Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis (MyClad)
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)
2 other identifiers
interventional
264
22 countries
133
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started Jun 2024
Longer than P75 for phase_3
133 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 11, 2024
CompletedFirst Posted
Study publicly available on registry
June 18, 2024
CompletedStudy Start
First participant enrolled
June 25, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 19, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
August 19, 2030
April 22, 2026
April 1, 2026
3.9 years
June 11, 2024
April 21, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change from Baseline in Myasthenia Gravis - Activities of Daily Living (MG-ADL) Scale Score at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period
Baseline, Week 24
Secondary Outcomes (10)
Change from Baseline in Quantitative Myasthenia Gravis (QMG) Scale Score at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period
Baseline, Week 24
Percentage of MG-ADL Responders at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period
At Week 24
Change from Baseline in Myasthenia Gravis Composite (MGC) Scale Score at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period
Baseline, Week 24
Percentage of Quantitative Myasthenia Gravis (QMG) Scale Responders at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period
At Week 24
Time From Initial Cladribine Full Dose Treatment to First Retreatment or Rescue Treatment up to end of Study
Up to End of Study (Week 144)
- +5 more secondary outcomes
Study Arms (3)
Placebo
PLACEBO COMPARATORDBPC Period: Participants will be administered with Placebo, orally as 2 separate treatment courses starting on Day 1 and at the beginning of Week 5. BE Period: Participants initially randomized to placebo matched to cladribine in DBPC period will receive cladribine Low Dose or High Dose, orally as 2 separate treatment courses starting at the beginning of Week 25 and at the beginning of Week 29 and retreated if clinically justified with placebo matched to cladribine. RT Period: Participants requiring retreatment with cladribine Low Dose or High Dose or retreated with cladribine supplemental dose if clinically justified.
Cladribine Low Dose
EXPERIMENTALDBPC Period: Participants will be administered with cladribine Low Dose, orally as 2 separate treatment courses starting on Day 1 and at the beginning of Week 5. BE Period: Participants initially randomized to cladribine Low Dose in DBPC period will receive placebo matched to cladribine as 2 separate treatment courses starting at the beginning of Week 25 and at the beginning of Week 29 and retreated with cladribine supplemental dose if clinically justified. RT Period: Participants requiring retreatment with cladribine Low Dose regimen and/or supplemental dose will receive the selected dose of cladribine if clinically justified.
Cladribine High Dose
EXPERIMENTALDBPC Period: Participants will be administered cladribine High Dose, orally as 2 separate treatment courses starting on Day 1 and at the beginning of Week 5. BE Period: Participants initially randomized to cladribine High Dose in DBPC period will receive placebo matched to cladribine as 2 separate treatment courses starting at the beginning of Week 25 and at the beginning of Week 29 and retreated with cladribine supplemental dose if clinically justified. RT Period: Participants requiring retreatment with cladribine High Dose regimen and/or supplemental dose will receive the selected dose of cladribine if clinically justified.
Interventions
Participants will receive placebo matched to cladribine in two courses separated by 4 weeks.
Participants will receive cladribine low dose in two courses separated by 4 weeks.
Participants will receive cladribine high dose in two courses separated by 4 weeks.
Eligibility Criteria
You may qualify if:
- 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 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 or 16 mg/day for methylprednisolone
- If treated with acetylcholinesterase inhibitor should be on a stable daily dose (pyridostigmine dose ≤ 480 mg/day) for at least 3 months prior to and during screening
- Have a body weight \>= 40 kilograms
You may not qualify if:
- 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
- Has a history of or current diagnosis of active tuberculosis (TB)
- Active malignancy, or history of cancer
- Treatment with nonsteroidal immunosuppressants, used in gMG, such as azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus within 4 weeks prior to randomization
- Treatment with eculizumab, rozanolixizumab efgartigimod, ravulizumab, or zilucoplan within 8 weeks prior to randomization
- History of thymectomy within 6 months prior to Screening.
- History of generalized seizures (except for history of infantile febrile seizures)
- Negative 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) within the last 2 years
- Discontinuation of treatment with any non-steroidal immunosuppressants used in gMG, such as azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus within the last 6 months prior to Screening
- If treated with non-steroidal immunosuppressants for gMG, the dose at Screening should not exceed 50 mg/day for azathioprine, 500 mg/day for mycophenolate mofetil, 1 mg/day for tacrolimus, 50 mg/day for cyclosporine, 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (133)
Arizona Neuroscience Research, LLC
Phoenix, Arizona, 85032, United States
Advanced Neurosciences Research LLC
Longmont, Colorado, 80501, United States
The George Washington University Medical Faculty Associates Foggy Bottom South Pavilion
Washington D.C., District of Columbia, 20037, United States
Neurology of Central Florida Research Center, LLC
Altamonte Springs, Florida, 32714, United States
SFM Clinical Research, LLC
Boca Raton, Florida, 33487, United States
University of Florida Health Science Center - 300120311
Jacksonville, Florida, 32209, United States
Neurology Associates, P. A.
Maitland, Florida, 32751, United States
University of South Florida - PARENT - PARENT
Tampa, Florida, 33612, United States
University of Kansas Medical Center Research Institute, Inc. - 3901 Rainbow (MAIN)
Kansas City, Kansas, 66160, United States
UNC Hospitals - PARENT
Chapel Hill, North Carolina, 27599, United States
Atrium Health Wake Forest Baptist
Winston-Salem, North Carolina, 27157-1078, United States
Miami Valley Hospital South
Centerville, Ohio, 45459, United States
Clinical Trials of South Carolina - Charleston
Charleston, South Carolina, 29406, United States
Erlanger Health, Inc. - 1123255
Chattanooga, Tennessee, 37403, United States
Baylor Scott & White Research Institute - Baylor Scott & White Research Institute
Dallas, Texas, 75246, United States
ANESC Research
El Paso, Texas, 79912, United States
Baylor Scott & White Neurology - McKinney
McKinney, Texas, 75071, United States
The University of Vermont Medical Center
Burlington, Vermont, 05401, United States
Integrated Neurology Services - Dr. Simon Fishman's Office
Falls Church, Virginia, 22043, United States
Expertia S.A- Mautalen Salud e Investigación
Ciudad Autonoma Buenos Aires, 1427, Argentina
Instituto de Investigaciones Neurologicas Raul Carrea, FLENI - de Investigaciones Clinicas del área de Cardiología y enfermedades metabólicas asociadas
Ciudad Autonoma Buenos Aires, 1428, Argentina
Instituto de Investigaciones Metabolicas (IDIM)
Ciudad Autonoma Buenos Aires, C1012AAR, 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, 5400, Argentina
Centro de Investigaciones Medicas Tucuman
San Miguel de Tucumán, Argentina
Sunshine Coast University Hospital
Birtinya, Queensland, 4575, Australia
John Hunter Hospital - PARENT
City of Burnside, South Australia, SA 5072, Australia
Box Hill Hospital - PARENT
Box Hill, Victoria, 3128, Australia
Gold Coast University Hospital - PARENT
Southport, Australia
UZ Leuven
Leuven, 3000, Belgium
C. H. R. de la Citadelle - Account 1
Liège, 4000, Belgium
Medical Center Hera - branch Montana
Montana, 3400, Bulgaria
UMHAT 'Dr. Georgi Stranski', EAD
Pleven, 5800, Bulgaria
UMHAT "Sv. Georgi", EAD
Plovdiv, 4000, Bulgaria
MHATNP "Sv.Naum", EAD - Third Neurology Clinic
Sofia, 1113, Bulgaria
UMHATEM 'N.I. Pirogov', EAD
Sofia, 1606, Bulgaria
The First Affiliated Hospital of Bengbu Medical College
Anhui Sheng, Anhui, 233000, China
Xuanwu Hospital Capital Medical University
Beijing, Beijing Municipality, 100053, China
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730, China
The First Affiliated Hospital of Guangzhou University of Chinese Medicine - 300164595
Guangzhou, Guangdong, 510405, China
Affiliated Hospital of Zunyi Medical University
Zunyi, Guizhou, 563099, China
Shijiazhuang People's Hospital
Shijiazhuang, Hebei, 050000, China
Henan Provincial People's Hospital
Zhengzhou, Henan, 450003, China
Xiangya Hospital, Central South University
Changsha, Hu'nan, 410008, China
Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology
Wuhan, Hubei, 430030, China
The First Hospital of Jilin University - The First Hospital of Jilin University
Changchun, Jilin, 130021, China
China-Japan Union Hospital of Jilin University - 300173736
Changchun, Jilin, 130033, China
Shengjing Hospital of China Medical University
Shenyang, Liaoning, 110004, China
Huashan Hospital, Fudan University - Neurology
Shanghai, Shanghai Municipality, 200040, China
Xi 'an International Medical Center
Xi’an, Shanxi, 710038, China
West China Hospital, Sichuan University
Chengdu, Sichuan, 610041, China
CHU Nice - Hôpital Pasteur - Cardiology
Nice, Alpes Maritimes, 06002, France
CHU Strasbourg - Hopital Hautepierre - Service Anesthesie-Reanimation&Medecine Peri-Opera
Strasbourg, Bas Rhin, 67098, France
CHU de Grenoble - Hôpital Albert Michallon - Centre de Références des Maladies Neuromusculaires
La Tronche, Isere, 38700, France
Fondation Ophtalmologique Adolphe de Rothschild - Service de neurologie
Paris, Paris, 75940, France
CHU Clermont Ferrand - Hopital Gabriel Montpied - service de neurologie B
Clermont-Ferrand, Puy De Dome, 63003, France
Hopital Neurologique Pierre Wertheimer - Pathologies neuro-musculaires. Electromyographie
Bron, Rhone, 69677, France
