Cabaletta Bio
PI: Dr. Dimachkie University of Kansas Health Center Clinicaltrials.gov identifier: NCT05451212
A Phase 1, Open-label, Safety and Dose-finding Study of Autologous Muscle-specific Tyrosine Kinase Chimeric Autoantibody Receptor T Cells (MuSK-CAART) in Subjects With Anti-MuSK-antibody-positive Myasthenia Gravis
Experimental: MuSK-CAART
Cohort A: Infusion of MuSK-CAART at various dose levels with or without pre-treatment (6 groups planned).
Cohort B: Infusion of MuSK-CAART at the dose regimen selected from Part A
Total: 24 patients
For more information contact:
Andrew Heim aheim2@kumc.edu
Clinical Trials
KATALYST MG
Dr. Pasnoor
University of Kansas Health Center & University of Missouri
ClinicalTrials.gov Identifier: NCT06064695
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Myasthenia Gravis
Experimental: N/A- Device
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.
18+ gMG via sero+ test or sero- test with clinical symptoms consistent with gMG
Total enrollment: 16 participants
For more information contact:
at University of Kansas- Abby Davis adavis54@kumc.edu
at University of Missouri- Kristina Kelly, PT, DPT, MS, EdM, NCS, CPT, PES: kristina.kelly@health.missouri.edu.
ME & MG Open
Eligibility Critiera
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
For more information click here.
Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)
Eligibility Critiera
Child, Adult, Older Adult
Diagnosis of gMG confirmed by a positive serologic test for anti-AChR antibodies (Abs) obtained at Screening and/or during Screening Period
Myasthenia Gravis Foundation of America (MGFA) Clinical Classification of Class II to Class IV at Screening
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.
All participants must be vaccinated against meningococcal infection
For more information click here.
ClinicalTrials.gov Identifier: NCT06607627
An Open-Label, Single-arm Study to Evaluate the Pharmacokinetics (PK), Pharmacodynamics (PD), Safety, and Efficacy of Gefurulimab in Pediatric Patients (6 to < 18 Years of Age) With Generalized Myasthenia Gravis (gMG) Who Express Acetylcholine Receptor Antibodies (AChR+).
For more information contact:
Alexion Pharmaceuticals, Inc. (Sponsor)
Phone Number:1-855-752-2356
Email: clinicaltrials@alexion.com
NMD Pharma
University of Missouri- Columbia
ClinicalTrials.gov Identifier: NCT06414954
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.
For more information contact:
Kristina Kelly, kristina.kelly@health.missouri.edu
The MGA maintains communication with representatives from clinical research studies in efforts to keep the public informed about potential trials MG patients can participate in. Take a look at some of the latest studies in our area below or visit the clinical trials database to discover all the research studies conducted around the world.
Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis (MyClad)
ClinicalTrials.gov ID NCT06463587
Sponsor Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany
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)
For more information
Phone Number:888-275-7376
Email: eMediUSA@emdserono.com
For more information click here
ALXN1720 – MG
PI: Dr. Dimachkie University of Kansas Health Center ClinicalTrials.gov Identifier: NTC
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
Experimental: ALXN1720
Participants will receive a weight-based maintenance treatment with ALXN1720 on Day 1, followed by weight-based maintenance treatment of ALXN1720 on Week 1 (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 96 weeks.
Placebo Comparator: Placebo
Participants will receive placebo during the 26-week RCT period, after which they will enter the OLE period of the study and receive ALXN1720.
Total Enrollment: 254 participants
For more information:
Nick Staudenmier nstaudenmier@kumc.edu
ARGX-113-2315
Argenx
University of Kansas Medical Center
ClinicalTrials.gov Identifier: NCT06558279
A Phase III, Study to evaluate the efficacy and safety of efgartigimod PH20 SC given by a pre-filled syringe in adult patients with ocular myasthenia gravis.
Experimental: Efgartigimod PH20 SC
Participants must be at least 18 years of age, has been diagnosed with myasthenia gravis and supported by seropositivity for AChR-Ab, presents ocular muscle weakness, and 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
Total enrollment: 124 participants
For more information contact:
Argenx (Sponsor)
Name: Sabine Coppieters, MD
Phone Number:1-857-350-4834
Email: clinicaltrials@argenx.com
COUR Pharmaceutical Development Company, Inc.
University of Missouri- Columbia
University of Kansas Medical Center
ClinicalTrials.gov Identifier: NCT06106672
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.
For more information contact:
University of Missouri - Kristina Kelly, kristina.kelly@health.missouri.edu
University of Kansas - Abby Davis adavis54@kumc.edu
ARGX-113-2308
Argenx
University of Kansas Medical Center
Saint Louis University
ClinicalTrials.gov Identifier: NCT06298552
A Phase III, Study to measure the efficacy and safety of efgartigimod intravenously (IV) compared to placebo in participants with Acetylcholine Receptor Binding Antibody (AChR-Ab) seronegative Generalized Myasthenia Gravis (gMG). Other objectives are to assess long-term efficacy, safety, and tolerability of efgartigimod.
