Clinical Trials

 University of Kansas Health Center   

 NCT05556096 

254 participants  

Abby Davis adavis54@kumc.edu 

A Phase 3, Randomized, Double-blind, Placebo-controlled, Parallel, Multicenter Study to Evaluate the Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis 

Participants will receive a weight-based initial (loading) dose of ALXN1720 on Day 1, followed by weight-based maintenance treatment with ALXN1720 on Day 8 and once every week (Q1W) thereafter for a total of 26 weeks. Following this randomized controlled treatment (RCT) period, all participants will receive ALXN1720 in an open-label extension (OLE) period of 105 weeks. 

Participants will receive placebo during the 26-week RCT period, after which they will enter the OLE period of the study and receive ALXN1720 

-Age 18 or older

-Diagnosis of MG with generalized muscle weakness meeting the clinical criteria defined by Myasthenia Gravis Foundation of America (MGFA) Class II, III or IV

-Positive serological test for autoantibodies against AChR

 NCT06799247 

100 participants 

Cartesian Clinical Trials

617-231-8102

trials@cartesiantx.com

The AURORA Study is evaluating the safety, tolerability, and efficacy of an investigational mRNA CAR T-cell therapy known as Descartes-08 in adults with acetylcholine receptor autoantibody -positive generalized myasthenia gravis. Part 1 of the study will last around 6 months. For eligible participants, Part 2 will last around 8 months. 

Group will undergo leukapheresis and receive manufactured Decartes-08 

-Aged 18 years old and older

- Generalized myasthenia gravis (gMG), MGFA clinical classification grades 2-4 at the time of Sscreening.

-MG ADL total score ≥ 6.

-Concomitant immunosuppressive drugs must be deemed necessary by the investigator. 

-Corticosteroids, the daily dose should not exceed 40 mg/day of prednisone equivalent.

-Anti-nAChR titer or anti-AChR cluster antibody positive

-Willing to return for all study visits.

-Women of childbearing potential must agree to use birth control 

BioSensics 

 NCT07224386 

50 participants 

Amanda Guidon, MD 

617-726-3642  

aguidon@mgb.org 

The study includes five clinical visits, complemented by biweekly digital assessments and continuous monitoring using wearable sensors. The primary outcomes are changes in physical activity and posture, measured using the PAMSysTM wearable sensors. Secondary outcomes will assess changes in Myasthenia Gravis–related patient-reported outcomes, as well as digital speech and video-based assessments. 

Biweekly digital assessments and continuous monitoring using wearable sensors

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

-Diagnosed gMG 

-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

 University of Kansas Health Center   

NCT06359041 

12 participants  

Andrew Heim aheim2@kumc.edu   

RESET-MG: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Participants with Generalized Myasthenia Gravis 

AChR Antibody-Positive Cohort 

AChR Antibody-Negative Cohort

-Age ≥18 and ≤70 years of age

-Diagnosis of MG with generalized muscle weakness meeting criteria as defined by the MGFA class II, III , IVa, and IVb.

-Diagnosis of seropositive (autoantibodies AChR, MuSK and/or LRP4) or seronegative MG

NCT04146051 

30 participants 

Ali Russo aciersdorff@kumc.edu 

Autologous T-Cells Expressing A Chimeric Antigen Receptor Directed To B-Cell Maturation Antigen (BCMA) In Patients With Generalized Myasthenia Gravis (MG) 

In Part 3, the primary objective is to compare the effect of Descartes-08 versus placebo, as measured by the change in MG ADL score from baseline to Week 12.

Descartes will be administered as a tablet  

-Patient must be at least 18 years of age.

-Patient must have Generalized Myasthenia Gravis at the time of screening.

-Concomitant immunosuppressive drugs must be deemed necessary by the investigator.

-Seronegative Patients are included

University of Missouri

NCT06106672 

30 participants 

Neetha Gali
(573) 882-3065  

ngdcd@health.missouri.edu

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. 

 200 mL intravenous infusion on Day 1 and Day 8: CNP-106 

-Men and non-pregnant women, ages 18-75 years inclusive.

-Female subjects of childbearing potential must agree not to become pregnant during the clinical study, have a negative pregnancy test at the Screening Visit

-Subjects with a MGFA Clinical Classification Class III-IV (Cohort 1).

-Positive for anti-AChR antibodies

-MG-ADL Score ≥ 6 at Screening and Baseline Visit

-QMG Score ≥ 11 at Screening and Baseline Visit. 8. For subjects on any medication used to treat the symptoms of MG

University of Kansas Health Center   

NCT04226170 

24 participants 

Lilli Saavedra lsaavedra2@kumc.edu 

A Phase II, Study to Evaluate the Safety and Tolerability of Pyridostigmine When Given with Ondansetron to Subjects with anti-AchR positive Myasthenia Gravis 

Group A: Patients currently taking pyridostigmine and experiencing pyridostigmine-related gastrointestinal (GI) adverse events (AEs) within the past 7 days

Pyridostigmine + Ondansetron will be administered as an oral tablet 

-Aged 18 years old and older

-Diagnosed with anti-AchR antibody positive MG.

Subjects must complete a GI symptom diary on a daily basis.

-Must be clinically stable for past 3 months.

-Must be able to swallow liquid.

-Must be in otherwise good health as determined by their medical history, physical examination, vital signs, and laboratory tests. 

University of Kansas Health Center   

NCT05265273 

12 participants  

Katie Lillig  kjennens2@kumc.edu  

An Open-Label Uncontrolled Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Activity of Nipocalimab in Children Aged 2 to Less Than 18 Years with Generalized Myasthenia Gravis 

Nipocalimab

Participants aged 2 to less than [<] 18 years of age will receive nipocalimab once every two weeks for 24 weeks. After Week 24, all participants will have the option to enroll in long term extension (LTE).
Nipocalimab will be administered as an IV infusion 

-8 to <18 years

-Diagnosis of gMG 

-MGFA Clinical Classification Class IIa/b, IIIa/b, or IVa/b

-Has a positive serologic test for acetylcholine receptor (anti-AChR) or muscle-specific tyrosine kinase (anti-MuSK) antibodies

-Access to drug administration by infusion and blood sampling 

-Body weight and body mass index between 5th and 95th percentile for age and sex.

University of Kansas Health Center & University of Missouri 

NCT06064695 

16 participants  

University of Kansas- Abby Davis adavis54@kumc.edu

University of Missouri- Kristina Kelly, PT, DPT, MS, EdM, NCS, CPT, PES: kristina.kelly@health.missouri.edu. 

Investigators will examine the effects of whole-body electrical muscle stimulation (WB-EMS) exercise on neuromuscular junction transmission and fatigability in adults with gMG. Participants will undergo clinical and electrophysiologic testing before and after the WB-EMS Exercise intervention. The WB-EMS Exercise intervention will be delivered 2 times per week for 4 weeks.

All participants will receive the WB-EMS Exercise intervention 2 times per week for 4 weeks. Participants will only perform Level 1 exercise programs (simple movements) in the "Strength" Training Mode. 

-Age 18 or older

-Diagnosed with gMG 

-On stable MG therapy for at least 1 month

-Ability to stand for approximately 15 minutes continuously

-Score of 1 (Mild) or 2 (Moderate) on at least one side of the "arm outstretched" and "leg outstretched" items of the QMG, demonstrating fatigability

-At least some anti-gravity strength in major muscle groups as assessed by manual muscle testing

-Medical clearance

-Ability to conform to the requirements of the study 

NCT06193889 

66 participants 

 Kyverna Therapeutics, Inc.  Clinicaltrials@kyvernatx.com 

 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 

Phase 2: Dosing with KYV-101 CAR-T cells
Phase 3:  KYV-101 Treatment 

-Aged 18-75 years old.

-Presence of autoantibodies to AChR or MuSK at screening.

- Myasthenia Gravis Foundation of America (MGFA) Class II-IV 

- Failed treatment with 2 or more immunosuppressive/immunomodulatory therapies, or failed at least 1 immunosuppressive therapy and required chronic plasmapheresis, or IVIG (>4 times/year over ≥12 months) to control symptoms 

N/A

N/A

www.meandmgopen.com

ME&MGopen is a research study which aims to better understand and manage Myasthenia gravis. Participants will get free access to the ME&MGopen app, which allows participants to complete assessments and take part in the study remotely. Participants will be compensated up to $500 USD for taking part!

- Aged 18 + years old
-Diagnosis of gMG with positive serologic test for anti-AChR autoantibody at screening who has read the information sheet and signed the informed consent form
-Owns a personal smartphone which version is above 14 for IOS and 8 for Android included
-Able to use a smartphone
-Able to perform the ME&MG tests (based on investigator's judgment)
-Able to read language in which the mobile application is available (English, Spanish, German) and able to understand pictograms

University of Kansas Medical Center 

NCT04951622 

198 participants 

Ali Russo aciersdorff@kumc.edu 

Phase 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 

Double-blind Placebo-controlled Phase: Participants will receive nipocalimab intravenous (IV) infusions once every 2 weeks (q2w) up to 24 weeks during double-blind placebo-controlled phase.

