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En undersøgelse af sikkerhed, tolerabilitet og effektivitet af VRDN 001 i raske frivillige og personer med skjoldbruskkirteløjensygdom (TED) (THRIVE)

22. juli 2026 opdateret af: Viridian Therapeutics, Inc.

En Multiple Ascending Dose (MAD) undersøgelse af sikkerhed, tolerabilitet og effektivitet af VRDN 001, et humaniseret monoklonalt antistof rettet mod IGF-1-receptoren, hos normale sunde frivillige (NHV'er) og forsøgspersoner med skjoldbruskkirteløjensygdom (TED)

Undersøgelseslægemidlet, VRDN-001, er et monoklonalt antistof, der hæmmer aktiviteten af ​​en celleoverfladereceptor kaldet insulinlignende vækstfaktor-1-receptor (IGF-1R). Hæmning af IGF-1R kan bidrage til at reducere inflammation og associeret vævshævelse, der opstår hos patienter med skjoldbruskkirteløjensygdom (TED). Dette kliniske forsøg vil evaluere sikkerheden, tolerabiliteten og farmakokinetikken (koncentrationen af ​​lægemiddel i blodet over tid) af VRDN-001 hos raske frivillige og hos patienter med TED. Studiedeltagere med TED vil også blive evalueret over tid for ændringer i deres tegn og symptomer på TED sammenlignet med deres baseline målinger.

Studieoversigt

Status

Afsluttet

Betingelser

Intervention / Behandling

Undersøgelsestype

Interventionel

Tilmelding (Faktiske)

113

Fase

  • Fase 3

Kontakter og lokationer

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

Studiesteder

  • Australien
      • Sydney, Australien, 2000
        • Sydney Eye Hospital
      • Sydney, Australien, 2065
        • North Shore Eye Surgery
  • Det Forenede Kongerige
    • United Kingdon
      • London, United Kingdon, Det Forenede Kongerige, HA13UJ
        • Northwick Park Hospital
      • London, United Kingdon, Det Forenede Kongerige, NW1 5QH
        • Western Eye Hospital Imperial College NHS trust
      • London, United Kingdon, Det Forenede Kongerige, SE1 7EH
        • Guy's and St. Thomas NHS Trust
  • Forenede Stater
    • California
      • Aurora, California, Forenede Stater, 80045
        • University of Colorado - Department of Ophthalmology
      • Beverly Hills, California, Forenede Stater, 90210
        • The Private Office of Raymond Douglas
      • Los Angeles, California, Forenede Stater, 90033
        • USC Roski Eye Institute
      • Los Angeles, California, Forenede Stater, 90049
        • MACRO Trials, Inc.
      • Newport Beach, California, Forenede Stater, 92260
        • Amy Patel Jain, MD
      • Palo Alto, California, Forenede Stater, 94303
        • Byers Eye Institute/Stanford University
      • San Diego, California, Forenede Stater, 95207
        • Senta Clinic
      • San Francisco, California, Forenede Stater, 94158
        • University of California, San Francisco
    • Colorado
      • Aurora, Colorado, Forenede Stater, 80045
        • University of Colorado
    • Florida
      • Homestead, Florida, Forenede Stater, 33034
        • Sina Medical Center
      • Miami, Florida, Forenede Stater, 33125
        • Med-Care Research Inc.
      • Plantation, Florida, Forenede Stater, 33317
        • Edward Jenner Research Group Center LLC
      • Sarasota, Florida, Forenede Stater, 34231
        • Sarasota Retina Institute
    • Illinois
      • Orland Park, Illinois, Forenede Stater, 60462
        • Vision Medical Research Inc.
    • Kansas
      • Kansas City, Kansas, Forenede Stater, 64108
        • University Health Diabetes, Endocrinology & Nephrology Center
      • Kansas City, Kansas, Forenede Stater, 66103
        • KU Medical Center, University of Kansas
    • Massachusetts
      • Boston, Massachusetts, Forenede Stater, 02114
        • Massachusetts Eye and Ear
      • Weymouth, Massachusetts, Forenede Stater, 02189
        • Ophthalmic Consultants of Boston
    • Michigan
      • Fraser, Michigan, Forenede Stater, 48026
        • Fraser Eye Care Center
      • Livonia, Michigan, Forenede Stater, 48152
        • Kahana Oculoplastic & Orbital Surgery
    • Minnesota
      • Minneapolis, Minnesota, Forenede Stater, 55455
        • University of Minnesota, Department of Ophthalmology and Visual Neurosciences
    • Nevada
      • Las Vegas, Nevada, Forenede Stater, 89144
        • Advanced Research International, LLC
    • New Jersey
      • Newark, New Jersey, Forenede Stater, 07103
        • Rutgers-New Jersey Medical School-Newark
    • New York
      • New York, New York, Forenede Stater, 10003
        • New York Eye Ear Infirmary of Mount Sinai
    • Texas
      • Houston, Texas, Forenede Stater, 77074
        • Neuro-Eye Clinical Trials
      • Houston, Texas, Forenede Stater, 77074
        • Neuro-Ophthalmology of Texas PLLC
      • Houston, Texas, Forenede Stater, 77030
        • Baylor College of Medicine (BCM)-Ophthalmology
    • Vermont
      • Burlington, Vermont, Forenede Stater, 05401
        • University of Vermont Medical Center
    • West Virginia
      • Morgantown, West Virginia, Forenede Stater, 26506
        • West Virgina University Eye Institute
  • Frankrig
      • Angers, Frankrig, 49100
        • CHU d'Angers
      • Nice, Frankrig, 06000
        • CH Nice
      • Paris, Frankrig, 75012
        • Centre Hospitalier National D'ophtalmologie
      • Paris, Frankrig, 75013
        • AP-HP- Hopital de la Pitie Salpetriere
  • Polen
      • Bialystok, Polen, 15-879
        • NZOZ E-Vita
      • Bydgoszcz, Polen, 85-870
        • Specjakistyczny Osrodek Okulistyczny Oculomedica
      • Lodz, Polen, 90-302
        • Santa Familia PTG Lodz
      • Warsaw, Polen, 02-507
        • Panstwowy Instytut Medycsny MSWiA
      • Wroclaw, Polen, 53-114
        • 4 Wojskowy Szpital Kliniczny z Polikinika SP ZOZ
  • Spanien
      • Barcelona, Spanien, 08022
        • Clinica Bonanova de Cirugia Ocular
      • Córdoba, Spanien, 14012
        • Hospital Arruzafa
      • Madrid, Spanien, 28034
        • Hospital Universitario Ramon y Cajal
      • Madrid, Spanien, 28027
        • Clinica Universidad de Navarra
      • Pamplona, Spanien, 31008
        • Clinica Universidad de Navarra
      • Santiago, Spanien, 15706
        • Complexo Hospiralario Universitario de Santiago-Hospital Medico-Ciruxico de Conxo
      • Seville, Spanien, 41071
        • Hospital Universitatio Virgen De La Macarena, Servicio de Oftalmologia
      • Zaragoza, Spanien, 50009
        • Hospital Universitario Miguel Servet, Servicio de Paseo Isabel La Catdlica1
  • Tyskland
      • Göttingen, Tyskland, 37075
        • Universitätsmedizin Göttingen
      • Mainz, Tyskland, 55131
        • Johannes Gutenberg-University Medical Center

