ACTIVE_NOT_RECRUITING

A Phase 3 Study to Evaluate the Efficacy and Safety of Efgartigimod IV in Patients With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

The primary purpose of this study is to measure the efficacy and safety of efgartigimod IV compared to placebo in participants with Acetylcholine Receptor Binding Antibody (AChR-Ab) seronegative Generalized Myasthenia Gravis (gMG). The study consists of a Part A where participants will be randomized to receive either efgartigimod IV or placebo IV and a Part B where participants completing part A will receive open-label efgartigimod IV. Participants will be in the study for up to (approximately) 2.5 years.

Official Title

A Randomized, Double-Blinded, Placebo-Controlled, Phase 3, Parallel-Group Design Study Evaluating the Efficacy and Safety of Efgartigimod IV in Adult Participants With Acetylcholine Receptor Binding Antibody Seronegative Generalized Myasthenia Gravis

Quick Facts

Study Start:2024-04-16
Study Completion:2027-07-23
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT06298552

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.

Ages Eligible for Study:18 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * The participant is at least 18 years of age and the local legal age of consent for clinical studies when signing the ICF.
  2. * The participant is capable of providing signed informed consent and following with protocol requirements.
  3. * The participant agrees to use contraceptive measures consistent with local regulations and the women of child-bearing potential (WOCBP) must have a negative serum pregnancy test result at screening and a negative urine pregnancy test result at baseline before receiving the study drug.
  4. * The participant has no known weakness in infancy and later develop fatigable weakness after aged 16 years and diagnosed with acquired gMG of both of the following:
  5. 1. History of abnormal neuromuscular transmission demonstrated by single fiber electromyography or repetitive nerve stimulation (RNS) or is anti-muscle-specific kinase antibodies (MuSK-Ab) seropositive
  6. 2. Either a history of positive edrophonium chloride test OR a demonstrated improvement in MG signs with treatments such as oral acetylcholinesterase (AChE) inhibitors, plasma exchange (PLEX), immunoabsorption, or intravenous immunoglobulin (IVIg)/ subcutaneous immunoglobulin (SCIg) treatment
  7. * The participant is receiving a stable dose of MG therapy before screening that includes acetylcholinesterase (AChE) inhibitors, steroids, or nonsteroidal immunosuppressive therapies (NSISTs) in combination or alone.
  1. * Known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of gMG or puts the participant at undue risk
  2. * History of malignancy, cancer, unless considered cured by adequate treatment with no evidence of recurrence for ≥3 years. Adequately treated participants with the following cancers can be included at any time: Basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast, Incidental histological findings of prostate cancer
  3. * Clinically significant active infection that is not sufficiently resolved in the investigator's opinion or positive serum test at screening for active infection with any of the following: Hepatitis B virus (HBV), Hepatitis C virus (HCV), HIV
  4. * History of or current alcohol, drug, or medication abuse as assessed by the investigator
  5. * Pregnant or lactating state or intention to become pregnant during the study
  6. * Live or live-attenuated vaccine received \<4 weeks before screening
  7. * Worsening muscle weakness secondary to concurrent infections or medications
  8. * Received a thymectomy less than 3 months before screening or thymectomy is planned during the study

Contacts and Locations

Study Locations (Sites)

HonorHealth Neurology - Bob Bove Neuroscience Institute
Scottsdale, Arizona, 85251
United States
Loma Linda University Health
Fresno, California, 93701-2234
United States
First Choice Neurology Boca Raton
Boca Raton, Florida, 33428
United States
SFM Clinical Research LLC
Boca Raton, Florida, 33487
United States
The Neurology Institute / Healthcare Innovations Institute - Coral Springs
Coral Springs, Florida, 33067
United States
Neurology Associates PA
Maitland, Florida, 32751
United States
Desai Sethi Medical Center
Miami, Florida, 33136
United States
Medsol Clinical Research Center Inc
Port Charlotte, Florida, 33952
United States
BayCare - St. Anthony's Hospital
Saint Petersburg, Florida, 33705-1410
United States
University of South Florida (USF) Health - Morsani Center for Advanced Healthcare
Tampa, Florida, 33612
United States
Wellstar - Augusta University Medical Center
Augusta, Georgia, 30912
United States
Kansas University Medical Center - Kansas City
Fairway, Kansas, 66205
United States
Rutgers-Robert Wood Johnson Medical School
New Brunswick, New Jersey, 08901-1962
United States
University of North Carolina - Chapel Hill
Chapel Hill, North Carolina, 27599-7025
United States
Duke University School of Medicine - Duke Early Phase Clinical Research Unit
Durham, North Carolina, 27710
United States
Cleveland Clinic
Cleveland, Ohio, 44195
United States
Erlanger Neuroscience Institute
Chattanooga, Tennessee, 37403-2173
United States
National Neuromuscular Research Institute
Austin, Texas, 78756
United States
University of Washington Medical Center - Montlake
Seattle, Washington, 98195
United States

Collaborators and Investigators

Sponsor: argenx

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 Registration Dates

Study Start Date2024-04-16
Study Completion Date2027-07-23

Study Record Updates

Study Start Date2024-04-16
Study Completion Date2027-07-23

Terms related to this study

Additional Relevant MeSH Terms

  • Generalized Myasthenia Gravis