An Efficacy and Safety Study of ARGX-113 in Patients With Myasthenia Gravis Who Have Generalized Muscle Weakness (ADAPT)

January 14, 2022 updated by: argenx

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
167

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Expanded Access

Expanded Access

A way for patients with serious diseases or conditions who cannot participate in a clinical trial to gain access to a medical product that has not been approved by the U.S. Food and Drug Administration (FDA). Also called compassionate use. There are different expanded access types.
Approved

Approved

  • Available: Expanded access is currently available for this investigational treatment, and patients who are not participants in the clinical study may be able to gain access to the drug, biologic, or medical device being studied.
  • No longer available: Expanded access was available for this intervention previously but is not currently available and will not be available in the future.
  • Temporarily not available: Expanded access is not currently available for this intervention but is expected to be available in the future.
  • Approved for marketing: The intervention has been approved by the U.S. Food and Drug Administration for use by the public.
 for sale to the public. See expanded access record.

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Edegem, Belgium, 2650
        • Investigator Site 11
      • Ghent, Belgium, 9000
        • Investigator Site 8
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 2G3
        • Investigator Site 38
    • Ontario
      • Toronto, Ontario, Canada, M5G 2C4
        • Investigator Site 24
    • Quebec
      • Montréal, Quebec, Canada, H3A 2B4
        • Investigator Site 22
  • Czechia
      • Brno, Czechia, 62500
        • Investigator Site 32
      • Ostrava-Poruba, Czechia, 70852
        • Investigator Site 35
      • Praha 2, Czechia, 128 00
        • Investigator Site 51
  • Denmark
      • Aarhus, Denmark, 8200
        • Investigator Site 36
      • Copenhagen, Denmark, 2100
        • Investigator Site 15
  • France
      • Bordeaux Cedex, France, 33076
        • Investigator Site 13
      • Marseille, France, 13385
        • Investigator Site 52
  • Georgia
      • Tbilisi, Georgia, 0112
        • Investigator Site 46
      • Tbilisi, Georgia, 0114
        • Investigator Site 45
      • Tbilisi, Georgia, 0114
        • Investigator Site 47
  • Germany
      • Berlin, Germany, 10117
        • Investigator Site 33
  • Hungary
      • Budapest, Hungary, 1204
        • Investigator Site 55
      • Szeged, Hungary, 6725
        • Investigator Site 54
  • Italy
      • Milano, Italy, 20133
        • Investigator Site 10
      • Napoli, Italy, 80131
        • Investigator Site 12
  • Japan
    • Chiba
      • Chiba-shi, Chiba, Japan, 260-8677
        • Investigator Site 42
    • Hokkaido
      • Sapporo, Hokkaido, Japan, 0608543
        • Investigator Site 26
    • Iwate
      • Hanamaki, Iwate, Japan, 025-0075
        • Investigator Site 19
    • Miyagi
      • Sendai, Miyagi, Japan, 983-8520
        • Investigator Site 43
    • Osaka
      • Suita, Osaka, Japan, 565-0871
        • Investigator Site 50
      • Ōsaka-sayama, Osaka, Japan, 5898511
        • Investigator Site 28
    • Tokyo
      • Meguro, Tokyo, Japan, 1538515
        • Investigator Site 31
      • Minato-Ku, Tokyo, Japan, 108-8329
        • Investigator Site 41
      • Shinjuku-Ku, Tokyo, Japan, 160-0023
        • Investigator Site 39
  • Netherlands
      • Leiden, Netherlands, 2333 ZA
        • Investigator Site 37
  • Poland
      • Gdańsk, Poland, 80-952
        • Investigator Site 7
      • Katowice, Poland, 40-123
        • Investigator Site 57
      • Kraków, Poland, 31-505
        • Investigator Site 14
      • Warszawa, Poland, 02-097
        • Investigator Site 23
  • Russian Federation
      • Krasnoyarsk, Russian Federation, 660037
        • Investigator Site 64
      • Nizhny Novgorod, Russian Federation, 603126
        • Investigator Site 62
      • Novosibirsk, Russian Federation, 630087
        • Investigator Site 65
      • Samara, Russian Federation, 443095
        • Investigator Site 60
  • Serbia
      • Belgrade, Serbia, 11000
        • Investigator Site 61
  • United Kingdom
      • Edgbaston, United Kingdom, B15 2TH
        • Investigator Site 63
      • Liverpool, United Kingdom, L9 7LJ
        • Investigator Site 56
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85018
        • Investigator Site 29
    • California
      • Carlsbad, California, United States, 92011
        • Investigator Site 66
      • Los Angeles, California, United States, 90033
        • Investigator Site 5
      • Los Angeles, California, United States, 90095
        • Investigator Site 49
      • Orange, California, United States, 92868
        • Investigator Site 18
      • Palo Alto, California, United States, 94304
        • Investigator Site 40
      • San Francisco, California, United States, 94115
        • Investigator Site 59
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Investigator Site 58
    • Florida
      • Jacksonville, Florida, United States, 32209
        • Investigator Site 34
      • Tampa, Florida, United States, 33612
        • Investigator Site 4
    • Illinois
      • Springfield, Illinois, United States, 62702
        • Investigator Site 30
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • Investigator Site 25
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • Investigator Site 21
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Investigator Site 27
    • Michigan
      • Detroit, Michigan, United States, 48201
        • Investigator Site 48
    • New York
      • Buffalo, New York, United States, 14202
        • Investigator Site 53
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • Investigator Site 3
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Investigator Site 20
    • Oregon
      • Portland, Oregon, United States, 97239
        • Investigator Site 9
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Investigator Site 17
    • Tennessee
      • Cordova, Tennessee, United States, 38018
        • Investigator Site
    • Texas
      • Houston, Texas, United States, 77030
        • Investigator Site 44
      • San Antonio, Texas, United States, 78229
        • Investigator Site 6
    • Virginia
      • Charlottesville, Virginia, United States, 22908
        • Investigator Site 2
    • Washington
      • Seattle, Washington, United States, 98195
        • Investigator Site 16

