Not yet recruiting

Last updated information on 5-2-2026 from clinicaltrials.gov

ClinicalTriails.gov ID: NCT07224386

Sponsor: BioSensics

Study Start (Actual): 2026-01-20

Phase 3 

Evaluate the feasibility of using digital health technologies to monitor symptoms in myasthenia gravis (MG).

Study subjects will be screened and enrolled at Massachusetts General Brigham Hospital to participate in this 12 month observational study. Study subjects will be asked to wear multiple wearable sensors to monitor their physical activity and PPG during daily activities. Participants will also complete speech, video, and ePRO and eCOA digital assessments at home and during study visits.

The primary objective for this observational study is to measure the correlation of sensor-derived measures of physical activity to MG-specific ratings of MG-ADL, QMG, MGC, and Neuro-QoL Fatigue

Inclusion Criteria:

• Autoimmune MG with or without history of thymoma, MGFA severity Class IIa/b, IIIa/b or IVa/b at the screening visit

• Diagnosed gMG through ONE of the following methods:

  • Positive acetylcholine receptor antibody (AChR Ab) test
  • Positive muscle specific kinase receptor antibody (MuSK Ab) test
  • Positive LRP4 antibody and abnormal neuromuscular transmission demonstrated by single-fiber electromyography (SFEMG) or repetitive nerve stimulation (RNS) OR has maintained a positive response to treatments such as AChE inhibitors, IVIG/PLEX, FcRn antagonists or C5 inhibitors
  • Abnormal neuromuscular transmission demonstrated by single-fiber electromyography (SFEMG) or repetitive nerve stimulation (RNS) OR has maintained a positive response to treatments such as AChE inhibitors, IVIG/PLEX, FcRn antagonists or C5 inhibitors.
• Physically and cognitively able to provide informed consent and adhere to the protocol, as determined by the investigator's judgment
• Ambulatory status defined as the ability to walk a distance of 10 meters independently, with or without the use of an assistive device
• Male or female, between the ages of 18 years old and 80 years old
• Speaks and reads English fluently

Exclusion Criteria:

• Inability to engage in activities that are essential for independent living, such as dressing, bathing, toileting, or eating independently.
• Neurological or orthopedic problems independent of myasthenia which significantly affect gait and ADLs in the investigator's judgement.
• Any significant medical, laboratory, or psychiatric condition that, in the judgment of the investigators, would potentially interfere with the ability to participate in the study.
• Residence in long-term care centers or institutions, nursing facilities, skilled nursing facilities, or recipients of hospice care, or incarceration.
• MGFA severity class I or V (MG crisis)
• Pregnant women.
• Concurrent participation in an interventional clinical trial (observational studies, biomarker studies and registries are acceptable)

Click here to open study  

Recruiting

Last updated information on 5-2-2026 from clinicaltrials.gov

ClinicalTriails.gov ID: NCT07250750

Sponsor: ImmunAbs Inc.

Study Start (Actual): 2026-02-05

Phase: 1 & 2 

The goal of this clinical trial is to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and potential efficacy of IM-101 in adult participants with AChR antibody-positive gMG. Subsequently, the safety and efficacy of the selected IM-101 dose-regimen will be tested in participants with AChR antibody-negative gMG and participants with AChR antibody-positive or AChR antibody-negative oMG.

A Phase 1b/2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Investigate A) the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Ascending Doses of IM-101 in Adult Participants With Generalized Myasthenia Gravis, and B) the Efficacy and Safety of Treatment of IM-101 in Adult Participants With Generalized Myasthenia Gravis and Ocular Myasthenia Gravis.

Inclusion Criteria:

1. Able and willing to provide signed informed consent
2. Willingness to consent to screening for genetic muscular diseases
3. Male or female aged ≥ 18 years and < 75 years
4. Diagnosed with MG
5. On a stable dose of background therapy for the treatment of MG
6. Body weight ≥ 40 kg at screening
7. Vaccinated against meningococcal infection (Neisseria meningitidis), streptococcus pneumoniae, and haemophilus influenzae type B

Exclusion Criteria:

1. Previous exposure to IM-101
2. Anti-MuSK antibody Positive
3. History of malignant thymoma, or history of cancer within the past 5 years of screening
4. History of N. meningitidis infection
5. Has been treated with any complement inhibitor, but failed due to intolerability or lack of efficacy

Full eligibility criteria is available in the study protocol.

Current Locations: United States

Florida Locations

Altamonte Springs, Florida, United States, 32714

Recruiting

Neurology of Central Florida Research Center, LLC

Boca Raton, Florida, United States, 33487

Recruiting

SFM Clinical Research, LLC

Texas Locations

Houston, Texas, United States, 77030

Recruiting

Nerve & Muscle Center of Texas

Houston, Texas, United States, 77070

Not yet recruiting

Houston Methodist Neurological Institute

Click here to open study

Recruiting

Last updated information on 5-2-2026 from clinicaltrials.gov

ClinicalTriails.gov ID: NCT06414954

Sponsor: NMD Pharma A/S

Study Start (Actual): 2026-02-05

This Phase 2 proof-of-concept, dose range finding study aims to evaluate the safety and efficacy of 3 dose levels of NMD670 vs placebo in adult patients with MG with antibodies against AChR or MuSK, administered twice a day (BID) for 21 days.

A Phase 2b, Randomised, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of 3 Dose Levels of NMD670 Over 21 Days in Adult Patients With AChR/MuSK-Ab+ Myasthenia Gravis

Inclusion Criteria:

• Participant must be a male or female being 18 or more, at the time of signing the informed consent
• Diagnosis of MG, MGFA class II, III or IV
• Documented positive AChR or MuSK antibody test.
• Participant must be able to swallow tablets
• Body mass index between 18 and 35 kg/m2, inclusive, at screening, and with a minimum weight of 40 kg
• Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
• Participant is capable of and has given signed informed consent

Exclusion Criteria:

• Known medical or psychological condition(s) or risk factor that, in the opinion of the Investigator, might interfere with the patient's full participation in the study, pose any additional risk for the patient, or confound the assessment of the patient or outcome of the study
• Participants with other significant clinical and/or laboratory safety findings that may interfere with the conduction or interpretation of the study
• Participants that received treatment with an investigational medical product within 30 days or 5 half-lives of the medication, whichever is longer prior to Day 1
• Participants with history of poor compliance with relevant MG therapy
• Female patients who plan to become pregnant during the study or are currently pregnant or breastfeeding

Current Locations: United States

California Locations

Irvine, California, United States, 92868

Recruiting

University of California Irvine Medical Center

Aurora, Colorado, United States, 80045

Recruiting

University of Colorado Neuromuscular Division

Recruiting

Last updated information on 5-2-2026 from clinicaltrials.gov

ClinicalTriails.gov ID: NCT06193889

Sponsor: Kyverna Therapeutics

Study Start (Actual): 2024-08-28

Phase 2 & 3 

A Study of the Anti-CD 19 Chimeric Antigen Receptor T Cell Therapy for Patients with Myasthenia Gravis.

Myasthenia gravis (MG) is a chronic autoimmune disease that affects the neuromuscular junction and is characterized by muscle weakness. B cells play a role in MG, and the disease is characterized by the presence of autoantibodies such as anti-AChR and anti-MuSK antibodies. CD-19 target chimeric antigen receptor (CAR) T cells harness the ability of cytotoxic T cells to directly and specifically lyse target cells to effectively deplete both normal and autoreactive B cells in the circulation as well as impacted lymphoid and potentially non-lymphoid tissues. KYV-101, a fully human anti-CD19 CAR T-cell therapy, will be investigated in adult subjects with myasthenia gravis (MG).

