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

NCT06307613 | Terminated Phase 3 DMC FDA Drug

• 2 other identifiers: ARGX-113-23012023-509197-35-00
• Study Type: interventional
• Target: 102
• Locations: 15 countries
• Sites: 73

Brief Summary

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.

Conditions

Thyroid Eye Disease

Outcome Measures

Primary Outcomes (1)

• Percentage of participants who were proptosis responders

  At week 24 of the Double-Blinded Treatment Period

Secondary Outcomes (3)

• Change in proptosis measurement in the study eye from baseline

  Up to week 24 of the Double-Blinded Treatment Period

• Change in the total Graves' Orbitopathy Quality of Life (GO-QoL) score from baseline

  Up to week 24 of the Double-Blinded Treatment Period

• Percentage of participants with a resolution of diplopia (responders)

  At week 24 of the Double-Blinded Treatment Period

Study Arms (2)

Efgartigimod arm

EXPERIMENTAL

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

Combination Product: Efgartigimod PH20 SC

Placebo arm

PLACEBO COMPARATOR

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

Other: Placebo PH20 SC

Interventions

Efgartigimod PH20 SC COMBINATION_PRODUCT

Subcutaneous efgartigimod PH20 SC given by prefilled syringe

Efgartigimod arm

Placebo PH20 SC OTHER

Subcutaneous placebo given by prefilled syringe

Placebo arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• argenx: lead

Study Sites (77)

Thrive Health - Beverly Hills

Beverly Hills, California, 90210, United States

Location

UCI Health - Gavin Herbert Eye Institute

Irvine, California, 92697, United States

Location

University Of Southern California - Keck School Of Medicine

Los Angeles, California, 90033, United States

Location

Silkiss Eye Surgery

San Francisco, California, 94102, United States

Location

Advanced Research LLC

Coral Springs, Florida, 33067, United States

Location

Levenson Eye Associates

Jacksonville, Florida, 32204, United States

Location

Bascom Palmer Eye Institute

Miami, Florida, 33136, United States

Location

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

Tampa, Florida, 33612, United States

Location

University of Illinois Chicago

Chicago, Illinois, 60612, United States

Location

Wilmer Eye Institute

Baltimore, Maryland, 21205, United States

Location

University of Michigan

Ann Arbour, Michigan, 48105, United States

Location

Moyes Eye Center - Northland

Kansas City, Missouri, 64154, United States

Location

Quest Diagnostics - Mercy Smith Glynn Callaway

Springfield, Missouri, 65807, United States

Location

Duke University

Durham, North Carolina, 27705, United States

Location

Penn Medicine - Penn Presbyterian Medical Center - Scheie Eye Institute

Philadelphia, Pennsylvania, 19104, United States

Location

West Virginia University Eye Institute

Morgantown, West Virginia, 26506, United States

Location

Centre for Eye Research Australia

Melbourne, 3002, Australia

Location

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

Nedlands, 6009, Australia

Location

North Shore Private Hospital

St Leonards, 2065, Australia

Location

Sydney Eye Hospital

Sydney, 2000, Australia

Location

CHU UVC Brugmann - Site Victor Horta

Brussels, 1020, Belgium

Location

Oxycom Medical Centre

Burgas, 8000, Bulgaria

Location

UMHAT Aleksandrovska - Diagnostic Consultative Center (DCC) Aleksandrova

Sofia, 1431, Bulgaria

Location

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

Stara Zagora, 6008, Bulgaria

Location

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

Varna, 9010, Bulgaria

Location

Peking Union Medical College Hospital - Dongdan campus

Beijing, 100730, China

Location

Shunde Hospital of Southern Medical University

Foshan, 528399, China

Location

Fujian Medical University Union Hospital

Fuzhou, 350001, China

Location

The First Affiliated Hospital of Zhejiang University School of Medicine

Hangzhou, 310003, China

Location

The Second Hospital of Anhui Medical University

Hefei, 230601, China

Location

The First Affiliated Hospital of Henan University of Science and Technology

Luoyang, 471003, China

Location

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

Shanghai, 200011, China

Location

The First Hospital of China Medical University

Shenyang, 110002, China

Location

Fakultni Nemocnice Olomouc - Ocni Klinika

Olomouc, 77900, Czechia

Location

OFTEX - Ocni klinika

Pardubice, 53002, Czechia

Location

Vseobecna fakultni nemocnice v Praze

Prague, 128 00, Czechia

Location

Axon Clinical s.r.o

Prague, 15000, Czechia

Location

Dr Liina Viitas Endokrinoloog

Pärnu, 80018, Estonia

Location

Tartu Ulikooli Kliinikum

Tartu, 50406, Estonia

Location

Charite Universitaetsmedizin Berlin - Campus Charite Mitte (CCM)