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 og ældre (Voksen, Ældre voksen)

Tager imod sunde frivillige

Ingen

Beskrivelse

Nøgleinklusionskriterier for sunde frivillige:

  • Skal være fri for klinisk signifikant sygdom eller medicinske tilstande som bestemt af investigator
  • Kvindelige frivillige må ikke være i den fødedygtige alder

Nøgleudelukkelseskriterier for raske frivillige:

• Må ikke have en historie med eller tegn på diabetes mellitus, nyligt diagnosticeret nyreinsufficiens eller inflammatorisk tarmsygdom eller klinisk signifikant ørepatologi eller hørenedsættelse

Nøgleinklusionskriterier for deltagere med TED:

  • Skal have moderat til svær aktiv TED med dokumenteret tegn på okulære symptomer eller tegn, der begyndte inden for 1 år før screening
  • Skal have Clinical Activity Score (CAS) på ≥ 4 på 7-element-skalaen for undersøgelsens (mere proptotisk) øje
  • Skal acceptere at bruge højeffektiv prævention som specificeret i protokollen
  • Kvindelige TED-deltagere skal have en negativ serumgraviditetstest

Nøgleudelukkelseskriterier for deltagere med TED:

  • Må ikke have modtaget tidligere behandling med et andet anti-IGF-1R monoklonalt antistof
  • Må ikke have brugt orale kortikosteroider inden for 4 uger før dag 1
  • Må ikke have modtaget rituximab, tocilizumab eller andre immunsuppressive midler inden for 90 dage før dag 1
  • Må ikke have tegn på optisk nervepåvirkning inden for de foregående 6 måneder
  • Må ikke have hornhindekompensation i undersøgelsesøjet, som ikke reagerer på medicinsk behandling
  • Må ikke have haft tidligere orbital bestråling eller operation for TED i undersøgelsesøjet
  • Må ikke have en tidligere inflammatorisk tarmsygdom eller klinisk signifikant ørepatologi eller hørenedsættelse
  • Må ikke have modtaget en undersøgelsesagent for nogen tilstand inden for 60 dage
  • Kvindelige TED-deltagere må ikke være gravide eller ammende

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: Veligrotug
Participants will receive veligrotug 10 mg/kg as an intravenous (IV) infusion every 3 weeks (Q3W) for 12 weeks.
Medicin: Veligrotug
5 IV Infusions of veligrotug 10 mg/kg
Andre navne:
  • VRDN-001
Placebo komparator: Placebo
Participants will receive placebo matched to veligrotug as an IV infusion Q3W for 12 weeks.
Medicin: Placebo
5 IV Infusions of veligrotug matched placebo

