Clinical trial NCT06558279: A Study to Assess Efficacy and Safety of Efgartigimod PH20 SC in Adults With Ocular Myasthenia Gravis
Official title: A Randomized, Double-Blinded, Placebo-Controlled, Phase 3, Parallel-Group Design Study Evaluating the Efficacy and Safety of Efgartigimod PH20 SC Administered by Prefilled Syringe in Adult Participants With Ocular Myasthenia Gravis
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Myasthenia Gravis, Ocular
Keywords: Myasthenia Gravis
Ocular myasthenia
Lead sponsor: argenx
Sponsor class: INDUSTRY
First posted: 2024-08-16
Enrollment: 124
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: The purpose of this study is to evaluate the efficacy and safety of efgartigimod PH20 SC given by a pre-filled syringe in adult patients with ocular myasthenia gravis. The study consists of a part A (approximately 7 weeks) and a part B (up to 2 years). In part A, half of the participants will receive efgartigimod PH20 SC and the other half will receive placebo. In part B, all participants will receive efgartigimod PH20 SC. The participants will be in the study for about up to 2 years and 12 weeks.


Eligibility criteria: Inclusion Criteria:

* Is at least 18 years of age and the local legal age of consent for clinical studies
* Has been diagnosed with myasthenia gravis and supported by seropositivity for AChR-Ab; or abnormal neuromuscular transmission demonstrated by abnormal neurophysiology testing and history on positive edrophonium chloride testing or demonstrated improvement on MG therapy"
* Is MGFA Class I (any ocular muscle weakness)
* Has a screening and baseline MGII (PRO) ocular score of at least 6 with at least 2 ocular items with a score of at least 2

Exclusion Criteria:

* Other diseases that lead to eyelid drooping, peripheral muscle weakness, or diplopia
* Known autoimmune disease or any medical condition other than indication under study that would interfere with an accurate assessment of clinical symptoms of ocular myasthenia gravis or puts the participant at undue risk

