Clinical trial NCT03669588: An Efficacy and Safety Study of ARGX-113 in Patients With Myasthenia Gravis Who Have Generalized Muscle Weakness
Official title: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Phase 3 Trial to Evaluate the Efficacy, Safety and Tolerability of ARGX-113 in Patients With Myasthenia Gravis Having Generalized Muscle Weakness
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Generalized Myasthenia Gravis
Lead sponsor: argenx
Sponsor class: INDUSTRY
First posted: 2018-09-13
Enrollment: 167
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: A randomized, double-blind, placebo controlled, multicenter Phase 3 trial to evaluate the efficacy, safety, tolerability, quality of life and impact on normal daily activities of ARGX-113 in patients with gMG.


Eligibility criteria: Inclusion Criteria:

1. Patients with the ability to understand the requirements of the trial, provide written informed consent, and comply with the trial protocol procedures.
2. Male or female patients aged ≥ 18 years.
3. Diagnosis of MG with generalized muscle weakness meeting the clinical criteria for diagnosis of MG as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, IVa and IVb.

Other, more specific inclusion criteria are defined in the protocol

Exclusion Criteria:

1. Pregnant and lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing.
2. Male patients who are sexually active and do not intend to use effective methods of contraception during the trial or within 90 days after the last dosing or male patients who plan to donate sperm during the trial or within 90 days after the last dosing.
3. MGFA Class I and V patients.
4. Patients with worsening muscle weakness secondary to concurrent infections or medications.
5. Patients with known seropositivity or who test positive for an active viral infection at Screening with:

   * Hepatitis B Virus (HBV) (except patients who are seropositive because of HBV vaccination)
   * Hepatitis C Virus (HCV)
   * Human Immunodeficiency Virus (HIV)

Other, more specific exclusion criteria are further defined in the protocol.