Hôpital Henri Mondor - Service de Neurologie
Créteil, Val De Marne, 94010, France
Groupe Hospitalier Pitie-Salpetriere - Centre Référent Maladies Rares SLA - Neurologie
Paris, 75013, France
Ltd. Pineo Medical Ecosystem
Tbilisi, 0114, Georgia
New Hospitals LLC
Tbilisi, 0114, Georgia
Aversi Clinic Ltd
Tbilisi, 0160, Georgia
Universitaetsmedizin Goettingen - Abteilung fuer Nephrologie und Rheumatologie
Göttingen, Lower Saxony, 37075, Germany
Medizinische Hochschule Hannover - Klinik fur Neurologie mit Klinischer Neurophysiol
Hanover, Lower Saxony, 30625, Germany
St. Joseph Hospital - St. Joseph Hospital Haan
Bochum, North Rhine-Westphalia, 44791, Germany
Universitaetsklinikum Carl Gustav Carus TU Dresden - Medizinische Klinik I
Dresden, Saxony, 01307, Germany
Universitaetsklinikum Leipzig AoeR - Klinik und Poliklinik fuer Neurologie
Leipzig, Saxony, 04103, Germany
Universitaetsklinikum Schleswig-Holstein - Campus Luebeck - Neurologie
Lübeck, Schleswig-Holstein, 23562, Germany
Charité Universitaetsmedizin Berlin - Campus Charité Mitte - Klinik fuer Neurologie
Berlin, 10117, Germany
University General Hospital of Larissa - Rheumatology Clinic
Larissa, 41500, Greece
University Hospital of Patra
Pátrai, 26504, Greece
AHEPA General Hospital of Thessaloniki
Thessaloniki, 54636, Greece
Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont - Neurologiai Klinika
Szeged, 6725, Hungary
Nizam's Institute of Medical Sciences
Hyderabad, Andhra Pradesh, 500082, India
Yashoda Super Speciality Hospital - 300158817
Hyderabad, Andhra Pradesh, 500082, India
Bangalore Medical College and Victoria Hospital - 300148268
Bangalore, Karnataka, 560002, India
St John's Medical College Hospital
Bangalore, Karnataka, 560034, India
Mallikatta Neurocentre
Mangalore, Karnataka, 575001, India
Chopda Medicare & Research Centre - 300188248
Nashik, Maharashtra, 422005, India
Deenanath Mangeshkar Hospital and Research Centre - Department of Neurology
Pune, Maharashtra, 411030, India
Policlinico Universitario Agostino Gemelli - UOC Neurologia
Rome, Roma, 00168, Italy
Azienda Socio Sanitaria Territoriale Papa Giovanni
Bergamo, 24127, Italy
IRCCS Istituto delle Scienze Neurologiche di Bologna - Unità Operativa Complessa Clinica Neurologica
Bologna, 40139, Italy
IRCCS Ospedale Policlinico San Martino - Neuroradiologia
Genova, 16132, Italy
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, 20133, Italy
Fondazione Istituto Neurologico Casimiro Mondino - Neurology
Pavia, 27100, Italy
Azienda Ospedaliera Universitaria Policlinico Tor Vergata - U.O.C. Neurologia
Roma, 00133, Italy
Ospedale ca' Foncello - Neurology
Treviso, 31100, Italy
Chiba University Hospital - Dept of Neurology
Chiba, Chiba, 260-8677, Japan
IUHW Narita Hospital - Dept of Neurology
Narita-shi, Chiba, 286-8520, Japan
Fukuoka University Hospital - Dept of Neurology/Health Care
Fukuoka, Fukuoka, 814-0180, Japan
Southern Tohoku Medical Clinic - Dept of Neurology
Koriyama-shi, Fukushima, 963-8563, Japan
Hakodate Municipal Hospital - Dept of Neurology
Hakodate-shi, Hokkaido, 392006, Japan
General Hanamaki Hospital - Dept of Neurology
Hanamaki-shi, Iwate, 025-0082, Japan
Kagawa University Hospital - Dept of Neurology
Kita-gun, Kagawa-ken, 7610793, Japan
Nagasaki University Hospital - Dept of Neurology
Nagasaki, Nagasaki, 852-8501, Japan
Saitama Medical University Hospital - Dept of Neurology/Stroke Care Unit
Iruma-gun, Saitama, 350-0495, Japan
Toho University Ohashi Medical Center - Dept of Neurology
Meguro-ku, Tokyo-To, 153-8515, Japan
Tokyo Medical University Hospital - Dept of Neurology
Shinjuku-ku, Tokyo-To, 160-0023, Japan
Osaka University Hospital - Dept of Neurology/Stroke Care Unit
Suita-shi, Japan
Uniwersyteckie Centrum Kliniczne - Dept of Neurology
Gdansk, 80-214, Poland
SP ZOZ Szpital Uniwersytecki w Krakowie - Zespó
Krakow, 30-688, Poland
Krakowska Akademia Neurologii Sp z o.o. Centrum Neurologii Klinicznej
Krakow, 31-505, Poland
MTZ Clinical Research Powered By Pratia
Warsaw, 02-091, Poland
Wielospecjalistyczne Centrum Medyczne Ibismed - Dept of Neurology
Zabrze, Poland
Institutul Clinic Fundeni - parent
Bucharest, 022328, Romania
S.C Neurocity S.R.L
Bucharest, 040215, Romania
Brainaxy Clinic S.R.L.
