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A Safety and Tolerability Study of ARGX-113 in Patients With Myasthenia Gravis Who Have Generalized Muscle Weakness.

Phase 3
Completed
Conditions
Generalized Myasthenia Gravis
Interventions
Biological: ARGX-113
Registration Number
NCT03770403
Lead Sponsor
argenx
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.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
151
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.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
ARGX-113ARGX-113-
Primary Outcome Measures
NameTimeMethod
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 ParticipantsTEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years

An AE was any untoward medical occurrence in a clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. Any clinically significant abnormal laboratory test results (hematology, clinical chemistry, or urinalysis) or other safety assessments (electrocardiogram \[ECG\], radiological scans, vital signs measurements) were collected as AEs. All AEs starting on or after first dose administered and until completion of participant's last visit were considered as TEAEs. A serious AE (SAE) was any AE that resulted in death, was life-threatening, required inpatient hospitalization, resulted in persistent or significant disability/incapacity, was a congenital abnormality, or was medically significant.

Secondary Outcome Measures
NameTimeMethod
Number of Participants With TEAEs, Treatment-Emergent SAEs, TEAEs Leading to Study Drug Discontinuation and Fatal TEAE in the Overall PopulationTEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years

Overall population included both AChR-Ab seropositive and AChR-Ab seronegative participants. An AE was any untoward medical occurrence in a clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. Any clinically significant abnormal laboratory test results (hematology, clinical chemistry, or urinalysis) or other safety assessments (ECG, radiological scans, vital signs measurements) were collected as AEs. All AEs starting on or after first dose administered and until completion of participant's last visit were considered as TEAEs. An SAE was any AE that resulted in death, was life-threatening, required inpatient hospitalization, resulted in persistent or significant disability/incapacity, was a congenital abnormality, or was medically significant.

Trial Locations

Locations (52)

Investigator Site 8

🇺🇸

Los Angeles, California, United States

Investigator Site 16

🇨🇿

Brno, Czechia

Investigator Site 51

🇫🇷

Marseille, France

Investigator Site 19

🇨🇿

Ostrava-Poruba, Czechia

Investigator Site 30

🇨🇿

Praha, Czechia

Investigator Site 7

🇧🇪

Ghent, Belgium

Investigator Site 49

🇩🇰

Aarhus, Denmark

Investigator Site 50

🇫🇷

Bordeaux, France

Investigator Site 20

🇨🇦

Toronto, Ontario, Canada

Investigator Site 52

🇭🇺

Szeged, Hungary

Investigator Site 13

🇯🇵

Hanamaki-shi, Iwate, Japan

Investigator Site 27

🇯🇵

Sendai, Miyagi, Japan

Investigator Site 28

🇩🇪

Berlin, Germany

Investigator Site 33

🇺🇸

Orange, California, United States

Investigator Site 21

🇺🇸

Palo Alto, California, United States

Investigator Site 35

🇭🇺

Budapest, Hungary

Investigator Site 38

🇮🇹

Roma, Italy

Investigator Site 24

🇯🇵

Sapporo, Hokkaido, Japan

Investigator Site 44

🇯🇵

Hiroshima, Japan

Investigator Site 14

🇺🇸

Kansas City, Kansas, United States

Investigator Site 32

🇺🇸

Detroit, Michigan, United States

Investigator Site 18

🇺🇸

Cleveland, Ohio, United States

Investigator Site 3

🇺🇸

San Antonio, Texas, United States

Investigator Site 17

🇩🇰

Copenhagen, Denmark

Investigator Site 10

🇮🇹

Milano, MI, Italy

Investigator Site 5

🇮🇹

Napoli, Italy

Investigator Site 1

🇺🇸

Cordova, Tennessee, United States

Investigator Site 34

🇷🇺

Novosibirsk, Russian Federation

Investigator Site 43

🇯🇵

Shinjuku-Ku, Tokyo, Japan

Investigator Site 11

🇧🇪

Edegem, Belgium

Investigator Site 45

🇬🇪

Tbilisi, Georgia

Investigator Site 31

🇬🇪

Tbilisi, Georgia

Investigator Site 46

🇬🇪

Tbilisi, Georgia

Investigator Site 23

🇯🇵

Ōsaka-sayama, Osaka, Japan

Investigator Site 29

🇵🇱

Katowice, Poland

Investigator Site 25

🇨🇦

Montréal, Quebec, Canada

Investigator Site 47

🇯🇵

Chiba, Japan

Investigator Site 22

🇯🇵

Suita, Osaka, Japan

Investigator Site 40

🇯🇵

Meguro, Tokyo, Japan

Investigator Site 48

🇯🇵

Minato, Japan

Investigator Site 36

🇳🇱

Leiden, Netherlands

Investigator Site 9

🇵🇱

Gdańsk, Poland

Investigator Site 6

🇵🇱

Kraków, Poland

Investigator Site 15

🇵🇱

Warszawa, Poland

Investigator Site 39

🇷🇺

Samara, Russian Federation

Investigator Site 26

🇷🇸

Belgrade, Serbia

Investigator Site 42

🇺🇸

Carlsbad, California, United States

Investigator Site 41

🇺🇸

Jacksonville, Florida, United States

Investigator Site 37

🇺🇸

Charlottesville, Virginia, United States

Investigator Site 4

🇺🇸

Tampa, Florida, United States

Investigator Site 12

🇺🇸

Portland, Oregon, United States

Investigator Site 2

🇺🇸

Chapel Hill, North Carolina, United States

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