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

NCT03770403 | Completed Phase 3 Results DMC FDA Drug

• 2 other identifiers: ARGX-113-17052018-002133-37
• Study Type: interventional
• Target: 151
• Locations: 15 countries
• Sites: 52

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.

Conditions

Generalized Myasthenia Gravis

Outcome Measures

Primary Outcomes (1)

• 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

  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.

  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 (1)

• Number of Participants With TEAEs, Treatment-Emergent SAEs, TEAEs Leading to Study Drug Discontinuation and Fatal TEAE in the Overall Population

  TEAEs were collected from the start of first administered study treatment (Day 1) up to end of follow-up, approximately up to 3 years

Study Arms (1)

ARGX-113

EXPERIMENTAL

Biological: ARGX-113

Interventions

ARGX-113 BIOLOGICAL

Intravenous administration of ARGX-113

Also known as: efgartigimod

ARGX-113

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with the ability to understand the requirements of the trial, provide written informed consent, and comply with the trial protocol procedures.
• Patients who participated in trial ARGX-113-1704 and are eligible for roll over, as specified in the protocol.

You may not qualify if:

• 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.
• 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.
• 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.
• Patients with known Hepatitis B Virus (HBV), Hepatitis C Virus (HCV) or Human Immunodeficiency Virus (HIV) seropositivity.

Sponsors & Collaborators

• argenx: lead

Study Sites (52)

Investigator Site 42

Carlsbad, California, 92011, United States

Location

Investigator Site 8

Los Angeles, California, 90033, United States

Location

Investigator Site 33

Orange, California, 92868, United States

Location

Investigator Site 21

Palo Alto, California, 94304, United States

Location

Investigator Site 41

Jacksonville, Florida, 32209, United States

Location

Investigator Site 4

Tampa, Florida, 33612, United States

Location

Investigator Site 14

Kansas City, Kansas, 66160, United States

Location

Investigator Site 32

Detroit, Michigan, 48201, United States

Location

Investigator Site 2

Chapel Hill, North Carolina, 27599, United States

Location

Investigator Site 18

Cleveland, Ohio, 44195, United States

Location

Investigator Site 12

Portland, Oregon, 97239, United States

Location

Investigator Site 1

Cordova, Tennessee, 38018, United States

Location

Investigator Site 3

San Antonio, Texas, 78229, United States

Location

Investigator Site 37

Charlottesville, Virginia, 22908, United States

Location

Investigator Site 11

Edegem, 2650, Belgium

Location

Investigator Site 7

Ghent, 9000, Belgium

Location

Investigator Site 20

Toronto, Ontario, M5G 2C4, Canada

Location

Investigator Site 25

Montreal, Quebec, H3A 2B4, Canada

Location

Investigator Site 16

Brno, 625 00, Czechia

Location

Investigator Site 19

Ostrava-Poruba, 70852, Czechia

Location

Investigator Site 30

Prague, 12800, Czechia

Location

Investigator Site 49

Aarhus, Denmark

Location

Investigator Site 17

Copenhagen, DK-2100, Denmark

Location

Investigator Site 50

Bordeaux, France

Location

Investigator Site 51

Marseille, France

Location

Investigator Site 45

Tbilisi, 0112, Georgia

Location

Investigator Site 31

Tbilisi, 0114, Georgia

Location

Investigator Site 46

Tbilisi, 0114, Georgia

Location

Investigator Site 28

Berlin, 10117, Germany

Location

Investigator Site 35

Budapest, 1204, Hungary

Location

Investigator Site 52

Szeged, Hungary

Location

Investigator Site 10

Milan, MI, 20133, Italy

Location

Investigator Site 5

Napoli, 80131, Italy

Location

Investigator Site 38

Roma, 00189, Italy

Location

Investigator Site 24

Sapporo, Hokkaido, 060-8543, Japan

Location

Investigator Site 13

Hanamaki-shi, Iwate, 025-0075, Japan

Location

Investigator Site 27

Sendai, Miyagi, 983-8520, Japan

Location

Investigator Site 23

Ōsaka-sayama, Osaka, 589-8511, Japan

Location

Investigator Site 22

Suita, Osaka, 565-0871, Japan

Location

Investigator Site 40

Meguro City, Tokyo, 153-8515, Japan

Location

Investigator Site 43

Shinjuku-Ku, Tokyo, 160-0023, Japan

Location

Investigator Site 47

Chiba, Japan

Location

Investigator Site 44

Hiroshima, 730-8518, Japan

Location

Investigator Site 48

Minato, Japan

Location

Investigator Site 36

Leiden, 2333 ZA, Netherlands

Location

Investigator Site 9

Gdansk, 80-952, Poland

Location

Investigator Site 29

Katowice, 40-123, Poland

Location

Investigator Site 6

Krakow, 31-505, Poland

Location

Investigator Site 15

Warsaw, 02-097, Poland

Location

Investigator Site 34

Novosibirsk, 630087, Russia

Location

Investigator Site 39

Samara, 443095, Russia

Location

Investigator Site 26

Belgrade, 11000, Serbia

Location

Related Publications (2)

• Howard JF Jr, Bril V, Vu T, Karam C, Peric S, De Bleecker JL, Murai H, Meisel A, Beydoun SR, Pasnoor M, Guglietta A, Van Hoorick B, Steeland S, T'joen C, Utsugisawa K, Verschuuren J, Mantegazza R; ADAPT+ Study Group. Long-term safety, tolerability, and efficacy of efgartigimod (ADAPT+): interim results from a phase 3 open-label extension study in participants with generalized myasthenia gravis. Front Neurol. 2024 Jan 17;14:1284444. doi: 10.3389/fneur.2023.1284444. eCollection 2023.

  PMID: 38318236 DERIVED

• 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.

  PMID: 34146511 DERIVED

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

efgartigimod alfa

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Results Point of Contact

• Title: Regulatory Manager
• Organization: argenx BVBA

regulatory@argenx.com

+32 93103400

Study Officials

• Sabine Coppieters, MD

  argenx

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: November 16, 2018
• First Posted: December 10, 2018
• Study Start: March 1, 2019
• Primary Completion: June 23, 2022
• Study Completion: June 30, 2022
• Last Updated: July 14, 2023
• Results First Posted: July 14, 2023

Record last verified: 2023-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (14); NL (1); CA (2); RU (2); BE (2); IT (3); JP (10); FR (2); DE (1); DK (2); CZ (3); HU (2); GE (3); PL (4); SE (1)