Constanța, 900591, Romania
Spitalul Clinic Judetean de Urgenta Targu Mures - Sectia Clinica Neurologie I
Târgu Mureş, 540136, Romania
Kyungpook National University Chilgok Hospital
Daegu, 41404, South Korea
Chonnam National University Hospital
Gwangju, 61469, South Korea
Samsung Medical Center
Seoul, 06351, South Korea
Korea University Anam Hospital
Seoul, 136-705, South Korea
Asan Medical Center
Seoul, 138-736, South Korea
Seoul Metropolitan Government Seoul National University Boramae Medical Center
Seoul, 156707, South Korea
Keimyung University Dongsan Hospital
Seoul, South Korea
Hospital Universitari Germans Trias i Pujol - Servicio de Neurologia
Badalona, Barcelona, 08916, Spain
Hospital Universitario Reina Sofia - Neurology Dept
Córdoba, Córdoba, 14004, Spain
Hospital Universitario Central de Asturias - Servicio de Neurologia
Oviedo, Principality of Asturias, 33011, Spain
Hospital Universitari Vall d'Hebron - Neurology Dept
Barcelona, 08035, Spain
Hospital Universitari i Politecnic La Fe - Pediatry Department
Valencia, 46026, Spain
Sahlgrenska Sjukhuset
Göteborg, 41345, Sweden
Centralsjukhuset Karlstad
Karlstad, 65230, Sweden
Danderyd Sjukhus - 300147691
Stockholm, 18288, Sweden
Inselspital - Universitaetsspital Bern - Neuropsychologische Rehabilitation, Neurologie
Bern, 3010, Switzerland
Kaohsiung Chang Gung Memorial Hospital
Kaohsiung City, 83301, Taiwan
China Medical University Hospital
Taichung, 404327, Taiwan
Shin Kong Wu Ho-Su Memorial Hospital
Taipei, 111, Taiwan
Taipei Veterans General Hospital
Taipei, 11217, Taiwan
Nottingham University Hospitals NHS Trust
Nottingham, Nottinghamshire, NG7 2UH, United Kingdom
Royal Hallamshire Hospital - Dept of Neurology
Sheffield, South Yorkshire, S10 2JF, United Kingdom
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Medical Responsible
EMD Serono Research & Development Institute, Inc.
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 11, 2024
First Posted
June 18, 2024
Study Start
June 25, 2024
Primary Completion (Estimated)
May 19, 2028
Study Completion (Estimated)
August 19, 2030
Last Updated
April 22, 2026
Record last verified: 2026-04
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, CSR, ANALYTIC CODE
- Time Frame
- Within six months after the approval of a new product or a new indication for an approved product in both the United States and the European Union
- Access Criteria
- Qualified scientific and medical researchers can request the data. Such requests must be submitted in writing to the company's portal and will be internally reviewed regarding criteria for researchers' qualification and legitimacy of the research proposal.
We are committed to enhancing public health through responsible sharing of clinical trial data. Following approval of a new product or a new indication for an approved product in both the US and European Union, the study sponsor and/or its affiliated companies will share study protocols, anonymized patient data and study level data, and redacted clinical study reports with qualified scientific and medical researchers, upon request, as necessary for conducting legitimate research. Further information on how to request data can be found on our website bit.ly/IPD21