A Study of Efgartigimod PH20 SC Given by Prefilled Syringe in Adults With Thyroid Eye Disease

NCT ID: NCT06307613

Last Updated: 2026-01-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

102 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-03-27

Study Completion Date

2026-03-31

Brief Summary

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This study aims to evaluate the efficacy, safety and tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of efgartigimod PH20 SC in participants with active, moderate-to-severe TED, compared with placebo PH20 SC.

After the screening period, eligible participants will be randomized in a 2:1 ratio to receive efgartigimod PH20 SC or placebo PH20 SC, respectively during the double-blinded treatment period (DBTP). At the end of the DBTP, participants may enter a follow-up observational period while off study drug. Some participants may also enter the open-label treatment period with efgartigimod PH20 SC. The study duration varies from approximately 60 to 110 weeks.

An alternative list of clinical sites open for recruitment could be found in the other UplighTED study record (https://www.clinicaltrials.gov/study/NCT06307626).

This study was terminated early on 15 December 2025 as the pre-defined interim analysis concluded that continuing the trials is unlikely to demonstrate the intended efficacy. This decision is not related to safety concerns, and the safety profile of efgartigimod remains unchanged. End-of-study and Safety-Follow-Up visits are ongoing for the participants of this trial.

Detailed Description

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Conditions

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Thyroid Eye Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Efgartigimod arm

Participants with active, moderate-to-severe TED receiving Efgartigimod PH20 SC (subcutaneously) via pre-filled syringe (PFS)

Group Type EXPERIMENTAL

Efgartigimod PH20 SC

Intervention Type COMBINATION_PRODUCT

Subcutaneous efgartigimod PH20 SC given by prefilled syringe

Placebo arm

Participants with active, moderate-to-severe TED receiving Placebo PH20 SC (subcutaneously) via pre-filled syringe (PFS)

Group Type PLACEBO_COMPARATOR

Placebo PH20 SC

Intervention Type OTHER

Subcutaneous placebo given by prefilled syringe

Interventions

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Efgartigimod PH20 SC

Subcutaneous efgartigimod PH20 SC given by prefilled syringe

Intervention Type COMBINATION_PRODUCT

Placebo PH20 SC

Subcutaneous placebo given by prefilled syringe

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* The participant is at least 18 years of age
* The participant is capable of providing signed informed consent and following with protocol requirements
* The investigator determines active, moderate-to-severe thyroid eye disease (TED) associated with autoimmune thyroid conditions (Graves' disease or Hashimoto's thyroiditis) for the most severely affected eye
* The participant has first onset of active TED symptoms within 12 months before screening
* The participant must have normal thyroid function with the baseline disease under control or have mild hypo or hyperthyroidism at screening. Every effort should be made to correct the mild hypo or hyperthyroidism promptly and to maintain the normal thyroid function for the full duration of the study
* The participant agrees to use birth control consistent with local regulations and the people of child-bearing potential must have a negative blood pregnancy test at screening and a negative urine pregnancy test before receiving the study drug

Exclusion Criteria

* Optic neuropathy (damage to optic nerve), defined as new visual field defect (blind spot), relative afferent pupillary defect (pupils respond differently to light), or color defect secondary to optic nerve involvement within the 6 months before screening
* Corneal decompensation (swelling of the cornea) unresponsive to medical management
* Previous orbital irradiation or surgery for TED
* Use of some medications before screening (more information is found in the protocol)
* Known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of TED or puts the participant at undue risk
* History of malignancy, cancer, unless considered cured by adequate treatment with no evidence of recurrence for ≥3 years. Adequately treated participants with the following cancers can be included at any time: Basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast, Incidental histological findings of prostate cancer
* Clinically significant active infection that is not sufficiently resolved in the investigator's opinion or positive serum test at screening for active infection with any of the following: Hepatitis B virus (HBV), Hepatitis C virus (HCV), HIV
* Current participation in another interventional clinical study or previous participation in an efgartigimod clinical study and at least 1 dose of study drug received or has received at least 1 dose of commercially available efgartigimod
* Known hypersensitivity to study drug or one of its excipients (inactive ingredients)
* History of or current alcohol, drug, or medication abuse within 12 months before screening as assessed by the investigator
* Pregnant or lactating state or intention to become pregnant during the study
* Live or live-attenuated vaccine received \<4 weeks before screening
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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argenx

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Thrive Health - Beverly Hills

Beverly Hills, California, United States

Site Status

UCI Health - Gavin Herbert Eye Institute

Irvine, California, United States

Site Status

University Of Southern California - Keck School Of Medicine

Los Angeles, California, United States

Site Status

Silkiss Eye Surgery

San Francisco, California, United States

Site Status

Advanced Research LLC

Coral Springs, Florida, United States

Site Status

Levenson Eye Associates

Jacksonville, Florida, United States

Site Status

Bascom Palmer Eye Institute

Miami, Florida, United States

Site Status

University of South Florida (USF) Health - Morsani Center for Advanced Healthcare

Tampa, Florida, United States

Site Status

University of Illinois Chicago

Chicago, Illinois, United States

Site Status

Wilmer Eye Institute

Baltimore, Maryland, United States

Site Status

University of Michigan

Ann Arbour, Michigan, United States

Site Status

Moyes Eye Center - Northland

Kansas City, Missouri, United States

Site Status

Quest Diagnostics - Mercy Smith Glynn Callaway

Springfield, Missouri, United States

Site Status

Duke University

Durham, North Carolina, United States

Site Status

Penn Medicine - Penn Presbyterian Medical Center - Scheie Eye Institute

Philadelphia, Pennsylvania, United States

Site Status

West Virginia University Eye Institute

Morgantown, West Virginia, United States

Site Status

Centre for Eye Research Australia

Melbourne, , Australia

Site Status

The University of Western Australia - Lions Eye Institute (LEI)

Nedlands, , Australia

Site Status

North Shore Private Hospital

St Leonards, , Australia

Site Status

Sydney Eye Hospital

Sydney, , Australia

Site Status

CHU UVC Brugmann - Site Victor Horta

Brussels, , Belgium

Site Status

Oxycom Medical Centre

Burgas, , Bulgaria

Site Status

UMHAT Aleksandrovska - Diagnostic Consultative Center (DCC) Aleksandrova

Sofia, , Bulgaria

Site Status

Medical Center Vereya EOOD - Vereya Medical Center (former Military Hospital)

Stara Zagora, , Bulgaria

Site Status

St. Petka Eye Hospital - Eye Clinic St. Petka Varna

Varna, , Bulgaria

Site Status

Peking Union Medical College Hospital - Dongdan campus

Beijing, , China

Site Status

Shunde Hospital of Southern Medical University

Foshan, , China

Site Status

Fujian Medical University Union Hospital

Fuzhou, , China

Site Status

The First Affiliated Hospital of Zhejiang University School of Medicine

Hangzhou, , China

Site Status

The Second Hospital of Anhui Medical University

Hefei, , China

Site Status

The First Affiliated Hospital of Henan University of Science and Technology

Luoyang, , China

Site Status

Shanghai Ninth People's Hospital of Shanghai Jiaotong University School of Medicine

Shanghai, , China

Site Status

The First Hospital of China Medical University

Shenyang, , China

Site Status

Fakultni Nemocnice Olomouc - Ocni Klinika

Olomouc, , Czechia

Site Status

OFTEX - Ocni klinika

Pardubice, , Czechia

Site Status

Vseobecna fakultni nemocnice v Praze

Prague, , Czechia

Site Status

Axon Clinical s.r.o

Prague, , Czechia

Site Status

Dr Liina Viitas Endokrinoloog

Pärnu, , Estonia

Site Status

Tartu Ulikooli Kliinikum

Tartu, , Estonia

Site Status

Charite Universitaetsmedizin Berlin - Campus Charite Mitte (CCM)