Experimental: Efgartigimod IV
Participants must be at least 18 years of age, has no known weakness in infancy and later develop fatigable weakness after aged 16 years and diagnosed with acquired gMG by a 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 and either a history of positive edrophonium chloride test OR a demonstrated improvement in MG signs
Total enrollment: 110 participants
For more information contact:
Argenx (Sponsor)
Name: Sabine Coppieters, MD
Phone Number:1-857-350-4834
Email: clinicaltrials@argenx.com
MOM-M281-011
Dr. Farmakidis
University of Kansas Medical Center
ClinicalTrials.gov Identifier: NCT04951622
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
Experimental: Nipocalimab
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.
Nipocalimab will be administered as an IV infusion
Total enrollment: 198 participants
For more information contact:
Lillian Saavedra lsaavedra2@kumc.edu
DAS-MG
Dr. Dimachkie
University of Kansas Health Center
ClinicalTrials.gov Identifier: NCT04226170
A Phase II, Study to Evaluate the Safety and Tolerability of Pyridostigmine When Given with Ondansetron to Subjects with anti-AchR positive Myasthenia Gravis
Experimental: Pyridostigmine
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
Total enrollment: 24 participants
For more information contact:
Lilli Saavedra lsaavedra2@kumc.edu
ALXN2050 - MG
Eligibility Criteria
AGES ELIGIBLE FOR STUDY
18 Years and older (ADULT, OLDER_ADULT)
Key Inclusion Criteria:
Diagnosed with MG at least 3 months (90 days) prior to the date of the Screening Visit. Confirmation of MG must be made via the following:
Positive serologic test for anti AChR antibodies at the Screening Visit, and
Abnormal neuromuscular transmission demonstrated by single fiber electromyography or repetitive nerve stimulation, or
Positive response to an AChEI test (eg, edrophonium chloride test), or
Improvement of signs or symptoms related to MG during treatment with an oral AChEI, as determined by the treating physician
Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at the Screening Visit.
MG-ADL total score must be ≥ 5 (with at least 50% of the score attributed to non-ocular elements) at the Screening Visit and at randomization (Day 1).
Participants receiving treatment with protocol-specified immunosuppressive therapies, corticosteroids, or acetylcholinesterase inhibitors must have been receiving treatment and on a stable dose prior to the date of the Screening Visit, with no changes to the regimen expected during screening, the PEP, and/or the ETP.
Key Exclusion Criteria:
Estimated glomerular filtration rate ≤ 30 milliliters/minute/1.73 squared meters during Screening calculated by Chronic Kidney Disease Epidemiology Collaboration.
History of thymectomy, thymomectomy, or any other thymic surgery within 12 months prior to the Screening Visit.
Any untreated thymic malignancy, carcinoma, or thymoma. Participants with a history of treated thymic malignancy or carcinoma are eligible for enrollment if they meet pre-specified conditions outlined in the protocol.
Clinical features consistent with Clinical Deterioration at the time of the Screening Visit or at any time during the Screening Period prior to randomization (Day 1).
Use of the following within the time periods specified below:
Intravenous immunoglobulin G or subcutaneous immunoglobulin within the 4 weeks (28 days) prior to the Screening Visit.
Use of tacrolimus or cyclosporine within the 4 weeks (28 days) prior to the Screening Visit.
Recruiting for children
Argenx ClinicalTrials.gov Identifier: NCT05374590
Evaluating Long-term Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis
A Long-term, Single-Arm, Open-label, Multicenter, Follow-on Trial of ARGX-113-2006 to Evaluate Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis
Ages 2-18
Contact: Sabine Coppieters, MD 857-350-4834 ClinicalTrials@argenx.com
Recruiting in St. Louis, Missouri & Little Rock, Arkansas
Cabaletta Bio PI: Dr. Dimachkie ClinicalTrials.gov Identifier: NCT06359041
A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of Autologous CD19-specific Chimeric Antigen Receptor T Cells (CABA-201) in Participants With Generalized Myasthenia Gravis
Phase: 1/2
Open Label study:
Experimental: CABA-201
AChR Antibody-Positive Cohort AChR Antibody-Negative Cohort Biological: CABA-201
Single intravenous infusion of CABA-201 at a single dose level following preconditioning with fludarabine and cyclophosphamide
Enrollment Total: 12 participants
For more information contact:
Lillian Saavedra lsaavedra2@kumc.edu
Janssen
PI: Dr. Farmakidis
University of Kansas Health Center
ClinicalTrials.gov Identifier: NCT05265273
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
Experimental: 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
Total enrollment: 12 participants
For more information contact:
Samantha Colgan scolgan@kumc.edu