Open-label Extension (OLE) Phase: Participants who complete the double-blind placebo-controlled phase will enter the OLE phase and continue to receive nipocalimab q2w IV infusion till study end.

Double-blind Placebo-controlled Phase: Participants will receive matching placebo of nipocalimab IV infusion q2w up to 24 weeks during double-blind placebo-controlled phase.

-18 Years and older 

-Diagnosis of  gMG as defined by the MGFA Clinical Classification Class II a/b, III a/b, or IVa/b

-MG-ADL score >= 6 

-Access to drug administration by infusion and blood sampling 

 NCT06463587 

264 participants 

888-275-7376
eMediUSA@emdserono.com 

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, 3-Arm, 3-Period Study to Assess the Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis(MyClad) 

This study is divided into 3 periods: the double-blind placebo control (DBPC) pivotal period, and 2 extensions, the blinded extension (BE) and the retreatment (RT) period. 

-18 Years and older 

-Diagnosis of Myasthenia Gravis with generalized muscle weakness, meeting clinical criteria for Myasthenia Gravis Foundation of America Class II to IVa classification.
-In participants positive for Acetylcholine receptor antibody (anti-AChR) or muscle-specific kinase antibody(anti-MuSK)

-In participants that are autoantibody seronegative and participants who are positive for anti-low-density lipoprotein receptor-related protein 4 antibodies (anti-LRP4)

-Has a Screening and Base

NCT05644561 

Estimated 12 participants

Alexion Pharmaceuticals, Inc.   clinicaltrials@alexion.com 

A Phase 3, Open-label, Single-arm, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants (6 to < 18 Years of Age) With Generalized Myasthenia Gravis (gMG) 

All participants will receive a weight-based loading dose of ravulizumab IV on Day 1, followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for participants weighing ≥ 20 kg, or once every 4 weeks (q4w) for participants weighing < 20 kg, for a total of 122 weeks of treatment.

-Diagnosis of gMG

-MGFA Clinical Classification of Class II to Class IV

-Participants receiving treatment must be on a stable dosing regimen of adequate duration prior to Screening and during the Screening Period.

-Eculizumab-experienced participants must have been enrolled and treated with eculizumab in Study ECU-MG-303 for at least 6 months (180 days) and must have been on a stable dose for ≥ 2 months (60 days) prior to Screening.

-Vaccinated against meningococcal infection

University of Kansas Health Center    

NCT06456580 

180 participants

Courtney Richardson 

c969r831@kumc.edu 

A 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. 

Subcutaneous injection

-Aged ≥18 years at screening.

-Diagnosis of gMG with generalized muscle weakness as defined by the MGFA clinical classification II-IV.

-Positive antibodies against AChR or MuSK at screening.

-MG-ADL score ≥6 points at screening and baseline with ocular-related score <50% of the total score.

-QMG score ≥11 points at screening and baseline.

 Univ of Missouri School of Medicine 

NCT06414954 

80 participants

Kristina Kelly 

Kristina.kelly@health.missouri.edu 

Neetha Reddy Gali 

ngdcd@health.missouri.edu 

Phase 2 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 

Oral drug, targeted skeletal muscle chloride-1 ion channel inhibitor

-Participants age 18-75 

-Diagnosed with MG, MGFA class II, III or IV. 

-Participants can continue taking their current MG treatment(s). 

-You will be required to do a physical exam, muscle strength assessments, nerve and muscle tests and short questionnaires and complete a daily diary.

 BioSensics  

 NCT07224386 

50 participants

Amanda Guidon, MD 

617-726-3642  

aguidon@mgb.org 

The study includes five clinical visits, complemented by biweekly digital assessments and continuous monitoring using wearable sensors. The primary outcomes are changes in physical activity and posture, measured using the PAMSysTM wearable sensors. Secondary outcomes will assess changes in Myasthenia Gravis–related patient-reported outcomes, as well as digital speech and video-based assessments.

Biweekly digital assessments and continuous monitoring using wearable sensors

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

-Diagnosed gMG 

-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

University of Kansas Health Center    

NCT06456580 

180 participants

Courtney Richardson 

c969r831@kumc.edu 

A 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. 

Subcutaneous injection

-Aged ≥18 years at screening.

-Diagnosis of gMG with generalized muscle weakness as defined by the MGFA clinical classification II-IV.

-Positive antibodies against AChR or MuSK at screening.

-MG-ADL score ≥6 points at screening and baseline with ocular-related score <50% of the total score.

-QMG score ≥11 points at screening and baseline.

 Univ of Missouri School of Medicine 

NCT06414954 

80 participants

Kristina Kelly 

Kristina.kelly@health.missouri.edu 

Neetha Reddy Gali 

ngdcd@health.missouri.edu 

Phase 2 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 

Oral drug, targeted skeletal muscle chloride-1 ion channel inhibitor

-Participants age 18-75 

-Diagnosed with MG, MGFA class II, III or IV. 

-Participants can continue taking their current MG treatment(s). 

-You will be required to do a physical exam, muscle strength assessments, nerve and muscle tests and short questionnaires and complete a daily diary.

University of Kansas Health Center

 NCT06558279 

124 participants  

Sabine Coppieters, MD
1-857-350-4834
clinicaltrials@argenx.com 

The purpose of this study is to evaluate the efficacy and safety of efgartigimod PH20 SC given by a pre-filled syringe in adult patients with ocular myasthenia gravis. The study consists of a part A (approximately 7 weeks) and a part B (up to 2 years). In part A, half of the participants will receive efgartigimod PH20 SC and the other half will receive placebo. In part B, all participants will receive efgartigimod PH20 SC. The participants will be in the study for about up to 2 years and 12 week

Participants receiving efgartigimod PH20 SC during part A and part B Combination. 

Participants receiving placebo PH20 SC in Part A and Efgartigimod PH20 SC in Part B 

- Age 18 or older

- Has been diagnosed with myasthenia gravis and supported by seropositivity for AChR-Ab; or abnormal neuromuscular transmission demonstrated by abnormal neurophysiology testing and history on positive edrophonium chloride testing or demonstrated improvement on MG therapy"

-Is MGFA Class I (any ocular muscle weakness)

-Has a screening and baseline MGII (PRO) ocular score of at least 6 with at least 2 ocular items with a score of at least 2

NCT05644561 

Estimated 12 participants

Alexion Pharmaceuticals, Inc.   clinicaltrials@alexion.com 

A Phase 3, Open-label, Single-arm, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants (6 to < 18 Years of Age) With Generalized Myasthenia Gravis (gMG) 

All participants will receive a weight-based loading dose of ravulizumab IV on Day 1, followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for participants weighing ≥ 20 kg, or once every 4 weeks (q4w) for participants weighing < 20 kg, for a total of 122 weeks of treatment.

-Diagnosis of gMG

-MGFA Clinical Classification of Class II to Class IV

-Participants receiving treatment must be on a stable dosing regimen of adequate duration prior to Screening and during the Screening Period.

-Eculizumab-experienced participants must have been enrolled and treated with eculizumab in Study ECU-MG-303 for at least 6 months (180 days) and must have been on a stable dose for ≥ 2 months (60 days) prior to Screening.

-Vaccinated against meningococcal infection

University of Missouri

NCT06414954 

84 participants 

Neetha Gali 

(573) 882-3065 

ngdcd@health.missouri.edu 

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. 

Group 1: NMD670 high dose tablets taken twice a day for 21 days

Group 2: NMD670 mild dose tablets taken twice a day for 21 days

Group 3: NMD670 low dose tablets taken twice a day for 21 days

Group 4: Placebo tablets taken twice a day for 21 days

-Aged 18 years old and older

-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

Pharmaceuticals, Inc. 

NCT06607627 

12 participants 

Alexion Pharmaceuticals, Inc. (Sponsor)
1-855-752-2356
clinicaltrials@alexion.com 

The primary objective of this study is to assess the pharmacokinetics and pharmacodynamics of gefurulimab in pediatric participants with AChR+ gMG for the duration of the study. 

On Day 1, participants will receive a weight based loading dose followed by a weekly maintenance dose for up to 122 weeks. 

- 12 to < 18 years of age at the time of signing the informed consent/assent.

-All participants must be vaccinated against meningococcal infection from serogroups A, B, C, W, and Y within 3 years and at least 2 weeks prior to the first dose of study intervention administration.

-Diagnosis of MG with generalized muscle weakness meeting the clinical criteria defined by Myasthenia Gravis Foundation of America (MGFA) Class II, III or IV

-Positive serological test for autoantibodies against AChR