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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03669588
Recruitment Status : Completed
First Posted : September 13, 2018
Results First Posted : February 8, 2022
Last Update Posted : February 8, 2022
Sponsor:
Information provided by (Responsible Party):
argenx

Study Description
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Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Generalized Myasthenia Gravis Biological: ARGX-113 Biological: Placebo Phase 3

Expanded Access : An investigational treatment associated with this study has been approved for sale to the public.   More info ...

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 167 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : August 22, 2018
Actual Primary Completion Date : April 6, 2020
Actual Study Completion Date : April 6, 2020

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: ARGX-113 Biological: ARGX-113
Intravenous administration of ARGX-113
Other Name: efgartigimod
Placebo Comparator: Placebo Biological: Placebo
Intravenous administration of placebo


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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).

  4. 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.

  5. 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).


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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. 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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03669588


Locations
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Sponsors and Collaborators
argenx
Investigators
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Study Director: Antonio Guglietta, MD argenx
  Study Documents (Full-Text)

Documents provided by argenx:
Study Protocol  [PDF] July 11, 2019
Statistical Analysis Plan  [PDF] May 8, 2020

More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: argenx
ClinicalTrials.gov Identifier: NCT03669588    
Other Study ID Numbers: ARGX-113-1704
2018-002132-25 ( EudraCT Number )
First Posted: September 13, 2018    Key Record Dates
Results First Posted: February 8, 2022
Last Update Posted: February 8, 2022
Last Verified: March 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Myasthenia Gravis
Muscle Weakness
Paresis
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Paraneoplastic Syndromes, Nervous System
 Nervous System Neoplasms
Neoplasms by Site
Neoplasms
Paraneoplastic Syndromes
Autoimmune Diseases of the Nervous System
Neurodegenerative Diseases
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases