intervention / Behandling

Undersøgelsestype

Interventionel

Tilmelding (Faktiske)

139

Fase

  • Fase 3

Kontakter og lokationer

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

Studiesteder

  • Det Forenede Kongerige
      • Bristol, Det Forenede Kongerige
        • University Hospital Bristol and Weston NHS Foundation Trust- Bristol Eye Hospital
      • London, Det Forenede Kongerige, NW15QH
        • Imperial College Healthcare NHS Trust Western Eye Hospital
      • London, Det Forenede Kongerige, SE17EH
        • Guy's and St. Thomas Trust
      • Newcastle, Det Forenede Kongerige
        • Newcastle Eye Centre
    • London
      • Norwich, London, Det Forenede Kongerige
        • Norfolk and Norwich University Hospital NHS Foundation Trust
  • Forenede Stater
    • California
      • Los Angeles, California, Forenede Stater, 90033
        • USC Eye Institute
      • Los Angeles, California, Forenede Stater, 90023
        • Advancing Research International, LLC
      • Newport Beach, California, Forenede Stater, 90023
        • Advancing Research International, LLC
      • Palo Alto, California, Forenede Stater, 94303
        • Stanford Byers Eye Institute
      • San Diego, California, Forenede Stater, 92108
        • Cockerham Eye Consultants, PC
    • Florida
      • Miami, Florida, Forenede Stater, 33136
        • University of Miami Miller School of Medicine, Bascom Palmer Eye Institute
      • Sarasota, Florida, Forenede Stater, 34239
        • Sarasota Retina Institute
    • Illinois
      • Oak Lawn, Illinois, Forenede Stater, 60453
        • Vision Medical Research
    • Massachusetts
      • East Weymouth, Massachusetts, Forenede Stater, 02189
        • Ophthalmic Consultants of Boston
    • Michigan
      • Livonia, Michigan, Forenede Stater, 48152
        • Kahana Oculoplastic & Orbital Surgery
    • Nevada
      • Las Vegas, Nevada, Forenede Stater, 89144
        • Ophthalmic Plastic, Reconstructive, Orbital and Cosmetic Surgery
    • New Jersey
      • Newark, New Jersey, Forenede Stater, 07103
        • Rutgers New Jersey Medical School
      • Somerset, New Jersey, Forenede Stater, 08873
        • The Center for Eye and Facial Plastic Surgery
    • Pennsylvania
      • Philadelphia, Pennsylvania, Forenede Stater, 19104
        • Hospital of the University of Pennsylvania Perleman Center
    • Texas
      • Houston, Texas, Forenede Stater, 77030
        • Baylor College of Medicine
      • Houston, Texas, Forenede Stater, 77074
        • Neuro-Eye Clinical Trials
    • Vermont
      • Burlington, Vermont, Forenede Stater, 05401
        • University of Vermont Medical Center
    • Washington
      • Seattle, Washington, Forenede Stater, 98104-2430
        • University of Washington Medical Center
  • Frankrig
      • Angers, Frankrig, 49933
        • CHU Angers
    • Hérault
      • Montpellier, Hérault, Frankrig, 34295
        • Centre Hospitalier Universitaire de Montpellier Hopital Lapeyronie
    • Loire-Atlantique
      • Saint-Herblain, Loire-Atlantique, Frankrig, 44800
        • Centre Hospitalier Universitaire De Nantes G.R. Laenne
  • Holland
      • Amsterdam, Holland
        • Amsterdam UMC
  • Polen
      • Bialystok, Polen, 15-276
        • Uniwersytecki Szpital Kliniczny w Bialymstoku
      • Gdansk, Polen, 80809
        • Optimum Profesorskie Centrum Okulistyki
      • Piaseczno, Polen, 05-500
        • Centrum Medyczne Pulawska
    • Piasta
      • Wałbrzych, Piasta, Polen, 58-304
        • Centrum Medyczne Piasta
  • Spanien
      • Córdoba, Spanien, 14012
        • Hospital Arruzafa. Servicio de Oftalmologia
      • Madrid, Spanien, 28034
        • Hospital Universitario Ramon y Cajal
      • Madrid, Spanien
        • Hospital Clinico San Carlos
      • Seville, Spanien, 41009
        • Hospital Universitario Virgen De La Macarena
      • Zaragoza, Spanien, 50009
        • Hospital Universitario Miguel Servel
  • Tyrkiet (Türkiye)
      • Ankara, Tyrkiet (Türkiye), 06230
        • Hacettepe Universitesi Tip Fakultesi
      • Ankara, Tyrkiet (Türkiye), 06560
        • Gazi University Medical Faculty Hospital
      • Antalya, Tyrkiet (Türkiye), 7070
        • Akdeniz University Medical Faculty Hospital
      • Istanbul, Tyrkiet (Türkiye), 34899
        • Marmara University Faculty of Medicine
  • Tyskland
      • Berlin, Tyskland
        • Charite - Universitatsmedizin Berlin KoR, Campus Virchow Klinikum, Klinik tor Augenheilkunde
      • Dresden, Tyskland
        • Universitatsklinikum Carl Gustav Carus
      • Essen, Tyskland
        • Universitatsklinikum Essen AoR - Klinik fur Augenheilkunde
      • Freiburg im Breisgau, Tyskland
        • University Medical Center Freiburg
    • Lower Saxony
      • Göttingen, Lower Saxony, Tyskland, 37075
        • Universitatmedizin Gottingen

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

  • Voksen
  • Ældre voksen

Tager imod sunde frivillige

Ingen

Beskrivelse

Inklusionskriterier:

  • Har gennemført mindst 5 infusioner og vurderinger, der kræves for at bestemme proptosis responder-status målt med exophthalmometer 3 uger efter den femte infusion (dvs. uge 15) som defineret i enten VRDN-001-101 eller VRDN-001-301 pivotale undersøgelser
  • Har været deltager i enten VRDN-001-101 eller VRDN-001-301 pivotale undersøgelser og fundet ikke-responderende
  • Skal acceptere at bruge højeffektiv prævention som specificeret i protokollen
  • Kvindelige deltagere i skjoldbruskkirteløjensygdomme (TED) skal have en negativ uringraviditetstest

Ekskluderingskriterier:

  • Må ikke have modtaget tidligere behandling med et andet anti-IGF-1R monoklonalt antistof eller noget andet forsøgsmiddel for TED
  • Må ikke have brugt systemiske kortikosteroider inden for 2 uger før dag 1
  • Må ikke have modtaget rituximab, tocilizumab eller andre immunsuppressive midler inden for 8 uger før dag 1
  • Må ikke have en anamnese eller screeningsaudiometrivurdering af klinisk signifikant (som bestemt af investigator) ørepatologi, relevant ørekirurgi eller høretab
  • Må ikke have modtaget et forsøgsmiddel for nogen tilstand (andre end VRDN-001 eller placebo forbundet med VRDN-001-101 eller VRDN-001-301 pivotale undersøgelser) inden for 8 uger før dag 1

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: N/A
  • Interventionel model: Parallel tildeling
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: Veligrotug/Veligrotug
Non-responder participants who received veligrotug in the parent Study VRDN-001-101 or VRDN-001-301 will receive veligrotug 10 milligrams (mg)/kilogram (kg) every 3 weeks (Q3W) for 12 weeks.
Medicin: Veligrotug
Veligrotug 10 mg/kg (5 infusions of Veligrotug 10 mg/kg)
Andre navne:
  • VRDN-001
Eksperimentel: Placebo/Veligrotug
Non-responder participants who received placebo in the parent Study VRDN-001-101 or VRDN-001-301 will receive veligrotug 10 mg/kg Q3W for 12 weeks.
Medicin: Veligrotug
Veligrotug 10 mg/kg (5 infusions of Veligrotug 10 mg/kg)
Andre navne:
  • VRDN-001

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Proptosis Responder Rate (PRR) in the Most Proptotic Eye as Measured by Exophthalmometer
Tidsramme: Baseline to Week 15
Proptosis response in the most proptotic eye was defined as a reduction of proptosis of ≥2 millimeters (mm) from baseline in the most proptotic eye (without a corresponding increase of ≥2 mm in the other eye) as measured by exophthalmometer. Missing data were not imputed.
Baseline to Week 15

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Change From Baseline in Proptosis in the Most Proptotic Eye as Measured by Exophthalmometer
Tidsramme: Baseline, Week 15
Proptosis was defined as distance between the lateral orbital rim and the most anterior position of the cornea in mm, measured using an exophthalmometer. Missing data were not imputed.
Baseline, Week 15
PRR in the Most Proptotic Eye as Measured by Magnetic Resonance Imaging (MRI)/Computed Tomography (CT)
Tidsramme: Baseline to Week 15
Proptosis responder in the most proptotic eye by MRI/CT was defined as a reduction of proptosis of ≥2 mm from baseline in the most proptotic eye by MRI/CT (without a corresponding increase of ≥2 mm in the other eye) as measured by MRI/CT. Missing data were not imputed.
Baseline to Week 15
Change From Baseline in Proptosis in the Most Proptotic Eye as Measured by MRI/CT
Tidsramme: Baseline, Week 15
Measurement of proptosis was conducted by the central imaging reading center using MRI/CT of the orbits acquired without contrast. Measurements were conducted by 2 independent readers with adjudication if the difference between the 2 primary readers in proptosis measurement exceeded 5% (calculated as the difference divided by the larger measurement). The average of 2 (or 3 if adjudicated) measurements were used for analyses. Missing data were not imputed.
Baseline, Week 15
Clinical Activity Responder Rate in the Most Proptotic Eye as Measured by Exophthalmometer
Tidsramme: Week 15
Clinical activity responder in the most proptotic eye was defined as no worsening in clinical activity score (CAS) from baseline in the most proptotic eye without a corresponding increase of ≥2 points in the other eye. Missing data were not imputed.
Week 15
ORR Comprising PRR and Clinical Activity Responder Rate in the Most Proptotic Eye as Measured by Exophthalmometer
Tidsramme: Baseline to Week 15
ORR was comprised of PRR in the most proptotic eye (reduction of proptosis of ≥2 mm from baseline in the most proptotic eye [without a corresponding increase of ≥2 mm in the other eye]) as measured by exophthalmometer at Week 15 and clinical activity responder rate in the most proptotic eye (no worsening in CAS from baseline in the most proptotic eye without a corresponding increase of ≥2 points in the other eye) as measured by exophthalmometer at Week 15. Missing data were not imputed.
Baseline to Week 15
Diplopia Responder Rate
Tidsramme: Week 15
A diplopia responder was defined as having a decrease of ≥1 from baseline for participants with a baseline Gorman subjective diplopia score >0. Gorman subjective diplopia score (range, 0 to 3) includes 4 categories: no diplopia (absent, scored as 0), diplopia in the primary position of gaze when the participant is tired or awakening (intermittent, scored as 1), diplopia at extremes of gaze (inconstant, scored as 2), and continuous diplopia in the primary or reading position (constant, scored as 3). Missing data were not imputed.
Week 15
Diplopia Resolution Rate
Tidsramme: Week 15
Diplopia resolution was defined as reduction in Gorman subjective diplopia score to 0 from baseline for participants with baseline Gorman subjective diplopia score >0. Gorman subjective diplopia score (range, 0 to 3) includes 4 categories: no diplopia (absent, scored as 0), diplopia in the primary position of gaze when the participant is tired or awakening (intermittent, scored as 1), diplopia at extremes of gaze (inconstant, scored as 2), and continuous diplopia in the primary or reading position (constant, scored as 3). Missing data were not imputed.
Week 15

Samarbejdspartnere og efterforskere

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

Sponsor

Viridian Therapeutics, Inc.

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)

31. januar 2024

Primær færdiggørelse (Faktiske)

25. april 2025

Studieafslutning (Faktiske)

23. juni 2025

Datoer for studieregistrering

Først indsendt

12. december 2023

Først indsendt, der opfyldte QC-kriterier

21. december 2023

Først opslået (Faktiske)

22. december 2023

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

14. august 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

22. juli 2026

Sidst verificeret

1. juli 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • VRDN-001-302

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

INGEN

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

produkt fremstillet i og eksporteret fra U.S.A.

Ingen

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 .

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