A study of efgartigimod PH20 SC given by prefilled syringe in adults with thyroid eye disease

Phase 3

Recruiting

• Conditions: Thyroid Eye Disease (TED)

• Registration Number: 2023-509197-35-00
• Lead Sponsor: Argenx

Brief Summary

To evaluate the efficacy of efgartigimod PH20 SC compared with placebo PH20 SC at week 24 of the Double Blind treatment period (DBTP) on the proptosis responder rate.

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: 2025-08
• Last Update Submitted: 2025-08-14
• Last Update Posted: 2025-08-18
• Primary Completion: 2026-02
• Study Completion: 2027-10-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Not specified
• Target Recruitment: 49

Inclusion Criteria

1. The participant is at least 18 years of age

2. The participant is capable of providing signed informed consent and following with protocol requirements.

3C. 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.

4A. The participant has first onset of active TED symptoms within 12 months before screening.

5. 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

6. 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

1. 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

2. History of or current alcohol, drug, or medication abuse within 12 months before screening as assessed by the investigator.

3. Pregnant or lactating state or intention to become pregnant during the study

4. Live or live-attenuated vaccine received <4 weeks before screening

5. Corneal decompensation (swelling of the cornea) unresponsive to medical management

6. Previous orbital irradiation or surgery for TED

7. Use of some medications before screening (more information is found in the protocol)

8. 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

9. 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.

10. 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

11. 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

12. Known hypersensitivity to study drug or one of its excipients (inactive ingredients)

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Percentage of participants who were proptosis responders at week 24 of the Double-Blind treatment period (DBTP).		Percentage of participants who were proptosis responders at week 24 of the Double-Blind treatment period (DBTP).

Secondary Outcome Measures

Name	Time	Method
Change in proptosis measurement in the study eye from baseline to week 24 of the Double-Blind Treatment Period (DBTP)		Change in proptosis measurement in the study eye from baseline to week 24 of the Double-Blind Treatment Period (DBTP)
Change in the total GO-QoL score from baseline to week 24 of the Double-Blind Treatment Period (DBTP)		Change in the total GO-QoL score from baseline to week 24 of the Double-Blind Treatment Period (DBTP)
Percentage of participants with a resolution of diplopia (responders) a at week 24 of the Double-Blind Treatment Period (DBTP).		Percentage of participants with a resolution of diplopia (responders) a at week 24 of the Double-Blind Treatment Period (DBTP).

Trial Locations

Locations (72)

Fondazione Irccs Ca' Granda Ospedale Maggioe Policlinico Di Milano; 🇮🇹 Milan, Italy

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

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

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

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

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

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