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

Phase 3

Recruiting

• Conditions: Thyroid Eye Disease
• Interventions: Combination Product: Efgartigimod PH20 SC; Other: Placebo PH20 SC

• Registration Number: NCT06307613
• Lead Sponsor: argenx

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)

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-03-06
• Study First Submitted QC: 2024-03-06
• Study First Posted: 2024-03-13
• Study Start: 2024-03-27
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-02
• Last Update Posted: 2025-05-04
• Primary Completion: 2026-02
• Study Completion: 2027-10

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 108

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Percentage of participants who were proptosis responders	At week 24 of the Double-Blinded Treatment Period

Secondary Outcome Measures

Name	Time	Method
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	Minimum value: 0 (most negative impact on Quality of Life); Maximum value: 100 (No impact on Quality of Life)
Percentage of participants with a resolution of diplopia (responders)	At week 24 of the Double-Blinded Treatment Period

Trial Locations

Locations (61)

University Of Southern California - Keck School Of Medicine; 🇺🇸 Los Angeles, California, United States

Wilmer Eye Institute; 🇺🇸 Baltimore, Maryland, United States

Universitaetsklinikum Muenster; 🇩🇪 Muenster, Germany

Markusovszky Teaching Hospital; 🇭🇺 Szombathely, Hungary

Santa Chiara Hospital; 🇮🇹 Pisa, Italy

Institutul National de Endocrinologie C.I.Parhon; 🇷🇴 Bucuresti, Romania

Faculty of Medicine Siriraj Hospital, Mahidol University; 🇹🇭 Bang Phlat, Thailand

King Chulalongkorn Memorial Hospital; 🇹🇭 Bangkok, Thailand

Maharaj Nakorn Chiang Mai Hospital; 🇹🇭 Chiang Mai, Thailand

Cardiff and Vale University Health Board - University Hospital of wales (UHW); 🇬🇧 Cardiff, United Kingdom

St Thomas' Hospital - Guy's & St Thomas' NHS Foundation Trust; 🇬🇧 London, United Kingdom

Thrive Health - Beverly Hills; 🇺🇸 Beverly Hills, California, United States

UCI Health - Gavin Herbert Eye Institute; 🇺🇸 Irvine, California, United States

Advanced Research LLC; 🇺🇸 Coral Springs, Florida, United States

Levenson Eye Associates; 🇺🇸 Jacksonville, Florida, United States

University of South Florida (USF) Health - Morsani Center for Advanced Healthcare; 🇺🇸 Tampa, Florida, United States

University of Michigan; 🇺🇸 Ann Arbour, Michigan, United States

Moyes Eye Center - Northland; 🇺🇸 Kansas City, Missouri, United States

Quest Diagnostics - Mercy Smith Glynn Callaway; 🇺🇸 Springfield, Missouri, United States

Penn Medicine - Penn Presbyterian Medical Center - Scheie Eye Institute; 🇺🇸 Philadelphia, Pennsylvania, United States

Centre for Eye Research Australia; 🇦🇺 Melbourne, Australia

North Shore Private Hospital; 🇦🇺 St Leonards, Australia

Sydney Eye Hospital; 🇦🇺 Sydney, Australia

CHU UVC Brugmann - Site Victor Horta; 🇧🇪 Bruxelles, Belgium

UMHAT Aleksandrovska - Diagnostic Consultative Center (DCC) Aleksandrova; 🇧🇬 Sofia, Bulgaria

Medical Center Vereya EOOD - Vereya Medical Center (former Military Hospital); 🇧🇬 Stara Zagora, Bulgaria

St. Petka Eye Hospital - Eye Clinic St. Petka Varna; 🇧🇬 Varna, Bulgaria

Peking Union Medical College Hospital - Dongdan campus; 🇨🇳 Beijing, China

Shunde Hospital of Southern Medical University; 🇨🇳 Foshan, China

Fujian Medical University Union Hospital; 🇨🇳 Fuzhou, China

The First Affiliated Hospital of Zhejiang University School of Medicine; 🇨🇳 Hangzhou, China

The Second Hospital of Anhui Medical University; 🇨🇳 Hefei, China

The First Affiliated Hospital of Henan University of Science and Technology; 🇨🇳 Luoyang, China

Shanghai Ninth People's Hospital of Shanghai Jiaotong University School of Medicine; 🇨🇳 Shanghai, China

The First Hospital of China Medical University; 🇨🇳 Shenyang, China

OFTEX - Ocni klinika; 🇨🇿 Pardubice, Czech Republic

Axon Clinical s.r.o; 🇨🇿 Praha, Czech Republic

Dr Liina Viitas Endokrinoloog; 🇪🇪 Parnu, Estonia

Tartu Ulikooli Kliinikum; 🇪🇪 Tartu, Estonia

Charite Universitaetsmedizin Berlin - Campus Charite Mitte (CCM); 🇩🇪 Berlin, Germany

General Hospital of Athens G Gennimatas; 🇬🇷 Athens, Greece

University General Hospital of Patras; 🇬🇷 Patras, Greece

Athens Medical Group - European Interbalkan Medical Center; 🇬🇷 Thessaloniki, Greece

Budapest Retina Intezet; 🇭🇺 Budapest, Hungary

Ganglion Orvosi Kozpont; 🇭🇺 Pecs, Hungary

Pecsi Tudomanyegyetem (PTE) Altalanos Orvostudomanyi Kar (AOK) University of Pecs Medical School; 🇭🇺 Pecs, Hungary

Azienda Ospedaliero Universitaria Careggi; 🇮🇹 Firenze, Italy

Ospedale Policlinico San Martino; 🇮🇹 Genova, Italy

ASST Santi Paolo e Carlo - Ospedale San Paolo; 🇮🇹 Milano, Italy

Azienda Ospedaliera Universitaria (AOU) Federico II; 🇮🇹 Naples, Italy

ASST Sete Laghi - Ospedale di Circolo e Fondazione Macchi di Varese; 🇮🇹 Varese, Italy

Centrul Medical Sana; 🇷🇴 Bucuresti, Romania

Spitalul Universitar De Urgenta Militar Central Dr. Carol Davila; 🇷🇴 Bucuresti, Romania

Spitalul Clinic Judetean de Urgenta Sf. Spiridon; 🇷🇴 Iasi, Romania

National University Hospital; 🇸🇬 Singapore, Singapore

Hospital Universitario Ramon y Cajal; 🇪🇸 Madrid, Spain

Hospital Universitario Puerta de Hierro Majadahonda; 🇪🇸 Majadahonda, Spain

Gaias Clinicas - Clinica Gaias Santiago; 🇪🇸 Santiago de Compostela, Spain

Hospital Universitario Virgen Macarena; 🇪🇸 Sevilla, Spain

Frimley Health NHS Foundation Trust - Frimley Park Hospital; 🇬🇧 Frimley, United Kingdom

Royal Victoria Infirmary - The Newcastle Upon Tyne Hospitals NHS Foundation Trust; 🇬🇧 Newcastle, United Kingdom