KYSA-6: A Phase 2/3, Open-Label, Randomized, Controlled, Multicenter Study of KYV-101, an Autologous Fully Human Anti-CD19 Chimeric Antigen Receptor T-cell (CD19 CAR T) Therapy, Versus Ongoing Standard-Of-Care Immunosuppressive Therapy in Patients With Generalized Myasthenia Gravis

Key Inclusion Criteria

1. Presence of autoantibodies to AChR or MuSK
2. Myasthenia Gravis Foundation of America (MGFA) Class II-IV
3. MG-Activities of Daily Living (MG-ADL) total score of ≥6 at screening and confirmed at baseline visit
4. QMG total score of ≥11 at screening an confirmed at baseline visit
5. Failed treatment with 2 or more immunosuppressive/immunomodulatory therapies, or failed at least 1 immunosuppressive therapy and required chronic plasmapheresis, or IVIG (or subcutaneous or intramuscular Ig) to control symptoms
6. On a stable dose of glucocorticoids and/or other immunotherapies for ≥1 month prior to screening. For patients treated with azathioprine, a stable dose for ≥2 months prior to screening is required
7. No change in dose of acetylcholinesterase inhibitors for ≥2 weeks prior to screening
8. No use of intravenous immune globulin (IVIG) or plasmapheresis (PLEX) within 4 weeks of screening or pre-dose baseline (unless this is part of their SOC treatment regimen)
9. No use of rituximab (or any other anti-CD20 or CD19 monoclonal antibody) within 12 weeks prior to screening
10. Able and willing to attend the necessary visits to the study site

Key Exclusion Criteria

1. Unable to washout or interrupt autoimmune disease therapy prior to apheresis and/or baseline if required
2. Co-occurring neurological autoimmune disease (ie, Lambert-Eaton Myasthenic Syndrome) or any disease affecting the neuromuscular junction or muscle causing weakness (eg, myositis, myopathy, motor neuropathy)
3. History of stroke (with residual sequalae and/or risk for recurrence), seizure (even if well controlled on antiepileptics), neurodegenerative disease, altered mental status (unexplained and/or recent/current), or uncontrolled/severe psychiatric disease
4. Any serious and/or uncontrolled medical condition that, in the investigator's judgment, would cause unacceptable safety risk, interfere with study procedures or results, or compromise compliance with the protocol, including but not limited to, clinically significant cardiac or pulmonary disease
5. History of primary immunodeficiency, organ or allogeneic bone marrow transplant, or splenectomy
6. Active, uncontrolled, viral, bacterial, or systemic fungal infection or recent history of repeated infections
7. Thymectomy <12 months of screening or planned during the study
8. Prior treatment with gene therapy product or cellular immunotherapy (eg, CAR T) requiring vector integration and directed at any target
9. Patients requiring chronic anticoagulation therapy that cannot be discontinued for medical procedures

United States Locations

California Locations

Orange, California, United States, 92868

Recruiting

University of California, Irvine

Recruiting 

Last updated information on 5-2-2026 from clinicaltrials.gov

ClinicalTriails.gov ID: NCT07217587

Sponsor: Janssen Research & Development, LLC

Study Start (Actual): 2026-01-05

Phase 3

The purpose of this study is to assess how well nipocalimab works when compared to efgartigimod in participants with generalized myasthenia gravis (a condition in which body's immune system mistakenly attacks and damages the connection between nerves and muscles causing muscle weakness).

Efficacy and Safety of Nipocalimab vs Efgartigimod for Patients With Generalized Myasthenia Gravis in a Randomized, Open-label, Phase 3b, Interventional Trial Including Within Class Switching From Efgartigimod to Nipocalimab

Inclusion criteria:

For all arms:

• Medically stable on the basis of physical examination, medical history, vital signs, clinical laboratory tests, and 12-lead electrocardiogram (ECG) performed at screening
• Diagnosis of myasthenia gravis (MG) with generalized muscle weakness meeting the clinical criteria for generalized MG (gMG) as defined by the Myasthenia gravis foundation of America (MGFA) clinical classification class II a/b, III a/b, or IV a/b at screening and positive for acetylcholine receptor (AChR) antibodies
• Myasthenia Gravis-Activities of Daily Living (MG-ADL) score of greater than or equal to (>=) 5 with less than (<) 50% of symptoms coming from ocular MG-ADL sub-scores at study screening and baseline (Day 1) visits

Criteria specific to Arms 1 and 2 only:

- Has suboptimal response to current stable therapy for gMG according to the investigator or has discontinued corticosteroids and/or immunosuppressants/immunomodulators including eculizumab or other novel approved immune agents at least 4 weeks prior to baseline due to intolerance or lack of efficacy

Criteria specific to Arm 3:

- Treatment with efgartigimod IV or subcutaneous (SC) for >=1 cycle, and the final cycle is consistent with product information

Exclusion criteria:

• Any confirmed or suspected clinical immunodeficiency syndrome not related to treatment of his/her gMG, or has a family history of congenital or hereditary immunodeficiency unless confirmed absent in the participant
• Had a thymectomy within 1 year prior to baseline, or thymectomy is planned during the study
• Currently has a malignancy or has a history of malignancy within 3 years before baseline

Criteria specific to Arms 1 and 2 only:

- Has received treatment for MG with an FcRn-targeting therapy

Criteria specific to Arm 3 only:

- Is currently taking IgG monoclonal antibody therapeutics, or Fc-conjugated therapeutic agents, including factor or enzyme replacement, with the exception of efgartigimod

United States Locations

Florida Locations

Boca Raton, Florida, United States, 33487

Recruiting

SFM Clinical Research LLC

Illnois Locations

O'Fallon, Illinois, United States, 62269

Recruiting

HSHS St. Elizabeth's Hospital

Michigan Locations

Detroit, Michigan, United States, 48202

Recruiting

Henry Ford Hospital

Click here to open

Recruiting

Last updated information on 5-2-2026 from clinicaltrials.gov

ClinicalTrials.gov ID: NCT07215949

Sponsor: Miriam Freimer

Study Start (Actual): 2026-01-20

Brief Summary

This is an open-label, multicenter, interventional phase 3b study in participants with AChR+ gMG and severe exacerbation that require hospitalization. Patients will receive subcutaneous zilucoplan injections daily for 12 weeks. Participation in the study will last for approximately 18 weeks.

Detailed Description

The primary objective of the study is to evaluate the efficacy of subcutaneous zilucoplan in participants with AChR antibody positive gMG who experience severe exacerbations requiring hospitalization. A total of 15 patients will be enrolled in the study with treatment lasting 12 weeks. Dosing will be weight based.

Patients will be presented with the option of undergoing standard of care plasma exchange or IVIG or to participate in the research study to determine if the use of zilucoplan would rapidly alleviate the most severe symptoms of gMG (respiratory dysfunction and/or bulbar dysfunction). Assessments will include medical history, physical exam, vital signs and bloodwork. MG assessments will also be conducted including the QMG and MG-ADL and patient reported outcome measures like the Myasthenia Gravis Quality of Life 15 item Revised (MGQoL15r) scale. Treatment will start in the hospital and once the patient is discharged, treatment will continue as an outpatient. Current medications and adverse events will also be reviewed and documented. Patients who undergo treatment with zilucoplan will be vaccinated against meningitis prior to starting the study drug and will continue with the recommended set of vaccines for 6 months. Antibiotics will be taken concurrently until patients are fully vaccinated.

The study aims to find a less invasive method to treat MG patients who are experiencing an exacerbation. Utilization of a subcutaneous medication with relatively rapid onset of action may provide a viable alternative to the current therapeutic approaches. If rapid complement inhibition were to prove efficacious in the treatment of acute exacerbations of gMG, this therapy would potentially obviate the need for transfer to tertiary care centers, avoid the potentially hazardous placement of a large bore central venous catheter for plasmapheresis, and potentially reduce hospital length of stay.

United States Locations

Ohio Locations

Columbus, Ohio, United States, 43210

Recruiting

The Ohio State University

Click here to open study

Recruiting 

Last updated information on 5-2-2026 from clinicaltrials.gov

ClinicalTrials.gov ID: NCT04951622

Sponsor: Janssen Research & Development, LLC

Study Start (Actual): 2021-07-15

Phase 3

Brief Summary

The purpose of this study is to evaluate the efficacy and safety of nipocalimab compared to placebo in participants with generalized myasthenia gravis (gMG). The purpose of the subcutaneous substudy is to evaluate how well it works in the body (pharmacodynamic [PD]) when given as an injection under the skin (subcutaneous) compared to when given through a vein (intravenous) in participants with gMG.

Official TitlePhase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis

Inclusion Criteria:

• Diagnosis of myasthenia gravis (MG) with generalized muscle weakness meeting the clinical criteria for generalized myasthenia gravis (gMG) as defined by the Myasthenia Gravis Foundation of America (MGFA) Clinical Classification Class II a/b, III a/b, or IVa/b at screening
• Myasthenia Gravis - Activities of Daily Living (MG-ADL) score of greater than or equal to (>=) 6 at screening and baseline
• Has sufficient venous access to allow drug administration by infusion and blood sampling as per the protocol
• A woman of childbearing potential must have a negative highly sensitive serum (beta-human chorionic gonadotropin [beta-hCG]) at screening and a negative urine pregnancy test at Day 1 prior to administration of study intervention
• A male participant must agree not to donate sperm for the purpose of reproduction during the study and for a minimum 90 days after receiving the last administration of study intervention
• For the SC Substudy (Cohort 1 and Cohort 2): Has reasonable abdominal skin area for SC administration
• For the SC Substudy (Cohort 1 and Cohort 2): Participants must be willing to comply with maintaining their stable dose of corticosteroids and/or immunosuppressants for the initial 8 weeks of the SC substudy, that is, through the SC Week 8 visit

Exclusion Criteria:

• Has any confirmed or suspected clinical immunodeficiency syndrome not related to treatment of his/her gMG, or has a family history of congenital or hereditary immunodeficiency unless confirmed absent in the participant
• Has MGFA Class I disease or presence of MG crisis (MGFA Class V) at screening, history of MG crisis within 1 month of screening, or fixed weakness (and/or 'burnt out' MG)
• Has had a thymectomy within 12 months prior to screening, or thymectomy is planned during the study
• Has known allergies, hypersensitivity, or intolerance to nipocalimab or its excipients
• Has experienced myocardial infarction, unstable ischemic heart disease, or stroke within 12 weeks of screening
• For the SC Substudy (Cohort 1): Participants who have undergone a recent tapering of their concomitant MG medication in the OLE
• For the SC Substudy (Cohort 1): Participants deteriorating during the OLE in the month prior to SC Dose 1 of the SC substudy such that they meet the criteria for clinical deterioration
• For the SC Substudy (Cohort 2): History of an unprovoked pulmonary embolism within 1 year prior to screening or history of recurrent deep vein thrombosis (DVT)

United States Locations

Ohio Locations

Cleveland, Ohio, United States, 44145

Recruiting

Cleveland Clinic

Recruiting

Last updated information on 5-2-2026 from clinicaltrials.gov

ClinicalTrials.gov ID: NCT04202341

Sponsor: Alexion Pharmaceuticals, Inc.

Study Start (Actual): 2019-12-02

Phase Observational [Patient Registry]

Brief Summary

Long-term, multicenter, multinational, observational, registry of patients with gMG that is designed to collect data on clinical outcomes and safety in patients prescribed Alexion C5 inhibitor therapies (C5IT) such as eculizumab (Soliris®) and ravulizumab (Ultomiris®).

Detailed Description

At the time of enrollment in the Registry, participant records will be queried for retrospective information about the participants' medical history and gMG disease treatment history. Following enrollment, prospective data collection will be performed using data obtained as part of the routine clinical care and through patient-reported outcome methods in use. Data will be collected using an electronic data capture system. The duration of data collection for the Registry will be up to 5 years from the day of enrollment.

Official TitleLong-Term, Observational, Global Registry of Patients With Generalized Myasthenia Gravis Who Have Received Treatment With Complement C5 Inhibition Therapies (C5ITs)

United States Locations

Ohio Locations

Dayton, Ohio, United States, 45459

Recruiting

Clinical Trial Site

Click here to open study

Recruiting

Last updated information on 5-2-2026 from clinicaltrials.gov

ClinicalTrials.gov ID: NCT06456580

Sponsor: Vor Biopharma

Study Start (Actual): 2024-07-17

Phase 3

Brief Summary

The purpose of this study is to evaluate the efficacy and safety of telitacicept in the treatment of generalized myasthenia gravis.

Detailed Description

Myasthenia gravis (MG) is an autoimmune disease in which autoantibodies disrupt the postsynaptic membrane, impairing nerve-to-muscle signal transmission. The predominant manifestation is muscle weakness, which typically worsens with repeated muscle exertion, such that function is usually the best in the morning with more pronounced weakness at the end of the day. A major challenge in MG is the lack of therapies that effectively treat the disease.

Telitacicept is a fully human TACI-Fc fusion protein that targets B-lymphocyte stimulator (BLyS) and A proliferating-inducing ligand (APRIL), neutralizing their interactions with receptors on B cells. The blockage of BLyS and APRIL interaction with their respective cell membrane receptors (transmembrane activator and CAML interactor [TACI], B-cell maturation antigen, and BLyS receptors) by telitacicept can inhibit B-cell proliferation and maturation. This suppression at the proximal portion of the immune response could alleviate autoimmune symptoms.

This is a randomized, double-blind, placebo-controlled Phase 3 study with an open-label extension (OLE) to evaluate the efficacy and safety of telitacicept in a global patient population with gMG. The total duration of the study is variable but will include an approximately 4-week screening period, a 24-week double-blind treatment period (Week 0 through Week 24), a 48-week OLE (Week 24 through Week 72), followed by an extended OLE period (E-OLE), and an 8-week end-of-study follow-up period. The E-OLE is variable duration, defined as the period after the 48-week OLE period until telitacicept is approved for MG in the country or the further development in the indication is concluded.

Official TitleA Phase 3, Randomized, Double-Blind, Placebo-Controlled Study With an Open-label Extension Period to Evaluate the Efficacy and Safety of Telitacicept in Patients With Generalized Myasthenia Gravis

Key Inclusion Criteria:

1. Male or female patient aged ≥18 years at screening.
2. Patients have prior confirmed diagnosis of gMG with generalized muscle weakness (typical pattern of weakness meeting the clinical criteria for diagnosis of MG as defined by the Myasthenia Gravis Foundation of America (MGFA) clinical classification II-IV.
3. Patients have positive antibodies against AChR or MuSK at screening.
4. MG-ADL score ≥6 points at screening and baseline with ocular-related score <50% of the total score.
5. QMG score ≥8 points, and ≥ 4 items score at least 2 points at screening and baseline.

Key Exclusion Criteria:

1. Patients have been diagnosed with any other autoimmune disease which can potentially pose a safety or efficacy confounding risk.
2. Patients having acute or chronic infection.
3. Patients having thymoma within 5 years or received thymectomy ≤6 months prior to screening. Patients with thymoma diagnosed 3-5 years prior to screening may be eligible if thymoma was at a localized stage and definitively treated with complete surgical resection.
4. Patients having current or history of primary immunodeficiency.
5. Patients having history of malignancy within the last 5 years.
6. Patient having prior or continuing diagnosis of serious cardiovascular, liver, kidney, respiratory system, endocrine or hematologic disease.

United States Locations

Ohio Locations

Cincinnati, Ohio, United States, 45219

Recruiting

University of Cincinnati Health Physicians - Clifton

Click here to open study

Recruiting

Last updated information on 5-2-2026 from clinicaltrials.gov

ClinicalTrials.gov ID: NCT06106672

Sponsor: COUR Pharmaceutical Development Company, Inc.

Study Start (Actual): 2024-05-30

Phase 1 & 2

Brief Summary

Phase 1b/2a First-in-Human (FIH) clinical trial to assess the safety, tolerability, pharmacodynamics (PD), and efficacy of multiple ascending doses of CNP-106.

Detailed Description

This is a Phase 1b/2a First-in-Human (FIH) clinical trial to assess the safety, tolerability, pharmacodynamics (PD), and efficacy of multiple ascending doses of CNP-106. The clinical study lasts 222-days (up to 42 days for Screening, 180 Study Days). Subjects ages 18-75 with generalized myasthenia gravis (MG) will be screened up to 42 days prior to enrollment into the clinical study.

Official TitleA Phase 1b/2a Double Blind, Placebo Controlled Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Efficacy of CNP-106 in Subjects Ages 18-75 With Generalized Myasthenia Gravis

Inclusion Criteria:

1. Subjects who are willing and able to provide Institutional Review Board (IRB) approved written informed consent and privacy language as per national regulations.
2. Men and non-pregnant women, ages 18-75 years inclusive.

3. Female subjects of childbearing potential must agree not to become pregnant during the clinical study, have a negative pregnancy test at the Screening Visit, and agree to one of the following:

   • Use two highly effective forms of birth control starting at initial screening and continuing throughout the study duration.
   • Practice abstinence starting at initial screening and continuing throughout the study duration.
4. Subjects with a Myasthenia Gravis Foundation of America Clinical Classification Class III-IV (Cohort 1). Upon successful DMC review and approval of preliminary safety data obtained from Cohort 1 through Day 15, Cohort 2 will enroll subjects with MGFA Clinical Classification Class II-IV.
5. Subjects positive for anti-AChR antibodies by radioimmunoassay (RIA) (Mayo Clinic).

6, Subjects with MG-ADL Score ≥ 6 at Screening and Baseline Visit with ≥ 50% of the score derived from non-ocular symptoms.

7. Subjects with QMG Score ≥ 11 at Screening and Baseline Visit. 8. For subjects on any medication used to treat the symptoms of MG (ex. Corticosteroids, pyridostigmine), subjects must be on a stable dose for a minimum of 90 days prior to enrollment and must agree not to increase their dose through clinical study duration unless reviewed and approved by the medical monitor and the site investigator.

9. Female subjects who agree to not breastfeed starting at initial screening and throughout the study duration.

10. Female subjects who agree to not donate ova starting at initial screening and throughout the study duration.

11. Male subjects with a spouse or partner of childbearing potential, who themselves and their spouse or partner agree to practice an effective form of birth control as discussed with the study doctor or study staff starting at Screening and throughout the study duration.

Exclusion Criteria:

1. Subjects with a Myasthenia Gravis Foundation of America Clinical Classification Class I or V.
2. Subjects with a history of cerebrovascular accident in the past 12 months.
3. Subjects with MG-ADL Score < 6 at Screen or Subjects with MG-ADL Score ≥ 6 at Screen with ˂ 50% of the score derived from non-ocular symptoms.
4. Subjects with QMG Score < 11 at Screen.

