Clinical Study in Thyroid Eye Disease (TED)
Phase 3 Study to Evaluate the Safety and Efficacy of Self-administered Efgartigimod SC injection for Adults with Thyroid Eye Disease (TED)1
About The Study
To evaluate efgartigimod PH20 SC given by prefilled syringe in adults with active, moderate-to-severe TED, compared with placebo PH20 SC.
Eligibility
Understand the inclusion/exclusion criteria of the study to see if one of your patients may be eligible to participate.
Participation
After the screening period, participants will be randomized to 1 of 2 parallel arms to receive either efgartigimod or placebo (2:1 randomization).
EUclinicaltrials.eu ID 2023-509197-35-00 | 2023-509198-22-00
About The Study
Study goal: To evaluate the efficacy and safety of efgartigimod PH20 SC given by prefilled syringe in adults with active, moderate-to-severe TED, compared with placebo PH20 SC.
Two phase 3, randomized, double-masked, placebo-controlled, multicentre studies of efgartigimod for subcutaneously injected efgartigimod are each enrolling approximately 108 adult participants with moderate-to-severe thyroid eye disease.
About Efgartigimod
Efgartigimod is being clinically developed to treat autoimmune diseases mediated by pathogenic IgG autoantibodies and is currently approved for the treatment of myasthenia gravis and CIDP. It has not been approved for TED and the purpose of this study is to establish its efficacy and safety in this disease.
Efgartigimod prevents the binding of endogenous IgG to FcRN, leading to a reduction in circulating disease-causing autoantibodies.2 Because efgartigimod reduces IgG levels, it could reduce the pathogenic TSHR autoantibodies that are linked to TED clinical manifestations, activity and severity.3
Study Eligibility
Key Participant Inclusion Criteria
Must meet all of the following criteria:
Age 18 years or older
Capable of providing signed informed consent
Agrees to use contraceptive measures consistent with local regulations
The participant has a physician's diagnosis of active, moderate-to-severe thyroid eye disease (TED) associated with autoimmune thyroid conditions (Graves' disease or Hashimoto's thyroiditis)
The participant has 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
Key Participant Exclusion Criteria
Patients may not be eligible to participate if the following applies:
Optic neuropathy, defined as new visual field defect (blind spot), relative afferent pupillary defect (pupils respond differently to light), or colour defect secondary to optic nerve involvement within the 6 months before screening
Corneal decompensation unresponsive to medical management
Previous orbital irradiation or surgery for TED, requires immediate eye surgery, or corrective surgery/irradiation therapy is planned during the study
Use of some medications before screening
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
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
Further information can be found in the study listings at euclinicaltrials.eu UplighTED 2301 and euclinicaltrials.eu UplighTED 2309
Study Participation
Study Design
Participants will be trained in self-injection by the study team, and will administer first injections under their supervision. After adequate training, participants can self-inject, or have a caregiver inject at home, without having to travel to the study site.
Study Centre Locations
Hospital La Arruzafa, Cordoba, Spain
Universidad de Sevilla - Hospital Universitario Virgen Macarena, Sevilla, Spain
Hospital Puerta de Hierro, Majadahonda, Spain
Hospital Universitario Ramon y Cajal, Madrid, Spain
Hospital Clinico Universitario De Santiago De Compostela, Santiago De Compostela, Spain
Institut Catala de la Retina - Sarria, Barcelona, Spain
Medical Center Hera EOOD, Sofia, Bulgaria
Medical Center Vereya EOOD, Stara Zagora, Bulgaria
Multi-Profile Hospital for Active Treatment (MHAT) Hadji Dimitar, Sliven, Bulgaria
Eye Clinic Sveta Petka, Varna, Bulgaria
Diagnostic-Consultative Center Aleksandrovska EOOD, Sofia, Bulgaria
AIPSMAED Sveti Luka EOOD, Plovdiv, Bulgaria
Interbalkan Medical Center, Thessaloniki, Greece
University Hospital of Patras, Rio, Patras, Greece
Gennimatas General Hospital, Athens, Greece
Office of Dr. Liina Viitas MD, Parnu, Estonia
Tartu University Hospital, Tartu, Estonia
CHU Brugmann – HUDERF, Brussels, Belgium
Budapest Retina Associates, Budapest, Hungary
University of Pécs Medical School, Pécs, Hungary
Ganglion Orvosi Kozpont, Pecs, Hungary
AOU Federico II, Naples, Italy
AOU Careggi, Firenze, Italy
Clinica Oculistica Universitaria di Genova, Genova, Italy
AST Santi Paolo e Carlo, Milan, Italy
(IRCCS) - Ospedale San Raffaele, Milan, Italy
Ospedale di Circolo ASST dei Sette Laghi, Varese, Italy
Ospedale Mauriziano Umberto I, Turin, Italy
Azienda Ospedaliero - Universitaria Sant’Andrea, Rome, Italy
Saint Spiridon County Hospital, Iași, Romania
Spitalul Dr. Carol Davila, Bucharest, Romania
Sana Monitoring, Bucharest, Romania
Szpital Sw. Rozy, Krakow, Poland
Royal Victoria Infirmary - The NUT Hospitals NHS Foundation Trust, Newcastle, UK
Frimley Park Hospital, Frimley, Camberley, UK
University Hospitals Bristol and Weston NHS Foundation Trust, Bristol, UK
Western Eye Hospital - Imperial College Healthcare NHS Trust, London, UK
Charité Universitätsmedizin, Berlin, Germany
Oftex, s.r.o. Prague, Czech Republic
AXON Clinical, s.r.o. Prague, Czech Republic
OFTEX Ocni klinika, Pardubice, Czech Republic
Vienna Institute for Research in Ocular Surgery - Hanusch Hospital, Vienna, Austria
Landeskrankenhaus - Universitaetskliniken Innsbruck, Innsbruck, Austria
Pauls Stradiņš Clinical University Hospital, Riga, Latvia
Hospital Clinico Universitario De Valladolid, Valladolid, Spain
GAIAS Research, Valencia, Spain
OMIQ Research, Spain
Hospices Civils de Lyon (HCL) - Hopital Louis Pradel, Bron, France
Refer Your Patients with Moderate-To-Severe TED
CHU de Saint-Étienne, Saint Priest en Jarez, France
Hôpital Claude Huriez, Lille, France
Hôpital National de la vision des Quinze-Vingt, Paris, France
Hospital Universitari General de Catalunya, Sant Cugat del Valles, Spain
Contact us to refer a patient
Print an HCP Study Fact Sheet
References:
1. euclinicaltrials.eu ID 2023-509197-35-00 and 2023-509198-22-00.
2. Ulrichts et al. Neonatal Fc receptor antagonist efgartigimod safely and sustainably reduces IgGs in humans J Clin Invest. 2018; 128(10):4372-4386.
3. Sarić Matutinović et al. The phenotype of Graves' orbitopathy is associated with thyrotropin receptor antibody levels. J Endocrinol Invest. 2023 Nov;46(11):2309-2317.
UplighTED Study HCP Website_CMS_MED-EU-ESC-2400016