Berlin, 10117, Germany

Location

Buergerhospital Frankfurt am Main

Frankfurt, 60318, Germany

Location

Universitaetsklinikum der Ernst-Moritz-Arndt-Universitaet Greifswald

Greifswald, 17475, Germany

Location

Universitaetsklinikum Muenster

Münster, 48149, Germany

Location

General Hospital of Athens G Gennimatas

Athens, 11527, Greece

Location

University General Hospital of Patras

Pátrai, 26504, Greece

Location

A.LOUKAS General Hospital of Thessaloniki

Thessaloniki, 55236, Greece

Location

Athens Medical Group - European Interbalkan Medical Center

Thessaloniki, 57001, Greece

Location

Budapest Retina Intezet

Budapest, 1133, Hungary

Location

Vadnay Szemeszet es Latszereszet

Eger, 3300, Hungary

Location

Ganglion Orvosi Kozpont

Pécs, 7621, Hungary

Location

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

Pécs, 7624, Hungary

Location

Markusovszky Teaching Hospital

Szombathely, 9700, Hungary

Location

Azienda Ospedaliero Universitaria Careggi

Florence, 50134, Italy

Location

Ospedale Policlinico San Martino

Genova, 16132, Italy

Location

Fondazione Irccs Ca' Granda Ospedale Maggioe Policlinico Di Milano

Milan, 20122, Italy

Location

ASST Santi Paolo e Carlo - Ospedale San Paolo

Milan, 20142, Italy

Location

Azienda Ospedaliera Universitaria (AOU) Federico II

Naples, 80131, Italy

Location

Santa Chiara Hospital

Pisa, 56126, Italy

Location

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

Varese, 21100, Italy

Location

Centrul Medical Sana

Bucharest, 011025, Romania

Location

Institutul National de Endocrinologie C.I.Parhon

Bucharest, 011863, Romania

Location

Spitalul Universitar De Urgenta Militar Central Dr. Carol Davila

Bucharest, 10825, Romania

Location

Spitalul Clinic Judetean de Urgenta Sf. Spiridon

Iași, 700111, Romania

Location

National University Hospital

Singapore, 119074, Singapore

Location

Hospital Universitario Ramon y Cajal

Madrid, 28034, Spain

Location

Hospital Universitario Puerta de Hierro Majadahonda

Majadahonda, 28222, Spain

Location

Hospital Clinico Universitario De Santiago De Compostela

Santiago de Compostela, 15702, Spain

Location

Hospital Universitario Virgen Macarena

Seville, 41009, Spain

Location

Faculty of Medicine Siriraj Hospital, Mahidol University

Bang Phlat, 10700, Thailand

Location

King Chulalongkorn Memorial Hospital

Bangkok, 10330, Thailand

Location

Maharaj Nakorn Chiang Mai Hospital

Chiang Mai, 52000, Thailand

Location

Srinagarind Hospital

Khon Kaen, 40002, Thailand

Location

Bradford Royal Infirmary - Bradford Teaching Hospitals NHS Foundation

Bradford, BD9 6RJ, United Kingdom

Location

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

Cardiff, CF14 4XW, United Kingdom

Location

Frimley Health NHS Foundation Trust - Frimley Park Hospital

Frimley, GU16 7UJ, United Kingdom

Location

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

London, SE1 7EH, United Kingdom

Location

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

Newcastle, NE1 4LP, United Kingdom

Location

MeSH Terms

Conditions

Graves Ophthalmopathy

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Graves Disease; Exophthalmos; Orbital Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Goiter; Thyroid Diseases; Endocrine System Diseases; Hyperthyroidism; Autoimmune Diseases; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 6, 2024
• First Posted: March 13, 2024
• Study Start: March 27, 2024
• Primary Completion: December 15, 2025
• Study Completion: February 12, 2026
• Last Updated: March 16, 2026

Record last verified: 2026-03

Locations

GB (5); BE (1); DE (4); CN (8); HU (5); US (16); CZ (4); BU (4); ES (2); SG (1); GR (4); RO (4); AU (4); IT (7); TH (4)