Primary outcomes:
measure: MGII (PRO) ocular score change from baseline to day 29 in part A
timeFrame: Up to 29 days
Secondary outcomes:
measure: MGII (PRO+PE) ocular score change from baseline to day 29 in part A
timeFrame: Up to 29 days
measure: MG-ADL ocular domain score change from baseline to day 29 in part A
timeFrame: Up to 29 days
measure: MGII total score change from baseline to day 29 in part A
timeFrame: Up to 29 days
measure: MGII (PE) ocular score change from baseline to day 29 in part A
timeFrame: Up to 29 days
measure: MG-ADL total score change from baseline to day 29 in part A
timeFrame: Up to 29 days
measure: MGII ocular scores changes (PRO) from baseline in part A+B
timeFrame: Up to 111 weeks
measure: Incidence of (serious) adverse events
timeFrame: Up to 111 weeks
measure: MG-QoL15r total score changes from baseline in part A+B
timeFrame: Up to 111 weeks
measure: NEI VFQ-25 score changes from baseline in part A+B
timeFrame: Up to 111 weeks
measure: Percent change from baseline in total IgG levels over time in part A+B
timeFrame: Up to 111 weeks
measure: Percent change from baseline in AChR-Ab levels in AChR-Ab seropositive participants over time in part A+B
timeFrame: Up to 111 weeks
measure: Incidence of Anti-Drug Antibodies and Neutralizing Antibodies against efgartigimod in part A+B
timeFrame: Up to 111 weeks
measure: Incidence of antibodies and Neutralizing Antibodies against rHuPH20 in part A+B
timeFrame: Up to 111 weeks
Other outcomes:
Locations:
city: Boca Raton
country: United States
facility: Neurology Offices of South Florida
state: Florida
city: Boca Raton
country: United States
facility: SFM Clinical Research, LLC
state: Florida
city: Maitland
country: United States
facility: Neurology Associates PA
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Chicago
country: United States
facility: Northwestern Memorial Hospital
state: Illinois
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Austin
country: United States
facility: National Neuromuscular Research Institute
state: Texas
city: Box Hill
country: Australia
facility: Box Hill Hospital
city: Melbourne
country: Australia
facility: The Alfred Hospital
city: Southport
country: Australia
facility: Gold Coast University Hospital
city: Leuven
country: Belgium
facility: UZ Leuven
city: Liège
country: Belgium
facility: Hôpital de la Citadelle
city: Halifax
country: Canada
facility: Maritime Neurology
city: Ottawa
country: Canada
facility: Ottawa Hospital Research Institute
city: Chengdu
country: China
facility: Sichuan Academy of Medical Sciences & Sichuan Provincial People's Hospital
city: Chongqing
country: China
facility: The First Affiliated Hospital of Chongqing Medical University
city: Guangzhou
country: China
facility: Guangdong Province Traditional Chinese Medical Hospital
city: Jinan
country: China
facility: Qilu Hospital of Shandong University
city: Shanghai
country: China
facility: Huashan Hospital Fudan University
city: Egkomi
country: Cyprus
facility: Cyprus Institute of Neurology and Genetics
city: Brno
country: Czechia
facility: Fakultni nemocnice Brno
city: Pardubice
country: Czechia
facility: Nemocnice Pardubickeho kraje, a.s., Pardubicka nemocnice
city: Aarhus N
country: Denmark
facility: Aarhus Universitetshospital
city: København
country: Denmark
facility: Rigshospitalet
city: Turku
country: Finland
facility: Turun Yliopistollinen Keskussairaala
city: Tbilisi
country: Georgia
facility: Petre Sarajishvili Institute of Neurology
city: Tbilisi
country: Georgia
facility: New Hospitals
city: Tbilisi
country: Georgia
facility: Pineo Medical Ecosystem
city: Tbilisi
country: Georgia
facility: LLC Caucasus Medical Centre
city: Milano
country: Italy
facility: Fondazione IRCCS Istituto Neurologico Carlo Besta
city: Chuo-Ku
country: Japan
facility: Chiba University Hospital
city: Fukuoka
country: Japan
facility: Kyushu University Hospital
city: Hanamaki-shi
country: Japan
facility: General Hanamaki Hospital
city: Hiroshima
country: Japan
facility: Hiroshima University Hospital
city: Kawagoe
country: Japan
facility: Saitama Medical Center
city: Kyoto-Shi
country: Japan
facility: National Hospital Organization Utano National Hospital
city: Narita
country: Japan
facility: IUHW Narita Hospital
city: Sapporo-Shi
country: Japan
facility: National Hospital Organization Hokkaido Medical Center
city: Shinjuku-Ku
country: Japan
facility: Tokyo Medical University Hospital
city: Suita-Shi
country: Japan
facility: Osaka University Hospital
city: Daegu
country: Korea, Republic of
facility: Kyungpook National University Chilgok hospital
city: Seoul
country: Korea, Republic of
facility: Korea University Anam Hospital
city: Seoul
country: Korea, Republic of
facility: Severance Hospital Yonsei University Health System
city: Leiden
country: Netherlands
facility: Leids Universitair Medisch Centrum
city: Bydgoszcz
country: Poland
facility: MICS Centrum Medyczne Bydgoszcz
city: Katowice
country: Poland
facility: Centrum Medyczne Neurologia Slaska
city: Kraków
country: Poland
facility: SP ZOZ Szpital Uniwersytecki
city: Kraków
country: Poland
facility: Krakowska Akademia Neurologii
city: Lublin
country: Poland
facility: CLINIREM Sp z o.o.
city: Belgrade
country: Serbia
facility: University Clinical Center of Serbia
city: Nis
country: Serbia
facility: University Clinical Center Nis
city: Barcelona
country: Spain
facility: Hospital Universitario Vall d'Hebron
city: Barcelona
country: Spain
facility: Hospital del Mar
city: Madrid
country: Spain
facility: Hospital Clinico San Carlos
city: Madrid
country: Spain
facility: Hospital Universitario Ramon y Cajal
city: San Sebastián
country: Spain
facility: Hospital Universitario de Donostia
city: Valencia
country: Spain
facility: Hospital Clinico Universitario de Valencia
city: Göteborg
country: Sweden
facility: Sahlgrenska Universitetssjukhuset
city: Glasgow
country: United Kingdom
facility: Panthera Biopartners - Glasgow
Study officials:
References: