Clinical trial NCT06307626: A Study of Efgartigimod PH20 SC Given by Prefilled Syringe in Adults With Thyroid Eye Disease.
Official title: A Phase 3, Randomized, Double-Masked, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Efgartigimod PH20 SC Administered by Prefilled Syringe in Adult Participants With Thyroid Eye Disease
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Thyroid Eye Disease
Lead sponsor: argenx
Sponsor class: INDUSTRY
First posted: 2024-03-13
Enrollment: 108
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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)


Eligibility criteria: 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.

Primary outcomes:
measure: Percentage of participants who were proptosis responders
timeFrame: At week 24 of the Double-Blinded Treatment Period
Secondary outcomes:
measure: Change in proptosis measurement in the study eye from baseline
timeFrame: Up to week 24 of the Double-Blinded Treatment Period
measure: Change in the total Graves' Orbitopathy Quality of Life (GO-QoL) score from baseline
timeFrame: Up to week 24 of the Double-Blinded Treatment Period
measure: Percentage of participants with a resolution of diplopia (responders)
timeFrame: At week 24 of the Double-Blinded Treatment Period
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: American Institute of Research
state: California
city: Mission Hills
country: United States
facility: North Valley Eye Medical Group, Inc.
state: California
city: Mission Hills
country: United States
facility: North Valley Eye Medical Group, Inc.
state: California
city: Rancho Cordova
country: United States
facility: Martel Eye Medical Group
state: California
city: San Diego
country: United States
facility: Cockerham Eye Consultants
state: California
city: Boynton Beach
country: United States
facility: Sibia Eye Institute
state: Florida
city: Louisville
country: United States
facility: Butchertown Clinical Trials
state: Kentucky
city: St. Louis
country: United States
facility: St. Louis University (SLU) Care - Center for Specialized Medicine
state: Missouri
city: Las Vegas
country: United States
facility: Advancing Research International, LLC
state: Nevada
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Innsbruck
country: Austria
facility: Landeskrankenhaus - Universitaetskliniken Innsbruck
city: Wien
country: Austria
facility: Hanusch-Krankenhaus - Vienna Institute for Research in Ocular Surgery
city: Plovdiv
country: Bulgaria
facility: AIPSMAED Sveti Luka EOOD
city: Sliven
country: Bulgaria
facility: Multi-Profile Hospital for Active Treatment (MHAT) Hadji Dimitar
city: Sofia
country: Bulgaria
facility: Medical Center Hera EOOD
city: Beijing
country: China
facility: Peking University Third Hospital
city: Chengdu
country: China
facility: The Second Affiliated Hospital of Chengdu Medical College / Nuclear Industry 416 Hospital
city: Dalian
country: China
facility: The Second Hospital of Dalian Medical University
city: Fuzhou
country: China
facility: Fujian Provincial Hospital
city: Guangzhou
country: China
facility: Guangdong Provincial People's Hospital
city: Jinan
country: China
facility: Shandong Provincial Hospital of Shandong First Medical University
city: Bron
country: France
facility: Hospices Civils de Lyon (HCL) - Hopital Louis Pradel
city: Lille
country: France
facility: CHU Lille - Hopital Huriez
city: Paris
country: France
facility: Centre Hospitalier National d'Ophtalmologie (Chno) Des Quinze-Vingts
city: Saint-Priest-en-Jarez
country: France
facility: CHU de Saint-Etienne - Hopital Nord
city: Tbilisi
country: Georgia
facility: JSC Curatio
city: Tbilisi
country: Georgia
facility: National Institute of Endocrinology
city: Tbilisi
country: Georgia
facility: New Hospitals
city: Tbilisi
country: Georgia
facility: Caucasus Medical Centre
city: Ulm
country: Germany
facility: Universitaetsaugenklinik Ulm
city: Milano
country: Italy
facility: Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale San Raffaele (HSR) (Istituto Scientifico Universitario San Raffaele)
city: Pisa
country: Italy
facility: Ospedale Cisanello
city: Roma
country: Italy
facility: Azienda Ospedaliero Universitaria Sant'Andrea
city: Torino
country: Italy
facility: Azienda Ospedaliero Ordine Mauriziano Torino - Ospedale Umberto I di Torino
city: Kanazawa
country: Japan
facility: Kanazawa University Hospital
city: Kurume-shi
country: Japan
facility: Koga Hospital Group - Shinkoga Hospital
city: Kurume
country: Japan
facility: Shinkoga clinic
city: Kyoto-City
country: Japan
facility: National Hospital Organization Kyoto Medical Center
city: Kyoto
country: Japan
facility: National Hospital Organization Kyoto Medical Center
city: Mito
country: Japan
facility: Kozawa Eye Hospital and Diabetes Center
city: Nagasaki
country: Japan
facility: Nagasaki University Hospital
city: Niigata
country: Japan
facility: Niigata University Medical & Dental Hospital
city: Osaka
country: Japan
facility: Osaka City General Hospital
city: Sapporo-shi
country: Japan
facility: Hokkaido University Hospital
city: Sendai
country: Japan
facility: Tohoku Medical and Pharmaceutical University - Fukumuro
city: Yonago
country: Japan
facility: Tottori University Hospital
city: Daugavpils
country: Latvia
facility: Daugavpils Regionala Slimnica
city: Riga
country: Latvia
facility: Paula Stradina Kliniska universitates slimnica
city: Krakow
country: Poland
facility: Szpital Sw. Rozy
city: Lodz
country: Poland
facility: Instytut Centrum Zdrowia Matki Polki
city: Barcelona
country: Spain
facility: Institut Catala de Retina (ICR) Centre Oftalmologic - ICR Seu Central Ganduxer
city: Cordoba
country: Spain
facility: Hospital Arruzafa
city: Sant Cugat del Valles
country: Spain
facility: Quironsalud - Hospital Universitari General de Catalunya
city: Valladolid
country: Spain
facility: Hospital Clinico Universitario de Valladolid
city: Bristol
country: United Kingdom
facility: University Hospitals Bristol and Weston NHS Foundation Trust
city: Liverpool
country: United Kingdom
facility: Royal Liverpool University Hospital , St Paul's Eye Unit - Liverpool University Hospitals NHS Foundation Trust
city: London
country: United Kingdom
facility: Moorfields Eye Hospital
city: London
country: United Kingdom
facility: Western Eye Hospital - Imperial College Healthcare NHS Trust
Study officials:
References: