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RECRUITING

NCT06463587

PHASE3

Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis (MyClad)

Sponsor: Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany

View on ClinicalTrials.gov

Summary

The purpose of this clinical study is to determine the efficacy and safety of a new oral cladribine formulation in participants with Generalized Myasthenia Gravis (gMG) in comparison to placebo. It will also investigate the sustained efficacy, the need for retreatment, and the long-term safety of oral cladribine in gMG. An additional component is included to characterize the Pharmacokinetics (PK) of the new cladribine formulation in gMG participants. 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. Furthermore, in trial interviews will be conducted as a sub-study to MyClad with a sub-set of participants to gain an in depth understanding of the participant cladribine treatment and study experience.

Official title: 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)

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

264

Start Date

2024-06-25

Completion Date

2030-11-12

Last Updated

2026-10-21

Healthy Volunteers

Conditions

Generalized Myasthenia Gravis

Interventions

OTHER

Placebo

Participants will receive placebo matched to cladribine in two courses separated by 4 weeks.

DRUG

Cladribine Low Dose

Participants will receive cladribine low dose in two courses separated by 4 weeks.

DRUG

Cladribine High Dose

Participants will receive cladribine high dose in two courses separated by 4 weeks.

Inclusion Criteria: * Adults of ≥ 18 years of age at the time of signing the informed consent. * 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 i.e. not positive for anti-AChR and anti-MuSK antibodies and participants who are positive for anti-low-density lipoprotein receptor-related protein 4 antibodies (anti-LRP4) * Has a Screening and Baseline MG-ADL score more than or equal to (\>=) 6 with \>= 50 percentage (%) of the total score due to non-ocular symptoms. Screening and Baseline MG-ADL scores must be stable. The difference between the Screening and Baseline scores should not be more than 2 and there should be no reported MG exacerbation during the Screening period * If treated with oral corticosteroids: should be on a stable daily dose for at least 3 months prior to and during screening. In such case, the daily dose of oral steroids should not exceed 20 milligrams(mg)/day for prednisone/ prednisolone, 16 mg/day for methylprednisolone, 3 mg/day for dexamethasone, or 80 mg for hydrocortisone or equivalent doses for other corticosteroids. * If treated with acetylcholinesterase inhibitor should be on a stable daily dose (pyridostigmine dose ≤ 480 mg/day or neostigmine ≤ 300 mg/day) for at least 3 months prior to and during screening * Have a body weight \>= 40 kilograms * Other protocol defined inclusion criteria could apply Exclusion Criteria: * Immunologic disorder other than MG or any other condition requiring chronic oral, intravenous, intramuscular, or intraarticular corticosteroid therapy. Well-controlled thyroid disease, as per the Treating Investigator or the participants regular treating physician recorded in the source documents, is not exclusionary * Molecularly characterized or suspected congenital myasthenic syndrome, Lambert-Eaton myasthenic syndrome, inherited myopathy, muscular dystrophy, acquired myopathy or any other neurologic or systematic disease that mimics MG muscular weakness * Active, clinically significant viral, bacterial, or fungal infection, including brain MRI or chest X-ray findings consistent with signs of infection such as PML or TB, or any major episode of infection requiring hospitalization or treatment with parenteral anti-infectives within 8 weeks prior or during Screening, or completion of oral anti-infectives within 8 weeks prior or during Screening. Vaginal candidiasis, onychomycosis, and genital or oral herpes simplex virus considered by the Investigator to be sufficiently controlled would not be exclusionary * Has a history of or current diagnosis of active tuberculosis (TB) or is currently undergoing treatment for latent TB infection or has an untreated latent TB infection as determined by documented results within 3 months of the Screening Visit of a positive TB skin test . * Active malignancy, or history of cancer or signs of malignancy in any Screening assessment * Treatment with nonsteroidal immunosuppressants, used in gMG, such as azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, cyclophosphamide, tacrolimus within 4 weeks prior to randomization * Treatment with FcRn or complement inhibitors (such as eculizumab, rozanolixizumab efgartigimod, ravulizumab, zilucoplan or nipocalimab) within 8 weeks prior to randomization * History of thymectomy within 6 months prior to Screening. * History of generalized seizures (except for history of febrile seizures during the participant's childhood). * Negative or indeterminate for Varicella Zoster Virus antibodies at screening * History of myasthenic crisis in the last 12 months prior to and during screening * History of recurrent infections (that is 3 or more infections per year documented in available source data) within the last 2 years * Discontinuation of treatment with any non-steroidal immunosuppressants used in gMG, such as azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, cyclophosphamide, tacrolimus within the last 6 months prior to Screening * If treated with non-steroidal immunosuppressants for gMG, the dose at Screening higher than 50 mg/day for azathioprine, 500 mg/day for mycophenolate mofetil, 1 mg/day for tacrolimus, 50 mg/day for cyclosporine, 25 mg/day for cyclophosphamide, or 7.5 mg/week for methotrexate * Participation in clinical study of any investigational drug within 6 months, or 5 half-lives of the investigational drug used in the previous clinical study prior to randomization, whichever is longer. However, participants with any prior exposure to cladribine may not enter the study regardless of timing of exposure * Other protocol defined exclusion criteria could apply

