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

NCT ID: NCT06307626

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

105 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-03-28

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/NCT06307613).

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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American Institute of Research

Los Angeles, California, United States

Site Status

North Valley Eye Medical Group, Inc.

Mission Hills, California, United States

Site Status

Martel Eye Medical Group

Rancho Cordova, California, United States

Site Status

Cockerham Eye Consultants

San Diego, California, United States

Site Status

Sibia Eye Institute

Boynton Beach, Florida, United States

Site Status

Sarasota Retina Institute (SRI)

Sarasota, Florida, United States

Site Status

Butchertown Clinical Trials

Louisville, Kentucky, United States

Site Status

St. Louis University (SLU) Care - Center for Specialized Medicine

St Louis, Missouri, United States

Site Status

Advancing Research International, LLC

Las Vegas, Nevada, United States

Site Status

New York Eye & Ear Infirmary

New York, New York, United States

Site Status

Baylor College of Medicine

Houston, Texas, United States

Site Status

Landeskrankenhaus - Universitaetskliniken Innsbruck

Innsbruck, , Austria

Site Status

Hanusch-Krankenhaus - Vienna Institute for Research in Ocular Surgery

Vienna, , Austria

Site Status

AIPSMAED Sveti Luka EOOD

Plovdiv, , Bulgaria

Site Status

Multi-Profile Hospital for Active Treatment (MHAT) Hadji Dimitar

Sliven, , Bulgaria

Site Status

Medical Center Hera EOOD

Sofia, , Bulgaria

Site Status

Military Medical Academy (MMA)

Sofia, , Bulgaria

Site Status

Peking University Third Hospital

Beijing, , China

Site Status

The Second Affiliated Hospital of Chengdu Medical College / Nuclear Industry 416 Hospital

Chengdu, , China

Site Status

The Second Hospital of Dalian Medical University

Dalian, , China

Site Status

Fujian Provincial Hospital

Fuzhou, , China

Site Status

Guangdong Provincial People's Hospital

Guangzhou, , China

Site Status

Shandong Provincial Hospital of Shandong First Medical University

Jinan, , China

Site Status

Hospices Civils de Lyon (HCL) - Hopital Louis Pradel

Bron, , France

Site Status

CHU Lille - Hopital Huriez

Lille, , France

Site Status

Centre Hospitalier National d'Ophtalmologie (Chno) Des Quinze-Vingts

Paris, , France

Site Status

CHU de Saint-Etienne - Hopital Nord

Saint-Priest-en-Jarez, , France

Site Status

JSC Curatio

Tbilisi, , Georgia

Site Status

National Institute of Endocrinology

Tbilisi, , Georgia

Site Status

New Hospitals

Tbilisi, , Georgia

Site Status

Caucasus Medical Centre

Tbilisi, , Georgia

Site Status

Universitaetsklinikum Essen (AoR)

Essen, , Germany

Site Status

Klinikum Der Albert-Ludwigs Universitaet Freiburg

Freiburg im Breisgau, , Germany

Site Status

Philipps-Universitaet Marburg Universitaetsklinikum Giessen und Marburg Klinik fuer Augenheilkunde

Marburg, , Germany

Site Status

Universitaetsaugenklinik Ulm

Ulm, , Germany

Site Status

Universita degli Studi di Cagliari - Ospedale San Giovanni di Dio

Cagliari, , Italy

Site Status

Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale San Raffaele (HSR) (Istituto Scientifico Universitario San Raffaele)

Milan, , Italy

Site Status

Ospedale Cisanello

Pisa, , Italy

Site Status

Azienda Ospedaliero Universitaria Sant'Andrea

Roma, , Italy

Site Status

Azienda Ospedaliero Ordine Mauriziano Torino - Ospedale Umberto I di Torino

Torino, , Italy

Site Status

Kanazawa University Hospital

Kanazawa, , Japan

Site Status

Hospital of University of Occupational and Environmental Health

Kitakyushu, , Japan

Site Status

Shinkoga clinic

Kurume, , Japan

Site Status

Koga Hospital Group - Shinkoga Hospital

Kurume-shi, , Japan

Site Status

National Hospital Organization Kyoto Medical Center

Kyoto, , Japan

Site Status

Kozawa Eye Hospital and Diabetes Center

Mito, , Japan

Site Status

Nagasaki University Hospital

Nagasaki, , Japan

Site Status

Niigata University Medical & Dental Hospital

Niigata, , Japan

Site Status

Osaka City General Hospital

Osaka, , Japan

Site Status

Hokkaido University Hospital

Sapporo, , Japan

Site Status

Tohoku Medical and Pharmaceutical University - Fukumuro

Sendai, , Japan

Site Status

Tottori University Hospital

Yonago, , Japan

Site Status

Daugavpils Regionala Slimnica

Daugavpils, , Latvia

Site Status

Paula Stradina Kliniska universitates slimnica

Riga, , Latvia

Site Status

Uniwersytecki Szpital Kliniczny w Bialymstoku

Bialystok, , Poland

Site Status

Centrum Medycyny Inwazyjnej

Gdansk, , Poland

Site Status

Szpital Sw. Rozy

Krakow, , Poland

Site Status

Instytut Centrum Zdrowia Matki Polki

Lodz, , Poland

Site Status

Uniwersytecki Szpital Kliniczny w Poznaniu

Poznan, , Poland

Site Status

Centrum Zdrowia MDM

Warsaw, , Poland

Site Status

Clinical Center Vojvodina

Novi Sad, , Serbia

Site Status

OLHB d.o.o.

Ljubljana, , Slovenia

Site Status

University Medical Centre University Eye Hospital

Ljubljana, , Slovenia

Site Status

Institut Catala de Retina (ICR) Centre Oftalmologic - ICR Seu Central Ganduxer

Barcelona, , Spain

Site Status

Hospital La Arruzafa

Córdoba, , Spain

Site Status

Hospital Universitari General de Catalunya

Sant Cugat del Vallès, , Spain

Site Status

Hospital Clinico Universitario de Valladolid

Valladolid, , Spain

Site Status

Eye Clinic Linkoping

Linköping, , Sweden

Site Status

Universitaetsspital Bern - Inselspital

Bern, , Switzerland

Site Status

ADMEDICO Augenzentrum AG

Olten, , Switzerland

Site Status

Memorial Ankara Hospital

Ankara, , Turkey (Türkiye)

Site Status

Kocaeli University Research and Training Hospital

Kocaeli, , Turkey (Türkiye)

Site Status

University Hospitals Bristol and Weston NHS Foundation Trust

Bristol, , United Kingdom

Site Status

Royal Liverpool University Hospital , St Paul's Eye Unit - Liverpool University Hospitals NHS Foundation Trust

Liverpool, , United Kingdom

Site Status

Moorfields Eye Hospital

London, , United Kingdom

Site Status

Western Eye Hospital - Imperial College Healthcare NHS Trust

London, , United Kingdom

Site Status

Countries

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United States Austria Bulgaria China France Georgia Germany Italy Japan Latvia Poland Serbia Slovenia Spain Sweden Switzerland Turkey (Türkiye) United Kingdom

Related Links

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Other Identifiers

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2023-509198-22-00

Identifier Type: CTIS

Identifier Source: secondary_id

ARGX-113-2309

Identifier Type: -

Identifier Source: org_study_id

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