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

NCT06307626 | Terminated Phase 3 DMC FDA Drug

• 2 other identifiers: ARGX-113-23092023-509198-22-00
• Study Type: interventional
• Target: 105
• Locations: 17 countries
• Sites: 75

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/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.

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 (76)

American Institute of Research

Los Angeles, California, 90017, United States

Location

North Valley Eye Medical Group, Inc.

Mission Hills, California, 91345, United States

Location

Martel Eye Medical Group

Rancho Cordova, California, 95670, United States

Location

Cockerham Eye Consultants

San Diego, California, 91208, United States

Location

Sibia Eye Institute

Boynton Beach, Florida, 33437, United States

Location

Sarasota Retina Institute (SRI)

Sarasota, Florida, 34239, United States

Location

Butchertown Clinical Trials

Louisville, Kentucky, 40206, United States

Location

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

St Louis, Missouri, 63104, United States

Location

Advancing Research International, LLC

Las Vegas, Nevada, 89144, United States

Location

New York Eye & Ear Infirmary

New York, New York, 10003, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Landeskrankenhaus - Universitaetskliniken Innsbruck

Innsbruck, 6020, Austria

Location

Hanusch-Krankenhaus - Vienna Institute for Research in Ocular Surgery

Vienna, 1140, Austria

Location

AIPSMAED Sveti Luka EOOD

Plovdiv, 4000, Bulgaria

Location

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

Sliven, 8800, Bulgaria

Location

Medical Center Hera EOOD

Sofia, 1510, Bulgaria

Location

Military Medical Academy (MMA)

Sofia, 1606, Bulgaria

Location

Peking University Third Hospital

Beijing, 100191, China

Location

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

Chengdu, 610066, China

Location

The Second Hospital of Dalian Medical University

Dalian, 116021, China

Location

Fujian Provincial Hospital

Fuzhou, 350001, China

Location

Guangdong Provincial People's Hospital

Guangzhou, 510080, China

Location

Shandong Provincial Hospital of Shandong First Medical University

Jinan, 250021, China

Location

Hospices Civils de Lyon (HCL) - Hopital Louis Pradel

Bron, 69677, France

Location

CHU Lille - Hopital Huriez

Lille, 59000, France

Location

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

Paris, 75012, France

Location

CHU de Saint-Etienne - Hopital Nord

Saint-Priest-en-Jarez, 42270, France

Location

JSC Curatio

Tbilisi, 0114, Georgia

Location

National Institute of Endocrinology

Tbilisi, 0159, Georgia

Location

New Hospitals

Tbilisi, 0162, Georgia

Location

Caucasus Medical Centre

Tbilisi, 0186, Georgia

Location

Universitaetsklinikum Essen (AoR)

Essen, 45147, Germany

Location

Klinikum Der Albert-Ludwigs Universitaet Freiburg

Freiburg im Breisgau, 79106, Germany

Location

Philipps-Universitaet Marburg Universitaetsklinikum Giessen und Marburg Klinik fuer Augenheilkunde

Marburg, 35043, Germany

Location

Universitaetsaugenklinik Ulm

Ulm, 89075, Germany

Location

Universita degli Studi di Cagliari - Ospedale San Giovanni di Dio

Cagliari, 09124, Italy

Location

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

Milan, 20132, Italy

Location

Ospedale Cisanello

Pisa, 56124, Italy

Location

Azienda Ospedaliero Universitaria Sant'Andrea

Roma, 00189, Italy

Location

Azienda Ospedaliero Ordine Mauriziano Torino - Ospedale Umberto I di Torino

Torino, 10128, Italy

Location

Kanazawa University Hospital

Kanazawa, 920-8641, Japan

Location

Hospital of University of Occupational and Environmental Health

Kitakyushu, 807-8555, Japan

Location

Shinkoga clinic

Kurume, 830-8522, Japan

Location

Koga Hospital Group - Shinkoga Hospital

Kurume-shi, 830-8577, Japan

Location

National Hospital Organization Kyoto Medical Center

Kyoto, 612-0861, Japan

Location

Kozawa Eye Hospital and Diabetes Center

Mito, 310-0845, Japan

Location

Nagasaki University Hospital

Nagasaki, 852-8501, Japan

Location

Niigata University Medical & Dental Hospital

Niigata, 951-8520, Japan

Location

Osaka City General Hospital

Osaka, 534-0021, Japan

Location

Hokkaido University Hospital

Sapporo, 060-8648, Japan

Location

Tohoku Medical and Pharmaceutical University - Fukumuro

Sendai, 983-8536, Japan

Location

Tottori University Hospital

Yonago, 683-8504, Japan

Location

Daugavpils Regionala Slimnica

Daugavpils, 5417, Latvia

Location

Paula Stradina Kliniska universitates slimnica

Riga, 1002, Latvia

Location

Uniwersytecki Szpital Kliniczny w Bialymstoku

Bialystok, 15-276, Poland

Location

Centrum Medycyny Inwazyjnej

Gdansk, 80-952, Poland

Location

Szpital Sw. Rozy

Krakow, 30-394, Poland

Location

Instytut Centrum Zdrowia Matki Polki

Lodz, 93-338, Poland

Location

Uniwersytecki Szpital Kliniczny w Poznaniu

Poznan, 60-355, Poland

Location

Centrum Zdrowia MDM

Warsaw, 04-736, Poland

Location

Clinical Center Vojvodina

Novi Sad, 21000, Serbia

Location

OLHB d.o.o.

Ljubljana, 1000, Slovenia

Location

University Medical Centre University Eye Hospital

Ljubljana, 1000, Slovenia

Location

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

Barcelona, 08022, Spain

Location

Hospital La Arruzafa

Córdoba, 14012, Spain

Location

Hospital Universitari General de Catalunya

Sant Cugat del Vallès, 08915, Spain

Location

Hospital Clinico Universitario de Valladolid

Valladolid, 47003, Spain

Location

Eye Clinic Linkoping

Linköping, 581 85, Sweden

Location

Universitaetsspital Bern - Inselspital

Bern, 3010, Switzerland

Location

ADMEDICO Augenzentrum AG

Olten, 4600, Switzerland

Location

Memorial Ankara Hospital

Ankara, 06520, Turkey (Türkiye)

Location

Kocaeli University Research and Training Hospital

Kocaeli, 41380, Turkey (Türkiye)

Location

University Hospitals Bristol and Weston NHS Foundation Trust

Bristol, BS13NU, United Kingdom

Location

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

Liverpool, L7 8XP, United Kingdom

Location

Moorfields Eye Hospital

London, EC1V 2PD, United Kingdom

Location

Western Eye Hospital - Imperial College Healthcare NHS Trust

London, NW1 5QH, 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 28, 2024
• Primary Completion: December 15, 2025
• Study Completion: February 12, 2026
• Last Updated: March 16, 2026

Record last verified: 2026-03

Locations

ES (4); LA (2); JP (12); AU (2); GE (4); IT (5); SL (2); PL (6); GB (4); US (11); SE (1); CN (6); FR (4); TU (2); BU (4); CH (2); DE (4)