Clinical trial NCT03770403: A Safety and Tolerability Study of ARGX-113 in Patients With Myasthenia Gravis Who Have Generalized Muscle Weakness.
Official title: A Long-Term, Single-Arm, Open-Label, Multicenter, Phase 3 Follow-on Trial of ARGX-113-1704 to Evaluate the 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: 2018-12-10
Enrollment: 151
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a Long-Term, Single-Arm, Open-Label, Multicenter Phase 3 follow-on trial of the ARGX-113-1704 study to evaluate the safety and tolerability of ARGX-113 in patients with gMG. Patients who have completed at least 1 cycle of treatment and at least 1 year of trial ARGX-113-1705 and have started Part B are eligible to enroll in the open-label trial ARGX-113-2002 to receive efgartigimod by SC administration.


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. Patients who participated in trial ARGX-113-1704 and are eligible for roll over, as specified in the protocol.

Other more specific inclusion criteria are further defined in the protocol.

Exclusion Criteria:

1. Patients who discontinued early from trial ARGX-113-1704 or patients who discontinued early from randomized treatment for pregnancy or rescue reasons or an (S)AE that might jeopardize the safety of the patient in that trial.
2. Pregnant and lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing. Women or childbearing potential should have a negative urine pregnancy test at SEB.
3. 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.
4. Patients with known Hepatitis B Virus (HBV), Hepatitis C Virus (HCV) or Human Immunodeficiency Virus (HIV) seropositivity.

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

Primary outcomes:
measure: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious AEs, TEAEs Leading to Study Drug Discontinuation and Fatal TEAE in AChR-Positive Participants
timeFrame: TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
Secondary outcomes:
measure: Number of Participants With TEAEs, Treatment-Emergent SAEs, TEAEs Leading to Study Drug Discontinuation and Fatal TEAE in the Overall Population
timeFrame: TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years
Other outcomes:
Locations:
city: Carlsbad
country: United States
facility: Investigator Site 42
state: California
city: Los Angeles
country: United States
facility: Investigator Site 8
state: California
city: Orange
country: United States
facility: Investigator Site 33
state: California
city: Palo Alto
country: United States
facility: Investigator Site 21
state: California
city: Jacksonville
country: United States
facility: Investigator Site 41
state: Florida
city: Tampa
country: United States
facility: Investigator Site 4
state: Florida
city: Kansas City
country: United States
facility: Investigator Site 14
state: Kansas
city: Detroit
country: United States
facility: Investigator Site 32
state: Michigan
city: Chapel Hill
country: United States
facility: Investigator Site 2
state: North Carolina
city: Cleveland
country: United States
facility: Investigator Site 18
state: Ohio
city: Portland
country: United States
facility: Investigator Site 12
state: Oregon
city: Cordova
country: United States
facility: Investigator Site 1
state: Tennessee
city: San Antonio
country: United States
facility: Investigator Site 3
state: Texas
city: Charlottesville
country: United States
facility: Investigator Site 37
state: Virginia
city: Edegem
country: Belgium
facility: Investigator Site 11
city: Ghent
country: Belgium
facility: Investigator Site 7
city: Toronto
country: Canada
facility: Investigator Site 20
state: Ontario
city: Montréal
country: Canada
facility: Investigator Site 25
state: Quebec
city: Brno
country: Czechia
facility: Investigator Site 16
city: Ostrava-Poruba
country: Czechia
facility: Investigator Site 19
city: Praha
country: Czechia
facility: Investigator Site 30
city: Aarhus
country: Denmark
facility: Investigator Site 49
city: Copenhagen
country: Denmark
facility: Investigator Site 17
city: Bordeaux
country: France
facility: Investigator Site 50
city: Marseille
country: France
facility: Investigator Site 51
city: Tbilisi
country: Georgia
facility: Investigator Site 45
city: Tbilisi
country: Georgia
facility: Investigator Site 31
city: Tbilisi
country: Georgia
facility: Investigator Site 46
city: Berlin
country: Germany
facility: Investigator Site 28
city: Budapest
country: Hungary
facility: Investigator Site 35
city: Szeged
country: Hungary
facility: Investigator Site 52
city: Milano
country: Italy
facility: Investigator Site 10
state: MI
city: Napoli
country: Italy
facility: Investigator Site 5
city: Roma
country: Italy
facility: Investigator Site 38
city: Sapporo
country: Japan
facility: Investigator Site 24
state: Hokkaido
city: Hanamaki-shi
country: Japan
facility: Investigator Site 13
state: Iwate
city: Sendai
country: Japan
facility: Investigator Site 27
state: Miyagi
city: Suita
country: Japan
facility: Investigator Site 22
state: Osaka
city: Ōsaka-sayama
country: Japan
facility: Investigator Site 23
state: Osaka
city: Meguro
country: Japan
facility: Investigator Site 40
state: Tokyo
city: Shinjuku-Ku
country: Japan
facility: Investigator Site 43
state: Tokyo
city: Chiba
country: Japan
facility: Investigator Site 47
city: Hiroshima
country: Japan
facility: Investigator Site 44
city: Minato
country: Japan
facility: Investigator Site 48
city: Leiden
country: Netherlands
facility: Investigator Site 36
city: Gdańsk
country: Poland
facility: Investigator Site 9
city: Katowice
country: Poland
facility: Investigator Site 29
city: Kraków
country: Poland
facility: Investigator Site 6
city: Warszawa
country: Poland
facility: Investigator Site 15
city: Novosibirsk
country: Russian Federation
facility: Investigator Site 34
city: Samara
country: Russian Federation
facility: Investigator Site 39
city: Belgrade
country: Serbia
facility: Investigator Site 26
Study officials:
affiliation: argenx
name: Sabine Coppieters, 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.