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

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

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.

Study Overview

• Status: Completed
• Conditions: Generalized Myasthenia Gravis
• Intervention / Treatment: Biological: ARGX-113

• Study Type: Interventional
• Enrollment (Actual): 151
• Phase: Phase 3

Expanded Access

Approved for sale to the public. See expanded access record.

Contacts and Locations

Study Locations

• Belgium
  • Edegem, Belgium, 2650
    • Investigator Site 11
  • Ghent, Belgium, 9000
    • Investigator Site 7
• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5G 2C4
      • Investigator Site 20
  • Quebec
    • Montréal, Quebec, Canada, H3A 2B4
      • Investigator Site 25
• Czechia
  • Brno, Czechia, 625 00
    • Investigator Site 16
  • Ostrava-Poruba, Czechia, 70852
    • Investigator Site 19
  • Praha, Czechia, 12800
    • Investigator Site 30
• Denmark
  • Aarhus, Denmark
    • Investigator Site 49
  • Copenhagen, Denmark, DK-2100
    • Investigator Site 17
• France
  • Bordeaux, France
    • Investigator Site 50
  • Marseille, France
    • Investigator Site 51
• Georgia
  • Tbilisi, Georgia, 0112
    • Investigator Site 45
  • Tbilisi, Georgia, 0114
    • Investigator Site 31
  • Tbilisi, Georgia, 0114
    • Investigator Site 46
• Germany
  • Berlin, Germany, 10117
    • Investigator Site 28
• Hungary
  • Budapest, Hungary, 1204
    • Investigator Site 35
  • Szeged, Hungary
    • Investigator Site 52
• Italy
  • Napoli, Italy, 80131
    • Investigator Site 5
  • Roma, Italy, 00189
    • Investigator Site 38
  • MI
    • Milano, MI, Italy, 20133
      • Investigator Site 10
• Japan
  • Chiba, Japan
    • Investigator Site 47
  • Hiroshima, Japan, 730-8518
    • Investigator Site 44
  • Minato, Japan
    • Investigator Site 48
  • Hokkaido
    • Sapporo, Hokkaido, Japan, 060-8543
      • Investigator Site 24
  • Iwate
    • Hanamaki-shi, Iwate, Japan, 025-0075
      • Investigator Site 13
  • Miyagi
    • Sendai, Miyagi, Japan, 983-8520
      • Investigator Site 27
  • Osaka
    • Suita, Osaka, Japan, 565-0871
      • Investigator Site 22
    • Ōsaka-sayama, Osaka, Japan, 589-8511
      • Investigator Site 23
  • Tokyo
    • Meguro, Tokyo, Japan, 153-8515
      • Investigator Site 40
    • Shinjuku-Ku, Tokyo, Japan, 160-0023
      • Investigator Site 43
• Netherlands
  • Leiden, Netherlands, 2333 ZA
    • Investigator Site 36
• Poland
  • Gdańsk, Poland, 80-952
    • Investigator Site 9
  • Katowice, Poland, 40-123
    • Investigator Site 29
  • Kraków, Poland, 31-505
    • Investigator Site 6
  • Warszawa, Poland, 02-097
    • Investigator Site 15
• Russian Federation
  • Novosibirsk, Russian Federation, 630087
    • Investigator Site 34
  • Samara, Russian Federation, 443095
    • Investigator Site 39
• Serbia
  • Belgrade, Serbia, 11000
    • Investigator Site 26
• United States
  • California
    • Carlsbad, California, United States, 92011
      • Investigator Site 42
    • Los Angeles, California, United States, 90033
      • Investigator Site 8
    • Orange, California, United States, 92868
      • Investigator Site 33
    • Palo Alto, California, United States, 94304
      • Investigator Site 21
  • Florida
    • Jacksonville, Florida, United States, 32209
      • Investigator Site 41
    • Tampa, Florida, United States, 33612
      • Investigator Site 4
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • Investigator Site 14
  • Michigan
    • Detroit, Michigan, United States, 48201
      • Investigator Site 32
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27599
      • Investigator Site 2
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Investigator Site 18
  • Oregon
    • Portland, Oregon, United States, 97239
      • Investigator Site 12
  • Tennessee
    • Cordova, Tennessee, United States, 38018
      • Investigator Site 1
  • Texas
    • San Antonio, Texas, United States, 78229
      • Investigator Site 3
  • Virginia
    • Charlottesville, Virginia, United States, 22908
      • Investigator Site 37

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ARGX-113	Biological: ARGX-113; Intravenous administration of ARGX-113; Other Names: efgartigimod

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

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

Collaborators and Investigators

• Sponsor: argenx
• Investigators: Study Director: Sabine Coppieters, MD, argenx

Publications and helpful links

General Publications

• 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. Erratum In: Lancet Neurol. 2021 Aug;20(8):e5.

Study record dates

Study Major Dates

• Study Start (Actual): March 1, 2019
• Primary Completion (Actual): June 23, 2022
• Study Completion (Actual): June 30, 2022

Study Registration Dates

• First Submitted: November 16, 2018
• First Submitted That Met QC Criteria: December 7, 2018
• First Posted (Actual): December 10, 2018

Study Record Updates

• Last Update Posted (Actual): July 14, 2023
• Last Update Submitted That Met QC Criteria: June 21, 2023
• Last Verified: June 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Neoplasms; Autoimmune Diseases of the Nervous System; Autoimmune Diseases; Neoplasms by Site; Neurologic Manifestations; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Neuromuscular Manifestations; Nervous System Neoplasms; Paraneoplastic Syndromes, Nervous System; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Muscle Weakness; Myasthenia Gravis; Paresis
• Other Study ID Numbers: ARGX-113-1705; 2018-002133-37 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No