Primary outcomes:
measure: Percentage of MG-ADL Responders During Cycle 1 (C1); Analyzed in the AChR-Ab Seropositive Population
timeFrame: Baseline up to Day 63 (end of TC1)
Secondary outcomes:
measure: Percentage of Quantitative Myasthenia Gravis (QMG) Responders During C1; Analyzed in the AChR-Ab Seropositive Population
timeFrame: Baseline up to Day 63 (end of TC1)
measure: Percentage of MG-ADL Responders During C1; Analyzed in the Overall Population
timeFrame: Baseline up to Day 63 (end of TC1)
measure: Percentage of Time That Patients Had a Clinically Meaningful Improvement (CMI) in MG-ADL Total Score up to and Including Day 126; Analyzed in the AChR-Ab Seropositive Population
timeFrame: Baseline up to Day 126
measure: Time From Week 4 to Qualify for Retreatment; Analyzed in the AChR-Ab Seropositive Population
timeFrame: Week 4 up to Day 182 (end of study [EoS])
measure: Percentage of Early MG-ADL Responders During C1; Analyzed in the AChR-Ab Seropositive Population
timeFrame: Baseline up to Day 63 (end of TC1)
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Investigator Site 29
state: Arizona
city: Carlsbad
country: United States
facility: Investigator Site 66
state: California
city: Los Angeles
country: United States
facility: Investigator Site 5
state: California
city: Los Angeles
country: United States
facility: Investigator Site 49
state: California
city: Orange
country: United States
facility: Investigator Site 18
state: California
city: Palo Alto
country: United States
facility: Investigator Site 40
state: California
city: San Francisco
country: United States
facility: Investigator Site 59
state: California
city: Aurora
country: United States
facility: Investigator Site 58
state: Colorado
city: Jacksonville
country: United States
facility: Investigator Site 34
state: Florida
city: Tampa
country: United States
facility: Investigator Site 4
state: Florida
city: Springfield
country: United States
facility: Investigator Site 30
state: Illinois
city: Iowa City
country: United States
facility: Investigator Site 25
state: Iowa
city: Kansas City
country: United States
facility: Investigator Site 21
state: Kansas
city: Boston
country: United States
facility: Investigator Site 27
state: Massachusetts
city: Detroit
country: United States
facility: Investigator Site 48
state: Michigan
city: Buffalo
country: United States
facility: Investigator Site 53
state: New York
city: Chapel Hill
country: United States
facility: Investigator Site 3
state: North Carolina
city: Cleveland
country: United States
facility: Investigator Site 20
state: Ohio
city: Portland
country: United States
facility: Investigator Site 9
state: Oregon
city: Charleston
country: United States
facility: Investigator Site 17
state: South Carolina
city: Cordova
country: United States
facility: Investigator Site
state: Tennessee
city: Houston
country: United States
facility: Investigator Site 44
state: Texas
city: San Antonio
country: United States
facility: Investigator Site 6
state: Texas
city: Charlottesville
country: United States
facility: Investigator Site 2
state: Virginia
city: Seattle
country: United States
facility: Investigator Site 16
state: Washington
city: Edegem
country: Belgium
facility: Investigator Site 11
city: Ghent
country: Belgium
facility: Investigator Site 8
city: Edmonton
country: Canada
facility: Investigator Site 38
state: Alberta
city: Toronto
country: Canada
facility: Investigator Site 24
state: Ontario
city: Montréal
country: Canada
facility: Investigator Site 22
state: Quebec
city: Brno
country: Czechia
facility: Investigator Site 32
city: Ostrava-Poruba
country: Czechia
facility: Investigator Site 35
city: Praha 2
country: Czechia
facility: Investigator Site 51
city: Aarhus
country: Denmark
facility: Investigator Site 36
city: Copenhagen
country: Denmark
facility: Investigator Site 15
city: Bordeaux Cedex
country: France
facility: Investigator Site 13
city: Marseille
country: France
facility: Investigator Site 52
city: Tbilisi
country: Georgia
facility: Investigator Site 46
city: Tbilisi
country: Georgia
facility: Investigator Site 45
city: Tbilisi
country: Georgia
facility: Investigator Site 47
city: Berlin
country: Germany
facility: Investigator Site 33
city: Budapest
country: Hungary
facility: Investigator Site 55
city: Szeged
country: Hungary
facility: Investigator Site 54
city: Milano
country: Italy
facility: Investigator Site 10
city: Napoli
country: Italy
facility: Investigator Site 12
city: Chiba-shi
country: Japan
facility: Investigator Site 42
state: Chiba
city: Sapporo
country: Japan
facility: Investigator Site 26
state: Hokkaido
city: Hanamaki
country: Japan
facility: Investigator Site 19
state: Iwate
city: Sendai
country: Japan
facility: Investigator Site 43
state: Miyagi
city: Suita
country: Japan
facility: Investigator Site 50
state: Osaka
city: Ōsaka-sayama
country: Japan
facility: Investigator Site 28
state: Osaka
city: Meguro
country: Japan
facility: Investigator Site 31
state: Tokyo
city: Minato-Ku
country: Japan
facility: Investigator Site 41
state: Tokyo
city: Shinjuku-Ku
country: Japan
facility: Investigator Site 39
state: Tokyo
city: Leiden
country: Netherlands
facility: Investigator Site 37
city: Gdańsk
country: Poland
facility: Investigator Site 7
city: Katowice
country: Poland
facility: Investigator Site 57
city: Kraków
country: Poland
facility: Investigator Site 14
city: Warszawa
country: Poland
facility: Investigator Site 23
city: Krasnoyarsk
country: Russian Federation
facility: Investigator Site 64
city: Nizhny Novgorod
country: Russian Federation
facility: Investigator Site 62
city: Novosibirsk
country: Russian Federation
facility: Investigator Site 65
city: Samara
country: Russian Federation
facility: Investigator Site 60
city: Belgrade
country: Serbia
facility: Investigator Site 61
city: Edgbaston
country: United Kingdom
facility: Investigator Site 63
city: Liverpool
country: United Kingdom
facility: Investigator Site 56
Study officials:
affiliation: argenx
name: Antonio Guglietta, MD
role: STUDY_DIRECTOR
References:
citation: Howard JF Jr, Bril V, Vu T, Karam C, Peric S, Margania T, Murai H, Bilinska M, Shakarishvili R, Smilowski M, Guglietta A, Ulrichts P, Vangeneugden T, Utsugisawa K, Verschuuren J, Mantegazza R; ADAPT Investigator Study Group. Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial. Lancet Neurol. 2021 Jul;20(7):526-536. doi: 10.1016/S1474-4422(21)00159-9.