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

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. 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Placebo Comparator: Placebo
Biological: Placebo
Intravenous administration of placebo
Experimental: ARGX-113
Biological: ARGX-113
Intravenous administration of ARGX-113
Other Names:
  • efgartigimod

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Percentage of MG-ADL Responders During Cycle 1 (C1); Analyzed in the AChR-Ab Seropositive Population
Time Frame: Baseline up to Day 63 (end of TC1)
The MG-ADL is an 8-item patient-reported scale to assess MG symptoms and their effects on daily activities. The scale comprises 2 items on daily life activities and 6 items on symptoms. The MG-ADL total score range is 0-24, with higher scores indicative of greater disease severity. A patient was considered an MG-ADL responder during C1 if there was a reduction of ≥2 points on the MG-ADL total score (compared to baseline of C1 [C1B]) for ≥4 consecutive weeks with the first reduction occurring no later than 1 week after the last infusion of IMP in C1.
Baseline up to Day 63 (end of TC1)

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Percentage of Quantitative Myasthenia Gravis (QMG) Responders During C1; Analyzed in the AChR-Ab Seropositive Population
Time Frame: Baseline up to Day 63 (end of TC1)
The QMG scale quantifies disease severity based on impairments of body functions and structures as defined by the International Classification of Disability and Health. The QMG scale consists of 13 items that measure endurance or fatigability, and accounts for fluctuations in disease state. The QMG total score range is 0-39, with higher scores indicative of greater disease severity. A patient was considered a QMG responder during C1 if there was a reduction of ≥3-points on the QMG total score (compared to C1B) for ≥4 consecutive weeks with the first reduction occurring no later than 1 week after the last infusion of IMP in C1.
Baseline up to Day 63 (end of TC1)
Percentage of MG-ADL Responders During C1; Analyzed in the Overall Population
Time Frame: Baseline up to Day 63 (end of TC1)
The percentage of MG-ADL responders during C1 in the overall population is reported for this secondary end point; percentage of MG-ADL responders during C1 in the AChR-Ab seropositive population is reported previously as a primary end point.
Baseline up to Day 63 (end of TC1)
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
Time Frame: Baseline up to Day 126
An MG-ADL CMI was defined as a reduction of ≥2 points on the total MG-ADL score compared to study entry baseline (SEB).
Baseline up to Day 126
Time From Week 4 to Qualify for Retreatment; Analyzed in the AChR-Ab Seropositive Population
Time Frame: Week 4 up to Day 182 (end of study [EoS])
Time to qualify for retreatment was defined as time from the Week 4 assessment until the first visit with a <2-point reduction compared to SEB in the MG-ADL total score and MG-ADL total score ≥5 points with >50% of the total score attributable to nonocular symptoms.
Week 4 up to Day 182 (end of study [EoS])
Percentage of Early MG-ADL Responders During C1; Analyzed in the AChR-Ab Seropositive Population
Time Frame: Baseline up to Day 63 (end of TC1)
A patient was considered an early MG-ADL responder during C1 if there was a reduction of ≥2 points on the MG-ADL total score (compared to C1B) for ≥4 consecutive weeks with the first reduction occurring no later than Week 2 (ie, after 1 or maximum 2 infusions of IMP in C1).
Baseline up to Day 63 (end of TC1)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
argenx

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Antonio Guglietta, MD, argenx

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
August 22, 2018

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 6, 2020

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
April 6, 2020

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 6, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 11, 2018

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 13, 2018

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
February 8, 2022

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 14, 2022

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • ARGX-113-1704
  • 2018-002132-25 (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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings