Clinical trial NCT06307613: 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/NCT06307626)


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: Beverly Hills
country: United States
facility: Thrive Health - Beverly Hills
state: California
city: Irvine
country: United States
facility: UCI Health - Gavin Herbert Eye Institute
state: California
city: Los Angeles
country: United States
facility: University Of Southern California - Keck School Of Medicine
state: California
city: Coral Springs
country: United States
facility: Advanced Research LLC
state: Florida
city: Jacksonville
country: United States
facility: Levenson Eye Associates
state: Florida
city: Tampa
country: United States
facility: University of South Florida (USF) Health - Morsani Center for Advanced Healthcare
state: Florida
city: Chicago
country: United States
facility: University of Illinois Chicago
state: Illinois
city: Baltimore
country: United States
facility: Wilmer Eye Institute
state: Maryland
city: Ann Arbour
country: United States
facility: University of Michigan
state: Michigan
city: Kansas City
country: United States
facility: Moyes Eye Center - Northland
state: Missouri
city: Springfield
country: United States
facility: Quest Diagnostics - Mercy Smith Glynn Callaway
state: Missouri
city: Philadelphia
country: United States
facility: Penn Medicine - Penn Presbyterian Medical Center - Scheie Eye Institute
state: Pennsylvania
city: Morgantown
country: United States
facility: West Virginia University Eye Institute
state: West Virginia
city: Melbourne
country: Australia
facility: Centre for Eye Research Australia
city: Nedlands
country: Australia
facility: The University of Western Australia - Lions Eye Institute (LEI)
city: St Leonards
country: Australia
facility: North Shore Private Hospital
city: Sydney
country: Australia
facility: Sydney Eye Hospital
city: Bruxelles
country: Belgium
facility: CHU UVC Brugmann - Site Victor Horta
city: Burgas
country: Bulgaria
facility: Oxycom Medical Centre
city: Sofia
country: Bulgaria
facility: UMHAT Aleksandrovska - Diagnostic Consultative Center (DCC) Aleksandrova
city: Stara Zagora
country: Bulgaria
facility: Medical Center Vereya EOOD - Vereya Medical Center (former Military Hospital)
city: Varna
country: Bulgaria
facility: St. Petka Eye Hospital - Eye Clinic St. Petka Varna
city: Beijing
country: China
facility: Peking Union Medical College Hospital - Dongdan campus
city: Foshan
country: China
facility: Shunde Hospital of Southern Medical University
city: Fuzhou
country: China
facility: Fujian Medical University Union Hospital
city: Hangzhou
country: China
facility: The First Affiliated Hospital of Zhejiang University School of Medicine
city: Hefei
country: China
facility: The Second Hospital of Anhui Medical University
city: Luoyang
country: China
facility: The First Affiliated Hospital of Henan University of Science and Technology
city: Shanghai
country: China
facility: Shanghai Ninth People's Hospital of Shanghai Jiaotong University School of Medicine
city: Shenyang
country: China
facility: The First Hospital of China Medical University
city: Olomouc
country: Czechia
facility: Fakultni Nemocnice Olomouc - Ocni Klinika
city: Pardubice
country: Czechia
facility: OFTEX - Ocni klinika
city: Praha
country: Czechia
facility: Axon Clinical s.r.o
city: Parnu
country: Estonia
facility: Dr Liina Viitas Endokrinoloog
city: Tartu
country: Estonia
facility: Tartu Ulikooli Kliinikum
city: Berlin
country: Germany
facility: Charite Universitaetsmedizin Berlin - Campus Charite Mitte (CCM)
city: Frankfurt
country: Germany
facility: Buergerhospital Frankfurt am Main
city: Muenster
country: Germany
facility: Universitaetsklinikum Muenster
city: Athens
country: Greece
facility: General Hospital of Athens G Gennimatas
city: Patras
country: Greece
facility: University General Hospital of Patras
city: Thessaloniki
country: Greece
facility: Athens Medical Group - European Interbalkan Medical Center
city: Budapest
country: Hungary
facility: Budapest Retina Intezet
city: Eger
country: Hungary
facility: Vadnay Szemeszet es Latszereszet
city: Pecs
country: Hungary
facility: Ganglion Orvosi Kozpont
city: Pecs
country: Hungary
facility: Pecsi Tudomanyegyetem (PTE) Altalanos Orvostudomanyi Kar (AOK) University of Pecs Medical School
city: Szombathely
country: Hungary
facility: Markusovszky Teaching Hospital
city: Firenze
country: Italy
facility: Azienda Ospedaliero Universitaria Careggi
city: Genova
country: Italy
facility: Ospedale Policlinico San Martino
city: Milano
country: Italy
facility: ASST Santi Paolo e Carlo - Ospedale San Paolo
city: Naples
country: Italy
facility: Azienda Ospedaliera Universitaria (AOU) Federico II
city: Pisa
country: Italy
facility: Santa Chiara Hospital
city: Varese
country: Italy
facility: ASST Sete Laghi - Ospedale di Circolo e Fondazione Macchi di Varese
city: Bucuresti
country: Romania
facility: Centrul Medical Sana
city: Bucuresti
country: Romania
facility: Institutul National de Endocrinologie C.I.Parhon
city: Bucuresti
country: Romania
facility: Spitalul Universitar De Urgenta Militar Central Dr. Carol Davila
city: Iasi
country: Romania
facility: Spitalul Clinic Judetean de Urgenta Sf. Spiridon
city: Singapore
country: Singapore
facility: National University Hospital
city: Madrid
country: Spain
facility: Hospital Universitario Ramon y Cajal
city: Majadahonda
country: Spain
facility: Hospital Universitario Puerta de Hierro Majadahonda
city: Santiago de Compostela
country: Spain
facility: Gaias Clinicas - Clinica Gaias Santiago
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen Macarena
city: Bang Phlat
country: Thailand
facility: Faculty of Medicine Siriraj Hospital, Mahidol University
city: Bangkok
country: Thailand
facility: King Chulalongkorn Memorial Hospital
city: Chiang Mai
country: Thailand
facility: Maharaj Nakorn Chiang Mai Hospital
city: Khon Kaen
country: Thailand
facility: Srinagarind Hospital
city: Bradford
country: United Kingdom
facility: Bradford Royal Infirmary - Bradford Teaching Hospitals NHS Foundation
city: Cardiff
country: United Kingdom
facility: Cardiff and Vale University Health Board - University Hospital of wales (UHW)
city: Frimley
country: United Kingdom
facility: Frimley Health NHS Foundation Trust - Frimley Park Hospital
city: London
country: United Kingdom
facility: St Thomas' Hospital - Guy's & St Thomas' NHS Foundation Trust
city: Newcastle
country: United Kingdom
facility: Royal Victoria Infirmary - The Newcastle Upon Tyne Hospitals NHS Foundation Trust
Study officials:
References: