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

March 13, 2026 updated by: argenx

A Phase 3, Randomized, Double-Masked, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Efgartigimod PH20 SC Administered by Prefilled Syringe in Adult Participants With Thyroid Eye Disease

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

105

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Austria
      • Innsbruck, Austria, 6020
        • Landeskrankenhaus - Universitaetskliniken Innsbruck
      • Vienna, Austria, 1140
        • Hanusch-Krankenhaus - Vienna Institute for Research in Ocular Surgery
  • Bulgaria
      • Plovdiv, Bulgaria, 4000
        • AIPSMAED Sveti Luka EOOD
      • Sliven, Bulgaria, 8800
        • Multi-Profile Hospital for Active Treatment (MHAT) Hadji Dimitar
      • Sofia, Bulgaria, 1510
        • Medical Center Hera EOOD
      • Sofia, Bulgaria, 1606
        • Military Medical Academy (MMA)
  • China
      • Beijing, China, 100191
        • Peking University Third Hospital
      • Chengdu, China, 610066
        • The Second Affiliated Hospital of Chengdu Medical College / Nuclear Industry 416 Hospital
      • Dalian, China, 116021
        • The Second Hospital of Dalian Medical University
      • Fuzhou, China, 350001
        • Fujian Provincial Hospital
      • Guangzhou, China, 510080
        • Guangdong Provincial People's Hospital
      • Jinan, China, 250021
        • Shandong Provincial Hospital of Shandong First Medical University
  • France
      • Bron, France, 69677
        • Hospices Civils de Lyon (HCL) - Hopital Louis Pradel
      • Lille, France, 59000
        • CHU Lille - Hopital Huriez
      • Paris, France, 75012
        • Centre Hospitalier National d'Ophtalmologie (Chno) Des Quinze-Vingts
      • Saint-Priest-en-Jarez, France, 42270
        • CHU de Saint-Etienne - Hopital Nord
  • Georgia
      • Tbilisi, Georgia, 0114
        • JSC Curatio
      • Tbilisi, Georgia, 0159
        • National Institute of Endocrinology
      • Tbilisi, Georgia, 0162
        • New Hospitals
      • Tbilisi, Georgia, 0186
        • Caucasus Medical Centre
  • Germany
      • Essen, Germany, 45147
        • Universitaetsklinikum Essen (AoR)
      • Freiburg im Breisgau, Germany, 79106
        • Klinikum Der Albert-Ludwigs Universitaet Freiburg
      • Marburg, Germany, 35043
        • Philipps-Universitaet Marburg Universitaetsklinikum Giessen und Marburg Klinik fuer Augenheilkunde
      • Ulm, Germany, 89075
        • Universitaetsaugenklinik Ulm
  • Italy
      • Cagliari, Italy, 09124
        • Universita degli Studi di Cagliari - Ospedale San Giovanni di Dio
      • Milan, Italy, 20132
        • Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale San Raffaele (HSR) (Istituto Scientifico Universitario San Raffaele)
      • Pisa, Italy, 56124
        • Ospedale Cisanello
      • Roma, Italy, 00189
        • Azienda Ospedaliero Universitaria Sant'Andrea
      • Torino, Italy, 10128
        • Azienda Ospedaliero Ordine Mauriziano Torino - Ospedale Umberto I di Torino
  • Japan
      • Kanazawa, Japan, 920-8641
        • Kanazawa University Hospital
      • Kitakyushu, Japan, 807-8555
        • Hospital of University of Occupational and Environmental Health
      • Kurume, Japan, 830-8522
        • Shinkoga Clinic
      • Kurume-shi, Japan, 830-8577
        • Koga Hospital Group - Shinkoga Hospital
      • Kyoto, Japan, 612-0861
        • National Hospital Organization Kyoto Medical Center
      • Mito, Japan, 310-0845
        • Kozawa Eye Hospital And Diabetes Center
      • Nagasaki, Japan, 852-8501
        • Nagasaki University Hospital
      • Niigata, Japan, 951-8520
        • Niigata University Medical & Dental Hospital
      • Osaka, Japan, 534-0021
        • Osaka City General Hospital
      • Sapporo, Japan, 060-8648
        • Hokkaido University Hospital
      • Sendai, Japan, 983-8536
        • Tohoku Medical and Pharmaceutical University - Fukumuro
      • Yonago, Japan, 683-8504
        • Tottori University Hospital
  • Latvia
      • Daugavpils, Latvia, 5417
        • Daugavpils Regionala Slimnica
      • Riga, Latvia, 1002
        • Paula Stradiņa klīniskā universitates slimnīca
  • Poland
      • Bialystok, Poland, 15-276
        • Uniwersytecki Szpital Kliniczny w Bialymstoku
      • Gdansk, Poland, 80-952
        • Centrum Medycyny Inwazyjnej
      • Krakow, Poland, 30-394
        • Szpital Sw. Rozy
      • Lodz, Poland, 93-338
        • Instytut Centrum Zdrowia Matki Polki
      • Poznan, Poland, 60-355
        • Uniwersytecki Szpital Kliniczny w Poznaniu
      • Warsaw, Poland, 04-736
        • Centrum Zdrowia MDM
  • Serbia
      • Novi Sad, Serbia, 21000
        • Clinical Center Vojvodina
  • Slovenia
      • Ljubljana, Slovenia, 1000
        • OLHB d.o.o.
      • Ljubljana, Slovenia, 1000
        • University Medical Centre University Eye Hospital
  • Spain
      • Barcelona, Spain, 08022
        • Institut Catala de Retina (ICR) Centre Oftalmologic - ICR Seu Central Ganduxer
      • Córdoba, Spain, 14012
        • Hospital La Arruzafa
      • Sant Cugat del Vallès, Spain, 08915
        • Hospital Universitari General de Catalunya
      • Valladolid, Spain, 47003
        • Hospital Clinico Universitario de Valladolid
  • Sweden
      • Linköping, Sweden, 581 85
        • Eye Clinic Linkoping
  • Switzerland
      • Bern, Switzerland, 3010
        • Universitaetsspital Bern - Inselspital
      • Olten, Switzerland, 4600
        • ADMEDICO Augenzentrum AG
  • Turkey (Türkiye)
      • Ankara, Turkey (Türkiye), 06520
        • Memorial Ankara Hospital
      • Kocaeli, Turkey (Türkiye), 41380
        • Kocaeli University Research and Training Hospital
  • United Kingdom
      • Bristol, United Kingdom, BS13NU
        • University Hospitals Bristol and Weston NHS Foundation Trust
      • Liverpool, United Kingdom, L7 8XP
        • Royal Liverpool University Hospital , St Paul's Eye Unit - Liverpool University Hospitals NHS Foundation Trust
      • London, United Kingdom, EC1V 2PD
        • Moorfields Eye Hospital
      • London, United Kingdom, NW1 5QH
        • Western Eye Hospital - Imperial College Healthcare NHS Trust
  • United States
    • California
      • Los Angeles, California, United States, 90017
        • American Institute of Research
      • Mission Hills, California, United States, 91345
        • North Valley Eye Medical Group, Inc.
      • Rancho Cordova, California, United States, 95670
        • Martel Eye Medical Group
      • San Diego, California, United States, 91208
        • Cockerham Eye Consultants
    • Florida
      • Boynton Beach, Florida, United States, 33437
        • Sibia Eye Institute
      • Sarasota, Florida, United States, 34239
        • Sarasota Retina Institute (SRI)
    • Kentucky
      • Louisville, Kentucky, United States, 40206
        • Butchertown Clinical Trials
    • Missouri
      • St Louis, Missouri, United States, 63104
        • St. Louis University (SLU) Care - Center for Specialized Medicine
    • Nevada
      • Las Vegas, Nevada, United States, 89144
        • Advancing Research International, LLC
    • New York
      • New York, New York, United States, 10003
        • New York Eye & Ear Infirmary
    • Texas
      • Houston, Texas, United States, 77030
        • Baylor College of Medicine

