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: NCT06307626
• 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/NCT06307613)

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-03-06
• Study First Submitted QC: 2024-03-11
• Study First Posted: 2024-03-13
• Study Start: 2024-03-28
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-02
• Last Update Posted: 2025-05-05
• 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 (51)

Kozawa Eye Hospital and Diabetes Center; 🇯🇵 Mito, Japan

Universitaetsaugenklinik Ulm; 🇩🇪 Ulm, Germany

Ospedale Cisanello; 🇮🇹 Pisa, Italy

Shinkoga clinic; 🇯🇵 Kurume, Japan

Niigata University Medical & Dental Hospital; 🇯🇵 Niigata, Japan

Daugavpils Regionala Slimnica; 🇱🇻 Daugavpils, Latvia

Instytut Centrum Zdrowia Matki Polki; 🇵🇱 Lodz, Poland

American Institute of Research; 🇺🇸 Los Angeles, California, United States

North Valley Eye Medical Group, Inc.; 🇺🇸 Mission Hills, California, United States

Martel Eye Medical Group; 🇺🇸 Rancho Cordova, California, United States

Cockerham Eye Consultants; 🇺🇸 San Diego, California, United States

Sibia Eye Institute; 🇺🇸 Boynton Beach, Florida, United States

Butchertown Clinical Trials; 🇺🇸 Louisville, Kentucky, United States

St. Louis University (SLU) Care - Center for Specialized Medicine; 🇺🇸 St. Louis, Missouri, United States

Advancing Research International, LLC; 🇺🇸 Las Vegas, Nevada, United States

Baylor College of Medicine; 🇺🇸 Houston, Texas, United States

Landeskrankenhaus - Universitaetskliniken Innsbruck; 🇦🇹 Innsbruck, Austria

Hanusch-Krankenhaus - Vienna Institute for Research in Ocular Surgery; 🇦🇹 Wien, Austria

AIPSMAED Sveti Luka EOOD; 🇧🇬 Plovdiv, Bulgaria

Multi-Profile Hospital for Active Treatment (MHAT) Hadji Dimitar; 🇧🇬 Sliven, Bulgaria

Medical Center Hera EOOD; 🇧🇬 Sofia, Bulgaria

Peking University Third Hospital; 🇨🇳 Beijing, China

The Second Affiliated Hospital of Chengdu Medical College / Nuclear Industry 416 Hospital; 🇨🇳 Chengdu, China

The Second Hospital of Dalian Medical University; 🇨🇳 Dalian, China

Fujian Provincial Hospital; 🇨🇳 Fuzhou, China

Guangdong Provincial People's Hospital; 🇨🇳 Guangzhou, China

Shandong Provincial Hospital of Shandong First Medical University; 🇨🇳 Jinan, China

Hospices Civils de Lyon (HCL) - Hopital Louis Pradel; 🇫🇷 Bron, France

CHU Lille - Hopital Huriez; 🇫🇷 Lille, France

Centre Hospitalier National d'Ophtalmologie (Chno) Des Quinze-Vingts; 🇫🇷 Paris, France

CHU de Saint-Etienne - Hopital Nord; 🇫🇷 Saint-Priest-en-Jarez, France

Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale San Raffaele (HSR) (Istituto Scientifico Universitario San Raffaele); 🇮🇹 Milano, Italy

Azienda Ospedaliero Universitaria Sant'Andrea; 🇮🇹 Roma, Italy

Azienda Ospedaliero Ordine Mauriziano Torino - Ospedale Umberto I di Torino; 🇮🇹 Torino, Italy

Kanazawa University Hospital; 🇯🇵 Kanazawa, Japan

Koga Hospital Group - Shinkoga Hospital; 🇯🇵 Kurume-shi, Japan

National Hospital Organization Kyoto Medical Center; 🇯🇵 Kyoto, Japan

Nagasaki University Hospital; 🇯🇵 Nagasaki, Japan

Osaka City General Hospital; 🇯🇵 Osaka, Japan

Hokkaido University Hospital; 🇯🇵 Sapporo-shi, Japan

Tohoku Medical and Pharmaceutical University - Fukumuro; 🇯🇵 Sendai, Japan

Tottori University Hospital; 🇯🇵 Yonago, Japan

Paula Stradina Kliniska universitates slimnica; 🇱🇻 Riga, Latvia

Szpital Sw. Rozy; 🇵🇱 Krakow, Poland

Institut Catala de Retina (ICR) Centre Oftalmologic - ICR Seu Central Ganduxer; 🇪🇸 Barcelona, Spain

Hospital Arruzafa; 🇪🇸 Cordoba, Spain

Quironsalud - Hospital Universitari General de Catalunya; 🇪🇸 Sant Cugat del Valles, Spain

Hospital Clinico Universitario de Valladolid; 🇪🇸 Valladolid, Spain

University Hospitals Bristol and Weston NHS Foundation Trust; 🇬🇧 Bristol, United Kingdom

Royal Liverpool University Hospital , St Paul's Eye Unit - Liverpool University Hospitals NHS Foundation Trust; 🇬🇧 Liverpool, United Kingdom

Western Eye Hospital - Imperial College Healthcare NHS Trust; 🇬🇧 London, United Kingdom