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Proptosis Responder Rate (PRR) in the Study Eye As Measured by Exophthalmometer
Tidsramme: Baseline to Week 15
Proptosis responder in the study eye was defined as a reduction of proptosis of ≥2 millimeters (mm) from baseline in the study eye (without a corresponding increase of ≥2 mm in the fellow eye) as measured by exophthalmometer. Missing data were imputed with the Multiple Imputation method.
Baseline to Week 15

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Change From Baseline in Proptosis in the Study 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 imputed with the Multiple Imputation method.
Baseline, Week 15
PRR in the Most Proptotic Eye As Measured by Magnetic Resonance Imaging (MRI)/Computed Tomography (CT)
Tidsramme: 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 imputed using the Exophthalmometer Imputation method, as applicable.
Week 15
Change From Baseline in Proptosis in the Most Proptotic Eye As Measured by MRI/CT
Tidsramme: Baseline, Week 15
Measurement of proptosis 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 imputed using the Exophthalmometer Imputation method, as applicable.
Baseline, Week 15
Change From Baseline in CAS in the Study Eye
Tidsramme: Baseline, Week 15
The CAS was based on 7 components: spontaneous retrobulbar pain, pain on attempted eye movements (upward, side to-side, and downward gazes), conjunctival redness, redness of the eyelids, chemosis, swelling of the caruncle or plica and swelling of the eyelids. Each component was scored as present or absent (scored as 1 or 0, respectively), and the CAS was given as the sum of the scores (range, 0 to 7, with higher scores indicating greater level of inflammation). Missing data were imputed with the Multiple Imputation method.
Baseline, Week 15
Overall Responder Rate (ORR) Comprising of PRR and Clinical Activity Responder Rate in the Study Eye
Tidsramme: Baseline to Week 15
ORR in the study eye was comprised of PRR in the study eye (reduction of proptosis of ≥ 2 mm from baseline [without a corresponding increase of ≥ 2 mm in the fellow eye]) and clinical activity responder in the study eye (reduction in clinical activity score [CAS] ≥ 2 points from baseline [without a corresponding increase of ≥ 2 points in the fellow eye]). CAS ranged from 0 to 7, with higher scores indicating greater level of inflammation. Proptosis (distance between the lateral orbital rim and the most anterior position of the cornea in mm) was measured using an exophthalmometer. Missing data were imputed with Multiple Imputation method.
Baseline to Week 15
Clinical Activity Responder Rate in the Study Eye
Tidsramme: Week 15
Clinical activity responder rate in the study eye was defined as a reduction in CAS ≥2 points from baseline in the study eye (without a corresponding increase of ≥2 points in the fellow eye). Missing data were imputed with the Multiple Imputation method.
Week 15
Diplopia Responder Rate
Tidsramme: Week 15
Diplopia responder was defined as reduction in Gorman subjective diplopia score of ≥1 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 imputed with the Multiple Imputation method.
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 imputed with the Multiple Imputation method.
Week 15
Percentage of Participants With a CAS of 0 or 1 in the Study Eye
Tidsramme: Week 15
The CAS was based on 7 components: spontaneous retrobulbar pain, pain on attempted eye movements (upward, side to-side, and downward gazes), conjunctival redness, redness of the eyelids, chemosis, swelling of the caruncle or plica and swelling of the eyelids. Each component was scored as present or absent (scored as 1 or 0, respectively), and the CAS was given as the sum of the scores (range, 0 to 7, with higher scores indicating greater level of inflammation). Missing data were imputed with the Multiple Imputation method.
Week 15

Andre resultatmål

Resultatmål
Tidsramme
Veligrotug (VRDN-001) koncentrationer i blodet over tid
Tidsramme: Op til dag 50 for HV- og TED MAD-deltagere og op til uge 24 for fase 3-deltagere
Op til dag 50 for HV- og TED MAD-deltagere og op til uge 24 for fase 3-deltagere
Forekomst af anti-drug antistof (ADA) udvikling hos veligrotug (VRDN-001)-behandlede deltagere over tid
Tidsramme: Op til dag 50 for HV- og TED MAD-deltagere og op til uge 24 for fase 3-deltagere
Op til dag 50 for HV- og TED MAD-deltagere og op til uge 24 for fase 3-deltagere

Samarbejdspartnere og efterforskere

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

Sponsor

Viridian Therapeutics, Inc.

Efterforskere

  • Studieleder: Thomas Ciulla, MD, MBA, 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)

16. november 2022

Primær færdiggørelse (Faktiske)

15. juli 2024

Studieafslutning (Faktiske)

27. marts 2025

Datoer for studieregistrering

Først indsendt

15. december 2021

Først indsendt, der opfyldte QC-kriterier

15. december 2021

Først opslået (Faktiske)

4. januar 2022

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

13. 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-001-101

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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