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

Undersøgelse af ARO-ANG3 hos deltagere med homozygot familiær hyperkolesterolæmi (HOFH) (Gateway)

20. maj 2026 opdateret af: Arrowhead Pharmaceuticals

Fase 2-undersøgelse til evaluering af sikkerheden og effektiviteten af ​​ARO-ANG3 hos forsøgspersoner med homozygot familiær hyperkolesterolæmi (HOFH)

Deltagere med dokumenteret homozygot familiær hyperkolesterolæmi (HoFH), som har givet informeret samtykke, vil modtage 2 åbne doser af ARO-ANG3 og blive evalueret for sikkerheds- og effektparametre gennem 36 uger. Deltagere, der gennemfører den første 36 ugers behandlingsperiode, kan vælge at fortsætte i en yderligere 24-måneders forlængelsesperiode, hvor de vil modtage op til 8 doser åbne doser af ARO-ANG3.

Studieoversigt

Status

Afsluttet

Betingelser

Intervention / Behandling

Undersøgelsestype

Interventionel

Tilmelding (Faktiske)

18

Fase

  • Fase 2

Kontakter og lokationer

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

Studiesteder

  • Australien
    • New South Wales
      • Camperdown, New South Wales, Australien, 2050
        • Research Site 8
    • Western Australia
      • Nedlands, Western Australia, Australien, 6009
        • Research Site 3
  • Canada
    • Quebec
      • Chicoutimi, Quebec, Canada, G7H 7K9
        • Research Site 2
      • Québec, Quebec, Canada, G1V 4W2
        • Research Site 1
  • Forenede Stater
    • New York
      • Mount Sinai, New York, Forenede Stater, 10029
        • Research Site 4
    • Ohio
      • Cincinnati, Ohio, Forenede Stater, 45227
        • Research Site 5
  • Sydafrika
      • Johannesburg, Sydafrika, 2193
        • Research Site 7

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

16 år og ældre (Barn, Voksen, Ældre voksen)

Tager imod sunde frivillige

Ingen

Beskrivelse

Inklusionskriterier:

  • Fastende LDL-C >100 mg/dL ved screening
  • Vægt på ≥ 40 kg og kropsmasseindeks ≥ 18,5 og ≤ 40 kg/m2
  • Diagnose af HoFH baseret på en understøttende genetisk test eller klinisk diagnose
  • Ved stabil maksimalt tolereret lipidsænkende behandling
  • Villig til at overholde en stabil kost med lavt fedtindhold, lavt kolesterol, hjertesund kost i mindst 4 uger før dag 1
  • Deltagere i den fødedygtige alder (mænd og kvinder) skal acceptere at bruge højeffektiv prævention under undersøgelsen og i mindst 24 uger fra den sidste dosis af undersøgelsesmedicin.
  • Kvinder i den fødedygtige alder skal have en negativ graviditetstest og kan ikke amme
  • Kvinder i den fødedygtige alder på hormonelle præventionsmidler skal være stabile på medicinen i > 2 menstruationscyklusser før dag 1
  • Villig til at give skriftligt informeret samtykke og til at overholde studiekrav

Ekskluderingskriterier:

  • Nuværende brug eller brug inden for 365 dage fra dag 1 af ethvert hepatocytmålrettet små interfererende RNA-oligonukleotider (siRNA) eller antisense-oligonukleosidmolekyle
  • Brug af evinacumab (nogle undtagelser gælder)
  • Fastende TG > 300 mg/dL ved screening
  • Tilstedeværelse af enhver klinisk signifikant ukontrolleret endokrin sygdom, der vides at påvirke serumlipider eller lipoproteiner
  • Nydiagnosticeret (inden for 3 måneder før informeret samtykke) eller dårligt kontrolleret diabetes (hæmoglobin A1c > 9 %)
  • Brug af systemiske kortikosteroider (nogle undtagelser gælder)
  • Symptomer på myokardieiskæmi eller alvorlig venstre ventrikulær dysfunktion
  • Anamnese med metastatisk malignitet inden for 3 år efter dag 1 (nogle undtagelser gælder)
  • Planlagt hjerteindgreb/-kirurgi såsom koronararterie-bypass-operation (CABG), perkutan koronar intervention (PCI), carotiskirurgi eller stenting, eller carotis revaskularisering

Bemærk: Yderligere inklusions-/eksklusionskriterier kan gælde pr. protokol

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: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: ARO-ANG3 Dosis 1
ARO-ANG3 Dosisniveau 1 subkutant (SC)
Medicin: ARO-ANG 3 Injection
Deltagerne vil blive randomiseret til at modtage ARO-ANG3 SC på dag 1 og dag 84 i løbet af de første 36 uger af undersøgelsen og på dag 1 og måned 3, 6, 9, 12, 15, 18 og 21 af forlængelsesperioden
Eksperimentel: ARO-ANG3 Dosis 2
ARO-ANG3 Dosisniveau 2 SC
Medicin: ARO-ANG 3 Injection
Deltagerne vil blive randomiseret til at modtage ARO-ANG3 SC på dag 1 og dag 84 i løbet af de første 36 uger af undersøgelsen og på dag 1 og måned 3, 6, 9, 12, 15, 18 og 21 af forlængelsesperioden

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Percent Change From Baseline in Fasting LDL-C at Week 24
Tidsramme: Baseline, Week 24
LDL-C, computed using Friedewald formula, Martin-Hopkins methodology and preparative ultracentrifugation (PUC).
Baseline, Week 24

