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

Phase 3

Recruiting

Conditions: Thyroid Eye Disease (TED)

Interventions: Combination Product: Efgartigimod PH20 SC
Other: Placebo PH20 SC

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

Recruitment & Eligibility

Status: Ongoing, recruiting

Sex: Not specified

Target Recruitment: 49

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.

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.

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
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
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)

Study & Design

Study Type: INTERVENTIONAL

Study Design: Not specified

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-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 (41): Hospital Universitario Virgen De La Macarena
🇪🇸
Sevilla, Spain
Hospital Universitario Ramon Y Cajal
🇪🇸
Madrid, Spain
Hospital Universitario Puerta De Hierro De Majadahonda
🇪🇸
Majadahonda, Spain
Clinica Gaias Santiago
🇪🇸
Santiago De Compostela, Spain
St. Luke's Hospital S.A.
🇬🇷
Thessaloniki, Greece
General University Hospital Of Patras
🇬🇷
Patras, Greece
Athens Medical Center S.A.
🇬🇷
Thessaloniki, Greece
General Hospital Of Athens G Gennimatas
🇬🇷
Athens, Greece
Budapest Retina Associates Kft.
🇭🇺
Budapest XIII, Hungary
Vas Varmegyei Markusovszky Egyetemi Oktatokorhaz
🇭🇺
Szombathely, Hungary
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Hospital Universitario Virgen De La Macarena
🇪🇸Sevilla, Spain
Antonio Manuel Garrido Hermosilla
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