5. Subjects who have used the following medications:

   • Tacrolimus within 6 months prior to the first dosing;
   • Methotrexate within 5 half-lives or 90 days after last dose (whichever is longer);
   • Anti-FcRn inhibitors (ex. Efgartigimod) within 5 half-lives or 90 days after last dose (whichever is longer);
   • C5 complement inhibitor (ex. Eculizumab) within 5 half-lives or 90 days after last dose (whichever is longer);
   • Anti-CD20 (ex. Rituximab) within 5 half-lives for 90 days after last dose (whichever is longer);
   • Inclusion of subjects on other immunomodulatory drugs will be at the discretion of the medical monitor and study site investigator.
6. Subjects who have used immunoglobulins given SC or IV (SCIg or IVIg) or plasmapheresis/plasma exchange (PE) within 4 weeks before Screening.
7. Subjects who have had thymectomy or any other thymic surgery performed within 12 months prior to Screening.
8. Subjects with untreated thymic malignancy, carcinoma, or thymoma.
9. Subjects with a history of tuberculosis or positive PPD skin test.
10. Subjects who have received administration of any live vaccine (other than intranasal Influenza) within 28 days or subunit vaccine within 14 days prior to Screening or are planning to receive any vaccination throughout the study duration.
11. Subjects who have received any COVID-19 vaccine within 14 days prior to Screening. Subjects who have received the first dose of any COVID-19 vaccine may not screen for the study until 14 days following their last dose of the vaccine if applicable.
12. Subjects with laboratory test results at Screening or prior to study dosing that are outside the normal limits and considered by the investigator to be clinically significant. Note: Clinically significant laboratory test results at screening that are related to the condition (MG) are acceptable as long as all inclusion and no other exclusion criteria are met.
13. Subjects with positive test results for hepatitis B surface antigen (HbsAg), hepatitis C virus (HCV) antibody, or human immunodeficiency virus (HIV) antigen/antibody as determined at Screening.
14. Subjects with a history of or currently active immune disorders other than MG (including autoimmune disease) unless the condition, after discussion with the medical monitor and study site investigator, has been deemed to be acceptable for the subject's participation in this clinical study.
15. Subjects with a history of or current active diseases other than myasthenia gravis requiring immunosuppressive drugs (including azathioprine, prednisone, prednisolone, budesonide, cyclosporine, tacrolimus, methotrexate, or mycophenolate mofetil) unless the condition, after discussion with the medical monitor and site investigator, has been deemed to be acceptable for the subject's participation in this clinical study.
16. Subjects with a clinical history of significant cardiovascular disease as determined by the Investigator.
17. Subjects with a complication or medical history of malignancy within the past 5 years which, in the investigator's opinion, makes the subject unsuitable for study participation.
18. Subjects with a history of mast cell activation disease.
19. Subjects who, in the investigator's opinion, will be unable to adhere to study procedures.
20. Subjects who have received an investigational therapy other than CNP-106 within 28 days or 5 half-lives, whichever is longer, prior to Screening.
21. Subjects with any known active condition which, in the investigator's opinion, makes the subject unsuitable for study participation.
22. Known sensitivity to any components of CNP-106 (PLGA, sucrose, mannitol or sodium citrate).

United States Locations

Michigan Locations

Dearborn, Michigan, United States, 48126

Recruiting

Insight Research Institute, Dearborn

Ohio Locations

Colombus, Ohio, United States, 43221

Not yet recruiting

Ohio State University Wexner Medical Center

Recruiting

Last updated information on 5-2-2026 from clinicaltrials.gov

ClinicalTrials.gov ID: NCT06909214

Sponsor: argenx

Study Start (Actual): 2025-04-17

Phase 4

Brief Summary

The main purpose of this study is to measure how well adults with new-onset gMG (which means they've had generalized disease signs and/or symptoms for less than 1 year) respond to treatment with efgartigimod PH20 SC. The study consists of a treatment period of 51 weeks. The study duration for each participant will be approximately 58 weeks.

Official TitleA Phase 4, Open-Label, Prospective, Single-Group, Multicenter Study to Evaluate the Clinical Outcomes of Efgartigimod PH20 SC in Adult Participants With New-Onset Generalized Myasthenia Gravis

Inclusion Criteria:

• Is at least 18 years when signing the ICF
• Has been diagnosed with gMG of MGFA class II, III, or IV
• Is seropositive for AChR-Ab
• Is treatment-naive for gMG or has been administered AChEI for the treatment of gMG
• Had onset of generalized MG signs and/or symptoms within 12 months before screening; candidates who also had onset of ocular MG signs and/or symptoms within 24 months before screening may be enrolled in the study
• Has an MG-ADL score ≥5

Exclusion Criteria:

• gMG diagnosis of MGFA class I or V
• Underwent a thymectomy prior to screening, except thymectomy for treatment of nonmalignant thymoma prior to the gMG diagnosis
• Prior or current use of any of any systemic corticosteroid therapy or nonsteroidal immunosuppressive therapy for the treatment of gMG

United States Locations

Ohio Locations

Cleveland, Ohio, United States, 44195

Recruiting

Cleveland Clinic

Click here to open study