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Clinical Trials in the UK / NCT06463587
Recruiting Phase 3

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

NCT06463587 · tracked via the Priya Life Science UK tracker
Sponsor
Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany
Phase
Phase 3
Started
2024-06-25
Last updated
2026-09-16

Condition(s) studied

Generalized Myasthenia Gravis

Investigational drug(s) / intervention(s)

PlaceboCladribine Low DoseCladribine High Dose

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

Cladribine Low Dose: Participants will receive cladribine low dose in two courses separated by 4 weeks.

Cladribine High Dose: Participants will receive cladribine high dose in two courses separated by 4 weeks.

Study 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.

Eligibility

Sex
ALL
Min age
18 Years
Max age
Healthy volunteers
No
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

Primary outcome measure(s)

Trial sites (153)

FacilityCityRegionStatus
Arizona Neuroscience Research, LLC Phoenix Arizona Recruiting
Advanced Neurosciences Research LLC Longmont Colorado Active Not Recruiting
University of Connecticut Health Center - Department of Medicine Farmington Connecticut Recruiting
The George Washington University Medical Faculty Associates Foggy Bottom South Pavilion Washington D.C. District of Columbia Recruiting
Neurology of Central Florida Research Center, LLC Altamonte Springs Florida Recruiting
SFM Clinical Research, LLC Boca Raton Florida Recruiting
University of Florida Health Science Center - 300120311 Jacksonville Florida Recruiting
Neurology Associates, P. A. Maitland Florida Recruiting
University of South Florida - PARENT - PARENT Tampa Florida Recruiting
University of Kansas Medical Center Research Institute, Inc. - 3901 Rainbow (MAIN) Kansas City Kansas Recruiting
UNC Hospitals - PARENT Chapel Hill North Carolina Recruiting
Atrium Health Wake Forest Baptist Winston-Salem North Carolina Recruiting
Miami Valley Hospital South Centerville Ohio Recruiting
University of Cincinnati Cincinnati Ohio Recruiting
Clinical Trials of South Carolina - Charleston Charleston South Carolina Recruiting
Erlanger Health, Inc. - 1123255 Chattanooga Tennessee Recruiting
Baylor Scott & White Research Institute - Baylor Scott & White Research Institute Dallas Texas Recruiting
ANESC Research El Paso Texas Recruiting
Baylor Scott & White Neurology - McKinney McKinney Texas Recruiting
The University of Vermont Medical Center Burlington Vermont Recruiting
Integrated Neurology Services - Dr. Simon Fishman's Office Falls Church Virginia Active Not Recruiting
Expertia S.A- Mautalen Salud e Investigación Ciudad Autonoma Buenos Aires Argentina Recruiting
Instituto de Investigaciones Neurologicas Raul Carrea, FLENI - de Investigaciones Clinicas del área de Cardiología y enfermedades metabólicas asociadas Ciudad Autonoma Buenos Aires Argentina Recruiting
Instituto de Investigaciones Metabolicas (IDIM) Ciudad Autonoma Buenos Aires Argentina Recruiting
Hospital Cordoba Córdoba Argentina Recruiting
Fundacion Rosarina de Neurorehabilitacion Rosario Argentina Recruiting
INECO Neurociencias Oroño Rosario Argentina Recruiting
CER San Juan Centro Polivalente de Asistencia e Inv. Clinica San Juan Argentina Recruiting
Centro de Investigaciones Medicas Tucuman San Miguel de Tucumán Argentina Recruiting
Brain and Mind Research Institute Camperdown New South Wales Recruiting
Sunshine Coast University Hospital Birtinya Queensland Recruiting
Princess Alexandra Hospital - PARENT South Brisbane Queensland Recruiting
John Hunter Hospital - PARENT City of Burnside South Australia Recruiting
Box Hill Hospital - PARENT Box Hill Victoria Recruiting
Gold Coast University Hospital - PARENT Southport Australia Recruiting
UZ Leuven Leuven Belgium Recruiting
C. H. R. de la Citadelle - Account 1 Liège Belgium Recruiting
Medical Center Hera - branch Montana Montana Bulgaria Recruiting
UMHAT 'Dr. Georgi Stranski', EAD Pleven Bulgaria Recruiting
UMHAT "Sv. Georgi", EAD Plovdiv Bulgaria Recruiting

+ 113 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06463587 on ClinicalTrials.gov ↗ ← All trials in the UK