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Tidsramme: From first dose of study drug up to Week 36 (initial treatment period), and up to Month 24 (extension period)
An adverse event (AE) is any untoward medical occurrence, which does not necessarily have to have a causal relationship with this treatment. A serious adverse event (SAE) is an AE occurring during any study phase that: results in death; is immediately life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability or incapacity; results in a congenital abnormality or birth defect; is an important medically important event or reaction that may require medical intervention to prevent one of the outcomes listed above. AEs are considered treatment-related if the relationship to the study drug is 'possibly related', 'probably related'. A TEAE is defined as an AE that occurs following IP administration or a pre-existing condition exacerbated following IP administration. Injection site reactions are assessed at every visit starting on Day 1 and include any Preferred Term containing 'Injection Site'.
From first dose of study drug up to Week 36 (initial treatment period), and up to Month 24 (extension period)
Percentage of Participants Meeting United States National Lipid Association Apheresis Eligibility Criteria of LDL-C ≥ 300 mg/dL at Week 24
Tidsramme: Week 24
Apheresis is the extracorporeal process of removing one or more blood constituents from whole blood and returning the remainder to the circulation.The National Lipid Association outlines eligibility criteria for apheresis, particularly for patients with familial hypercholesterolemia who have not achieved target LDL cholesterol levels despite maximally tolerated pharmacotherapy. LDL-C ≥ 300 mg/dL is a criterion.
Week 24
Percentage of Participants Meeting European Union (EU) Apheresis Eligibility Criteria Per German Apheresis Working Group at Week 24
Tidsramme: Week 24

Apheresis is the extracorporeal process of removing one or more blood constituents from whole blood and returning the remainder to the circulation. The European Union (EU) apheresis eligibility criteria (per German Apheresis Working Group) include the following categories:

  • A patient with primary cardiovascular disease (CVD) prevention is considered as meeting German apheresis eligibility criteria if LDL-C >160 mg/dL
  • A patient with secondary CVD prevention is considered as meeting German apheresis eligibility criteria if LDL-C >120 mg/dL
Week 24
Percent Change From Baseline in Fasting LDL-C (PUC) Over Time
Tidsramme: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Absolute Change From Baseline in Fasting LDL-C (PUC) Over Time
Tidsramme: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Percent Change From Baseline in Fasting Calculated LDL-C (Friedewald Formula) Over Time
Tidsramme: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Absolute Change From Baseline in Fasting Calculated LDL-C (Friedewald Formula) Over Time
Tidsramme: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Percent Change From Baseline in Fasting Calculated LDL-C (Martin-Hopkins Methodology) Over Time
Tidsramme: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Absolute Change From Baseline in Fasting Calculated LDL-C (Martin-Hopkins Methodology) Over Time
Tidsramme: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Percent Change From Baseline in Fasting Angiopoietin-like 3 (ANGPTL3) Over Time
Tidsramme: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Absolute Change From Baseline in Fasting ANGPTL3 Over Time
Tidsramme: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Percent Change From Baseline in Fasting Total Apolipoprotein B (ApoB) Over Time
Tidsramme: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Absolute Change From Baseline in Fasting Total ApoB Over Time
Tidsramme: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Percent Change From Baseline in Fasting High-Density Lipoprotein-Cholesterol (HDL-C) Over Time
Tidsramme: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Absolute Change From Baseline in Fasting HDL-C Over Time
Tidsramme: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Percent Change From Baseline in Fasting Non-HDL-C Over Time
Tidsramme: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Absolute Change From Baseline in Fasting Non-HDL-C Over Time
Tidsramme: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Percent Change From Baseline in Fasting Very-Low-Density Lipoprotein-Cholesterol (VLDL-C) Over Time
Tidsramme: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Absolute Change From Baseline in Fasting VLDL-C Over Time
Tidsramme: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Percent Change From Baseline in Fasting Total Cholesterol (TC) Over Time
Tidsramme: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Absolute Change From Baseline in Fasting TC Over Time
Tidsramme: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Percent Change From Baseline in Fasting Triglycerides (TG) Over Time
Tidsramme: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Absolute Change From Baseline in Fasting TG Over Time
Tidsramme: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36 (Day 1 of Extension Period), Extension Months 1, 2, 3, 6, 9, 12, 15, 18, 21
Number of Participants With Anti-Drug Antibodies (ADAs) to ARO-ANG3 Over Time
Tidsramme: Baseline, Day 1, Weeks 4, 12, 16, 24 (initial treatment period), Week 36/Day 1, Months 1, 3, 6, 9, 12, 15, 18, 21 (extension period)
Baseline, Day 1, Weeks 4, 12, 16, 24 (initial treatment period), Week 36/Day 1, Months 1, 3, 6, 9, 12, 15, 18, 21 (extension period)

Samarbejdspartnere og efterforskere

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

Sponsor

Arrowhead 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.

Generelle publikationer

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)

22. april 2022

Primær færdiggørelse (Faktiske)

2. maj 2023

Studieafslutning (Faktiske)

13. november 2025

Datoer for studieregistrering

Først indsendt

20. januar 2022

Først indsendt, der opfyldte QC-kriterier

20. januar 2022

Først opslået (Faktiske)

1. februar 2022

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

12. juni 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

20. maj 2026

Sidst verificeret

1. maj 2026

Mere information

Begreber relateret til denne undersøgelse

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • AROANG3-2003

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

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 Homozygot familiær hyperkolesterolæmi

Kliniske forsøg med ARO-ANG 3 Injection

Søg i lignende forsøg

Sponsorer og samarbejdspartnere

Medicinske tilstande

Narkotikainterventioner

CROs by country

CROs in Latvia

Betingelser

Sjældne sygdomme

Narkotikainterventioner

Kosttilskud

Sponsor / samarbejdspartnere

Placeringer

Abonner