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

The complete list of exclusion criteria can be found in the protocol.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Efgartigimod arm
Participants with active, moderate-to-severe TED receiving Efgartigimod PH20 SC (subcutaneously) via pre-filled syringe (PFS)
Combination product: Efgartigimod PH20 SC
Subcutaneous efgartigimod PH20 SC given by prefilled syringe
Placebo Comparator: Placebo arm
Participants with active, moderate-to-severe TED receiving Placebo PH20 SC (subcutaneously) via pre-filled syringe (PFS)
Other: Placebo PH20 SC
Subcutaneous placebo given by prefilled syringe

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Percentage of participants who were proptosis responders
Time Frame: At week 24 of the Double-Blinded Treatment Period
At week 24 of the Double-Blinded Treatment Period

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in proptosis measurement in the study eye from baseline
Time Frame: Up to week 24 of the Double-Blinded Treatment Period
Up to week 24 of the Double-Blinded Treatment Period
Percentage of participants with a resolution of diplopia (responders)
Time Frame: At week 24 of the Double-Blinded Treatment Period
At week 24 of the Double-Blinded Treatment Period
Change in the total Graves' Orbitopathy Quality of Life (GO-QoL) score from baseline
Time Frame: Up to week 24 of the Double-Blinded Treatment Period
Minimum value: 0 (most negative impact on Quality of Life); Maximum value: 100 (No impact on Quality of Life)
Up to week 24 of the Double-Blinded Treatment Period

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

argenx

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 28, 2024

Primary Completion (Actual)

December 15, 2025

Study Completion (Actual)

February 12, 2026

Study Registration Dates

First Submitted

March 6, 2024

First Submitted That Met QC Criteria

March 11, 2024

First Posted (Actual)

March 13, 2024

Study Record Updates

Last Update Posted (Actual)

March 16, 2026

Last Update Submitted That Met QC Criteria

March 13, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ARGX-113-2309
  • 2023-509198-22-00 (Ctis)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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