Locations (154)

Arizona Neuroscience Research, LLC

Phoenix, Arizona, United States

Advanced Neurosciences Research LLC

Longmont, Colorado, United States

University of Connecticut Health Center - Department of Medicine

Farmington, Connecticut, United States

The George Washington University Medical Faculty Associates Foggy Bottom South Pavilion

Washington D.C., District of Columbia, United States

Neurology of Central Florida Research Center, LLC

Altamonte Springs, Florida, United States

SFM Clinical Research, LLC

Boca Raton, Florida, United States

University of Florida Health Science Center - 300120311

Jacksonville, Florida, United States

Neurology Associates, P. A.

Maitland, Florida, United States

University of South Florida - PARENT - PARENT

Tampa, Florida, United States

University of Kansas Medical Center Research Institute, Inc. - 3901 Rainbow (MAIN)

Kansas City, Kansas, United States

UNC Hospitals - PARENT

Chapel Hill, North Carolina, United States

Atrium Health Wake Forest Baptist

Winston-Salem, North Carolina, United States

Miami Valley Hospital South

Centerville, Ohio, United States

University of Cincinnati

Cincinnati, Ohio, United States

Clinical Trials of South Carolina - Charleston

Charleston, South Carolina, United States

Erlanger Health, Inc. - 1123255

Chattanooga, Tennessee, United States

Baylor Scott & White Research Institute - Baylor Scott & White Research Institute

Dallas, Texas, United States

ANESC Research

El Paso, Texas, United States

Baylor Scott & White Neurology - McKinney

McKinney, Texas, United States

The University of Vermont Medical Center

Burlington, Vermont, United States

Integrated Neurology Services - Dr. Simon Fishman's Office

Falls Church, Virginia, United States

BCN Research

Greenfield, Wisconsin, United States

Expertia S.A- Mautalen Salud e Investigación

Ciudad Autonoma Buenos Aires, Argentina

Instituto de Investigaciones Neurologicas Raul Carrea, FLENI - de Investigaciones Clinicas del área de Cardiología y enfermedades metabólicas asociadas

Ciudad Autonoma Buenos Aires, Argentina

Instituto de Investigaciones Metabolicas (IDIM)

Ciudad Autonoma Buenos Aires, Argentina

Hospital Cordoba

Córdoba, Argentina

Fundacion Rosarina de Neurorehabilitacion

Rosario, Argentina

INECO Neurociencias Oroño

Rosario, Argentina

CER San Juan Centro Polivalente de Asistencia e Inv. Clinica

San Juan, Argentina

Centro de Investigaciones Medicas Tucuman

San Miguel de Tucumán, Argentina

Brain and Mind Research Institute

Camperdown, New South Wales, Australia

Sunshine Coast University Hospital

Birtinya, Queensland, Australia

Princess Alexandra Hospital - PARENT

South Brisbane, Queensland, Australia

John Hunter Hospital - PARENT

City of Burnside, South Australia, Australia

Box Hill Hospital - PARENT

Box Hill, Victoria, Australia

Gold Coast University Hospital - PARENT

Southport, Australia

UZ Leuven

Leuven, Belgium

C. H. R. de la Citadelle - Account 1

Liège, Belgium

Medical Center Hera - branch Montana

Montana, Bulgaria

UMHAT 'Dr. Georgi Stranski', EAD

Pleven, Bulgaria

UMHAT "Sv. Georgi", EAD

Plovdiv, Bulgaria

MHATNP "Sv.Naum", EAD - Third Neurology Clinic

Sofia, Bulgaria

UMHATEM 'N.I. Pirogov', EAD

Sofia, Bulgaria

Burnaby Hospital - Fraser Health Multiple Sclerosis Clinic

Burnaby, British Columbia, Canada

The First Affiliated Hospital of Bengbu Medical College

Anhui Sheng, Anhui, China

Xuanwu Hospital Capital Medical University

Beijing, Beijing Municipality, China

Peking Union Medical College Hospital

Beijing, Beijing Municipality, China

The First Affiliated Hospital of Guangzhou University of Chinese Medicine - 300164595

Guangzhou, Guangdong, China

Affiliated Hospital of Zunyi Medical University

Zunyi, Guizhou, China

Shijiazhuang People's Hospital

Shijiazhuang, Hebei, China

Henan Provincial People's Hospital

Zhengzhou, Henan, China

Xiangya Hospital, Central South University

Changsha, Hu'nan, China

Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology

Wuhan, Hubei, China

The First Hospital of Jilin University - The First Hospital of Jilin University

Changchun, Jilin, China

China-Japan Union Hospital of Jilin University - 300173736

Changchun, Jilin, China

Shengjing Hospital of China Medical University

Shenyang, Liaoning, China

Huashan Hospital, Fudan University - Neurology

Shanghai, Shanghai Municipality, China

Xi 'an International Medical Center

Xi’an, Shanxi, China

West China Hospital, Sichuan University

Chengdu, Sichuan, China

CHU Nice - Hôpital Pasteur - Cardiology

Nice, Alpes Maritimes, France

CHU Strasbourg - Hopital Hautepierre - Service Anesthesie-Reanimation&Medecine Peri-Opera

Strasbourg, Bas Rhin, France

CHU de Grenoble - Hôpital Albert Michallon - Centre de Références des Maladies Neuromusculaires

La Tronche, Isere, France

Fondation Ophtalmologique Adolphe de Rothschild - Service de neurologie

Paris, Paris, France

CHU Clermont Ferrand - Hopital Gabriel Montpied - service de neurologie B

Clermont-Ferrand, Puy De Dome, France

Hopital Neurologique Pierre Wertheimer - Pathologies neuro-musculaires. Electromyographie

Bron, Rhone, France

Hôpital Henri Mondor - Service de Neurologie

Créteil, Val De Marne, France

Groupe Hospitalier Pitie-Salpetriere - Centre Référent Maladies Rares SLA - Neurologie

Paris, France

Ltd. Pineo Medical Ecosystem

Tbilisi, Georgia

New Hospitals LLC

Tbilisi, Georgia

Aversi Clinic Ltd

Tbilisi, Georgia

Universitaetsklinikum Ulm - Klinik fuer Neurologie

Ulm, Baden-Wurttemberg, Germany

Universitaetsmedizin Goettingen - Abteilung fuer Nephrologie und Rheumatologie

Göttingen, Lower Saxony, Germany

Medizinische Hochschule Hannover - Klinik fur Neurologie mit Klinischer Neurophysiol

Hanover, Lower Saxony, Germany

St. Joseph Hospital - St. Joseph Hospital Haan

Bochum, North Rhine-Westphalia, Germany

Universitaetsklinikum Duesseldorf AoeR - Klinik fuer Neurologie

Düsseldorf, North Rhine-Westphalia, Germany

Universitaetsklinikum Carl Gustav Carus TU Dresden - Medizinische Klinik I

Dresden, Saxony, Germany

Universitaetsklinikum Leipzig AoeR - Klinik und Poliklinik fuer Neurologie

Leipzig, Saxony, Germany

Universitaetsklinikum Schleswig-Holstein - Campus Luebeck - Neurologie

Lübeck, Schleswig-Holstein, Germany

Charité Universitaetsmedizin Berlin - Campus Charité Mitte - Klinik fuer Neurologie

Berlin, Germany

University General Hospital of Larissa - Rheumatology Clinic

Larissa, Greece

University Hospital of Patra

Pátrai, Greece

AHEPA General Hospital of Thessaloniki

Thessaloniki, Greece

Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont - Neurologiai Klinika

Szeged, Hungary

Nizam's Institute of Medical Sciences

Hyderabad, Andhra Pradesh, India

Yashoda Super Speciality Hospital - 300158817

Hyderabad, Andhra Pradesh, India

Bangalore Medical College and Victoria Hospital - 300148268

Bangalore, Karnataka, India

St John's Medical College Hospital

Bangalore, Karnataka, India

Mallikatta Neurocentre

Mangalore, Karnataka, India

Chopda Medicare & Research Centre - 300188248

Nashik, Maharashtra, India

Deenanath Mangeshkar Hospital and Research Centre - Department of Neurology

Pune, Maharashtra, India

Azienda Socio Sanitaria Territoriale Papa Giovanni

Bergamo, Italy

IRCCS Istituto delle Scienze Neurologiche di Bologna - Unità Operativa Complessa Clinica Neurologica

Bologna, Italy

IRCCS Ospedale Policlinico San Martino - Neuroradiologia

Genova, Italy

Fondazione IRCCS Istituto Neurologico Carlo Besta

Milan, Italy

Fondazione Istituto Neurologico Casimiro Mondino - Neurology

Pavia, Italy

Azienda Ospedaliera Universitaria Policlinico Tor Vergata - U.O.C. Neurologia

Rome, Italy

Policlinico Universitario Agostino Gemelli - UOC Neurologia

Rome, Italy

Ospedale ca' Foncello - Neurology

Treviso, Italy

Chiba University Hospital - Dept of Neurology

Chiba, Chiba, Japan

IUHW Narita Hospital - Dept of Neurology

Narita-shi, Chiba, Japan

And 54 more locations on ClinicalTrials.gov

Clinical Research Directory

Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis (MyClad)

Summary

Key Details

Conditions

Interventions

Eligibility Criteria

Locations (154)