Berlin, , Germany

Site Status

Buergerhospital Frankfurt am Main

Frankfurt, , Germany

Site Status

Universitaetsklinikum der Ernst-Moritz-Arndt-Universitaet Greifswald

Greifswald, , Germany

Site Status

Universitaetsklinikum Muenster

Münster, , Germany

Site Status

General Hospital of Athens G Gennimatas

Athens, , Greece

Site Status

University General Hospital of Patras

Pátrai, , Greece

Site Status

A.LOUKAS General Hospital of Thessaloniki

Thessaloniki, , Greece

Site Status

Athens Medical Group - European Interbalkan Medical Center

Thessaloniki, , Greece

Site Status

Budapest Retina Intezet

Budapest, , Hungary

Site Status

Vadnay Szemeszet es Latszereszet

Eger, , Hungary

Site Status

Ganglion Orvosi Kozpont

Pécs, , Hungary

Site Status

Pecsi Tudomanyegyetem (PTE) Altalanos Orvostudomanyi Kar (AOK) University of Pecs Medical School

Pécs, , Hungary

Site Status

Markusovszky Teaching Hospital

Szombathely, , Hungary

Site Status

Azienda Ospedaliero Universitaria Careggi

Florence, , Italy

Site Status

Ospedale Policlinico San Martino

Genova, , Italy

Site Status

Fondazione Irccs Ca' Granda Ospedale Maggioe Policlinico Di Milano

Milan, , Italy

Site Status

ASST Santi Paolo e Carlo - Ospedale San Paolo

Milan, , Italy

Site Status

Azienda Ospedaliera Universitaria (AOU) Federico II

Naples, , Italy

Site Status

Santa Chiara Hospital

Pisa, , Italy

Site Status

ASST Sete Laghi - Ospedale di Circolo e Fondazione Macchi di Varese

Varese, , Italy

Site Status

Centrul Medical Sana

Bucharest, , Romania

Site Status

Institutul National de Endocrinologie C.I.Parhon

Bucharest, , Romania

Site Status

Spitalul Universitar De Urgenta Militar Central Dr. Carol Davila

Bucharest, , Romania

Site Status

Spitalul Clinic Judetean de Urgenta Sf. Spiridon

Iași, , Romania

Site Status

National University Hospital

Singapore, , Singapore

Site Status

Hospital Universitario Ramon y Cajal

Madrid, , Spain

Site Status

Hospital Universitario Puerta de Hierro Majadahonda

Majadahonda, , Spain

Site Status

Hospital Clinico Universitario De Santiago De Compostela

Santiago de Compostela, , Spain

Site Status

Hospital Universitario Virgen Macarena

Seville, , Spain

Site Status

Faculty of Medicine Siriraj Hospital, Mahidol University

Bang Phlat, , Thailand

Site Status

King Chulalongkorn Memorial Hospital

Bangkok, , Thailand

Site Status

Maharaj Nakorn Chiang Mai Hospital

Chiang Mai, , Thailand

Site Status

Srinagarind Hospital

Khon Kaen, , Thailand

Site Status

Bradford Royal Infirmary - Bradford Teaching Hospitals NHS Foundation

Bradford, , United Kingdom

Site Status

Cardiff and Vale University Health Board - University Hospital of wales (UHW)

Cardiff, , United Kingdom

Site Status

Frimley Health NHS Foundation Trust - Frimley Park Hospital

Frimley, , United Kingdom

Site Status

St Thomas' Hospital - Guy's & St Thomas' NHS Foundation Trust

London, , United Kingdom

Site Status

Royal Victoria Infirmary - The Newcastle Upon Tyne Hospitals NHS Foundation Trust

Newcastle, , United Kingdom

Site Status

Countries

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United States Australia Belgium Bulgaria China Czechia Estonia Germany Greece Hungary Italy Romania Singapore Spain Thailand United Kingdom

Related Links

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Other Identifiers

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2023-509197-35-00

Identifier Type: CTIS

Identifier Source: secondary_id

ARGX-113-2301

Identifier Type: -

Identifier Source: org_study_id

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